256. 筋型糖原病 ［臨床試験数：81，薬物数：71（DrugBank：20），標的遺伝子数：32，標的パスウェイ数：97］
Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha 1,4 glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04138277 | December 2019 | 4 November 2019 | A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease | Pompe Disease (Late-onset) | Drug: AT2221;Biological: ATB200 | Amicus Therapeutics | Not recruiting | 18 Years | N/A | All | 110 | Phase 3 | ||
| 2 | NCT04094948 | October 1, 2019 | 30 September 2019 | Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease | Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Stably Treated With Enzyme Replacement Therapy | Pompe Disease (Late-onset) | Drug: Clenbuterol;Drug: Placebos | Duke University | Not recruiting | 18 Years | N/A | All | 40 | Phase 2 | United States | |
| 3 | NCT03911505 | August 2019 | 2 September 2019 | A Study to Evaluate the PK, Safety, Efficacy, and PD With ATB200/AT2221 in LOPD Subjects Aged 12 to <18 | An Open-label Study of the Pharmacokinetics, Safety, Efficacy, and Pharmacodynamics of ATB200/AT2221 in Pediatric Subjects Aged 12 to < 18 Years With Late-onset Pompe Disease | Pompe Disease (Late-onset) | Biological: ATB200;Drug: AT2221 | Amicus Therapeutics | Recruiting | 12 Years | 17 Years | All | 14 | Phase 3 | United States | |
| 4 | NCT03945370 | May 6, 2019 | 20 May 2019 | Oral Ketone Body Supplementation in Patients With McArdle Disease | Oral Ketone Body Supplementation in Patients With McArdle Disease | McArdle Disease | Dietary Supplement: ß-hydroxybuturate esters;Dietary Supplement: Placebo drink | Rigshospitalet, Denmark | Not recruiting | 18 Years | N/A | All | 10 | N/A | ||
| 5 | NCT03865836 | March 1, 2019 | 18 March 2019 | Expanded Access for ATB200/AT2221 for the Treatment of LOPD | Expanded Access for ATB200/AT2221 for the Treatment of Late Onset Pompe Disease Patients That do Not Qualify for Pivotal Study ATB200-03 | Pompe Disease | Biological: ATB200;Drug: AT2221 | Amicus Therapeutics | Not recruiting | 1 Year | N/A | All | N/A | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | EUCTR2018-000755-40-HU | 28/02/2019 | 30 April 2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared with Alglucosidase Alfa/Placebo. | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0 Level: LLT Classification code 10075702 Term: Pompe's disease late onset System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 Pharmaceutical Form: Powder for concentrate for solution for injection/infusion INN or Proposed INN: ALGLUCOSIDASE ALFA CAS Number: 420784-05-0 Current Sponsor code: ATB200 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 15- Product Name: AT2221 Product Code: AT2221 Pharmaceutical Form: Capsule, hard INN or Proposed INN: MIGLUSTAT CAS Number: 72599-27-0 Current Sponsor code: AT2221 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 65- Pharmaceutical form of the placebo: Capsule, hard Route of administration of the placebo: Oral use Trade Name: Myozyme Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: ALGLUCOSIDASE ALFA CAS Number: 420784-05-0 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 5- | Amicus Therapeutics, Inc. | Authorised | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Israel;Italy;France;Australia;Denmark;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Brazil;Belgium;Poland;Romania;Bulgaria;Germany;Japan;New Zealand;Sweden | |||
| 7 | NCT03687333 | December 4, 2018 | 18 December 2018 | Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment | A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment | Glycogen Storage Disease Type II | Drug: ALGLUCOSIDASE ALFA (MYOZYME) | Genzyme, a Sanofi Company | Recruiting | N/A | 12 Months | All | 10 | Phase 4 | China | |
| 8 | NCT03729362 | December 4, 2018 | 4 November 2019 | PROPEL Study - A Study Comparing ATB200/AT2221 With Alglucosidase/Placebo in Adult Subjects With LOPD | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Pompe Disease (Late-onset) | Drug: AT2221;Biological: alglucosidase alfa;Biological: ATB200 | Amicus Therapeutics | Recruiting | 18 Years | N/A | All | 100 | Phase 3 | United States;Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Spain;Taiwan;United Kingdom | |
| 9 | ChiCTR1800018514 | 2018-11-06 | 24 September 2018 | A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa Treatment | A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa Treatment | Genetic and Metabolism | Case series:Alglucosidase Alfa treatment; | Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine | Not Recruiting | 0 | 1 | Both | Case series:10; | Phase 4 study | China | |
| 10 | NCT03533673 | November 1, 2018 | 28 October 2019 | AAV2/8-LSPhGAA in Late-Onset Pompe Disease | A Phase 1 Study of the Safety of AAV2/8-LSPhGAA in Late-onset Pompe Disease | Pompe Disease | Biological: AAV2/8LSPhGAA | Asklepios Biopharmaceutical, Inc. | Duke University;National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) | Recruiting | 18 Years | N/A | All | 6 | Phase 1/Phase 2 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | EUCTR2017-004370-34-HU | 29/08/2018 | 10 October 2018 | A study to evaluate changes in function such as walking and/or breathing in subjects taking enzyme replacement therapy (ERT) for late-onset Pompe disease | A Prospective Study in Subjects with Late-onset Pompe Disease who are Currently Being Treated with Enzyme Replacement Therapy | Late-onset Pompe disease (LOPD) in subjects receiving standard-of-care enzyme replacement therapy (ERT) MedDRA version: 20.0 Level: LLT Classification code 10075702 Term: Pompe's disease late onset System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: ALGLUCOSIDASE ALFA CAS Number: 420784-05-0 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 20- | Amicus Therapeutics, Inc. | Not Recruiting | Female: yes Male: yes | 125 | Phase 4 | United States;Taiwan;Slovakia;Spain;Korea, Democratic People's Republic of;Italy;United Kingdom;France;Hungary;Canada;Poland;Belgium;Brazil;Romania;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Sweden | |||
| 12 | NCT03642860 | August 15, 2018 | 9 October 2018 | The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses | Triheptanoin's Effect on Fatty Acid Oxidation and Exercise Tolerance in Patients With Debrancher Deficiency, Glycogenin-1 Deficiency and Phosphofructoinase Deficiency at Rest and During Exercise. A Randomized, Double-blind, Placebo-controlled, Cross-over Study | Tarui Disease;Debrancher Deficiency;GYG1 DEFICIENCY | Drug: Triheptanoin;Drug: Placebo Oil | Rigshospitalet, Denmark | Ultragenyx Pharmaceutical Inc | Recruiting | 15 Years | 85 Years | All | 20 | Phase 2 | Denmark |
| 13 | EUCTR2017-004153-17-DK | 03/05/2018 | 14 May 2018 | The effect of Triheptanoin on excercise in adults and adolescence with glycogenoses | Evaluation of the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest and during exercise. A randomized, double-blind, placebo-controlled, cross-over study. - Triheptanoin in Glycogenoses | Cori Forbe's Disease Also called: glycogen storage disease Type III or debrancher deficiency. Tarui's disease Also called: glycogen storage disease Type VII or phosphofructokinase deficiency. Glycogenin-1 deficiency or glycogen storage disease Type XV. MedDRA version: 20.1 Level: PT Classification code 10053241 Term: Glycogen storage disease type VII System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1 Level: PT Classification code 10053250 Term: Glycogen storage disease type III System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: LLT Classification code 10053255 Term: Tarui disease System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: LLT Classification code 10016983 Term: Forbes' disease System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Triheptanoin Product Code: UX007 Pharmaceutical Form: Oral liquid INN or Proposed INN: Not available CAS Number: 210-647-2 Current Sponsor code: UX0007 Other descriptive name: TRIHEPTANOIN Concentration unit: % percent Concentration type: equal Concentration number: 100- Pharmaceutical form of the placebo: Oral liquid Route of administration of the placebo: Oral use | Copenhagen Neuromuscular Center | Authorised | Female: yes Male: yes | 20 | Phase 2 | Denmark | |||
| 14 | EUCTR2017-004370-34-BE | 09/04/2018 | 7 January 2019 | A study to evaluate changes in function such as walking and/or breathing in subjects taking enzyme replacement therapy (ERT) for late-onset Pompe disease | A Prospective Study in Subjects with Late-onset Pompe Disease who are Currently Being Treated with Enzyme Replacement Therapy | Late-onset Pompe disease (LOPD) in subjects receiving standard-of-care enzyme replacement therapy (ERT) MedDRA version: 20.0 Level: LLT Classification code 10075702 Term: Pompe's disease late onset System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: ALGLUCOSIDASE ALFA CAS Number: 420784-05-0 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 20- | Amicus Therapeutics, Inc. | Not Recruiting | Female: yes Male: yes | 125 | Phase 4 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Italy;United Kingdom;France;Hungary;Canada;Belgium;Brazil;Australia;Denmark;Netherlands;Germany;Japan;Sweden | |||
| 15 | NCT02240407 | October 17, 2017 | 11 June 2019 | Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease | Evaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Patients With Late-Onset Pompe Disease (LOPD) | Pompe Disease | Genetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase;Drug: Rapamycin;Other: saline;Drug: Rituxan;Drug: Diphenhydramine;Drug: Acetaminophen;Drug: Lidocaine;Drug: LMX 4 Topical Cream | University of Florida | Lacerta Therapeutics, Inc | Recruiting | 18 Years | 50 Years | All | 7 | Phase 1 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT03019406 | October 12, 2017 | 28 October 2019 | A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa | An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response | Glycogen Storage Disease Type II-Pompe's Disease | Drug: alglucosidase alfa GZ419829;Drug: avalglucosidase alfa GZ402666 | Genzyme, a Sanofi Company | Not recruiting | 6 Months | 17 Years | All | 22 | Phase 2 | United States;France;Japan;Taiwan;United Kingdom | |
| 17 | NCT02898753 | June 21, 2017 | 11 November 2019 | VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease | A Three-Month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Versus Myozyme®/Lumizyme® in Patients With Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment With VAL-1221 in All Patients | Pompe Disease | Drug: VAL-1221;Drug: RhGAA | Valerion Therapeutics, LLC | Not recruiting | 18 Years | N/A | All | 12 | Phase 1/Phase 2 | United States;United Kingdom | |
| 18 | NCT03045042 | April 2017 | 16 December 2017 | Search for Serum/Plasma Biomarkers in Pompe's Disease | Search for Serum/Plasma Biomarkers in Pompe's Disease | Late Onset Pompe Disease;Pompe Disease | Drug: Enzyme Replacement Agent | Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | Genzyme, a Sanofi Company | Not recruiting | 2 Years | N/A | All | 35 | N/A | Spain |
| 19 | NCT02185651 | October 2016 | 18 December 2018 | A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction | A Pilot Study of the Effects of Oral Administration of Zavesca® on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy | Pompe Disease;Hypersensitivity Reaction | Drug: Zavesca® Prescription | University of Florida | Amicus Therapeutics | Not recruiting | 18 Years | 65 Years | All | 2 | Phase 1 | United States |
| 20 | NCT02782741 | October 2016 | 11 November 2019 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe Disease | Glycogen Storage Disease Type II;Pompe's Disease | Drug: alglucosidase alfa (GZ419829);Drug: avalglucosidase alfa(GZ402666) | Sanofi | Not recruiting | 3 Years | N/A | All | 102 | Phase 3 | United States;Argentina;Australia;Austria;Belgium;Brazil;Canada;Colombia;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Switzerland;Taiwan;Turkey;United Kingdom;Bulgaria;Czech Republic;Sweden | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT02919631 | October 2016 | 10 October 2016 | Triheptanoin in Mc Ardle | The Effect of Triheptanoin in Adults With Mc Ardle Disease (Glycogen Storage Disease Type V) | Glycogen Storage Disease Type V | Drug: Placebo oil;Drug: Triheptanoin | Institut National de la Santé Et de la Recherche Médicale, France | Rigshospitalet, Denmark;Rigshospitalet, Denmark | Not recruiting | 18 Years | 65 Years | Both | 12 | Phase 2 | |
| 22 | EUCTR2015-004798-34-GB | 01/08/2016 | 19 November 2018 | This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the safety, tolerability, PK, PD and efficacy of intravenous (IV) ATB200 when co-administered with oral AT2221. | AN OPEN-LABEL, FIXED-SEQUENCE, ASCENDING-DOSE, FIRST-IN-HUMAN STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF INTRAVENOUS INFUSIONS OF ATB200 CO-ADMINISTERED WITH ORAL AT2221 IN ADULT SUBJECTS WITH POMPE DISEASE | Pompe Disease - acid maltase deficiency or glycogen storage disease type II. MedDRA version: 20.0 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ATB200 Product Code: ATB200 Pharmaceutical Form: Powder for concentrate for solution for injection/infusion INN or Proposed INN: ALGLUCOSIDASE ALFA CAS Number: 420784-05-0 Current Sponsor code: ATB200 Concentration unit: mg/l milligram(s)/litre Concentration type: equal Concentration number: 15- Product Code: AT2221 Pharmaceutical Form: Capsule, hard INN or Proposed INN: MIGLUSTAT CAS Number: 72599-27-0 Current Sponsor code: AT2221 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 65- | Amicus Therapeutics, Inc. | Authorised | Female: yes Male: yes | 32 | Phase 1;Phase 2 | United States;Australia;Netherlands;Germany;United Kingdom | |||
| 23 | EUCTR2015-004798-34-NL | 25/07/2016 | 28 February 2019 | This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the safety, tolerability, PK, PD and efficacy of intravenous (IV) ATB200 when co-administered with oral AT2221. | AN OPEN-LABEL, FIXED-SEQUENCE, ASCENDING-DOSE, FIRST-IN-HUMAN STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF INTRAVENOUS INFUSIONS OF ATB200 CO-ADMINISTERED WITH ORAL AT2221 IN ADULT SUBJECTS WITH POMPE DISEASE | Pompe Disease - acid maltase deficiency or glycogen storage disease type II. MedDRA version: 20.0 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ATB200 Product Code: ATB200 Pharmaceutical Form: Powder for concentrate for solution for injection/infusion INN or Proposed INN: ALGLUCOSIDASE ALFA CAS Number: 420784-05-0 Current Sponsor code: ATB200 Concentration unit: mg/l milligram(s)/litre Concentration type: equal Concentration number: 15- Product Code: AT2221 Pharmaceutical Form: Capsule, hard INN or Proposed INN: MIGLUSTAT CAS Number: 72599-27-0 Current Sponsor code: AT2221 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 65- | Amicus Therapeutics, Inc. | Authorised | Female: yes Male: yes | 32 | Phase 1;Phase 2 | United States;Australia;Germany;Netherlands;United Kingdom | |||
| 24 | EUCTR2016-000942-77-GB | 15/07/2016 | 16 January 2017 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatment naïve Patients with Late-onset Pompe Disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 19.0 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 Pharmaceutical Form: Powder for concentrate for solution for infusion CAS Number: 1802558-87-7 Current Sponsor code: GZ402666 Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100- Trade Name: MYOZYME® Product Name: Myozyme® Pharmaceutical Form: Powder for concentrate for solution for infusion INN or Proposed INN: ALGLUCOSIDASE ALFA CAS Number: 420784-05-0 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 50- | Genzyme Corporation | Authorised | Female: yes Male: yes | 100 | Phase 3 | United States;Taiwan;Spain;Turkey;Austria;Russian Federation;Colombia;Italy;United Kingdom;Switzerland;France;Czech Republic;Mexico;Canada;Argentina;Belgium;Brazil;Denmark;Netherlands;Germany;Japan;Korea, Republic of;Sweden | |||
| 25 | NCT02824068 | June 2016 | 26 August 2019 | Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (ATBIG-Pompe-Study) | Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months | Pompe Disease | Drug: glucosidase alfa | Klinikum der Universitaet Muenchen | Genzyme, a Sanofi Company | Recruiting | 8 Years | N/A | All | 100 | Phase 1 | Argentina;Brazil;Germany;Italy;Taiwan |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | NCT02675465 | January 2016 | 6 May 2019 | First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 | An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease | Pompe Disease | Drug: ATB200;Drug: AT2221 | Amicus Therapeutics | Not recruiting | 18 Years | 75 Years | All | 32 | Phase 1/Phase 2 | United States;Australia;Germany;Netherlands;New Zealand;United Kingdom | |
| 27 | NCT02357225 | August 2015 | 11 June 2018 | A Pilot Study of Pyridostigmine in Pompe Disease | Evaluation of Respiratory and Skeletal Muscle Functions in Response to Acetylcholinesterase Inhibitors in Pompe Disease | Pompe Disease | Drug: Pyridostigmine Bromide | University of Florida | Not recruiting | 8 Years | 60 Years | All | 2 | Early Phase 1 | United States | |
| 28 | NCT02525172 | August 2015 | 25 April 2016 | Immune Modulation Therapy for Pompe Disease | Immune Modulation Therapy for ERT-naïve or ERT-treated Pompe Disease Patients | Pompe Disease | Drug: Rituximab;Drug: intravenous immune globulin;Drug: Bortezomib;Drug: Methotrexate | National Taiwan University Hospital | Recruiting | N/A | N/A | Both | 8 | Phase 4 | Taiwan | |
| 29 | NCT02432768 | April 2015 | 28 October 2019 | The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V) | The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V) | Glycogen Storage Disease Type V | Drug: Triheptanoin;Other: Placebo oil | Rigshospitalet, Denmark | Groupe Hospitalier Pitie-Salpetriere;University of Texas Southwestern Medical Center;Ultragenyx Pharmaceutical Inc | Not recruiting | 18 Years | 65 Years | All | 22 | Phase 2 | Denmark |
| 30 | NCT01710813 | March 20, 2015 | 28 October 2019 | Alglucosidase Alfa Pompe Safety Sub-Registry | A Prospective Safety Sub-Registry to Assess Anaphylaxis and Severe Allergic Reactions, and Severe Cutaneous and Systemic Immune Complex Mediated Reactions With Alglucosidase Alfa Treatment | Pompe Disease | Biological: alglucosidase alfa | Genzyme, a Sanofi Company | Not recruiting | N/A | N/A | All | 110 | Phase 1 | United States;Belgium;Czechia;Germany;Italy;Taiwan;Czech Republic | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | NCT03112889 | January 2015 | 12 February 2018 | Sodium Valproate for GSDV | A Phase II Pilot Study to Explore Treatment With Sodium Valproate in Adults With McArdle Disease (Glycogen Storage Disorder Type V, GSDV) | Glycogen Storage Disease Type V;McArdle Disease | Drug: Sodium Valproate | University College, London | Not recruiting | 18 Years | 64 Years | All | 8 | Phase 2 | Denmark;United Kingdom | |
| 32 | EUCTR2015-000582-31-Outside-EU/EEA | 10 July 2015 | An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (alglucosidase alfa) Treatment in Patients with Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen | An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (alglucosidase alfa) Treatment in Patients with Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 17.1 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Myozyme 50 mg powder for concentrate for solution for infusion Pharmaceutical Form: Powder for concentrate for solution for infusion INN or Proposed INN: ALGLUCOSIDASE ALFA CAS Number: 420784-05-0 Concentration unit: mg/g milligram(s)/gram Concentration type: equal Concentration number: 50- | Genzyme Corporation Inc | Not Available | Female: yes Male: yes | 14 | United States;Australia;Canada | |||||
| 33 | NCT01410890 | November 3, 2014 | 14 October 2019 | Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease | A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease | Pompe Disease (Late-Onset);Glycogen Storage Disease Type II (GSD II) | Biological: alglucosidase alfa | Genzyme, a Sanofi Company | Recruiting | N/A | N/A | All | 20 | Phase 4 | United States;Bulgaria;India;Russian Federation;Ukraine;United Kingdom;Germany | |
| 34 | EUCTR2014-001637-88-DK | 12/08/2014 | 2 October 2017 | Effects on muscle function, in persons with McArdle disease, when treated with the drug Valproate. | A phase 2a study to explore treatment with Sodium Valproate in adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV) - Valproate treatment in McArdle disease | McArdle disease (Glycogen storage disorder type V) MedDRA version: 17.0 Level: LLT Classification code 10026969 Term: McArdle's disease System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Deprakine Pharmaceutical Form: Prolonged-release tablet INN or Proposed INN: SODIUM VALPROATE CAS Number: 1069-66-5 Concentration unit: mg milligram(s) Concentration type: range Concentration number: 300-500 | John Vissing | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | Denmark | |||
| 35 | EUCTR2013-003321-28-BE | 12/05/2014 | 20 August 2018 | NeoGAA Extension Study | An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1 Level: PT Classification code 10053185 Term: Glycogen storage disease type II System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 Pharmaceutical Form: Powder for concentrate for solution for infusion Current Sponsor code: GZ402666 Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100- | Genzyme Corporation | Authorised | Female: yes Male: yes | 21 | Phase 2 | France;United States;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 36 | EUCTR2012-002933-12-GB | 07/04/2014 | 30 April 2019 | A Phase II Pilot Study to Assess Safety and Efficacy of Sodium Valproate in Adults with McArdle Disease | A Phase II pilot study to explore treatment with Sodium Valproate in Adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV) - Sodium Valproate for GSDV Version 1.0 13th January 2014 | McArdle disease (Glycogen storage disease type V, GSDV). The condition is an inherited disorder of skeletal muscle that causes exercise intolerance. The condition can give way to potential rhabdomyolysis which can cause acute renal failure and from middle age muscle wasting and weakness. Affected patients are unable to produce lactate during ischaemic exercise due to a congenital lack of the enzyme muscle glycogen phosphorylase which is essential for glycogen metabolism. MedDRA version: 16.1 Level: LLT Classification code 10026969 Term: McArdle's disease System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Epilim Chrono 200 Controlled Released tablets Product Name: Epilim Chrono 200 Controlled Released tablets Pharmaceutical Form: Prolonged-release tablet INN or Proposed INN: Sodium valproate CAS Number: 1069-66-5 Other descriptive name: Valproic acid Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 200- Trade Name: Epilim Chrono 300 Controlled Released tablets Product Name: Epilim Chrono 300 Controlled Released tablets Pharmaceutical Form: Prolonged-release tablet INN or Proposed INN: Sodium valproate Current Sponsor code: 11/0090 Other descriptive name: valproic acid Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 300- Trade Name: Epilim Chrono 500 Controlled Released tablets Product Name: Epilim Chrono 500 Controlled Released tablets Pharmaceutical Form: Prolonged-release tablet INN or Proposed INN: Sodium valproate Current Sponsor code: 11/0090 Other descriptive name: valproic acid Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 500- | University College London, | Not Recruiting | Female: yes Male: yes | 8 | Phase 2 | United States;Denmark;United Kingdom | |||
| 37 | NCT01924845 | April 2014 | 25 June 2018 | BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects With Late-onset Pompe Disease | Late-onset Pompe Disease | Drug: BMN 701 | BioMarin Pharmaceutical | Not recruiting | 18 Years | N/A | All | 24 | Phase 3 | United States;Belgium;France;Germany;Italy;Netherlands;Portugal;United Kingdom | |
| 38 | NCT02032524 | February 27, 2014 | 28 January 2019 | NeoGAA Extension Study | An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of NeoGAA In Patients With Pompe Disease | Glycogen Storage Disease Type II Pompe Disease | Drug: GZ402666 | Genzyme, a Sanofi Company | Recruiting | N/A | N/A | All | 21 | Phase 2/Phase 3 | United States;Belgium;Denmark;France;Germany;Netherlands;United Kingdom | |
| 39 | NCT02054832 | November 2013 | 6 October 2015 | Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch | A Comparison of Quality of Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard and Modified Uncooked Cornstarch | Glycogen Storage Disease Type IA;Glycogen Storage Disease Type IB;Glycogen Storage Disease Type III;Glycogen Storage Disease Type 0 | Dietary Supplement: Glycosade | John Mitchell | Not recruiting | 2 Years | 50 Years | Both | 11 | N/A | Canada | |
| 40 | NCT02405598 | October 2013 | 26 September 2016 | Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease | Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease | Pompe Disease | Drug: Salbutamol | National Taiwan University Hospital | Not recruiting | 2 Years | N/A | Both | 14 | Phase 4 | Taiwan | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 41 | NCT01942590 | September 2013 | 15 July 2019 | Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy | A Clinical Investigation of the Safety and Efficacy of Clenbuterol on Motor Function in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy | Pompe Disease | Drug: Clenbuterol;Drug: Placebo | Dwight Koeberl, M.D., Ph.D. | Not recruiting | 18 Years | N/A | All | 17 | Phase 1/Phase 2 | United States | |
| 42 | NCT01898364 | July 2013 | 23 March 2015 | Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients. | An Open-label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in naïve and Alglucosidase Alfa Treated Late-onset Pompe Disease Patients. | Pompe Disease;Glycogen Storage Disease Type II (GSD II);Acid Maltase Deficiency | Drug: GZ402666 | Genzyme, a Sanofi Company | Not recruiting | 18 Years | N/A | Both | 24 | Phase 1 | United States;Belgium;Denmark;France;Germany;Netherlands;United Kingdom;Italy | |
| 43 | NCT01885936 | June 2013 | 22 July 2019 | Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease | A Phase 1/2 Double-Blind Study of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement Therapy | Pompe Disease | Drug: Albuterol;Drug: Placebo | Duke University | Not recruiting | 18 Years | N/A | All | 16 | Phase 1/Phase 2 | United States | |
| 44 | EUCTR2013-002257-30-GB | 28 February 2019 | A dose finding study with intravenous administration of duvoglustat hydrochloride (AT2220) and Myozyme in Pompe patients | AN OPEN-LABEL SAFETY AND DOSE-FINDING STUDY OF INTRAVENOUS DUVOGLUSTAT CO-ADMINISTERED WITH RECOMBINANT HUMAN ACID a-GLUCOSIDASE IN SUBJECTS WITH POMPE DISEASE | Pompe disease MedDRA version: 20.0 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: duvoglustat hydrochloride Product Code: AT2220 Pharmaceutical Form: Solution for injection INN or Proposed INN: duvoglustat HCl CAS Number: 73285-50-4 Current Sponsor code: AT2220 HCl Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 30.6- Trade Name: Myozyme Product Name: Myozyme Pharmaceutical Form: Powder for concentrate for solution for infusion INN or Proposed INN: ALGLUCOSIDASE ALFA CAS Number: 420784-05-0 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: range Concentration number: 0.5-4 | Amicus Therapeutics, Inc. | Not Recruiting | Female: yes Male: yes | 24 | Phase 2 | United States;Netherlands;United Kingdom | ||||
| 45 | NCT01597596 | August 2012 | 19 October 2017 | A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease | A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease | Pompe Disease (Infantile-Onset);Glycogen Storage Disease Type II (GSD II);Glycogenosis 2;Acid Maltase Deficiency | Biological: alglucosidase alfa | Genzyme, a Sanofi Company | Not recruiting | N/A | 12 Months | All | 4 | Phase 4 | United States;Germany;Taiwan | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 46 | NCT01859624 | June 2012 | 15 July 2019 | Albuterol in Individuals With Late Onset Pompe Disease (LOPD) | A Clinical Investigation of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease, Whether or Not Receiving Enzyme Replacement Therapy | Pompe Disease | Drug: Albuterol | Duke University | Not recruiting | 18 Years | N/A | All | 8 | Phase 1 | United States | |
| 47 | NCT00566878 | March 2012 | 8 January 2018 | Pompe Lactation Sub-Registry | A Sub-Registry to Determine the Presence of Alglucosidase Alfa in Breast Milk From Women With Pompe Disease Treated With Alglucosidase Alfa. | Glycogen Storage Disease;Pompe Disease | Biological: alglucosidase alfa | Genzyme, a Sanofi Company | Recruiting | N/A | N/A | Female | 5 | N/A | United States | |
| 48 | NCT01526785 | March 2012 | 19 October 2017 | A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease | A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale | Pompe Disease | Drug: Alglucosidase alfa | Genzyme, a Sanofi Company | Not recruiting | 1 Year | N/A | All | 113 | Phase 4 | United States | |
| 49 | NCT00567073 | November 2011 | 24 June 2019 | Pompe Pregnancy Sub-Registry | A Sub-Registry to Observe the Effect of Myozyme (Alglucosidase Alfa) Treatment on Pregnancy and Infant Growth in Women With Pompe Disease | Glycogen Storage Disease Type II (GSD-II);Pompe Disease (Late-onset);Glycogenesis 2 Acid Maltase Deficiency | Biological: alglucosidase alpha;Other: No Treatment | Genzyme, a Sanofi Company | Recruiting | N/A | N/A | Female | 20 | Phase 2 | United States;Italy | |
| 50 | NCT01380743 | October 31, 2011 | 20 August 2018 | Drug-drug Interaction Study | An Open-Label, Multi-Center, International Study to Investigate Drug-Drug Interactions Between AT2220 and Alglucosidase Alfa in Patients With Pompe Disease | Pompe Disease | Drug: duvoglustat;Drug: rhGAA | Amicus Therapeutics | Not recruiting | 18 Years | 65 Years | All | 25 | Phase 2 | United States;Canada;France;United Kingdom | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 51 | NCT01435772 | August 15, 2011 | 11 June 2018 | Extension Study for Patients Who Have Participated in a BMN 701 Study | A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study | Pompe Disease | Biological: BMN 701 | BioMarin Pharmaceutical | Not recruiting | 13 Years | N/A | All | 21 | Phase 2 | United States;Australia;France;Germany;New Zealand;United Kingdom | |
| 52 | NCT01288027 | June 2011 | 19 October 2017 | Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa | A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa | Pompe Disease (Late-Onset);Glycogen Storage Disease Type II (GSD II);Glycogenesis 2 Acid Maltase Deficiency | Biological: Alglucosidase Alfa | Genzyme, a Sanofi Company | Not recruiting | 18 Years | N/A | All | 16 | Phase 4 | United States;Germany;Netherlands;United Kingdom | |
| 53 | EUCTR2010-024647-32-IT | 18/03/2011 | 19 March 2012 | Combined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease - ND | Combined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease - ND | Patients with Pompe Disease (Type II Glycogenosis) on therapy with Enzyme Replacement Therapy (ERT) for at least 6 months MedDRA version: 13.1 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders | Trade Name: ZAVESCA Pharmaceutical Form: Capsule, hard INN or Proposed INN: Miglustat Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100- | UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II | Authorised | Female: yes Male: yes | Italy | |||||
| 54 | NCT01230801 | December 2010 | 25 May 2015 | Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease | A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease | Pompe Disease | Biological: BMN 701 | BioMarin Pharmaceutical | Not recruiting | 13 Years | N/A | Both | 30 | Phase 1/Phase 2 | United States;Australia;France;Germany;United Kingdom | |
| 55 | NCT00976352 | September 2010 | 24 September 2018 | Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease | Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients With Pompe Disease | Pompe Disease | Drug: rAAV1-CMV-GAA (study agent) Administration;Other: RMST | University of Florida | National Heart, Lung, and Blood Institute (NHLBI) | Not recruiting | 2 Years | 18 Years | All | 9 | Phase 1/Phase 2 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 56 | NCT00701129 | October 2009 | 19 October 2017 | An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease | An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme®-Naive, CRIM(-) Patients With Infantile-onset Pompe Disease | Pompe Disease;Glycogen Storage Disease Type II | Biological: Alglucosidase Alfa;Drug: Methotrexate;Drug: Rituximab | Genzyme, a Sanofi Company | Not recruiting | N/A | N/A | All | 4 | Phase 4 | United States;Israel | |
| 57 | EUCTR2008-002302-18-DE | 16/02/2009 | 24 July 2012 | An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease - | Pompe Disease MedDRA version: 9.1 Level: LLT Classification code 10036143 Term: Pompe's disease | Product Code: AT2220 Pharmaceutical Form: Powder for oral solution CAS Number: 73285-50-4 Current Sponsor code: AT2220 Other descriptive name: 1-deoxynojirimycin hydrochloride Concentration unit: g gram(s) Concentration type: equal Concentration number: 2.5- Product Code: AT2220 Pharmaceutical Form: Powder for oral solution CAS Number: 73285-50-4 Current Sponsor code: AT2220 Other descriptive name: 1-deoxynojirimycin hydrochloride Concentration unit: g gram(s) Concentration type: equal Concentration number: 5.0- | Amicus Therapeutics, Inc. | Not Recruiting | Female: yes Male: yes | 18 | United Kingdom;Germany | |||||
| 58 | EUCTR2008-008293-31-NL | 10/02/2009 | 19 March 2012 | Early introduction in the Netherlands of alglucosidase alfa manufactured in a 4000 liter bioreactor | Early introduction in the Netherlands of alglucosidase alfa manufactured in a 4000 liter bioreactor | Glycogen Storage Disease Type II (Pompe's Disease) MedDRA version: 9.1 Level: LLT Classification code 10036143 Term: Pompe's disease | Product Name: alglucosidase alfa Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: alglucosidase alfa CAS Number: n.a. Current Sponsor code: rhGAA Other descriptive name: alglucosidase alfa Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 50- | Erasmus MC Sophia | Authorised | Female: yes Male: yes | Netherlands | |||||
| 59 | NCT00701701 | December 14, 2008 | 23 April 2019 | Immune Tolerance Induction Study | An Exploratory Study of the Safety and Efficacy of Immune Tolerance Induction (ITI) in Patients With Pompe Disease Who Have Previously Received Myozyme | Pompe Disease;Glycogen Storage Disease Type II (GSD-II);Glycogenesis 2 Acid Maltase Deficiency | Biological: Myozyme (alglucosidase alfa) | Genzyme, a Sanofi Company | Not recruiting | 1 Month | N/A | All | 4 | Phase 4 | United States;Israel;Canada | |
| 60 | NCT00688597 | December 8, 2008 | 20 August 2018 | Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease | An Open-Label, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Three Dosing Regimens of Oral AT2220 in Patients With Pompe Disease | Pompe Disease | Drug: Duvoglustat | Amicus Therapeutics | Not recruiting | 18 Years | 74 Years | All | 3 | Phase 2 | United States;Australia;Canada;France;Germany;Netherlands;United Kingdom | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 61 | NCT01451879 | October 2008 | 16 December 2017 | Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies | Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy | Pompe Disease | Drug: Rituximab;Drug: Miglustat | University of Florida | Not recruiting | N/A | 65 Years | All | 11 | N/A | United States | |
| 62 | NCT00486889 | August 2008 | 16 December 2017 | Growth and Development Study of Alglucosidase Alfa. | A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa. | Pompe Disease;Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency Disease | Biological: alglucosidase alfa | Genzyme, a Sanofi Company | Not recruiting | N/A | 24 Months | All | 30 | Phase 4 | United States | |
| 63 | NCT00520143 | July 2007 | 19 February 2015 | Alglucosidase Alfa Temporary Access Program | Alglucosidase Alfa Temporary Access Program | Glycogen Storage Disease Type II (GSD-II);Pompe Disease (Late-Onset);Acid Maltase Deficiency Disease;Glycogenosis 2 | Biological: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA]) | Genzyme, a Sanofi Company | Not recruiting | 18 Years | N/A | Both | N/A | United States | ||
| 64 | EUCTR2007-001375-11-NL | 16/05/2007 | 19 March 2012 | Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease Long-term follow-up of patients receiving commercially available Myozyme - not applicable | Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease Long-term follow-up of patients receiving commercially available Myozyme - not applicable | Pompe disease (glycogen storage disease type II) is a genetic, lysosomal storage disorder with a frequency of 1 in 40.000 newborns. The disease is caused by deficiency of alpha-glucosidase, a lysosomal hydrolase involved in the degradation of glycogen. | Trade Name: Myozyme Product Name: Myozyme Product Code: MYOZYME® Pharmaceutical Form: Intravenous infusion | Authorised | Female: yes Male: yes | Netherlands | ||||||
| 65 | NCT00483379 | May 2007 | 19 October 2017 | High Dose or High Dose Frequency Study of Alglucosidase Alfa | An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Alglucosidase Alfa Treatment in Patients With Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen | Pompe Disease;Glycogen Storage Disease Type II (GSD-II);Glycogenesis 2 Acid Maltase Deficiency | Biological: alglucosidase alfa | Genzyme, a Sanofi Company | Not recruiting | 6 Months | N/A | All | 13 | Phase 4 | United States;Australia;Canada | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 66 | EUCTR2006-003644-31-FR | 18/01/2007 | 19 March 2012 | An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 | An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 | Glycogen Storage Disease type II (Pompe's disease) | Trade Name: Myozyme Product Name: Myozyme Product Code: rhGAA, acid alpha glucosidase Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: Alglucosidase alfa CAS Number: n.a. Current Sponsor code: rhGAA Other descriptive name: alglucosidase alpha Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 50- | Genzyme Europe BV | Authorised | Female: yes Male: yes | 90 | Germany;Netherlands;France | ||||
| 67 | NCT00268944 | December 2005 | 19 February 2015 | Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support | Prospective, Open-label, Single-arm, Exploratory Study of the Effect and Safety of rhGAA in Patients With Advanced Late-onset Pompe Disease Who Are Receiving Respiratory Support | Pompe Disease (Late-onset);Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency Disease;Glycogenosis 2 | Biological: Myozyme | Genzyme, a Sanofi Company | Not recruiting | 18 Years | N/A | Both | 5 | Phase 3 | France | |
| 68 | NCT00158600 | September 2005 | 19 October 2017 | A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease | Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease. | Pompe Disease (Late-onset);Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency Disease;Glycogenosis 2 | Biological: alglucosidase alfa;Drug: Placebo | Genzyme, a Sanofi Company | Not recruiting | 8 Years | N/A | All | 90 | Phase 3 | United States;France;Netherlands | |
| 69 | EUCTR2005-002829-31-GB | 31/08/2005 | 24 April 2012 | Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTS | Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTS | Glycogen Storage Disease type II (Pompe´s disease) | Product Name: Myozyme Product Code: rhGAA, acid alpha glucosidase Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: Alglucosidase alfa CAS Number: n.a. Current Sponsor code: rhGAA Other descriptive name: alglucosidase alpha Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 5- | Genzyme Europe BV | Authorised | Female: yes Male: yes | 5 | United Kingdom | ||||
| 70 | EUCTR2005-001629-27-DE | 03/08/2005 | 19 March 2012 | A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602 | A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602 | Pompe disease is a rare metabolic muscle disease inherited in an autosomal recessive fashion. Pompe disease is caused by a deficiency of GAA, which is needed for the degradation of lysosomal glycogen. Pompe disease is characterized by organelle bound (lysosomal) accumulation of glycogen in many body tissues. In general, there is an inverse correlation between the amount of residual GAA activity in patients with Pompe disease and the severity of the disease. | Product Name: Myozyme Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: Alglucosidase alfa CAS Number: n.a. Current Sponsor code: rhGAA Other descriptive name: n.a. Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 5- | Genzyme Europe BV | Not Recruiting | Female: yes Male: yes | 17 | Germany;Italy | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 71 | NCT00125879 | June 2005 | 19 February 2015 | Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602 | A Long-Term Continuation Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602 | Glycogen Storage Disease Type II | Biological: Myozyme | Genzyme, a Sanofi Company | Not recruiting | N/A | N/A | Both | 16 | Phase 2/Phase 3 | United States;France;Germany;Israel;Italy;Netherlands;Taiwan | |
| 72 | NCT00250939 | February 2005 | 19 February 2015 | A Study of rhGAA in Patients With Late-Onset Pompe Disease | Single-center, Open-label Study of Safety, Pharmacokinetics and Efficacy of rhGAA in Patients With Late-Onset Pompe Disease | Pompe Disease (Late-onset);Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency Disease;Glycogenosis 2 | Biological: Myozyme | Genzyme, a Sanofi Company | Not recruiting | 5 Years | 18 Years | Both | 5 | Phase 2 | Netherlands | |
| 73 | NCT00074932 | November 2004 | 19 February 2015 | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease | Glycogen Storage Disease Type II;Glycogenosis 2 | Biological: Myozyme | Genzyme, a Sanofi Company | Not recruiting | N/A | N/A | Both | 9 | N/A | United States | |
| 74 | EUCTR2004-002168-59-IT | 14/10/2004 | 19 March 2012 | An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha- Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-Onset Pompe Disease (Glycogen Storage Disease Type II) | An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha- Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-Onset Pompe Disease (Glycogen Storage Disease Type II) | Treatment for Type II Glycogenosis MedDRA version: 6.1 Level: PT Classification code 10053185 | Product Name: Recombinant human acid alfa-glucosidase (rhGAA) Product Code: NA Pharmaceutical Form: Powder for infusion* INN or Proposed INN: alglucosidase alfa CAS Number: 420784-05-0 Current Sponsor code: myozyme Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 52.5- Product Name: Recombinant human acid alfa-glucosidase (rhGAA) Product Code: NA Pharmaceutical Form: Powder for infusion* INN or Proposed INN: alglucosidase alfa CAS Number: 420784-05-0 Current Sponsor code: myozyme Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 52.5- | GENZYME | Not Recruiting | Female: yes Male: yes | 20 | Italy | ||||
| 75 | NCT00074919 | December 2003 | 19 February 2015 | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease | Glycogen Storage Disease Type II;Glycogenosis 2 | Biological: alglucosidase alfa | Genzyme, a Sanofi Company | Not recruiting | N/A | N/A | Both | N/A | United States | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 76 | NCT00059280 | April 2003 | 19 February 2015 | A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease | An Open-label, Multicenter, Multinational Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid Alpha-glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-onset Pompe Disease | Glycogen Storage Disease Type II | Biological: Myozyme | Genzyme, a Sanofi Company | Not recruiting | N/A | 26 Weeks | Both | 16 | Phase 2/Phase 3 | United States;France;Israel;Taiwan;United Kingdom | |
| 77 | NCT00763932 | April 2003 | 19 February 2015 | Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies | A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid a-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies | Pompe Disease Infantile-Onset;Glycogen Storage Disease Type II | Biological: Myozyme | Genzyme, a Sanofi Company | Not recruiting | 18 Years | N/A | Both | 7 | Phase 2 | United States;France;South Africa | |
| 78 | NCT00765414 | April 2003 | 19 February 2015 | Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies. | An Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid a-Glucosidase (rhGAA) Given as Enyzme Replacement Therapy to a Single Patient With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies | Pompe Disease Late-Onset;Glycogen Storage Disease Type II GSD II | Biological: Myozyme | Genzyme, a Sanofi Company | Not recruiting | 16 Years | N/A | Female | 1 | Phase 2 | United States | |
| 79 | NCT00053573 | February 2003 | 19 February 2015 | rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) | An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of rhGAA Treatment in Patients Greater Than 6 Months and Less Than or Equal to 36 Months Old With Infantile-Onset GSD-II | Glycogen Storage Disease Type II;Pompe Disease;Acid Maltase Deficiency Disease;Glycogenosis 2 | Biological: Myozyme | Genzyme, a Sanofi Company | Not recruiting | 6 Months | 36 Months | Both | 20 | Phase 1/Phase 2 | United States;France;Israel;United Kingdom | |
| 80 | NCT00051935 | January 2003 | 19 February 2015 | A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II | Open-Label, Pilot Study of the Safety, Pharmacokinetics and Pharmacodynamics of Recombinant Human Acid Alpha-Glucosidase (rhGAA) as Enzyme Replacement Therapy in Siblings With Glycogen Storage Disease Type II (GSD-II). | Glycogen Storage Disease Type II;Pompe Disease;Acid Maltase Deficiency Disease;Glycogenosis 2 | Drug: Alglucosidase alfa | Genzyme, a Sanofi Company | Not recruiting | N/A | N/A | Both | 2 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 81 | NCT00025896 | May 2001 | 19 February 2015 | Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease | A Prospective Multinational, Multicenter, Clinical Trial of the Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) in Cross-Reacting Immunologic Material-Positive Patients With Classical Infantile Pompe Disease | Pompe Disease;Glycogen Storage Disease Type II;Acid Maltase Deficiency Disease;Glycogenosis 2 | Drug: recombinant human acid alpha-glucosidase (rhGAA) | Genzyme, a Sanofi Company | Not recruiting | N/A | N/A | Both | 8 | Phase 2 | United States |