PDY16894- ITP
Research type
Research Study
Full title
A multicenter, phase 2a, open label, non-randomized study evaluating the efficacy, safety, and tolerability of BIVV020 in adults with persistent/chronic immune thrombocytopenia (ITP)
IRAS ID
288502
Contact name
Paolo Caferra
Contact email
Sponsor organisation
Sanofi-Aventis Recherche & Développement
Eudract number
2020-004162-18
Duration of Study in the UK
2 years, 1 months, 4 days
Research summary
Research Summary
This is a single group treatment, Phase 2a, open-label, single arm study of BIVV020 in male and female participants with persistent/chronic ITP.
Study duration:
• Screening Period: up to 56 days
• Transition Period between last sutimlimab dose and first dose of BIVV020 (for
participants who were previously receiving sutimlimab): at least 14 days, included as part of the 56-day Screening Period.
Treatment duration: 52 weeks (minimum)
Visit frequency:
• Day 1
• Day 4
• Weeks 1 to 6: Weekly
• Weeks 7 to 12: Every other week
• Weeks 13 to 24: Every 4 weeks
• Weeks 25+: At least every 8 weeks
• End of Study visit: 22 weeks after the last dose of BIVV020
Number of participants:
Approximately 12 participants will be enrolled. Since there is no statistical hypothesis to be tested in this study.
Intervention groups and duration:
All participants will receive the study intervention.
Study intervention: BIVV020
Investigational medicinal product:
• Formulation: BIVV020 supplied in single use vials containing 750 mg (150 mg/mL;
extractable volume of 5 mL)
• Routes of administration: IV loading dose; SC maintenance administration
• Dose regimen: 50 mg/kg IV loading dose, followed by 600 mg SC weekly
Participants will continue to receive BIVV020 until the last participant enrolled has completed 52 weeks of treatment. After that time, a long-term extension study may be available for participants to continue to receive BIVV020.
• Interim analysis
An interim analysis will be conducted when approximately 12 participants have each been treated for 15 weeks.
• Primary endpoint
The proportion (and number) of responders fulfilling the criteria in the primary endpoint will be presented; the 95% confidence interval for the proportion of responders will be estimated with Clopper-Pearson method.
• Secondary endpoints
All secondary endpoints will be summarized using descriptive statistics.Summary of results
There were no clinically meaningful changes in either the vital signs, clinical laboratory parameters, or ECG parameters during the study. Overall, SAR445088 was well-tolerated in ITP participants. The majority of the participants discontinued the study early for reasons other than safety concerns. As a result, the primary efficacy endpoint for the study was not met. The Sponsor has decided to discontinue development of SAR445088 for the ITP. This Clinical Study Report is written as an abbreviated report.
REC name
London - London Bridge Research Ethics Committee
REC reference
20/LO/1278
Date of REC Opinion
21 Dec 2020
REC opinion
Further Information Favourable Opinion