The Natural History of Metachromatic Leukodystrophy Study (HOME Study) (NCT04628364) | Clinical Trial Compass
CompletedNot Applicable
The Natural History of Metachromatic Leukodystrophy Study (HOME Study)
United States21 participantsStarted 2020-10-01
Plain-language summary
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.
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
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.
Exclusion Criteria:
Patients will be excluded from the study if they do not meet inclusion criteria.
* Non-English speaking individuals
* No confirmed diagnosis of metachromatic leukodystrophy.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Gross Motor Function Classification System - Metachromatic Leukodystrophy (GMFC-MLD)