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A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

NCT03771898 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2026-04-15

Study results available
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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

  • Metachromatic Leukodystrophy (MLD)

Interventions

DRUG

SHP611

Participants will receive 150 mg of SHP611 IT via IDDD or LP once weekly for 106 weeks.

Sponsors & Collaborators

  • Takeda Development Center Americas, Inc.

    collaborator INDUSTRY

  • Shire

    lead INDUSTRY

Principal Investigators

  • Study Director · Shire

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
72 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-05-13
Primary Completion
2023-03-08
Completion
2026-05-01
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Belgium
  • Brazil
  • Canada
  • France
  • Germany
  • Greece
  • Israel
  • Italy
  • Japan
  • Netherlands
  • Spain
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03771898 on ClinicalTrials.gov