234. ペルオキシソーム病(副腎白質ジストロフィーを除く。) Peroxisomal disease (except Adrenoleukodystrophy) Clinical trials / Disease details


臨床試験数 : 41 薬物数 : 37 - (DrugBank : 12) / 標的遺伝子数 : 13 - 標的パスウェイ数 : 44

  
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Recruitment_
Status
Inclusion_
agemin
Inclusion_
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Inclusion_
gender
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PhaseCountries
1NCT05001269
(ClinicalTrials.gov)
October 202130/6/2021Nedosiran in Pediatric Patients From Birth to 5 Years of Age With PH and Relatively Intact Renal FunctionA Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients From Birth to 5 Years of Age With Primary Hyperoxaluria and Relatively Intact Renal FunctionPrimary Hyperoxaluria;Primary Hyperoxaluria Type 2;Primary Hyperoxaluria Type 1Drug: nedosiranDicerna Pharmaceuticals, Inc.NULLNot yet recruitingN/A5 YearsAll10Phase 2NULL
2EUCTR2019-001346-17-IT
(EUCTR)
14/05/202107/06/2021A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) - ILLUMINATE-C Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: [ALN-GO1]
INN or Proposed INN: Lumasiran
ALNYLAM PHARMACEUTICALS, INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 3United Arab Emirates;United States;Lebanon;Turkey;Israel;United Kingdom;Switzerland;Italy;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany
3EUCTR2020-002826-97-IT
(EUCTR)
12/05/202107/06/2021Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXCA Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCRPHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe RenalImpairment, With or Without Dialysis - PHYOX 7: Safety & Efficacy of DCR-PHXC in patients with PH1/2 and ESRD Primary Hyperoxaluria
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Nedosiran
Product Code: [DCR-PHXC]
INN or Proposed INN: DCR-L1360
Dicerna Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 2France;United States;Spain;Romania;Australia;Germany;United Kingdom;Italy
4EUCTR2020-002826-97-DE
(EUCTR)
20/04/202108/12/2020Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXCA Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis - PHYOX 7: Safety & Efficacy of DCR-PHXC in patients with PH1/2 and ESRD Primary Hyperoxaluria
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Nedosiran
Product Code: DCR-PHXC
INN or Proposed INN: DCR-L1360
Other descriptive name: Nedosiran
Dicerna Pharmaceuticals IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 2United States;France;Spain;Romania;Germany;United Kingdom;Italy
5NCT04580420
(ClinicalTrials.gov)
April 15, 202124/9/2020Safety & Efficacy of DCR-PHXC in Patients With PH1/2 and ESRDA Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without DialysisPrimary Hyperoxaluria;Primary Hyperoxaluria Type 2;End Stage Renal DiseaseDrug: DCR-PHXCDicerna Pharmaceuticals, Inc.NULLRecruitingN/AN/AAll12Phase 2United States;Spain;United Kingdom
6EUCTR2020-002826-97-FR
(EUCTR)
29/01/202130/09/2020Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXCA Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis - PHYOX 7: Safety & Efficacy of DCR-PHXC in patients with PH1/2 and ESRD Primary Hyperoxaluria
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Nedosiran
Product Code: DCR-PHXC
INN or Proposed INN: DCR-L1360
Other descriptive name: Nedosiran
Dicerna Pharmaceuticals IncNULLNot RecruitingFemale: yes
Male: yes
24Phase 2United States;France;Spain;Romania;Australia;Germany;United Kingdom;Japan;Italy
7EUCTR2019-001346-17-GB
(EUCTR)
22/01/202023/07/2019A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: Lumasiran
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 3United Arab Emirates;United States;Lebanon;Turkey;Israel;United Kingdom;Switzerland;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany
8NCT04152200
(ClinicalTrials.gov)
January 21, 202031/10/2019A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PH1)Primary Hyperoxaluria Type 1;Primary HyperoxaluriaDrug: LumasiranAlnylam PharmaceuticalsNULLActive, not recruitingN/AN/AAll21Phase 3United States;Australia;Belgium;France;Israel;Italy;Jordan;Lebanon;Netherlands;Turkey;United Arab Emirates;Switzerland;United Kingdom
9EUCTR2019-001346-17-NL
(EUCTR)
05/12/201922/08/2019A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: LUMASIRAN
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 3United States;United Arab Emirates;Lebanon;Turkey;Israel;Switzerland;United Kingdom;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany
10NCT03847909
(ClinicalTrials.gov)
October 28, 201915/2/2019A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary HyperoxaluriaPrimary Hyperoxaluria Type 1 (PH1);Primary Hyperoxaluria Type 2 (PH2);Kidney Diseases;Urologic Diseases;Genetic DiseaseDrug: DCR-PHXC;Drug: Sterile Normal Saline (0.9% NaCl)Dicerna Pharmaceuticals, Inc.NULLCompleted6 YearsN/AAll35Phase 2United States;Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom
11NCT04125472
(ClinicalTrials.gov)
October 15, 201910/10/2019Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1Primary HyperoxaluriaDrug: LumasiranAlnylam PharmaceuticalsNULLApproved for marketingN/AN/AAllBelgium;Canada;Italy;Netherlands;Spain;France;French Polynesia;Germany;Switzerland;United Kingdom;United States
12EUCTR2019-001346-17-BE
(EUCTR)
07/10/201921/08/2019A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: LUMASIRAN
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 3Germany;Netherlands;Australia;Belgium;Jordan;France;Egypt;India;United Kingdom;Switzerland;Israel;Turkey;Lebanon;United States;United Arab Emirates
13NCT04042402
(ClinicalTrials.gov)
July 9, 201910/7/2019Long Term Extension Study in Patients With Primary HyperoxaluriaAn Open-Label Roll-Over Study to Evaluate the Long-Term Safety and Efficacy of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary HyperoxaluriaPrimary Hyperoxaluria Type 1 (PH1);Primary Hyperoxaluria Type 2 (PH2);Kidney Diseases;Urologic Diseases;Genetic DiseaseDrug: DCR-PHXCDicerna Pharmaceuticals, Inc.NULLEnrolling by invitation6 YearsN/AAll50Phase 3United States;Australia;Canada;France;Germany;Italy;Japan;Lebanon;Netherlands;Spain;United Kingdom
14EUCTR2018-004014-17-DE
(EUCTR)
23/04/201927/12/2018A Study of an Investigational Drug, Lumasiran (ALN-GO1) in Infants and Young Children with Primary Hyperoxaluria Type 1ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: LUMASIRAN
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 3United States;France;Israel;Germany;United Kingdom
15NCT03905694
(ClinicalTrials.gov)
April 22, 20191/4/2019A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1Primary Hyperoxaluria;Primary Hyperoxaluria Type 1 (PH1)Drug: LumasiranAlnylam PharmaceuticalsNULLActive, not recruitingN/A5 YearsAll18Phase 3United States;France;Germany;Israel;United Kingdom
16EUCTR2018-001981-40-NL
(EUCTR)
21/03/201921/01/2019A Phase 3 Study of an Investigational Drug, Lumasiran (ALN-GO1) with an Extended Dosing Period in Children and Adults with Primary Hyperoxaluria Type 1 DiseaseILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study with an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: lumasiran
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3France;United States;United Arab Emirates;Jordan;Israel;Germany;Netherlands;United Kingdom;Japan;Switzerland
17EUCTR2018-004014-17-GB
(EUCTR)
01/03/201919/12/2018A Study of an Investigational Drug, Lumasiran (ALN-GO1) in Infants and Young Children with Primary Hyperoxaluria Type 1ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: LUMASIRAN
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
20 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;United States;Israel;Germany;United Kingdom
18EUCTR2018-004014-17-FR
(EUCTR)
19/02/201926/12/2018A Study of an Investigational Drug, Lumasiran (ALN-GO1) in Infants and Young Children with Primary Hyperoxaluria Type 1ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: LUMASIRAN
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
8 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;France;Israel;Germany;United Kingdom
19NCT03856866
(ClinicalTrials.gov)
January 11, 20195/11/2018Hydroxychloroquine Administration for Reduction of PexophagyHydroxychloroquine Administration for Reduction of PexophagyZellweger Syndrome;Peroxisome Biogenesis DisordersDrug: Hydroxychloroquine;Drug: PlaceboThe Hospital for Sick ChildrenNULLRecruiting6 Months40 YearsAll5Phase 2Canada
20EUCTR2018-001981-40-DE
(EUCTR)
17/12/201811/09/2018A Phase 3 Study of an Investigational Drug, Lumasiran (ALN-GO1) with an Extended Dosing Period in Children and Adults with Primary Hyperoxaluria Type 1 DiseaseILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study with an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: lumasiran
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3United States;United Arab Emirates;France;Jordan;Israel;Netherlands;Germany;United Kingdom;Switzerland
21NCT03681184
(ClinicalTrials.gov)
November 27, 201819/9/2018A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study With an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1Primary Hyperoxaluria Type 1 (PH1)Drug: Placebo;Drug: LumasiranAlnylam PharmaceuticalsNULLActive, not recruiting6 YearsN/AAll39Phase 3United States;France;Germany;Israel;Netherlands;Switzerland;United Arab Emirates;United Kingdom
22EUCTR2018-001981-40-GB
(EUCTR)
20/11/201807/08/2018A Phase 3 Study of an Investigational Drug, Lumasiran (ALN-GO1) with an Extended Dosing Period in Children and Adults with Primary Hyperoxaluria Type 1 DiseaseILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study with an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: lumasiran
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
39 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;United States;United Arab Emirates;Jordan;Israel;Netherlands;Germany;Switzerland;United Kingdom
23EUCTR2016-003134-24-NL
(EUCTR)
21/06/201828/09/2017Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.0;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: ALN-GO1
Product Code: ALN-GO1
INN or Proposed INN: ALN-65585
Other descriptive name: ALN-65585
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2France;United States;United Arab Emirates;Jordan;Israel;Germany;Netherlands;United Kingdom
24NCT03350451
(ClinicalTrials.gov)
April 4, 201817/11/2017An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Patients With Primary Hyperoxaluria Type 1A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients With Primary Hyperoxaluria Type 1PH1;Primary Hyperoxaluria;RNAi Therapeutic;siRNA;AGTDrug: LumasiranAlnylam PharmaceuticalsNULLActive, not recruiting6 YearsN/AAll20Phase 2France;Germany;Israel;Netherlands;United Kingdom
25EUCTR2016-003134-24-DE
(EUCTR)
28/12/201711/07/2017Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: lumasiran
Other descriptive name: ALN-65585
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2United States;United Arab Emirates;France;Jordan;Israel;Netherlands;Germany;United Kingdom
26EUCTR2016-003134-24-GB
(EUCTR)
20/12/201730/08/2017Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: lumasiran
Other descriptive name: ALN-65585
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2France;Israel;Netherlands;Germany;United Kingdom
27EUCTR2016-003134-24-FR
(EUCTR)
09/10/201720/10/2017Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.0;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: ALN-GO1
Product Code: ALN-GO1
INN or Proposed INN: ALN-65585
Other descriptive name: ALN-65585
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2United States;United Arab Emirates;France;Jordan;Israel;Netherlands;Germany;United Kingdom
28EUCTR2015-004407-23-NL
(EUCTR)
06/06/201715/08/2016The First-in-Human Study of an Investigational Drug, ALN-GO1, in Healthy Adult Subjects and Patients with Primary Hyperoxaluria Type 1 DiseaseA Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 19.0;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: ALN-GO1
Product Code: ALN-GO1
INN or Proposed INN: ALN-65585
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
60Phase 1;Phase 2United States;France;Jordan;Israel;Germany;Netherlands;United Kingdom
29EUCTR2015-003142-51-NL
(EUCTR)
21/07/201617/11/2015An Early Phase Study of DCR-PH1 in Patients with an Inherited Disorder Resulting in Overproduction of OxalateA Phase 1 Study of DCR-PH1 in Patients with Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 1
MedDRA version: 18.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: DCR-PH1
INN or Proposed INN: Not Yet Assigned
Other descriptive name: DCR-1171X
Dicerna Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 1France;United States;Israel;Germany;Netherlands;United Kingdom
30NCT02795325
(ClinicalTrials.gov)
May 13, 201618/5/2016A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)A Phase 1 Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)Primary Hyperoxaluria Type 1Drug: DCR-PH1;Other: PlaceboDicerna Pharmaceuticals, Inc.NULLTerminated12 YearsN/AAll41Phase 1Germany;Netherlands
31EUCTR2015-003142-51-GB
(EUCTR)
11/04/201606/10/2015An Early Phase Study of DCR-PH1 in Patients with an Inherited Disorder Resulting in Overproduction of OxalateA Phase 1 Study of DCR-PH1 in Patients with Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 1
MedDRA version: 18.0;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: DCR-PH1
INN or Proposed INN: Not Yet Assigned
Other descriptive name: DCR-1171X
Dicerna Pharmaceuticals, Inc.NULLNot Recruiting Female: yes
Male: yes
42 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Israel;Netherlands;Germany;United Kingdom
32NCT02706886
(ClinicalTrials.gov)
March 8, 20163/3/2016Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients With Primary Hyperoxaluria Type 1Primary Hyperoxaluria Type 1 (PH1)Drug: Lumasiran;Drug: PlaceboAlnylam PharmaceuticalsNULLCompleted6 Years64 YearsAll52Phase 1/Phase 2France;Germany;Israel;Netherlands;United Kingdom;Jordan;United States
33EUCTR2015-004407-23-GB
(EUCTR)
10/02/201621/12/2015The First-in-Human Study of an Investigational Drug, ALN-GO1, in Healthy Adult Subjects and Patients with Primary Hyperoxaluria Type 1 DiseaseA Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: ALN-GO1
Product Code: ALN-GO1
INN or Proposed INN: ALN-65585
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
52Phase 1;Phase 2United Kingdom;Germany;Netherlands;Israel;Jordan;France;United States
34NCT02171104
(ClinicalTrials.gov)
July 10, 201420/6/2014MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisMT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGMucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome;Alpha-Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Glycoprotein Metabolic Disorders;Sphingolipidoses;Recessive Leukodystrophies;Globoid Cell Leukodystrophy;Metachromatic Leukodystrophy;Niemann-Pick B;Niemann-Pick C Subtype 2;Sphingomyelin Deficiency;Peroxisomal Disorders;Adrenoleukodystrophy With Cerebral Involvement;Zellweger Syndrome;Neonatal Adrenoleukodystrophy;Infantile Refsum Disease;Acyl-CoA Oxidase Deficiency;D-Bifunctional Enzyme Deficiency;Multifunctional Enzyme Deficiency;Alpha-methylacyl-CoA Racmase Deficiency;Mitochondrial Neurogastrointestingal Encephalopathy;Severe Osteopetrosis;Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);Inherited Metabolic DisordersBiological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparative Regimen;Drug: Osteopetrosis Haploidentical Only Preparative Regimen;Drug: cALD SR-A (Standard-Risk, Regimen A);Drug: cALD SR-B (Standard-Risk, Regimen B);Drug: cALD HR-D (High-Risk, Regimen C);Drug: cALD HR-D (High-Risk, Regimen D)Masonic Cancer Center, University of MinnesotaNULLRecruitingN/A55 YearsAll100Phase 2United States
35NCT01626092
(ClinicalTrials.gov)
July 11, 201220/6/2012Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal DisordersTreatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT)Lysosomal Storage Disease;Peroxisomal DisorderDrug: Campath-1H;Drug: Clofarabine;Drug: Melphalan;Radiation: Total Body Irradiation with Marrow Boosting;Biological: Hematopoietic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate mofetilMasonic Cancer Center, University of MinnesotaNULLCompletedN/A55 YearsAll3N/AUnited States
36NCT00004442
(ClinicalTrials.gov)
September 199718/10/1999Study of Bile Acids in Patients With Peroxisomal DisordersInfantile Refsum's Disease;Zellweger Syndrome;Bifunctional Enzyme Deficiency;AdrenoleukodystrophyDrug: chenodeoxycholic acid;Drug: cholic acid;Drug: ursodiolChildren's Hospital Research Foundation University of CincinnatiChildren's Hospital Medical Center, CincinnatiTerminatedN/A5 YearsBoth25N/ANULL
37NCT00007020
(ClinicalTrials.gov)
January 19926/12/2000Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic AcidInvestigation in the Pathogenesis of Liver Disease in Patients With Inborn Errors of Bile Acid MetabolismInfantile Refsum's Disease;Zellweger Syndrome;Adrenoleukodystrophy;Peroxisomal Disorders;CholestasisDrug: Cholic AcidsTravere Therapeutics, Inc.Children's Hospital Medical Center, CincinnatiCompletedN/AN/AAll85Phase 3United States
38EUCTR2019-001346-17-DE
(EUCTR)
15/07/2019A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: LUMASIRAN
Alnylam Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
20Phase 3United Arab Emirates;United States;Lebanon;Turkey;Israel;Switzerland;United Kingdom;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany
39EUCTR2015-004407-23-DE
(EUCTR)
07/03/2016The First-in-Human Study of an Investigational Drug, ALN-GO1, in Healthy Adult Subjects and Patients with Primary Hyperoxaluria Type 1 DiseaseA Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients with Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: ALN-GO1
Product Code: ALN-GO1
INN or Proposed INN: ALN-65585
Alnylam Pharmaceuticals, Inc.NULLNot Recruiting Female: yes
Male: yes
52 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Jordan;Israel;Netherlands;Germany;United Kingdom
40EUCTR2019-001346-17-FR
(EUCTR)
12/07/2019A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 1 (PH1)
MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lumasiran
Product Code: ALN-GO1
INN or Proposed INN: LUMASIRAN
Alnylam Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
16Phase 3United Arab Emirates;United States;Lebanon;Turkey;Israel;Switzerland;United Kingdom;India;France;Egypt;Belgium;Australia;Netherlands;Germany
41EUCTR2010-022046-25-NL
(EUCTR)
27/10/2010Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in ZellwegerCholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in Zellweger Peroxisomal biogenesis disorder Synonym: Zellweger spectrum disorderThis syndrome encompasses a spectrum of severity in symptomsPreviously the disorder was subdivided into three disorders based on the severity of symptoms; Infantile Refsum disease (IRD) is the mildest form, neonatal adrenoleucodystrofia (NALD) has more severe symptoms and Zellweger syndrome has the most severe clinical symptoms.
MedDRA version: 12.1;Level: LLT;Classification code 10053706;Term: Zellweger syndrome
MedDRA version: 12.1;Classification code 10038275;Term: Refsum's disease
Product Name: cholic acid
Product Code: CA
INN or Proposed INN: Cholic Acid
Other descriptive name: CHOLIC ACID
Department of Pediatric Gastroenterology, Academic Medical CentreNULLNAFemale: yes
Male: yes
Phase 2Netherlands