DDrare: Database of Drug Development for Rare Diseases

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.

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	NCT00007020 (ClinicalTrials.gov)	January 1992	6/12/2000	Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid	Investigation in the Pathogenesis of Liver Disease in Patients With Inborn Errors of Bile Acid Metabolism	Infantile Refsum's Disease;Zellweger Syndrome;Adrenoleukodystrophy;Peroxisomal Disorders;Cholestasis	Drug: Cholic Acids	Travere Therapeutics, Inc.	Children's Hospital Medical Center, Cincinnati	Completed	N/A	N/A	All	85	Phase 3	United States

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

NCT00007020
(ClinicalTrials.gov)

January 1992

6/12/2000

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Investigation in the Pathogenesis of Liver Disease in Patients With Inborn Errors of Bile Acid Metabolism

Infantile Refsum's Disease;Zellweger Syndrome;Adrenoleukodystrophy;Peroxisomal Disorders;Cholestasis

Drug: Cholic Acids

Travere Therapeutics, Inc.

Children's Hospital Medical Center, Cincinnati

Completed

N/A

All

Phase 3

United States