DDrare: Database of Drug Development for Rare Diseases

Searched query = "Peroxisomal disease (except Adrenoleukodystrophy)", "Peroxisomal disease", "Peroxisomal disorder", "Peroxisome biogenesis disorder", "PEX gene disord ... show all
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.

Drug = ALN-65585; ALN-GO1; CA; CALD HR-D (High-Risk, Regimen C); CALD HR-D (High-Risk, Regimen D); CALD SR-A (Standard-Risk, Regimen A); CALD SR-B (Standard-Risk, Regimen B); CHOLIC ACID; Campath; Campath-1H; Chenodeoxycholic acid; Cholic Acid; Cholic Acids; Cholic acid; Clofarabine; Cyclosporine; Cyclosporine A; DCR-1171X; DCR-L1360; DCR-PH1; DCR-PHXC; Hematopoietic stem cell transplantation; Hydroxychloroquine; IMD; IMD Preparative Regimen; LUMASIRAN; Lumasiran; Melphalan; Mycophenolate; Mycophenolate mofetil; Nedosiran; Osteopetrosis Haploidentical Only Preparative Regimen; Osteopetrosis Only Preparative Regimen; Radiation: Total Body Irradiation with Marrow Boosting; Stem Cell Transplantation; Sterile Normal Saline (0.9% NaCl); Ursodiol

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2020-002826-97-FR (EUCTR)		30/09/2020	Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXC	A 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 Inc	NULL	NA			Female: yes Male: yes	24	Phase 2	United States;France;Spain;Romania;Australia;Germany;United Kingdom;Japan;Italy
2	EUCTR2020-002826-97-DE (EUCTR)		08/12/2020	Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXC	A 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 Inc	NULL	NA			Female: yes Male: yes	24	Phase 2	United States;France;Spain;Romania;Australia;Germany;United Kingdom;Japan;Italy

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agemin

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EUCTR2020-002826-97-FR
(EUCTR)

30/09/2020

Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXC

A 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 Inc

NULL

Female: yes
Male: yes

Phase 2

United States;France;Spain;Romania;Australia;Germany;United Kingdom;Japan;Italy

EUCTR2020-002826-97-DE
(EUCTR)