Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03771898 · ClinicalTrials.gov registry record · Phase 2
A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
A Phase 2 study of Metachromatic Leukodystrophy (MLD), sponsored by Shire.
- Active
- Registry status
- Phase 2
- Development phase
- 36
- Enrollment target
- 20
- Study locations
NCT03771898: Active Phase 2 study of Metachromatic Leukodystrophy (MLD), sponsored by Shire.
NCT03771898 is a Phase 2 study of Metachromatic Leukodystrophy (MLD) that is active but no longer recruiting, run by Shire. The registered enrollment target is 36 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (73% lower). The trial reports 20 study locations across 16 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03771898, a Phase 2 study of Metachromatic Leukodystrophy (MLD), is active but no longer recruiting, sponsored by Shire.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 36 participants
- Enrollment target
- 20
- Study locations
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.
Primary Outcome
Loss of locomotion was estimated using interval censoring survival analysis. Survival probability free of loss of locomotion based on GMFC-MLD was estimated up to Week 106 (or two years), with associated 2-sided 95 percent (%) confidence interval (CI). GMFC-MLD scale consists of 7 categories, scores ranging from 0 (walking without support with quality of performance normal for age) to 6 (loss of any locomotion as well as loss of any head and trunk control). Higher scores mean a worse outcome. T
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 |
What the registry record for NCT03771898 still lists
NCT03771898 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 36 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (73% lower).
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.
NCT03771898 names 20 study sites across 16 states, led by Other, Pennsylvania, California.
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).
What interventions are being tested in trial NCT03771898?
The interventions under investigation include: SHP611 (DRUG).
Who is sponsoring clinical trial NCT03771898?
This trial is sponsored by Shire, which has 245 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03771898's enrollment target sits among peer trials
36 1496th of 2000 higher than 477 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT00929006 · 36 participants · Early Phase 1
Acute Progesterone Suppression of Wake vs. Sleep Luteinizing Hormone Pulse Frequency in Pubertal Girls With and Without Hyperandrogenism
- NCT01891318 · 36 participants · Phase 1
Neoadjuvant Radiosurgery for Resectable Brain Metastases: Phase I/II Study
- NCT02291848 · 36 participants · Phase 1
Tumor-Associated Antigen-Specific Cytotoxic T-Lymphocytes for Multiple Myeloma
- NCT02366819 · 36 participants · Phase 4
Genetic Analysis-Guided Irinotecan Hydrochloride Dosing of mFOLFIRINOX in Treating Patients With Locally Advanced Gastroesophageal or Stomach Cancer
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI