A Phase 2a Study Evaluating BIVV020 in Adults With Persistent/Chronic Immune Thrombocytopenia (ITP)

• ClinicalTrials.gov Identifier: NCT04669600
• Recruitment Status: Recruiting
• First Posted: December 17, 2020
• Last Update Posted: June 4, 2021
• Sponsor: Bioverativ, a Sanofi company
• Information provided by (Responsible Party): Sanofi ( Bioverativ, a Sanofi company )

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

Condition or disease	Intervention/treatment	Phase
Immune Thrombocytopenia (ITP)	Drug: BIVV020	Phase 2

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

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 12 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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)
• Actual Study Start Date: February 4, 2021
• Estimated Primary Completion Date: January 2022
• Estimated Study Completion Date: December 2023

Arms and Interventions

Arm	Intervention/treatment
Experimental: BIVV0020; All eligible participants will receive BIVV020 for at least 52 weeks.	Drug: BIVV020; Pharmaceutical form:solution for injection

Outcome Measures

Primary Outcome Measures :

1. Proportion of participants with a durable platelet response [ Time Frame: Week 3 to Week 24 ]

   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 :

1. Number of participants with treatment emergent adverse events [ Time Frame: Week 52 ]
2. Plasma concentrations of BIVV020 [ Time Frame: Week 52 ]
3. Response rate of treatment with BIVV020 [ Time Frame: Weeks 24 and 52 ]
   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.
4. Time to first platelet response [ Time Frame: Baseline to Week 52 ]
   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)
5. 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 ]
6. Number of participants with incidence and titer (if relevant) of anti-BIVV020 antibodies [ Time Frame: Week 24, Week 52 ]

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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 patient 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 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.

Contacts and Locations

Contacts

Contact: Trial Transparency email recommended (Toll free number for US & Canada); 800-633-1610 ext option 6; Contact-US@sanofi.com

Locations

United States, District of Columbia

Investigational Site Number 8400001; Recruiting

Washington, District of Columbia, United States, 20007

United States, Florida

Investigational Site Number 8400002; Recruiting

Tamarac, Florida, United States, 33321

Czechia

Investigational Site Number 2030002; Recruiting

Ostrava, Czechia, 70852

Germany

Investigational Site Number 2760001; Recruiting

Essen, Germany, 45147

Netherlands

Investigational Site Number 5280001; Recruiting

Leiden, Netherlands, 2333 ZA

Spain

Investigational Site Number 7240002; Recruiting

La Coruña, Spain, 15006

Investigational Site Number 7240001; Recruiting

Palma De Mallorca, Spain, 07120

Investigational Site Number 7240003; Recruiting

Sevilla, Spain, 41013

United Kingdom

Investigational Site Number 8260001; Recruiting

London, United Kingdom, WC1E 6BT

Sponsors and Collaborators

Bioverativ, a Sanofi company

Investigators

• Study Director: Clinical Sciences & Operations; Sanofi

More Information

• Responsible Party: Bioverativ, a Sanofi company
• ClinicalTrials.gov Identifier: NCT04669600
• Other Study ID Numbers: PDY16894; 2020-004162-18 ( EudraCT Number ); U1111-1253-2343 ( Other Identifier: UTN )
• First Posted: December 17, 2020
• Last Update Posted: June 4, 2021
• Last Verified: June 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• 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://www.clinicalstudydatarequest.com/

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Thrombocytopenia; Immune System Diseases; Purpura, Thrombocytopenic, Idiopathic; Blood Platelet Disorders; Hematologic Diseases; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Thrombotic Microangiopathies; Hemorrhagic Disorders; Autoimmune Diseases; Hemorrhage; Pathologic Processes; Skin Manifestations