IDES IN ASYMPTOMATIC ANTIBODY-MEDIATED TTP PATIENTS WITH LOW ADAMTS13
Research type
Research Study
Full title
A PHASE II PILOT STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACODYNAMICS AND PHARMACOKINETICS OF IDES IN ASYMPTOMATIC ANTIBODY-MEDIATED THROMBOTIC THROMBOCYTOPENIC PURPURA (TTP) PATIENTS WITH LOW ADAMTS13 ACTIVITY
IRAS ID
203461
Contact name
Marie Scully
Contact email
Sponsor organisation
Hansa Medical AB
Eudract number
2016-000249-30
Duration of Study in the UK
1 years, 3 months, 0 days
Research summary
We want to study a new drug called IdeS, which is an enzyme originally discovered in a common bacteria. This enzyme is known to break down Immunoglobulin G, an antibody in the human blood that is part of the human immune system.
We plan to find out if IdeS is safe and well tolerated when given to patients with asymptomatic antibody mediated TTP. We will also investigate whether IdeS has the potential to increase the activity of an enzyme, ADAMTS13, which is deficient in patients with TTP.
Patients will be treated at University College London Hospital and they will receive a single 30-minute infusion of IdeS into a vein. Blood samples will be taken over a 24-hour period following infusion to investigate the safety and efficacy of IdeS. Twenty four hours after dosing the patients will be discharged from the clinic. The six follow-up visits will be in the outpatient setting.
In order to minimise the risk of any allergic reaction associated with the infusion of IdeS, the patients will receive paracetamol, antihistamine and corticosteroids before the infusion. Before IdeS dosing all patients will also receive antibiotics to minimise any risk of infection, as a consequence of IdeS clearing natural antibodies.
The study will include 8 visits in total (1 screening visit, 1 dosing visit including a residential stay of one night and 6 follow-up visits) until 64 days after dosing.
We plan to treat a total of 6 patients with this rare condition. The first 3 patients will receive a dose 0.25 mg/kg. This will be evaluated for safety and efficacy and there will be a potential to increase the IdeS dose to 0.5 mg/kg in the remaining 3 patients.REC name
London - City & East Research Ethics Committee
REC reference
16/LO/0951
Date of REC Opinion
14 Jul 2016
REC opinion
Further Information Favourable Opinion