Clinical trial NCT04669600: A Phase 2a Study Evaluating BIVV020 in Adults With Persistent/Chronic Immune Thrombocytopenia (ITP)
Official 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Immune Thrombocytopenia (ITP)
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2020-12-17
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Primary Objective:

- To evaluate the effect of BIVV020 on the durability of platelet response in participants with persistent/chronic immune thrombocytopenia (ITP)

Secondary Objectives:

* To assess the safety and tolerability of BIVV020
* To assess the pharmacokinetics of BIVV020
* To assess the response rate of treatment with BIVV020
* To assess the time to response
* To assess the effect of treatment with BIVV020 on the requirement for rescue ITP therapy
* To assess the immunogenicity of BIVV020

Detailed description: 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): 14 days, included as part of the 56-day Screening period. Treatment duration: Minimum 52 weeks.

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

Eligibility criteria: Inclusion criteria :

* 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:

  1. Platelet count ≤30 × 10\^9/L on 2 occasions at least 5 days apart during the Screening Period;
  2. Lack of an adequate platelet count response (as defined by maintenance of sustained platelet count ≥30 × 109/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.
  3. 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
  4. If applicable, concurrent administration of ITP medications (eg. corticosteroids, IVIg, azathioprine, danazol, cyclosporin A, mycophenolate mofetil, or thrombopoietin receptor agonists) is acceptable provided the participant has been on a stable dose for at least 1 month.
  5. 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 were sexually active with a female partner of childbearing potential

Exclusion criteria:

Participants were 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

The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Primary outcomes:
measure: Percentage of Participants With a Durable Platelet Response
timeFrame: From Week 3 to Week 24
Secondary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious AEs (SAE)
timeFrame: From first study treatment administration (Day 1) up to Week 103
measure: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
timeFrame: On Days 1, 15, 29, at Weeks 8, 12, 24, and then every 8 weeks until the end of study (EOS) (Week 103)
measure: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
timeFrame: On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)
measure: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Coagulation
timeFrame: On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)
measure: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Urinalysis
timeFrame: On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)
measure: Plasma Concentrations of SAR445088 (BIVV020)
timeFrame: 1-hour post-dose on Day 1, on Days 8, 15, 29, 43, at Weeks 12, 16, 24, 32, 40, 48, 56, 64, 72, 80 and EOS visit, up to 103 weeks
measure: Number of Responders to SAR445088 (BIVV020)
timeFrame: At Weeks 24 and 56
measure: Time to First Platelet Response
timeFrame: From Baseline (Day 1) up to Week 56
measure: Percentage of Participants Who Did Not Require Rescue Therapy for an Acute Episode of Thrombocytopenia After Week 3
timeFrame: Up to Week 84
measure: Number of Participants With Anti-Drug Antibody (ADAs) Response to SAR445088 (BIVV020)
timeFrame: Up to Week 103
Other outcomes:
Locations:
city: Washington
country: United States
facility: Investigational Site Number :8400001
state: District of Columbia
city: Tamarac
country: United States
facility: Investigational Site Number :8400002
state: Florida
city: Ostrava - Poruba
country: Czechia
facility: Investigational Site Number :2030002
city: Essen
country: Germany
facility: Investigational Site Number :2760001
city: Leiden
country: Netherlands
facility: Investigational Site Number :5280001
city: La Coruña
country: Spain
facility: Investigational Site Number :7240002
state: A Coruña [La Coruña]
city: Palma de Mallorca
country: Spain
facility: Investigational Site Number :7240001
city: Sevilla
country: Spain
facility: Investigational Site Number :7240003
city: London
country: United Kingdom
facility: Investigational Site Number :8260002
state: London, City Of
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: