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)
- Conditions
- immune thrombocytopeniaITP100644771002142910003816
- Registration Number
- NL-OMON52011
- Lead Sponsor
- Genzyme Europe BV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 2
Male and female participants >=18 years of age at the time of signing the
informed consent
- Confirmed diagnosis of primary ITP; for participants who previously
received sutimlimab in study TDR16218 (NCT03275454), a response to
sutimlimab must have been obtained, as defined by platelet count >=30 ×
10^9/L on 2 visits at least 7 days apart
- For participants who have not previously received sutimlimab:
persistent/chronic ITP (ITP lasting for >= 6 months) and all the following
conditions:
a) Platelet count <=30 × 10^9/L on 2 occasions at least 5 days apart during
the Screening Period;
b) Lack of an adequate platelet count response (as defined by
maintenance of sustained platelet count >=30 × 10^9/L in the absence of
bleeding) to at least 2 ITP treatments, 1 of which was a thrombopoietin
receptor agonist. Other ITP treatments include: IVIg, anti-D immunoglobulin,
corticosteroids, splenectomy, rituximab,
cyclophosphamide, azathioprine, danazol, cyclosporin A, mycophenolate mofetil,
or fostamatinib;
c) If receiving weekly thrombopoietin receptor agonist dosing, the last dose
must have been administered >=7 days before the first dose of BIVV020. If
receiving daily thrombopoietin receptor agonist dosing, the last dose must have
been administered >=24 hours before the first dose of
BIVV020;
d) If applicable, concurrent administration of ITP medications (eg.
corticosteroids, IVIg, azathioprine, danazol, cyclosporin A, mycophenolate
mofetil, or thrombopoietin receptor agonists) is acceptable provided the
patient has been on a stable dose for at least 1 month;
e) If previously dosed with rituximab, the last dose of rituximab must have
been administered at least 12 weeks before the first dose of BIVV020
- Documented vaccinations against encapsulated bacterial pathogens (Neisseria
meningitidis, including serogroup B where available, Haemophilus influenzae,
and Streptococcus pneumoniae) within 5 years of enrollment
- Contraceptive use for women of childbearing potential and men who are
sexually active with a female partner of childbearing potential
Participants are excluded from the study if any of the following criteria apply:
- Clinically significant medical history or ongoing chronic illness that
would jeopardize the safety of the participant or compromise the quality of
the data derived from his/her participation in the study
- Clinical diagnosis of SLE
- Clinically relevant infection within the month prior to enrollment
- History of venous or arterial thrombosis within the year prior to
enrollment
- Secondary ITP from any cause including lymphoma, chronic
lymphocytic leukemia, and drug-induced thrombocytopenia
- Positive hepatitis B surface antigen (HBsAg) or active HCV
infection
- HIV infection
- Pregnant or lactating women
- Hemoglobin level <10 g/dL
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Naïve participants: Proportion of participants with<br /><br>a platelet count >=50 × 109/L at >=50% of scheduled<br /><br>visits, or for participants with baseline platelet count<br /><br><15 × 109/L, a >=20 × 109/L increase in platelet<br /><br>count from baseline at >=50% of scheduled visits,<br /><br>without receiving rescue ITP therapy, as assessed<br /><br>from Week 3 to Week 24.<br /><br>• Participants who previously received sutimlimab:<br /><br>Proportion of participants with maintenance of<br /><br>platelet count >=30 × 109/L at >=50% of scheduled<br /><br>visits, without receiving rescue ITP therapy, as<br /><br>assessed from Week 3 to Week 24.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Standard clinical and laboratory parameters and<br /><br>adverse events<br /><br>• Plasma concentrations of BIVV020<br /><br>• Response rate at Weeks 24 and 52, defined as<br /><br>a platelet count >=50 × 109/L and a greater than<br /><br>2 -fold increase from baseline, measured on<br /><br>2 occasions at least 7 days apart, with the absence<br /><br>of bleeding (bleeding is defined as bleeding with<br /><br>a score >=2 on the WHO bleeding scale), and the<br /><br>lack of combination ITP therapy during this period.<br /><br>• Time from baseline to first platelet response,<br /><br>defined as greater than or equal to each of the<br /><br>following values: 50 × 109/L and 100 × 109/L<br /><br>(confirmed by 2 measurements at least 7 days<br /><br>apart)<br /><br>• Proportion of participants who did not require<br /><br>rescue therapy for an acute episode of<br /><br>thrombocytopenia after Week 3<br /><br>• Incidence and titer (if relevant) of anti-BIVV020<br /><br>antibodies</p><br>