The Natural History of Metachromatic Leukodystrophy Study (HOME Study)
Public ClinicalTrials.gov record NCT04628364. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Systems-based Approach to Patient-focused Rare Disease Research and Product Development
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT04628364
- Recruitment status
- Completed
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 21 participants
Conditions and interventions
Conditions
Interventions
Not listed
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 30, 2020
- Primary completion
- Jan 30, 2024
- Completion
- Mar 29, 2025
- Last update posted
- Jun 7, 2025
2020 – 2025
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| National Organization for Rare Disorders | Danbury | Connecticut | 06810 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04628364, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 7, 2025 · Synced Sep 8, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04628364 live on ClinicalTrials.gov.