Trials / Active Not Recruiting
Active Not RecruitingNCT03771898
A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy
- Status
- Active Not Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 36 (actual)
- Sponsor
- Shire · Industry
- Sex
- All
- Age
- 6 Months – 72 Months
- Healthy volunteers
- Not accepted
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
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | SHP611 | Participants will receive 150 mg of SHP611 IT via IDDD or LP once weekly for 106 weeks. |
Timeline
- Start date
- 2019-05-13
- Primary completion
- 2023-03-08
- Completion
- 2026-05-01
- First posted
- 2018-12-11
- Last updated
- 2026-04-15
- Results posted
- 2024-06-06
Locations
30 sites across 14 countries: United States, Argentina, Belgium, Brazil, Canada, France, Germany, Greece, Israel, Italy, Japan, Netherlands, Spain, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03771898. Inclusion in this directory is not an endorsement.