Trial Outcomes & Findings for A Phase 2a Study Evaluating BIVV020 in Adults With Persistent/Chronic Immune Thrombocytopenia (ITP) (NCT NCT04669600)
NCT ID: NCT04669600
Last Updated: 2025-09-10
Results Overview
A naive participant was a participant who did not use sutimlimab prior to enrollment. A switcher was a participant who used sutimlimab prior to enrollment. A naive participant was a responder if the platelet count was \>=50 × 10\^9/liter (L) at \>=50 percent (%) of scheduled visits, or for participants with baseline platelet count \<15 × 10\^9/L, a \>=20 × 10\^9/L increase in platelet count from baseline at \>=50% of scheduled visits, without receiving rescue immune thrombocytopenia (ITP) therapy. A switcher was a responder if the maintenance platelet count was \>=30 × 10\^9/L at \>=50% of scheduled visits, without receiving rescue ITP therapy.
COMPLETED
PHASE2
12 participants
From Week 3 to Week 24
2025-09-10
Participant Flow
The study was conducted at 8 centers in 5 countries. A total of 20 participants were screened from 04 Feb 2021 to 07 Sep 2021, of which 8 were screen failures due to not meeting eligibility criteria.
The study consisted of a screening period (up to 56 days), treatment period (up to 81 weeks), and follow-up visits (up to 22 weeks). A total of 12 participants \[either switchers: who had received and responded to sutimlimab (BIVV009) in study TDR16218 (NCT03275454) or naïve: who have not previously received sutimlimab\] were enrolled in this study.
Participant milestones
| Measure |
SAR445088
Participants received a loading dose of SAR445088 (BIVV020) 50 milligram per kilogram (mg/kg) intravenously (IV) on Day 1, followed by maintenance doses of 600 mg subcutaneous (SC) weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
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|---|---|
|
Overall Study
STARTED
|
12
|
|
Overall Study
COMPLETED
|
5
|
|
Overall Study
NOT COMPLETED
|
7
|
Reasons for withdrawal
| Measure |
SAR445088
Participants received a loading dose of SAR445088 (BIVV020) 50 milligram per kilogram (mg/kg) intravenously (IV) on Day 1, followed by maintenance doses of 600 mg subcutaneous (SC) weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
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|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Other
|
6
|
Baseline Characteristics
A Phase 2a Study Evaluating BIVV020 in Adults With Persistent/Chronic Immune Thrombocytopenia (ITP)
Baseline characteristics by cohort
| Measure |
SAR445088
n=12 Participants
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
|
|---|---|
|
Age, Continuous
|
54.7 years
STANDARD_DEVIATION 10.1 • n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From Week 3 to Week 24Population: Results are based on the overall number of participants analyzed = intent-to-treat (ITT) population which consisted of all exposed participants. Number analyzed = number of participants for each category (naive participant and switcher).
A naive participant was a participant who did not use sutimlimab prior to enrollment. A switcher was a participant who used sutimlimab prior to enrollment. A naive participant was a responder if the platelet count was \>=50 × 10\^9/liter (L) at \>=50 percent (%) of scheduled visits, or for participants with baseline platelet count \<15 × 10\^9/L, a \>=20 × 10\^9/L increase in platelet count from baseline at \>=50% of scheduled visits, without receiving rescue immune thrombocytopenia (ITP) therapy. A switcher was a responder if the maintenance platelet count was \>=30 × 10\^9/L at \>=50% of scheduled visits, without receiving rescue ITP therapy.
Outcome measures
| Measure |
SAR445088
n=12 Participants
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
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|---|---|
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Percentage of Participants With a Durable Platelet Response
Naive participant
|
0 percentage of participants
Interval 0.0 to 36.9
|
|
Percentage of Participants With a Durable Platelet Response
Switcher
|
25 percentage of participants
Interval 19.4 to 99.4
|
SECONDARY outcome
Timeframe: From first study treatment administration (Day 1) up to Week 103Population: The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment.
An AE was defined as any untoward medical occurrence in a participant temporally associated with the use of study treatment, whether or not considered related to the study treatment. SAEs were any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. TEAEs were AEs that developed after the first study treatment administration in the safety analysis period which was defined as the period from the first study intervention administration to the end of study (EOS) visit (up to Week 103).
Outcome measures
| Measure |
SAR445088
n=12 Participants
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
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|---|---|
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Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious AEs (SAE)
Any TEAE
|
8 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious AEs (SAE)
Any Treatment Emergent SAE
|
2 Participants
|
SECONDARY outcome
Timeframe: On Days 1, 15, 29, at Weeks 8, 12, 24, and then every 8 weeks until the end of study (EOS) (Week 103)Population: Overall number of participants analyzed = safety population. Number analyzed = number of participants in the 'safety population' with available data for the corresponding categories. The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. Only those participants with data available were analyzed.
Criteria for potentially clinically significant laboratory abnormalities (PCSA): White blood cells: less than (\<)3.0 Giga/L (Non-Black \[NB\]) or \<2.0 Giga/L (Black \[B\]), greater than or equal to (\>=)16.0 Giga/L; Lymphocytes: greater than (\>)4.0 Giga/L; Neutrophils: \<1.5 Giga/L (NB) or \<1.0 Giga/L (B); Monocytes: \>0.7 Giga/L; Basophils: \>0.1 Giga/L; Eosinophils: \>0.5 Giga/L or \>upper limit of normal (ULN) (if ULN \>=0.5 Giga/L); Hemoglobin: less than or equal to (\<=) 115 grams per liter (g/L) (Male\[M\]) or \<=95 g/L (Female\[F\]), \>=185 g/L (M) or \>=165 g/L (F), Decrease from baseline (DFB) \>=20 g/L; Hematocrit: \<=0.37 volume/volume (v/v) (M) or \<=0.32 v/v (F); Red blood cells (RBC): \>=6 Tera/L; Platelets:\<100 Giga/L, \>=700 Giga/L. Only the worst case during the treatment-emergent (TE) period for each participant with worsening from baseline is presented.
Outcome measures
| Measure |
SAR445088
n=12 Participants
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
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|---|---|
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Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
White blood cells: <3.0 Giga/L (NB) or <2.0 Giga/L (B)
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
White blood cells: >=16.0 Giga/L
|
3 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Lymphocytes: >4.0 Giga/L
|
2 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Neutrophils: <1.5 Giga/L (NB) or <1.0 Giga/L (B)
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Monocytes: >0.7 Giga/L
|
7 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Basophils: >0.1 Giga/L
|
3 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Eosinophils: >0.5 Giga/L or >ULN (if ULN >=0.5 Giga/L)
|
1 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Hemoglobin: <=115 g/L (M) or <=95 g/L (F)
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Hemoglobin: >=185 g/L (M) or >=165 g/L (F)
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Hemoglobin: DFB >=20 g/L
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Hematocrit: <=0.37 v/v (M) or <=0.32 v/v (F)
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
RBC: >=6 Tera/L
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Platelets: <100 Giga/L
|
11 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Platelets: >=700 Giga/L
|
0 Participants
|
SECONDARY outcome
Timeframe: On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)Population: The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment.
Criteria for PCSA: Blood Urea Nitrogen: \>=17 millimole (mmol)/L; Creatinine: \>=150 micromole (mcmol)/L (Adults), \>=30% and \<100% change from baseline, \>=100% change from baseline; Potassium: \<3 mmol/L, \>=5.5 mmol/L; Sodium: \<=129 mmol/L, \>=160 mmol/L; Aspartate Aminotransferase (AST): \>3 ULN, \>5 ULN, \>10 ULN, \>20 ULN; Alanine Aminotransferase (ALT): \>3 ULN, \>5 ULN, \>10 ULN, \>20 ULN; Alkaline Phosphatase (ALP): \>1.5 ULN; Bilirubin: \>1.5 ULN, \>2 ULN; ALT and Total Bilirubin: ALT \>3 ULN and TBILI \>2 ULN. Only the worst case during the TE period for each participant with worsening from baseline is presented.
Outcome measures
| Measure |
SAR445088
n=12 Participants
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
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|---|---|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
Bilirubin: >1.5 ULN
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
Blood Urea Nitrogen: >=17 mmol/L
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
Creatinine: >=150 mcmol/L (Adults)
|
1 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
Creatinine: >=30% and < 100% from baseline
|
4 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
Creatinine:>=100% from baseline
|
1 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
Potassium: <3 mmol/L
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
Potassium: >=5.5 mmol/L
|
1 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
Sodium:<=129 mmol/L
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
Sodium: >=160 mmol/L
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
AST: >3 ULN
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
AST: >5 ULN
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
AST: >10 ULN
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
AST: >20 ULN
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
ALT: >3 ULN
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
ALT: >5 ULN
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
ALT: >10 ULN
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
ALT: >20 ULN
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
ALP: > 1.5 ULN
|
1 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
Bilirubin: >2 ULN
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
ALT and total bilirubin: ALT>3ULN and TBILI >2ULN
|
0 Participants
|
SECONDARY outcome
Timeframe: On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)Population: Overall number of participants analyzed = Number of participants in the 'safety population' with available data for this outcome measure. Number analyzed = number of participants in the 'safety population' with available data for the corresponding categories. Some participants in 'Number Analyzed' were common for 2 or for all the 3 categories (Prothrombin time, Prothrombin International Normalized Ratio and APTT).
The number of participants with PCSA for coagulation parameters during the TE period without PCSA definition by biological function are presented. The parameters evaluated were prothrombin time, prothrombin international normalized ratio and activated partial thromboplastin time (APTT).
Outcome measures
| Measure |
SAR445088
n=6 Participants
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
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|---|---|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Coagulation
Prothrombin time: <Lower limit of normal (LLN)
|
1 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Coagulation
Prothrombin time: >ULN
|
2 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Coagulation
Prothrombin International Normalized Ratio: <LLN
|
2 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Coagulation
Prothrombin International Normalized Ratio: >ULN
|
2 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Coagulation
APTT: <LLN
|
1 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Coagulation
APTT: >ULN
|
3 Participants
|
SECONDARY outcome
Timeframe: On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)Population: The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment.
Criteria for PCSA: potential of Hydrogen (pH) \<=4.6, \>=8. Only the worst case during the TE period for each participant with worsening from baseline is presented.
Outcome measures
| Measure |
SAR445088
n=12 Participants
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
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|---|---|
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Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Urinalysis
pH: <=4.6
|
0 Participants
|
|
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Urinalysis
pH: >=8
|
1 Participants
|
SECONDARY outcome
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 weeksPopulation: The Pharmacokinetic (PK) population consisted of all enrolled and treated participants (safety population) with at least 1 post-baseline PK sample. Only those participants with data available were included in the analysis.
Plasma samples were collected at specified timepoints.
Outcome measures
| Measure |
SAR445088
n=12 Participants
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
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|---|---|
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Plasma Concentrations of SAR445088 (BIVV020)
Week 64
|
615.80 microgram/milliliter (mcg/mL)
Standard Deviation 247.91
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Day 1: 1-hour post-dose
|
1186.00 microgram/milliliter (mcg/mL)
Standard Deviation 254.24
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Day 8
|
668.09 microgram/milliliter (mcg/mL)
Standard Deviation 139.19
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Day 15
|
657.64 microgram/milliliter (mcg/mL)
Standard Deviation 107.33
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Day 29
|
631.80 microgram/milliliter (mcg/mL)
Standard Deviation 64.52
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Day 43
|
627.56 microgram/milliliter (mcg/mL)
Standard Deviation 84.39
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Week 12
|
736.78 microgram/milliliter (mcg/mL)
Standard Deviation 164.07
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Week 16
|
786.00 microgram/milliliter (mcg/mL)
Standard Deviation NA
Standard deviation could not be derived for a single participant.
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Week 24
|
781.63 microgram/milliliter (mcg/mL)
Standard Deviation 276.73
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Week 32
|
695.67 microgram/milliliter (mcg/mL)
Standard Deviation 343.75
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Week 40
|
602.33 microgram/milliliter (mcg/mL)
Standard Deviation 385.64
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Week 48
|
559.33 microgram/milliliter (mcg/mL)
Standard Deviation 300.73
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Week 56
|
602.80 microgram/milliliter (mcg/mL)
Standard Deviation 224.08
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Week 72
|
584.25 microgram/milliliter (mcg/mL)
Standard Deviation 243.37
|
|
Plasma Concentrations of SAR445088 (BIVV020)
Week 80
|
602.50 microgram/milliliter (mcg/mL)
Standard Deviation 287.79
|
|
Plasma Concentrations of SAR445088 (BIVV020)
EOS (Week 103)
|
206.46 microgram/milliliter (mcg/mL)
Standard Deviation 157.42
|
SECONDARY outcome
Timeframe: At Weeks 24 and 56Population: The ITT population consisted of all exposed participants.
A participant was a responder if the platelet count was \>=50 × 10\^9/L and there was a greater than 2-fold increase from baseline, measured on 2 occasions at least 7 days apart with the absence of bleeding \[bleeding score \>=2 on the World Health Organization (WHO) bleeding scale\] while the platelet counts were maintained above the threshold and lack of combination ITP therapy during this period. WHO bleeding scores: 1=Petechiae; 2=Mild blood loss; 3=Gross blood loss; and 4=Debilitating blood loss.
Outcome measures
| Measure |
SAR445088
n=12 Participants
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
|
|---|---|
|
Number of Responders to SAR445088 (BIVV020)
Week 24
|
2 Participants
|
|
Number of Responders to SAR445088 (BIVV020)
Week 56
|
2 Participants
|
SECONDARY outcome
Timeframe: From Baseline (Day 1) up to Week 56Population: Overall number of participants analyzed = ITT Population which consisted of all exposed participants. Number analyzed = number of participants in the 'ITT Population' who met the specified platelet counts (\>=50 x10\^9/L or \>=100 x10\^9/L) as confirmed by 2 measurements at least 7 days apart. Participants who met the criteria 'Platelet count \>=100 x10\^9/L' were the same as who met the criteria 'Platelet count \>=50 x10\^9/L'.
Time to first platelet response was defined as greater than or equal to each of the following values: 50 × 10\^9/L or 100 × 10\^9/L (confirmed by 2 measurements at least 7 days apart). It was calculated as date of first occurrence of confirmed platelet count response before rescue therapy.
Outcome measures
| Measure |
SAR445088
n=12 Participants
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
|
|---|---|
|
Time to First Platelet Response
Platelet count>=50 x 10^9/L
|
18.5 weeks
Interval 13.5 to 49.5
|
|
Time to First Platelet Response
Platelet count>=100 x 10^9/L
|
18.5 weeks
Interval 13.5 to 49.5
|
SECONDARY outcome
Timeframe: Up to Week 84Population: The ITT population consisted of all exposed participants.
Data was collected to assess the effect of treatment with SAR445088 (BIVV020) on the requirement for rescue ITP therapy.
Outcome measures
| Measure |
SAR445088
n=12 Participants
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
|
|---|---|
|
Percentage of Participants Who Did Not Require Rescue Therapy for an Acute Episode of Thrombocytopenia After Week 3
Week 3 to Week 24 during treatment period
|
75.0 percentage of participants
|
|
Percentage of Participants Who Did Not Require Rescue Therapy for an Acute Episode of Thrombocytopenia After Week 3
Week 3 to Week 56 during treatment period
|
75.0 percentage of participants
|
|
Percentage of Participants Who Did Not Require Rescue Therapy for an Acute Episode of Thrombocytopenia After Week 3
Week 3 to the end of on-treatment period
|
75.0 percentage of participants
|
SECONDARY outcome
Timeframe: Up to Week 103Population: The ADA population consisted of all enrolled and treated participants (safety population) with at least 1 post-baseline ADA sample.
Plasma samples were analyzed for the presence of ADAs for SAR445088 (BIVV020) using validated assays. Treatment-induced ADA was defined as ADAs that developed during the treatment-emergent period and without pre-existing ADA. Pre-existing ADA was defined as ADAs present in samples drawn before first study treatment administration. Treatment-boosted ADA positive was defined as pre-existing ADA (i.e., ADA positive at baseline) that was boosted at least a 9-fold increase of titer values during the treatment-emergent period than the baseline. Treatment-emergent ADA positive was defined as either treatment-induced ADA positive or treatment-boosted ADA positive during the treatment-emergent period. Inconclusive ADA was defined as the one that could not irrefutably be classified as with or without treatment-emergent ADA.
Outcome measures
| Measure |
SAR445088
n=12 Participants
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
|
|---|---|
|
Number of Participants With Anti-Drug Antibody (ADAs) Response to SAR445088 (BIVV020)
Participants with ADA negative or missing at baseline
|
11 Participants
|
|
Number of Participants With Anti-Drug Antibody (ADAs) Response to SAR445088 (BIVV020)
Participants with treatment-induced ADA
|
0 Participants
|
|
Number of Participants With Anti-Drug Antibody (ADAs) Response to SAR445088 (BIVV020)
Participants with pre-existing ADA
|
1 Participants
|
|
Number of Participants With Anti-Drug Antibody (ADAs) Response to SAR445088 (BIVV020)
Participants with treatment-boosted ADA
|
0 Participants
|
|
Number of Participants With Anti-Drug Antibody (ADAs) Response to SAR445088 (BIVV020)
Participants with treatment-emergent ADA during 24-week treatment period
|
0 Participants
|
|
Number of Participants With Anti-Drug Antibody (ADAs) Response to SAR445088 (BIVV020)
Participants with treatment-emergent ADA during 52-week treatment period
|
0 Participants
|
|
Number of Participants With Anti-Drug Antibody (ADAs) Response to SAR445088 (BIVV020)
Participants without treatment-emergent ADA
|
12 Participants
|
|
Number of Participants With Anti-Drug Antibody (ADAs) Response to SAR445088 (BIVV020)
Participants with inconclusive ADA
|
0 Participants
|
Adverse Events
SAR445088
Serious adverse events
| Measure |
SAR445088
n=12 participants at risk
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
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Infections and infestations
Appendicitis
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Nervous system disorders
Cerebrovascular Accident
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Gastrointestinal disorders
Abdominal Pain Upper
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Other adverse events
| Measure |
SAR445088
n=12 participants at risk
Participants received a loading dose of SAR445088 (BIVV020) at 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last participant enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual participant was up to 81 weeks.
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|---|---|
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Infections and infestations
Covid-19
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25.0%
3/12 • Number of events 4 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Infections and infestations
Influenza
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8.3%
1/12 • Number of events 2 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Infections and infestations
Post-Acute Covid-19 Syndrome
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Infections and infestations
Sinusitis
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Blood and lymphatic system disorders
Lymphadenopathy
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Metabolism and nutrition disorders
Decreased Appetite
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Metabolism and nutrition disorders
Hyperglycaemia
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Nervous system disorders
Aphasia
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Nervous system disorders
Burning Sensation
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8.3%
1/12 • Number of events 2 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Nervous system disorders
Dizziness
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Nervous system disorders
Syncope
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Eye disorders
Eye Irritation
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Ear and labyrinth disorders
Tinnitus
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Vascular disorders
Hypertension
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Respiratory, thoracic and mediastinal disorders
Cough
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Respiratory, thoracic and mediastinal disorders
Sinus Congestion
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Gastrointestinal disorders
Abdominal Pain
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Gastrointestinal disorders
Constipation
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Gastrointestinal disorders
Diarrhoea
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16.7%
2/12 • Number of events 2 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Gastrointestinal disorders
Dyspepsia
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Hepatobiliary disorders
Cholecystitis Chronic
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Skin and subcutaneous tissue disorders
Dermatitis Contact
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Skin and subcutaneous tissue disorders
Psoriasis
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Musculoskeletal and connective tissue disorders
Arthralgia
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Musculoskeletal and connective tissue disorders
Arthritis
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Musculoskeletal and connective tissue disorders
Back Pain
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Musculoskeletal and connective tissue disorders
Myalgia
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Musculoskeletal and connective tissue disorders
Pain In Extremity
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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General disorders
Fatigue
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25.0%
3/12 • Number of events 3 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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General disorders
Injection Site Bruising
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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General disorders
Oedema Peripheral
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Investigations
Blood Cholesterol Increased
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8.3%
1/12 • Number of events 1 • The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
The Safety population consisted of all enrolled participants who took at least 1 dose (including partial dose) of study treatment. As prespecified, AEs recorded for doses 50 mg/kg (loading dose) and 600 mg (maintenance dose) of SAR445088 are presented under the reporting group, SAR445088.
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Additional Information
Trial Transparency Team
Sanofi aventis recherche & développement
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place