HGT-MLD-071 Study of HGT-1110 in Patients with MLD
Research type
Research Study
Full title
An Open-Label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy
IRAS ID
196462
Contact name
Anupam Chakrapani
Contact email
Sponsor organisation
Shire Pharmaceuticals
Eudract number
2012-003775-20
Duration of Study in the UK
6 years, 9 months, 0 days
Research summary
Metachromatic leukodystrophy (MLD) is a rare, inherited disorder characterised by deficient activity of an enzyme (a protein) known as arylsulfatase A (ASA). The deficient activity of ASA leads to a reduction in myelin, the fatty covering around nerve cells and fibres that helps them work properly. This causes a reduction in cellular function and cellular death. The patient suffers from a loss of motor and cognitive ability. There are no approved therapies for MLD. The only treatment option for MLD is a bone-marrow transplant (BMT) or haematopoietic stem cell transplantation (HSCT), which have shown little to no effectiveness.
The drug being studied, HGT-1110, is a protein produced from a genetically-engineered human cell that secretes ASA. The drug is a liquid solution that must be administered to the patient intrathecally (IT), via a device called the SOPH-A-PORT Mini S. Patients will have already been fitted in the previous study, HGT-MLD-070, with the PORT-A-CATH IDDD and this will remain but will be replaced with the SOPH-A-PORT Mini S if required.
Patients recruited into this study will be children no older than 13 years of age. They must have participated in the previous HGT-MLD-070 study through to week 40. There will be one local study site in the UK, but other sites in the EU.
Participants can visit their local UK study site every other week (up to 163 times). There will also be follow up phone calls 4 weeks, 3 months and 6 months after receiving the last dose of HGT-1110 or the removal of the port, whichever occurs later. The total time spent in this study will be approximately 6 years and 9 months. As well as receiving the study drug at specified visits, the patient will also need to undergo data collection, basic health checks, scans and X-rays at the main site.REC name
London - West London & GTAC Research Ethics Committee
REC reference
16/LO/0462
Date of REC Opinion
25 Apr 2016
REC opinion
Further Information Favourable Opinion