Sarizotan in Patients with Rett Syndrome with Respiratory Symptoms
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled, Six-Month Study to Evaluate the Efficacy, Safety and Tolerability of Sarizotan in Patients with RETT Syndrome with Respiratory Symptoms Protocol: 001II2015
IRAS ID
215456
Contact name
Paramala Santosh
Contact email
Sponsor organisation
Newron Pharmaceuticals S.p.A.
Eudract number
2015-004448-20
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
EMA/430803/2015, Orphan drug designation number; FDA/15-4840, Orphan drug designation number; 125,510, IND No
Duration of Study in the UK
1 years, 6 months, 2 days
Research summary
Rett syndrome (RTT) is a rare condition that affects the development of the brain and other parts of the body. It can cause severe physical and mental disability that begins in early childhood. Breathing problems are very common and can be life threatening in RTT patients.
Initial 24-Week Double-blind Treatment Period:
This is a 24-week, randomised, double-blind, placebo-controlled study designed to evaluate the safety, tolerability, and efficacy of multiple oral doses of sarizotan in patients with Rett Syndrome (RTT) with respiratory abnormalities.At least 129 patients globally (13+yrs) will be randomised equally (approximately 43/group) to receive either sarizotan 5 mg twice daily (bid), sarizotan 10 mg (bid) or placebo (bid). It is anticipated that the UK will recruit 10 patients.
24-Week Double-blind Extension Treatment Period:
All patients who have completed the final evaluations at Week 24, have no safety or tolerability issues that would preclude continuing on the study medication, have been compliant with the trial requirements, and have not worsened as judged by the Investigator, will have the option of continuing double-blind treatment with sarizotan for an additional 24 weeks. At the end of the initial 6-month period, patients randomised to sarizotan 5 or 10 mg (bid) will be treated with their current dose. Patients randomised to placebo will be randomly switched (1:1) to either sarizotan 5 mg bid or 10 mg (bid).Patients who complete 12 months of treatment, and are doing well from a safety and tolerability point of view, will be eligible to continue treatment with sarizotan in an open-label extension study.
The following assessments will be included: Vital signs, Electrocardiogram (ECGs), Physical, Neurological and Eye examinations, Blood and Urine sampling, Respiratory Monitoring in the clinic and at home, and Caregiver Questionnaires.
REC name
London - Riverside Research Ethics Committee
REC reference
16/LO/1919
Date of REC Opinion
28 Nov 2016
REC opinion
Further Information Favourable Opinion