Individualized prophylaxis with Human cl rhFVIII in haemophilia A
Research type
Research Study
Full title
Prospective, open-label, multicentre phase 3b study to assess the efficacy and safety of individually tailored prophylaxis with Human-cl rhFVIII in previously treated adult patients with severe haemophilia A
IRAS ID
130927
Contact name
Savita Rangarajan
Contact email
Sponsor organisation
Octapharma AG
Eudract number
2013-001556-35
ISRCTN Number
n/a
Clinicaltrials.gov Identifier
n/a
Research summary
This is a phase 3 international study investigating a new factor VIII product called Human-cl rhFVIII in patients with severe Heamophilia A.
Haemophilia A is an inherited sex-linked coagulation disorder in which affected males do not produce functional coagulation factor VIII (FVIII) in sufficient quantities, leading to bleeding episodes. People with haemophilia A are treated either with factor VIII products that are made from donated human blood or with ’recombinant’ factor VIII products made using hamster cells. Human-cl rhFVIII is the first form of recombinant factor VIII produced in human cells. It is hoped that Human cl rhFVIII is less likely to give rise to an immunogenic reaction and the development of inhibitors compared to products derived from hamster cells.
The study has three phases. In the PK Evaluation Phase the first dose of Human-cl rhFVIII will be infused and the aim will be to find out how the body uses study drug. This will take 3 days. Prophylactic Treatment—Phase I will take 1 to 3 months. In this phase, the study drug will be infused at home every other day or 3x/week to allow the doctor to determine the dose and treatment interval for the next phase based on the results of the PK evaluation. Prophylactic Treatment—Phase II will take 6 months. Human cl rhFVIII will be infused at the dose and time interval determined by the doctor.
Fifty-five patients with severe haemophilia A from several study centres in Europe will be enrolled in this study. The overall duration of the study will be between 7 and 9 months. The study is sponsored by Octapharma.REC name
South Central - Oxford C Research Ethics Committee
REC reference
13/SC/0325
Date of REC Opinion
23 Jul 2013
REC opinion
Further Information Favourable Opinion