A Single-arm Study of Intrathecal SHP611 in Subjects with Metachromatic Leukodystrophy
Research type
Research Study
Full title
A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy
IRAS ID
258313
Contact name
Suresh Vijayaraghavan
Contact email
Sponsor organisation
Shire Human Genetic Therapies, Inc.
Eudract number
2018-003291-12
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 11 months, 15 days
Research summary
Metachromatic leukodystrophy (MLD) is a rare inherited condition in which an enzyme that breaks down fat needed for nerve transmission is missing or not working properly. As a result, nerve transmission gradually decreases over time, producing progressive weakness and learning problems. Patients with the late infantile form of MLD develop symptoms at an early age and progress rapidly to a state of paralysis during early childhood. Death from complications of paralysis typically occurs before age 10 years.
There is no cure, and currently no approved therapies, for MLD.
The study drug, SHP611, is a recombinant (man-made) human form of the enzyme deficient in MLD. It is hoped that intrathecal administration (administered in the spinal fluid) of SHP611 will break down the accumulated fats and could slow or prevent further accumulation, which should lead to nerve system benefits.
SHP611 will be given to all participants intrathecally (IT; injection into the spinal canal) via an intrathecal drug delivery device (IDDD) once weekly for up to 105 weeks. The IDDD will be surgically implanted to permit weekly administration. No placebo will be used.
To date, 24 patients with MLD have received SHP611 in prior clinical research studies. IT administration of SHP611 in children with MLD was well tolerated in previous studies.
This study, sponsored by Shire Human Genetic Therapies, Inc., will evaluate the safety of SHP611 and its effect on motor function (the abilities required to control the large muscles of the body for walking, running, sitting, crawling, and other activities) in patients with late infantile MLD.
About 38 children, up to 6years old in North America, Europe, Middle East, Latin America, and Japan are expected to take part in this study. Participants are expected to be in the study for about 26months. During the study visits, participants will undergo procedures such as physical examinations, blood tests and motor function assessments.
REC name
West of Scotland REC 1
REC reference
19/WS/0155
Date of REC Opinion
12 Nov 2019
REC opinion
Further Information Favourable Opinion