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The Natural History of Metachromatic Leukodystrophy Study (HOME Study)

NCT04628364 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 21

Last updated 2025-06-08

No results posted yet for this study

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.

Conditions

  • Metachromatic Leukodystrophy

Sponsors & Collaborators

  • Food and Drug Administration (FDA)

    collaborator FED

  • National Organization for Rare Disorders

    lead OTHER

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-10-01
Primary Completion
2024-01-31
Completion
2025-03-30

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04628364 on ClinicalTrials.gov