The Natural History of Metachromatic Leukodystrophy Study (HOME Study)

Completed

Conditions studied: Metachromatic Leukodystrophy

In brief

The primary aims of the HOME Study are to: * Design and implement a natural history study for metachromatic leukodystrophy to serve as a source of external control data, to augment or replace concurrent controls in clinical trials; * Pilot test and develop guidance on how to design, conduct, and analyze the data from a natural history study to support adaptive trial designs for regulatory use; * Reduce burden of participation in trials and provide a potential solution to patient recruitment challenges, particularly for RCT's; and * Design approaches that support remote participation in studies.

Key facts

Study ID

NCT04628364

Run by

National Organization for Rare Disorders

People needed

21

Starts

2020-10-01

Expected to finish

2025-03-30

Last updated by the study team

2025-06-08

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• The study is open to English speaking individuals of all ages who have a diagnosis consistent with metachromatic leukodystrophy (MLD). MLD is defined as:
• Mutations in the ASA and PSAP genes identified by genetic testing;
• A diagnosis of MLD by MRI of the brain; or
• Sulfatase enzyme activity and urinary sulfatide excretion identified by biochemical testing.

You may not qualify if…

• Patients will be excluded from the study if they do not meet inclusion criteria.
• Non-English speaking individuals
• No confirmed diagnosis of metachromatic leukodystrophy.

Where it is running

• National Organization for Rare Disorders — Danbury, Connecticut, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.