Infantile Refsum's Disease

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Infantile Refsum's Disease trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

RecruitingNot Applicable

NCT01793168

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, i…

United States, Australia20,000 participantsStarted 2010-07

All recent trials (5 shown)

Active — Not RecruitingPhase 2

NCT02171104

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-rel…

United States149 participantsStarted 2014-07-10

Treatment: Stem Cell Transplantation, IMD Preparative Regimen

CompletedPhase 3

NCT00007020

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

OBJECTIVES: I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bil…

United States85 participantsStarted 1992-01

Treatment: Cholic Acids

Infantile Refsum's Disease

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

All recent trials (5 shown)

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

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

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

All recent trials (5 shown)

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

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

Betaine and Peroxisome Biogenesis Disorders

Study of Bile Acids in Patients With Peroxisomal Disorders