Phase 1 BAX 826 Dose-Escalation Safety Study, Protocol 291501
Research type
Research Study
Full title
A Phase 1, Prospective, Open Label, Two Period, Fixed Sequence, Dose-Escalation Study of the PK and Safety of BAX 826 (PSA-rFVIII) in Previously Treated Patients with Severe (FVIII <1%) Hemophilia A
IRAS ID
195532
Contact name
Pratima Chowdary
Contact email
Sponsor organisation
Baxalta Innovations GmBH
Eudract number
2015-004079-60
Duration of Study in the UK
1 years, 0 months, 25 days
Research summary
This is a Phase 1, open label, first in humans of study drug BAX826. BAX826 produced to last longer in the blood and is a recombinant Factor VIII (rFVIII) being developed for the treatment of haemophilia A and is based on the approved rFVIII product, ADVATE, which is widely prescribed for the treatment of haemophilia. The objective of this study is to assess the tolerability and safety of BAX 826 following a single sub-cutaneous injection and to determine the pharmacokinetics (PK) of BAX 826 compared with ADVATE.
Participants are to remain on their standard treatment (during the screening and follow up period after the PK assessment) and will undergo a minimum 4-day (96-hour) washout period prior to the first ADVATE infusion. Similarly, participants will revert back to their standard treatment if they have a bleeding episode and then undergo a minimum 4-day (96-hour) washout period before repeating the appropriate study regimen.
The study has 3 distinct cohorts, where they will receive an infusion of either 25 ±3 IU/kg, 50 ±5 IU/kg or 75 ±5 IU/kg of ADVATE, following the ADVATE infusion, the patient will undergo a minimum 4-day (96-hour) washout period, which includes a 3-day PK evaluation. Following the washout period, patients will receive a single dose of BAX 826, equivalent to the ADVATE dose they have received, followed by a 7-day PK evaluation. In Cohort 1 the first dose of BAX 826 (25 IU/kg) will be administered to the first 3 participants with a minimum 24-hour staggered interval and safety in these 3 participants will be monitored closely over a minimum of 24 hours of in-hospital observation. Further participants in Cohort 1 will only be dosed after it has been confirmed that there were no post infusion reactions or safety issues for the first 3 participants. After the BAX 826 infusion, participants will be monitored for safety on Day 1 Visit 3, Day 4 Visit 3, Day 8 Visit 3, Day 14 Visit 3 and at the termination visit (6 weeks±4 days; Visit 4). Safety assessments at these time points will include vital signs and clinical laboratory assessments of haematology and chemistry and recording of adverse events (AEs). The findings of this study will determine the recommended dose of BAX 826 to be used in phase 2/3 studies, based on a risk-benefit analysis of safety and PK data generated throughout all 3 cohorts of phase 1.REC name
London - City & East Research Ethics Committee
REC reference
16/LO/0022
Date of REC Opinion
21 Jan 2016
REC opinion
Favourable Opinion