Human Placental-Derived Stem Cell Transplantation
Mucopolysaccharidosis IMucopolysaccharidosis VI14 moreThe purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant disorders who require a stem cell transplant. Patients will get either full dose (high-intensity) or lower dose (low intensity) chemo- and immunotherapy followed by a stem cell transplantation with UCB and HPDSC.
MD1003-AMN MD1003 in Adrenomyeloneuropathy
AdrenomyeloneuropathyAdrenoleukodystrophy1 moreThe primary objective of the trial is to demonstrate the superiority of biotin at 300 mg/day over placebo in the clinical improvement (walking tests) of patients with adrenomyeloneuropathy
A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
X-linked AdrenoleukodystrophyIn this pilot study, the investigators will assess the safety of two high-dose regimens of oral vitamin D supplementation and measure the effects of vitamin D supplementation on markers of oxidative stress and inflammation in the blood and brain of study participants before, during, and after taking vitamin D supplements. The goal of the study is to establish research measures (i.e. biomarkers) and an optimal dose for vitamin D supplementation in boys with the X-linked adrenoleukodystrophy (ALD) genotype.
Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy...
X-Linked AdrenoleukodystrophyThis is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.
Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)
X-Linked AdrenoleukodystrophyAdrenomyeloneuropathyThe purpose of this study is to assess the safety, tolerance, pharmacokinetics, and pharmacodynamics of sobetirome, a selective thyroid hormone analog, in adult male X-ALD patients.
MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)
Cerebral AdrenoleukodystrophyThe purpose of this study is to determine the maximum tolerated dose (MTD) of mesenchymal stem cells (IT-MSC) in patients with active, advanced cerebral adrenoleukodystrophy (cALD).
Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)...
X-Linked Adrenoleukodystrophy (X-ALD)Cerebral Adrenoleukodystrophy (CALD)1 moreStudy ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.
Lentiviral Gene Therapy for X-ALD
X-linked AdrenoleukodystrophyThis is a Phase I/II clinical trial of gene therapy for treating X-linked adrenoleukodystrophy using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF-ABCD1 to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.
A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.
AdrenoleukodystrophyThis is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the AMN phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the efficacy and safety of MIN-102 treatment. Study sites will consist of specialist referral centers experienced in the management of adrenoleukodystrophy (ALD).
Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children
AdrenoleukodystrophyStudy the clinical manifestations and gene mutation of Taiwanese ALD patients