Adrenoleukodystrophy Clinical Trial
OBJECTIVES: I. Determine the effectiveness of oral bile acid therapy with cholic acid,
chenodeoxycholic acid, and ursodeoxycholic acid in patients with peroxisomal disorders
involving impaired primary bile acid synthesis.
II. Determine whether suppression of synthesis of atypical bile acids and enrichment of bile
acid pool with this regimen is effective in treating this patient population and improving
quality of life.
PROTOCOL OUTLINE: Patients receive oral cholic acid and oral chenodeoxycholic acid on day 1.
On day 4, patients receive oral cholic and ursodeoxycholic acids. Patients are assessed at 3
and 6 months for liver function response, neurologic status, and nutritional status.
Patients receive treatment until disease progression or unacceptable toxic effects are
observed.
Completion date provided represents the completion date of the grant per OOPD records
;
Primary Purpose: Treatment
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT02254863 -
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
|
Phase 1 | |
Completed |
NCT00383448 -
HSCT for High Risk Inherited Inborn Errors
|
Phase 2 | |
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
Completed |
NCT02961803 -
MD1003-AMN MD1003 in Adrenomyeloneuropathy
|
Phase 2/Phase 3 | |
Withdrawn |
NCT02948062 -
Early Diagnosis Of Childhood Cerebral ALD
|
||
Completed |
NCT02952482 -
Newborn Screening for Adrenoleukodystrophy
|
||
Recruiting |
NCT03789721 -
Adrenoleukodystrophy National Registry Study
|
||
Completed |
NCT04303416 -
Plasma Exchange With Albumin in AMN Patients
|
Phase 2/Phase 3 | |
Terminated |
NCT00545597 -
A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy
|
Phase 3 | |
Completed |
NCT03513328 -
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT02699190 -
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
|
||
Recruiting |
NCT02559830 -
Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
|
Phase 1/Phase 2 | |
Recruiting |
NCT00278044 -
Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children
|
N/A | |
Completed |
NCT00176904 -
Stem Cell Transplant for Inborn Errors of Metabolism
|
Phase 2/Phase 3 | |
Completed |
NCT00007020 -
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
|
Phase 3 | |
Active, not recruiting |
NCT03231878 -
A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.
|
Phase 2/Phase 3 | |
Recruiting |
NCT03047369 -
The Myelin Disorders Biorepository Project
|
||
Recruiting |
NCT04925349 -
Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
|
||
Active, not recruiting |
NCT00005900 -
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
|
N/A | |
Completed |
NCT00004450 -
Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
|
N/A |