A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

36

Participants

Timeline

Start Date

May 13, 2019

Primary Completion Date

March 8, 2023

Study Completion Date

January 31, 2026

Conditions
Metachromatic Leukodystrophy (MLD)
Interventions
DRUG

SHP611

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

Trial Locations (30)

165

IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN, Roma

2650

UZ Antwerpen, Edegem

8035

Hospital Vall d'Hebrón, Barcelona

10016

New York University Langone Medical Center, New York

15224

Children's Hospital of Pittsburgh, Pittsburgh

19104

Children's Hospital of Philadelphia, Philadelphia

20246

Universitätsklinikum Hamburg Eppendorf, Hamburg

30303

Rare Disease Research, LLC, Atlanta

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

48903

Hospital Universitario Cruces, Barakaldo

52242

University of Iowa Stead Family Children's Hospital, Iowa City

55905

Mayo Clinic - PPDS, Rochester

60611

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

64239

Tel Aviv Sourasky Medical Center, Tel Aviv

72076

Universitätsklinikum Tübingen, Tübingen

80045

Childrens Hospital Colorado, Aurora

84108

University of Utah, Salt Lake City

90502

Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance

94275

Hôpital Bicêtre - Paris Sud, Le Kremlin-Bicêtre

B1629AHJ

Hospital Universitario Austral - PIN, Ciudad Autónoma Buenos Aires

90035-903

Hospital de Clínicas de Porto Alegre, Porto Alegre

T6G 2R7

Stollery Children's Hospital University of Alberta, Edmonton

V6H 3V4

British Columbia Children's Hospital, Vancouver

M5G 1X8

Hospital for Sick Children, Toronto

H3H 1P3

Montreal Children's Hospital, Montreal

06200

CHU Lenval, Nice

124 62

Attikon University General Hospital, Chaïdári

920-8641

Kanazawa University Hospital, Kanazawa

1081 HV

VU Medisch Centrum, Amsterdam

B4 6NH

Birmingham Children's Hospital NHS Foundation Trust, Birmingham

Sponsors

Lead Sponsor

All Listed Sponsors
collaborator

Takeda Development Center Americas, Inc.

INDUSTRY

lead

Shire

INDUSTRY