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A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
NCT03771898 · View on ClinicalTrials.gov ↗
Study Summary
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.
Conditions Studied
Interventions
- DRUG SHP611
Study Locations (20)
Other
- UZ Antwerpen — Edegem
- Hospital de Clínicas de Porto Alegre — Porto Alegre
- Hôpital Bicêtre - Paris Sud — Le Kremlin-Bicêtre
- CHU Lenval — Nice
Pennsylvania
- Children's Hospital of Philadelphia — Philadelphia
- Children's Hospital of Pittsburgh — Pittsburgh
California
- Los Angeles Biomedical Research Institute at Harbor-UCLA — Torrance
Colorado
- Childrens Hospital Colorado — Aurora
Georgia
- Rare Disease Research, LLC — Atlanta
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
Iowa
- University of Iowa Stead Family Children's Hospital — Iowa City
Minnesota
- Mayo Clinic - PPDS — Rochester
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 36 participants |
| Start Date | 2019-05-13 |
| Est. Completion | 2026-03-16 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03771898
The ClinicalTrials.gov registry entry for NCT03771898 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 36 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Shire, which has 56 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Metachromatic Leukodystrophy (MLD) appearing as the primary indexed condition, and to 1 intervention — of which SHP611 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03771898 reports 20 study locations spanning 16 distinct geographic areas — top geographies include Other, Pennsylvania, California. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03771898 about?
NCT03771898 is a clinical study titled "A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy". 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 ar...
What is the current status of trial NCT03771898?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 36 participants. The study started on 2019-05-13. Estimated completion is 2026-03-16.
What conditions does trial NCT03771898 study?
This clinical trial studies the following conditions: Metachromatic Leukodystrophy (MLD). These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03771898?
The interventions under investigation include: SHP611 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03771898?
This trial is sponsored by Shire, which has 56 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03771898 being conducted?
This trial has 20 study locations across California, Colorado, Georgia, Illinois, Iowa. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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