234. ペルオキシソーム病(副腎白質ジストロフィーを除く。)
[臨床試験数:38,薬物数:37(DrugBank:12),標的遺伝子数:12,標的パスウェイ数:42

Searched query = "Peroxisomal disease (except Adrenoleukodystrophy)", "Peroxisomal disease", "Peroxisomal disorder", "Peroxisome biogenesis disorder", "PEX gene disorder", "Zellweger syndrome", "Neonatal adrenoleukodystrophy", "Rhizomelic chondrodysplasia punctata type 1", "RCDP type 1", "RCDP1", "Peroxisomal beta-oxidation enzyme deficiency", "Acyl-CoA oxidase deficiency", "AOX deficiency", "D-Bifunctional protein deficiency", "DBP deficiency", "Sterol carrier protein X deficiency", "SCPx deficiency", "2-methylacyl-CoA racemase deficiency", "Alpha-methylacyl-CoA racemase deficiency", "AMACR deficiency", "Refsum disease", "Plasmalogen biosynthesis enzyme deficiency", "Rhizomelic chondrodysplasia punctata type 2", "RCDP type 2", "RCDP2", "Rhizomelic chondrodysplasia punctata type 3", "RCDP type 3", "RCDP3", "Primary hyperoxaluria type 1", "PH1", "Acatalasemia", "Acatalasia", "Contiguous ABCD1/DXS1357E deletion syndrome", "CADDS"
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"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04580420
(ClinicalTrials.gov)
October 202024/9/2020Safety & 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.NULLNot yet recruitingN/AN/AAll12Phase 2NULL
2EUCTR2019-001346-17-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
3NCT04125472
(ClinicalTrials.gov)
January 22, 202010/10/2019Expanded 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 PharmaceuticalsNULLAvailableN/AN/AAllUnited States;Belgium;France;French Polynesia;Germany;Italy;Switzerland;United Kingdom
4NCT04152200
(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 PharmaceuticalsNULLRecruitingN/AN/AAll20Phase 3United States;Australia;Belgium;France;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom
5EUCTR2019-001346-17-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03847909
(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.NULLRecruiting6 YearsN/AAll36Phase 2United States;Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom
7EUCTR2019-001346-17-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 3United Arab Emirates;United States;Lebanon;Turkey;Israel;Switzerland;United Kingdom;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany
8NCT04042402
(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;France;Germany;Netherlands;United Kingdom
9EUCTR2018-004014-17-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
10NCT03905694
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2018-001981-40-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
12EUCTR2018-004014-17-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]
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
20Phase 3France;United States;Israel;Germany;United Kingdom
13EUCTR2018-004014-17-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]
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
8Phase 3United States;France;Israel;Germany;United Kingdom
14NCT03856866
(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
15EUCTR2018-001981-40-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT03681184
(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: Lumasiran;Drug: Sterile Normal Saline (0.9% NaCl)Alnylam PharmaceuticalsNULLActive, not recruiting6 YearsN/AAll39Phase 3United States;France;Germany;Israel;Netherlands;Switzerland;United Arab Emirates;United Kingdom
17EUCTR2018-001981-40-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]
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
39Phase 3France;United States;United Arab Emirates;Jordan;Israel;Netherlands;Germany;Switzerland;United Kingdom
18EUCTR2016-003134-24-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
19NCT03350451
(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
20EUCTR2016-003134-24-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2016-003134-24-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
22EUCTR2016-003134-24-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
23EUCTR2015-004407-23-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
24EUCTR2015-003142-51-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
25NCT02795325
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26EUCTR2015-003142-51-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]
Dicerna Pharmaceuticals, Inc.NULLNot Recruiting Female: yes
Male: yes
42Phase 1France;United States;Israel;Netherlands;Germany;United Kingdom
27NCT02706886
(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
28EUCTR2015-004407-23-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 States;France;Jordan;Israel;Netherlands;Germany;United Kingdom
29NCT02171104
(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
30NCT01626092
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT00004442
(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
32NCT00007020
(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
33EUCTR2020-002826-97-DE
(EUCTR)
08/12/2020Study 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 IncNULLNAFemale: yes
Male: yes
24Phase 2United States;France;Spain;Romania;Australia;Germany;United Kingdom;Japan;Italy
34EUCTR2019-001346-17-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
35EUCTR2010-022046-25-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
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36EUCTR2015-004407-23-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]
Alnylam Pharmaceuticals, Inc.NULLNot Recruiting Female: yes
Male: yes
52Phase 1;Phase 2France;United States;Jordan;Israel;Netherlands;Germany;United Kingdom
37EUCTR2020-002826-97-FR
(EUCTR)
30/09/2020Study 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 IncNULLNAFemale: yes
Male: yes
24Phase 2United States;France;Spain;Romania;Australia;Germany;United Kingdom;Japan;Italy
38EUCTR2019-001346-17-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