A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
Public ClinicalTrials.gov record NCT03771898. 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 Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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 the time for children with Metachromatic Leukodystrophy (MLD) to retain the ability to move from place to place. Other aims of the study are to determine the effects of intrathecal administration of SHP611 on movement and speech functions and to learn how well SHP611 injected in the spinal fluid that surrounds the brain and spinal cord is tolerated. Study participants will receive SHP611 for about 2 years with the possibility of an extended treatment period.
Study identification
- NCT ID
- NCT03771898
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 36 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 6 Months to 72 Months
- 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
- May 12, 2019
- Primary completion
- Mar 7, 2023
- Completion
- May 5, 2026
- Last update posted
- Jul 9, 2026
2019 – 2026
United States locations
- U.S. sites
- 11
- U.S. states
- 10
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Los Angeles Biomedical Research Institute at Harbor-UCLA | Torrance | California | 90502 | — |
| Childrens Hospital Colorado | Aurora | Colorado | 80045 | — |
| Rare Disease Research, LLC | Atlanta | Georgia | 30303 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| University of Iowa Stead Family Children's Hospital | Iowa City | Iowa | 52242 | — |
| Mayo Clinic - PPDS | Rochester | Minnesota | 55905 | — |
| New York University Langone Medical Center | New York | New York | 10016 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| University of Utah | Salt Lake City | Utah | 84108 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 19 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03771898, 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 Jul 9, 2026 · Synced Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03771898 live on ClinicalTrials.gov.