Extension HGT1410 Stdy in Pediatric Patients with Early Stage MPS IIIA
Research type
Research Study
Full title
A Open-Label Extension of Study HGT-SAN-093 Evaluating the Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Paediatric Patients with Mucopolysaccharidosis Type IIIA Disease
IRAS ID
172862
Contact name
Maureen Cleary
Contact email
Sponsor organisation
Shire Human Genetic Therapies, Inc.
Eudract number
2014-003960-20
Duration of Study in the UK
3 years, 0 months, 28 days
Research summary
Mucopolysaccharidosis Type IIIA (MPS IIIA, also called Sanfilippo Syndrome Type A) is an uncommon, lysosomal storage disease (LSD) causing progressive loss of structure or function of neurons associated with behavioural and developmental problems. Clinical manifestations are most commonly apparent in the nervous system. There are currently no approved treatments for this devastating disease.
The main goal of this study is to collect the long-term safety and tolerability data in patients with Sanfilippo Syndrome Type A (MPS IIIA) who received the study drug (HGT-1410) through a surgically placed tiny tube (IDDD) in main study (HGT-SAN-093) and wish to continue the study treatment. In addition, patients who were on the “no treatment” arm in the HGT-SAN-093 study and who are suitable to participate in the SHP-610-201 extension trial, will be invited to participate. These patients will have an IDDD placed and will also receive the study drug. 21 participants are expected in the study globally and 1 participant from the UK for about 30 months.
Participants who provide written consent, complete study HGT-SAN-093 and meet all inclusion and exclusion criteria will be allowed to participant in this extension study (SHP-610-210).Patients who have received study drug in study HGT-SAN-093 will remain on the same 2 or 4 week dose administration schedule (groups 1 or 2 respectively). Patients who received no-treatment in study HGT-SAN-093 will be re-assigned with a 50% chance of being assigned to either treatment group and will receive the study drug either every 2 weeks or every 4 weeks.
All patients in this study will receive the investigational study drug through the IDDD.
The study involves procedures including: ECG’s, Vital signs, Blood/urine tests, reasoning and behavioural assessments, X-rays, Anaesthesia and brain MRI.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
15/LO/0898
Date of REC Opinion
10 Aug 2015
REC opinion
Further Information Favourable Opinion