Late Infantile Metachromatic Leukodystrophy

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Late Infantile Metachromatic Leukodystrophy trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

RecruitingNot Applicable

NCT01793168

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, i…

United States, Australia20,000 participantsStarted 2010-07

All recent trials (8 shown)

Active — Not RecruitingPhase 2

NCT03771898

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs…

United States36 participantsStarted 2019-05-13

Treatment: SHP611

CompletedPhase 2

NCT01303146

Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation

There is currently no effective treatment for late infantile MLD once clinical symptoms are evident. METAZYM is a recombinant human arylsulfatase A developed fo…

France1 participantsStarted 2008-10

Treatment: rhARSA

Late Infantile Metachromatic Leukodystrophy

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

All recent trials (8 shown)

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

All recent trials (8 shown)

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation

Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy

Baby Detect : Genomic Newborn Screening

Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD