Back to resultsA Phase 2a Study Evaluating BIVV020 in Adults With Persistent/Chronic Immune Thrombocytopenia (ITP)
Primary Purpose
Immune Thrombocytopenia (ITP)
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
BIVV020
Sponsored by
About this trial
This is an interventional treatment trial for Immune Thrombocytopenia (ITP)
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:
- Platelet count ≤30 × 10^9/L on 2 occasions at least 5 days apart during the Screening Period;
- 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.
- 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
- 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.
- 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
Exclusion criteria:
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
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Sites / Locations
- Investigational Site Number :8400001
- Investigational Site Number :8400002
- Investigational Site Number :2030002
- Investigational Site Number :2760001
- Investigational Site Number :5280001
- Investigational Site Number :7240002
- Investigational Site Number :7240001
- Investigational Site Number :7240003
- Investigational Site Number :8260002
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
BIVV0020
Arm Description
All eligible participants will receive BIVV020 for at least 52 weeks.
Outcomes
Primary Outcome Measures
Proportion of participants with a durable platelet response
Durable platelet response is defined for naïve participants as the proportion of participants with a platelet count ≥50 × 109/L at ≥50% of scheduled visits, or for participants with baseline platelet count <15 × 109/L, a ≥20 × 109/L increase in platelet count from baseline at ≥50% of scheduled visits, without receiving rescue ITP therapy, as assessed from Week 3 to Week 24.
Durable platelet response is defined for participants who previously received sutimlimab as maintenance of platelet count ≥30 × 109/L at ≥50% of scheduled visits, without receiving rescue ITP therapy, as assessed from Week 3 to Week 24.
Secondary Outcome Measures
Number of participants with treatment emergent adverse events
Plasma concentrations of BIVV020
Response rate of treatment with BIVV020
Response rate at Weeks 24 and 52, defined as a platelet count ≥50 × 109/L and a greater than 2-fold increase from baseline, measured on 2 occasions at least 7 days apart, with the absence of bleeding (bleeding is defined as bleeding with a score ≥2 on the WHO bleeding scale), and the lack of combination ITP therapy during this period.
Time to first platelet response
Time from baseline to first platelet response, defined as greater than or equal to each of the following values: 50 × 109/L and 100 × 109/L (confirmed by 2 measurements at least 7 days apart)
Proportion of patients who did not require rescue therapy for an acute episode of thrombocytopenia after Week 3
Number of participants with incidence and titer (if relevant) of anti-BIVV020 antibodies
Full Information
NCT ID
NCT04669600
First Posted
December 9, 2020
Last Updated
February 13, 2023
Sponsor
Bioverativ, a Sanofi company
1. Study Identification
Unique Protocol Identification Number
NCT04669600
Brief Title
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
2. Study Status
Record Verification Date
February 2023
Overall Recruitment Status
Completed
Study Start Date
February 4, 2021 (Actual)
Primary Completion Date
February 15, 2022 (Actual)
Study Completion Date
February 7, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bioverativ, a Sanofi company
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Immune Thrombocytopenia (ITP)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)
8. Arms, Groups, and Interventions
Arm Title
BIVV0020
Arm Type
Experimental
Arm Description
All eligible participants will receive BIVV020 for at least 52 weeks.
Intervention Type
Drug
Intervention Name(s)
BIVV020
Intervention Description
Pharmaceutical form:solution for injection
Primary Outcome Measure Information:
Title
Proportion of participants with a durable platelet response
Description
Durable platelet response is defined for naïve participants as the proportion of participants with a platelet count ≥50 × 109/L at ≥50% of scheduled visits, or for participants with baseline platelet count <15 × 109/L, a ≥20 × 109/L increase in platelet count from baseline at ≥50% of scheduled visits, without receiving rescue ITP therapy, as assessed from Week 3 to Week 24.
Durable platelet response is defined for participants who previously received sutimlimab as maintenance of platelet count ≥30 × 109/L at ≥50% of scheduled visits, without receiving rescue ITP therapy, as assessed from Week 3 to Week 24.
Time Frame
Week 3 to Week 24
Secondary Outcome Measure Information:
Title
Number of participants with treatment emergent adverse events
Time Frame
Week 52
Title
Plasma concentrations of BIVV020
Time Frame
Week 52
Title
Response rate of treatment with BIVV020
Description
Response rate at Weeks 24 and 52, defined as a platelet count ≥50 × 109/L and a greater than 2-fold increase from baseline, measured on 2 occasions at least 7 days apart, with the absence of bleeding (bleeding is defined as bleeding with a score ≥2 on the WHO bleeding scale), and the lack of combination ITP therapy during this period.
Time Frame
Weeks 24 and 52
Title
Time to first platelet response
Description
Time from baseline to first platelet response, defined as greater than or equal to each of the following values: 50 × 109/L and 100 × 109/L (confirmed by 2 measurements at least 7 days apart)
Time Frame
Baseline to Week 52
Title
Proportion of patients who did not require rescue therapy for an acute episode of thrombocytopenia after Week 3
Time Frame
From Week 3 to Week 52
Title
Number of participants with incidence and titer (if relevant) of anti-BIVV020 antibodies
Time Frame
Week 24, Week 52
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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:
Platelet count ≤30 × 10^9/L on 2 occasions at least 5 days apart during the Screening Period;
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.
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
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.
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
Exclusion criteria:
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
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Sciences & Operations
Organizational Affiliation
Sanofi
Official's Role
Study Director
Facility Information:
Facility Name
Investigational Site Number :8400001
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Facility Name
Investigational Site Number :8400002
City
Tamarac
State/Province
Florida
ZIP/Postal Code
33321
Country
United States
Facility Name
Investigational Site Number :2030002
City
Ostrava - Poruba
ZIP/Postal Code
70852
Country
Czechia
Facility Name
Investigational Site Number :2760001
City
Essen
ZIP/Postal Code
45147
Country
Germany
Facility Name
Investigational Site Number :5280001
City
Leiden
ZIP/Postal Code
2333 ZA
Country
Netherlands
Facility Name
Investigational Site Number :7240002
City
La Coruña
State/Province
A Coruña [La Coruña]
ZIP/Postal Code
15006
Country
Spain
Facility Name
Investigational Site Number :7240001
City
Palma de Mallorca
ZIP/Postal Code
07120
Country
Spain
Facility Name
Investigational Site Number :7240003
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Facility Name
Investigational Site Number :8260002
City
London
State/Province
London, City Of
ZIP/Postal Code
W12 0HS
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Learn more about this trial
A Phase 2a Study Evaluating BIVV020 in Adults With Persistent/Chronic Immune Thrombocytopenia (ITP)
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