Trials / Conditions / Metachromatic Leukodystrophy

Metachromatic Leukodystrophy

24 registered clinical trials studyying Metachromatic Leukodystrophy — 4 currently recruiting.

Status	Trial	Sponsor	Phase
Completed	Baby Detect : Genomic Newborn Screening NCT05687474	Centre Hospitalier Universitaire de Liege	—
Active Not Recruiting	OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) NCT04283227	Orchard Therapeutics	Phase 3
Recruiting	Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adu NCT04925349	Assistance Publique - Hôpitaux de Paris	—
Enrolling By Invitation	ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program NCT05368038	Albert Einstein College of Medicine	—
Completed	The Natural History of Metachromatic Leukodystrophy Study (HOME Study) NCT04628364	National Organization for Rare Disorders	—
Enrolling By Invitation	Early Check: Expanded Screening in Newborns NCT03655223	RTI International	—
Completed	A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) NCT03392987	Orchard Therapeutics	Phase 2
Completed	LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies NCT02699190	Children's Hospital of Philadelphia	—
Recruiting	The Myelin Disorders Biorepository Project NCT03047369	Children's Hospital of Philadelphia	—
Unknown	Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy NCT02559830	Shenzhen Second People's Hospital	Phase 1 / Phase 2
Active Not Recruiting	MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis NCT02171104	Masonic Cancer Center, University of Minnesota	Phase 2
Completed	Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy NCT01801709	Institut National de la Santé Et de la Recherche Médicale, France	Phase 1 / Phase 2
Completed	Human Placental-Derived Stem Cell Transplantation NCT01586455	New York Medical College	Phase 1
Completed	Gene Therapy for Metachromatic Leukodystrophy (MLD) NCT01560182	Orchard Therapeutics	Phase 1 / Phase 2
Completed	Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplan NCT01303146	Assistance Publique - Hôpitaux de Paris	Phase 2
Withdrawn	The Natural History of Metachromatic Leukodystrophy NCT00639132	University of Pittsburgh	—
Completed	Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy NCT00683189	The Cooper Health System	N/A
Completed	HSCT for High Risk Inherited Inborn Errors NCT00383448	Masonic Cancer Center, University of Minnesota	Phase 2
Unknown	Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoie NCT00005900	Fairview University Medical Center	—
Completed	Stem Cell Transplantation (SCT) for Genetic Diseases NCT00004378	National Center for Research Resources (NCRR)	N/A
Completed	Stem Cell Transplant for Inborn Errors of Metabolism NCT00176904	Masonic Cancer Center, University of Minnesota	Phase 2 / Phase 3
No Longer Available	Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use) NCT02084121	Talaris Therapeutics Inc.	—
No Longer Available	BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BP NCT03639844	Bellicum Pharmaceuticals	—
No Longer Available	Single Patient Expanded Access Protocol: Metabolic Boost NCT02021266	Talaris Therapeutics Inc.	—