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Active clinical trials for "Leukodystrophy, Metachromatic"

Results 31-36 of 36

Natural History Study of Children With Metachromatic Leukodystrophy

Lipid Metabolism DisordersMetachromatic Leukodystrophy (MLD)14 more

The purpose of this study is evaluate the natural course of disease progression related to gross motor function in children with metachromatic leukodystrophy (MLD).

Terminated12 enrollment criteria

Single Patient Expanded Access Protocol: Metabolic Boost

Metachromatic Leukodystrophy

This is a single patient expanded access protocol to investigate the effects of a second dose of facilitating cell-enhanced hematopoietic stem cell product.

No longer available20 enrollment criteria

Biomarker for Metachromatic Leukodystrophy (BioMeta) Disease

Peripheral NeuropathyMuscle Weakness

Development of a new MS-based biomarker for the early and sensitive diagnosis of Metachromatic Leu-kodystrophy disease from blood (plasma)

Withdrawn10 enrollment criteria

Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)...

Metachromatic Leukodystrophy

A subject was treated under compassionate use provisions under this study with facilitating cell therapy (FCRx) product manufactured using the CliniMACS (Miltenyi Biotec) device, rather than the Max Sep (Baxter) device.

No longer available25 enrollment criteria

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible...

Hurler SyndromeInherited Metabolic Disorder3 more

Providing access of BPX-501 gene modified T cells and rimiducid to pediatric patients who do not meet the eligibility criteria of the BP-U-004 study.

No longer available30 enrollment criteria

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic...

I Cell DiseaseFucosidosis10 more

OBJECTIVES: I. Evaluate bronchoalveolar lavage fluid and serum obtained from pediatric patients with storage disorders prior to allogeneic hematopoietic stem cell transplantation (HSCT) for the presence of proinflammatory cytokines and for the production of nitric oxide by alveolar macrophages to identify possible risk factors for pulmonary complications. II. Investigate the underlying mechanism for the development of significant pulmonary complications in these patients during HSCT. III. Evaluate bronchoalveolar lavage fluid and serum obtained from these same patients at the time a pulmonary complication develops post-HSCT, or at 60 days post-HSCT if there has been no pulmonary complications.

Unknown status1 enrollment criteria
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