RA101495 Open-Label Study of Paroxysmal Nocturnal Haemoglobinuria
Research type
Research Study
Full title
A Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria
IRAS ID
211876
Contact name
Anita Hill
Contact email
Sponsor organisation
Ra Pharmaceuticals, Inc.
Eudract number
2016-003522-16
Duration of Study in the UK
1 years, 2 months, 1 days
Research summary
The study drug to be investigated is RA101495, which is being developed for paroxysmal nocturnal haemoglobinuria (PNH). PNH is a rare disease of the blood which is characterised by the destruction of red blood cells and blood clots in veins.
The study will include two separate groups of patients based on prior history with eculizumab which is the standard treatment:
Group A, will include patients who have not received eculizumab for treatment of PNH.
Group B will include patients who have received treatment with eculizumab for at least 6 months prior to Screening.
The planned enrolment is approximately 8-12 participants in Group A and approximately 6-8 participants in Group B, for a total of up to 20 participants.
Group A will start the enrolment of the participants prior to Group B. Group B will not be opened until safety and efficacy of the drug from at least 2 participants completing the Week 2 visit in group A has been reviewed by the Sponsor and Study Investigators, and evidence of haemolysis (breakdown in red blood cells) suppression is demonstrated.The total duration of the study for all participants will include a Screening Period of up to 8 weeks and a 12-week Treatment Period for a total of up to 20 weeks in multiple sites in Europe, Australia and Canada.
During the Treatment Period, the participants will return to the clinic weekly for the first 4 weeks followed by visits every 2 weeks to evaluate the safety, tolerability, efficacy, Pharmacokinetics (what the body does to the drug) and Pharmacodynamics (drug does to the body).
The study is being funded by Ra Pharmaceuticals, Inc.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
16/YH/0503
Date of REC Opinion
24 Jan 2017
REC opinion
Further Information Favourable Opinion