Adrenoleukodystrophy Clinical Trial
Official title:
Newborn Screening for Adrenoleukodystrophy
To test if the routine newborn screening dried blood spots can be used to test if elevation of C26:0 lysophosphatidylcholine (C26:0-lyso-PC), a status indicating adrenoleukodystrophy (ALD)
Parents of newborns will be invited to test if their newborns are affected with ALD. The routine newborn screening dried blood spots sample will be used to test the concentration of C26:0-lyso-PC . If positive of a screening test, further confirmation tests including physical examination and other methodology for ALD confirmation will be provided. Genetic counseling and treatment option will be provided, too. ;
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Withdrawn |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
|
||
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 | |
Recruiting |
NCT04090268 -
Precision Exercise in Children With Malignant Hemopathies
|
N/A |