19. Lysosomal storage disease
784 clinical trials,   673 drugs   (DrugBank: 101 drugs),   68 drug target genes,   184 drug target pathways

Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
23 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02998879
(ClinicalTrials.gov)
December 201625/11/2016Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-MannosidosisA 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated Velmanase Alfa (Recombinant Human Alpha-mannosidase) Treatment in Pediatric Patients Below 6 Years of Age With Alpha-MannosidosisAlpha-MannosidosisDrug: Velmanase Alfa (e.g. Lamazym)Chiesi Farmaceutici S.p.A.CromsourceCompletedN/A6 YearsAll5Phase 2Austria;Denmark;France;Germany;Italy
2NCT02478840
(ClinicalTrials.gov)
February 20159/2/2015Evaluation of Long-term Efficacy of Treatment With LamazymA Single Center, Open Label Clinical Trial Investigating the Long-term Efficacy of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) Treatment in Subjects With Alpha-Mannosidosis Who Previously Participated in Lamazym TrialsAlpha-MannosidosisDrug: LamazymZymenex A/SNULLCompletedN/AN/AAll18Phase 3Denmark
3EUCTR2013-000336-97-DK
(EUCTR)
30/01/201503/12/2014A trial of the long-term safety of Lamazym aftercare treatment of subjects with alpha-Mannosidosis who previously participated in Lamazym-trialsA multi-center, un-controlled, open-labeled trial of the long-term safety of Lamazym aftercare treatment of subjects with alpha-Mannosidosis Alpha-Mannosidosis
MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specified disorders of carbohydrate transport and metabolism;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Lamazym
Product Code: rhLAMAN
INN or Proposed INN: Velmanase alfa
Other descriptive name: LAMAZYM
Chiesi Farmaceutici S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
13Phase 3France;Denmark
4EUCTR2014-003950-15-DK
(EUCTR)
22/01/201525/11/2014Medical research trial for testing drug treatment of alpha-MannosidosisA single center, open label clinical trial investigating the long-term efficacy of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) treatment in subjects with alpha-Mannosidosis who previously participated in Lamazym trials alpha-Mannosidosis
MedDRA version: 17.1;Level: LLT;Classification code 10032658;Term: Other specified disorders of carbohydrate transport and metabolism;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Lamazym
Product Code: rhLAMAN
INN or Proposed INN: recombinant human lysosomal alpha-mannosidase
Other descriptive name: LAMAZYM
Zymenex A/SNULLNot RecruitingFemale: yes
Male: yes
Denmark
5NCT01908712
(ClinicalTrials.gov)
August 201316/7/2013Lamazym Aftercare Study FR Designed to Provide Treatment for French PatientsA Multi-center, Un-controlled, Open-labeled Trial of the Long-term Safety of Lamazym Aftercare Treatment of Subjects With Alpha-Mannosidosis Whom Previously Participated in Lamazym TrialsAlpha-MannosidosisDrug: LamazymChiesi Farmaceutici S.p.A.NULLEnrolling by invitationN/AN/AAll10Phase 3France
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2012-000979-17-SE
(EUCTR)
30/07/201307/06/2013A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-MannosidosisA Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects with alpha-Mannosidosis - Phase III Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Product Name: Lamazym
Product Code: rhLAMAN
INN or Proposed INN: Lamazym
Other descriptive name: recombinant human alpha-mannosidase
Zymenex A/SNULLNot RecruitingFemale: yes
Male: yes
25Phase 3Spain;Belgium;Denmark;Germany;Sweden
7EUCTR2013-000336-97-FR
(EUCTR)
26/06/201328/09/2015A trial of the long-term safety of Lamazym aftercare treatment of subjects with alpha-Mannosidosis who previously participated in Lamazym-trialsA multi-center, un-controlled, open-labeled trial of the long-term safety of Lamazym aftercare treatment of subjects with alpha-Mannosidosis who previously participated in Lamazym-trials Treatment of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Zymenex A/SNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
7Phase 3France;Denmark
8NCT01908725
(ClinicalTrials.gov)
June 201312/6/2013Lamazym Aftercare StudyA Single-center, Un-controlled, Open-labeled Trial of the Long-term Safety of Lamazym Aftercare Treatment of Subjects With Alpha-Mannosidosis Whom Previously Participated in Lamazym TrialsAlpha-MannosidosisDrug: LamazymChiesi Farmaceutici S.p.A.NULLActive, not recruitingN/AN/AAll5Phase 3Denmark
9EUCTR2012-000979-17-BE
(EUCTR)
26/03/201307/01/2013A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-MannosidosisA Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects with alpha-Mannosidosis - Phase III Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Product Name: Lamazym
Product Code: rhLAMAN
INN or Proposed INN: recombinant human alpha-mannosidase
Other descriptive name: recombinant human alpha-mannosidase
Zymenex A/SNULLNot RecruitingFemale: yes
Male: yes
25Phase 3Spain;Belgium;Denmark;Germany;Sweden
10EUCTR2012-000979-17-ES
(EUCTR)
27/02/201309/01/2013A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-MannosidosisA Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects with alpha-Mannosidosis - Phase III Treatment of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Product Name: Lamazym
Product Code: rhLAMAN
INN or Proposed INN: Lamazym
Other descriptive name: recombinant human alpha-mannosidase
Zymenex A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 3Spain;Denmark;Germany
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2012-000979-17-DE
(EUCTR)
17/12/201209/07/2012A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-MannosidosisA Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects with alpha-Mannosidosis - Phase III Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Product Name: Lamazym
Product Code: rhLAMAN
INN or Proposed INN: rhLAMAN
Other descriptive name: recombinant human alpha-mannosidase
Zymenex A/SNULLNot RecruitingFemale: yes
Male: yes
20Phase 3Spain;Belgium;Denmark;Germany;Sweden
12EUCTR2012-000979-17-DK
(EUCTR)
06/08/201206/08/2012A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-MannosidosisA Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects with alpha-Mannosidosis - Phase III Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Product Name: Lamazym
Product Code: rhLAMAN
Other descriptive name: recombinant human alpha-mannosidase
Zymenex A/SNULLNot RecruitingFemale: yes
Male: yes
20Phase 3Spain;Belgium;Denmark;Germany;Sweden
13NCT01681953
(ClinicalTrials.gov)
August 201222/8/2012A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-MannosidosisA Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis.Alpha-MannosidosisDrug: Lamazym;Drug: PlaceboZymenex A/SEuropean CommissionCompleted5 Years35 YearsAll25Phase 3Denmark;France;Germany;Poland;United Kingdom
14EUCTR2011-004355-40-DE
(EUCTR)
23/07/201218/01/2012A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis.A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. - Phase 2b Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Product Name: Lamazym
Product Code: rhLAMAN
Other descriptive name: recombinant human alpha-mannosidase
Zymenex A/SNULLNot RecruitingFemale: yes
Male: yes
9Phase 2bSpain;Belgium;Denmark;Germany;United Kingdom
15EUCTR2011-004355-40-ES
(EUCTR)
19/04/201217/04/2012A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis.A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. - Phase 2b Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Product Name: Lamazym
Product Code: rhLAMAN
Other descriptive name: alfa-manosidadas humana recombinante
Zymenex A/SNULLNot RecruitingFemale: yes
Male: yes
9Phase 2bBelgium;Spain;Denmark;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2011-004355-40-GB
(EUCTR)
28/03/201214/02/2012A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis.A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. - Phase 2b Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Product Name: Lamazym
Product Code: rhLAMAN
Other descriptive name: recombinant human alpha-mannosidase
Zymenex A/SNULLNot RecruitingFemale: yes
Male: yes
9Phase 2bSpain;Belgium;Denmark;Germany;United Kingdom
17EUCTR2011-004355-40-BE
(EUCTR)
30/01/201219/01/2012A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis.A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. - Phase 2b Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Product Name: Lamazym
Product Code: rhLAMAN
Other descriptive name: recombinant human alpha-mannosidase
Zymenex A/SNULLNot RecruitingFemale: yes
Male: yes
9Phase 2bSpain;Belgium;Denmark;Germany;United Kingdom
18EUCTR2011-004355-40-DK
(EUCTR)
17/01/201218/01/2012A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis.A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. - Phase 2b Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Product Name: Lamazym
Product Code: rhLAMAN
Other descriptive name: recombinant human alpha-mannosidase
Zymenex A/SNULLNot RecruitingFemale: yes
Male: yes
9Phase 2Spain;Belgium;Denmark;Germany;United Kingdom
19NCT01681940
(ClinicalTrials.gov)
January 201222/8/2012Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-MannosidosisA Multi-Center, Open-Label Trial of the Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-MannosidosisAlpha-MannosidosisDrug: LamazymZymenex A/SEuropean CommissionCompleted5 Years21 YearsAll10Phase 2Belgium;Denmark;Spain;United Kingdom
20NCT01285700
(ClinicalTrials.gov)
January 201125/1/2011Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosisA Single Center, Randomized, Open-label, Multiple-dose Study of the Efficacy and Long-term Safety of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-mannosidosis.Alpha MannosidosisDrug: LamazymZymenex A/SEuropean CommissionActive, not recruiting5 Years21 YearsBoth10Phase 2Denmark
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2010-022085-26-DK
(EUCTR)
05/10/201023/08/2010A single center, randomized, open-label, multiple-dose study of the efficacy and long-term safety of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) for the treatment of patients with alpha-mannosidosisA single center, randomized, open-label, multiple-dose study of the efficacy and long-term safety of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) for the treatment of patients with alpha-mannosidosis Treatment of alpha-MannosidosisProduct Name: Lamazym
Product Code: rhLAMAN
INN or Proposed INN: recombinant human alpha-mannosidase
Zymenex A/SNULLNot RecruitingFemale: yes
Male: yes
10Denmark
22EUCTR2010-022084-36-DK
(EUCTR)
05/10/201020/08/2010A single center, open-label, dose escalation study of the safety and pharmacokinetics of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) for the treatment of patients with alpha-mannosidosis.A single center, open-label, dose escalation study of the safety and pharmacokinetics of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) for the treatment of patients with alpha-mannosidosis. Treatment of alpha-MannosidosisProduct Name: Lamazym
Product Code: rhLAMAN
INN or Proposed INN: recombinant human alpha-mannosidase
Zymenex A/SNULLNot RecruitingFemale: yes
Male: yes
10Denmark
23NCT01268358
(ClinicalTrials.gov)
October 201029/12/2010Safety Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosisA Single Center, Open-label, Dose Escalation Study of the Safety and Pharmacokinetics of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-mannosidosis.Alpha MannosidosisDrug: LamazymZymenex A/SEuropean CommissionCompleted5 Years20 YearsAll10Phase 1NULL