A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS)
Research type
Research Study
Full title
CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS)
IRAS ID
187262
Contact name
Simon Jones
Contact email
Sponsor organisation
PTC Therapeutics Inc.
Eudract number
2015-003105-41
Duration of Study in the UK
0 years, 11 months, 30 days
Research summary
Mucopolysaccharidosis type I (MPS I) is a disease caused by lack of a critical enzyme (chemical) in the body which is required for breaking down large sugar molecules called glycosaminoglycans (GAGs). Without this enzyme, GAGs build up in the body causing organ and tissue damage. It has been estimated that the severe form of MPS I, which is the most common, occurs in 1 in 100,000 births and is characterised by signs such as delayed mental development, impaired vision and hearing, and bone abnormalities resulting in resticted movement.
There is currently no cure for MPS I. Current treatments include bone marrow or stem cell transplantation (BMT/HSCT) to provide patients with new cells that produce the missing enzyme, and enzyme replacement therapy (ERT) whereby the missing enzyme is injected in to the patient to ease symptoms. Both procedures target many of the clinical features of MPS I, however there are serious risks involved with bone marrow transplant, and ERT does not have the ability to treat brain and bone abnormalities associated with the disease.
This study is being carried out to evaluate the safety of ataluren, a powder substance developed by PTC Therapeutics, and how the drug is broken down when taken orally in patients with MPS I.
There is no placebo for this open-label trial. All participants will receive 10 mg/kg ataluren in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for 12 weeks. Patients on ERT will receive further ataluren treatment for an additional 4 weeks.
Both the participant and the study staff know the drug and dosage that is being taken.
It is anticipated that at least 5 patients ≥2 years old will be enrolled in the UK.REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
15/YH/0402
Date of REC Opinion
24 Sep 2015
REC opinion
Favourable Opinion