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Active clinical trials for "Adrenoleukodystrophy"

Results 41-50 of 55

Human Placental-Derived Stem Cell Transplantation

Mucopolysaccharidosis IMucopolysaccharidosis VI14 more

The 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.

Completed15 enrollment criteria

MD1003-AMN MD1003 in Adrenomyeloneuropathy

AdrenomyeloneuropathyAdrenoleukodystrophy1 more

The 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

Completed19 enrollment criteria

A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy

X-linked Adrenoleukodystrophy

In 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.

Completed13 enrollment criteria

Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy...

X-Linked Adrenoleukodystrophy

This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.

Withdrawn6 enrollment criteria

Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)

X-Linked AdrenoleukodystrophyAdrenomyeloneuropathy

The 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.

Withdrawn11 enrollment criteria

MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)

Cerebral Adrenoleukodystrophy

The 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).

Withdrawn10 enrollment criteria

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)...

X-Linked Adrenoleukodystrophy (X-ALD)Cerebral Adrenoleukodystrophy (CALD)1 more

Study 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.

Terminated9 enrollment criteria

Lentiviral Gene Therapy for X-ALD

X-linked Adrenoleukodystrophy

This 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.

Unknown status11 enrollment criteria

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.

Adrenoleukodystrophy

This 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).

Unknown status11 enrollment criteria

Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children

Adrenoleukodystrophy

Study the clinical manifestations and gene mutation of Taiwanese ALD patients

Unknown status2 enrollment criteria
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