Clinical Trial Results:
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)
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Summary
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EudraCT number |
2020-004162-18 |
Trial protocol |
DE CZ NL ES |
Global end of trial date |
07 Feb 2023
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Results information
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Results version number |
v2(current) |
This version publication date |
28 Mar 2024
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First version publication date |
21 Feb 2024
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Other versions |
v1 |
Version creation reason |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
PDY16894
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT04669600 | ||
WHO universal trial number (UTN) |
U1111-1253-2343 | ||
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Sponsors
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Sponsor organisation name |
Sanofi aventis recherche & développement
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Sponsor organisation address |
1 avenue Pierre Brossolette, Chilly-Mazarin, France, 91380
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Public contact |
Trial Transparency Team, Sanofi aventis recherche & développement, Contact-US@sanofi.com
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Scientific contact |
Trial Transparency Team, Sanofi aventis recherche & développement, Contact-US@sanofi.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
05 May 2023
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
07 Feb 2023
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
To evaluate the effect of SAR445088 on the durability of platelet response in subjects with persistent/chronic immune thrombocytopenia (ITP).
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Protection of trial subjects |
Subjects were fully informed of all pertinent aspects of clinical trial as well as the possibility to discontinue at any time in language and terms appropriate for the subject and considering the local culture. During the course of the trial, subjects were provided with individual subject cards indicating the nature of the trial the subject is participating, contact details and any information needed in the event of
a medical emergency.
Collected personal data and human biological samples were processed in compliance with the Sanofi-Aventis Group Personal Data Protection Charter ensuring that the Group abides by the laws governing personal data protection in force in all countries in which it operates.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
04 Feb 2021
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Germany: 1
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Country: Number of subjects enrolled |
Netherlands: 2
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Country: Number of subjects enrolled |
Spain: 2
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Country: Number of subjects enrolled |
United States: 7
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Worldwide total number of subjects |
12
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EEA total number of subjects |
5
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
10
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From 65 to 84 years |
2
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85 years and over |
0
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Recruitment
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Recruitment details |
The study was conducted at 8 centres in 5 countries. A total of 20 subjects were screened from 04 Feb 2021 to 07 Sep 2021, of which 8 were screen failures due to not meeting eligibility criteria. | ||||||||||||
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Pre-assignment
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Screening details |
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 subjects [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. | ||||||||||||
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Period 1
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Period 1 title |
Overall study (overall period)
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Is this the baseline period? |
Yes | ||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||
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Arms
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Arm title
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SAR445088 | ||||||||||||
Arm description |
Subjects 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 subject enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual subject was up to 81 weeks. | ||||||||||||
Arm type |
Experimental | ||||||||||||
Investigational medicinal product name |
SAR445088
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Investigational medicinal product code |
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Other name |
BIVV020
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Pharmaceutical forms |
Concentrate for solution for injection/infusion
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Routes of administration |
Intravenous use, Subcutaneous use
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Dosage and administration details |
BIVV020 was supplied in single use vial and administered as a loading dose of 50 mg/kg IV infusion on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last subject completed 52 weeks of treatment.
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Baseline characteristics reporting groups
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Reporting group title |
SAR445088
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Reporting group description |
Subjects 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 subject enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual subject was up to 81 weeks. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
SAR445088
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Reporting group description |
Subjects 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 subject enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual subject was up to 81 weeks. | ||
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End point title |
Percentage of Subjects With a Durable Platelet Response [1] | ||||||||||||
End point description |
A naive subject was a subject who did not use sutimlimab prior to enrollment. A switcher was a subject who used sutimlimab prior to enrollment. A naive subject was a responder if the platelet count was >=50 × 10^9/litre (L) at >=50 percent (%) of scheduled visits, or for subjects 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 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. Results are based on the number of subjects analysed = intent-to-treat (ITT) population which consisted of all exposed subjects. Number analysed (n) = number of subjects for each category (naive subject and switcher).
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End point type |
Primary
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End point timeframe |
From Week 3 to Week 24
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: As the endpoint is descriptive in nature, no statistical analysis is provided. |
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious AEs (SAEs) | ||||||||||||
End point description |
An AE was defined as any untoward medical occurrence in a subject 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 hospitalisation or prolongation of existing hospitalisation, 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). The Safety population consisted of all enrolled subjects who took at least 1 dose (including partial dose) of study treatment.
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End point type |
Secondary
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End point timeframe |
From first study treatment administration (Day 1) up to Week 103
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Number of Subjects With Potentially Clinically Significant Laboratory Abnormalities: Hematology | ||||||||||||||||||||||||||||||||||||
End point description |
Criteria for potentially clinically significant laboratory abnormalities: White blood cells (WBCs): less than (<)3.0 Giga (G)/L(Non-Black [NB]) or <2.0 G/L(Black [B]),greater than or equal to(>=)16.0 G/L; Lymphocytes: greater than (>)4.0 G/L; Neutrophils:<1.5 G/L (NB) or <1.0 G/L (B);Monocytes:>0.7 G/L; Basophils:>0.1 G/L; Eosinophils:>0.5 /L or >upper limit of normal(ULN)(if ULN >=0.5 G/L);Hemoglobin: less than or equal to(<=)115 grams (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 G/L, >=700 G/L. Number of subjects analysed=safety population. Number analysed (n) = number of subjects in 'safety population' with available data for the corresponding categories. Safety population consisted of all enrolled subjects who took at least 1 dose (including partial dose) of study treatment.
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End point type |
Secondary
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End point timeframe |
On Days 1, 15, 29, at Weeks 8, 12, 24, and then every 8 weeks until the end of study (EOS) (Week 103)
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||
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End point title |
Number of Subjects With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry | ||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
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 (TBILI): ALT >3 ULN and TBILI >2 ULN. Only the worst case during the TE period for each subject with worsening from baseline is presented. The Safety population consisted of all enrolled subjects who took at least 1 dose (including partial dose) of study treatment.
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End point type |
Secondary
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End point timeframe |
On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||
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End point title |
Number of Subjects With Potentially Clinically Significant Laboratory Abnormalities: Coagulation | ||||||||||||||||||||
End point description |
The number of subjects with PCSA for coagulation parameters during the TE period without PCSA definition by biological function are presented. The parameters evaluated were prothrombin time (PT), prothrombin international normalised ratio (INR) and activated partial thromboplastin time (APTT). The Safety population consisted of all enrolled subjects who took at least 1 dose (including partial dose) of study treatment. Number of subjects analysed = Number of subjects in the 'safety population' with available data for this outcome measure. Number analysed (n) = Number of subjects in the 'safety population' with available data for the corresponding categories. Some subjects in 'Number Analysed' were common for 2 or for all the 3 categories (Prothrombin time, Prothrombin International Normalized Ratio and APTT).
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End point type |
Secondary
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End point timeframe |
On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)
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| No statistical analyses for this end point | |||||||||||||||||||||
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End point title |
Number of Subjects With Potentially Clinically Significant Laboratory Abnormalities: Urinalysis | ||||||||||||
End point description |
Criteria for PCSA: potential of Hydrogen (pH) <=4.6, >=8. Only the worst case during the TE period for each subject with worsening from baseline is presented. The Safety population consisted of all enrolled subjects who took at least 1 dose (including partial dose) of study treatment.
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End point type |
Secondary
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End point timeframe |
On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Plasma Concentrations of SAR445088 (BIVV020) | ||||||||||||||||||||||||||||||||||||||||
End point description |
Plasma samples were collected at specified timepoints. The Pharmacokinetic (PK) population consisted of all enrolled and treated subjects (safety population) with at least 1 post-baseline PK sample. Only those subjects with data available were included in the analysis and are denoted by 'n' in the category titles. Here '99999'= Standard deviation could not be derived for a single subject.
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End point type |
Secondary
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End point 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
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||
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End point title |
Number of Responders to SAR445088 (BIVV020) | ||||||||||||
End point description |
A subject 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. The ITT population consisted of all exposed subjects.
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End point type |
Secondary
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End point timeframe |
At Weeks 24 and 56
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Time to First Platelet Response | ||||||||||||
End point description |
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. Number of subjects analysed = ITT Population which consisted of all exposed subjects. Number analysed (n) = number of subjects 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. Subjects who met the criteria ‘Platelet count >=100 x10^9/L’ were the same as who met the criteria ‘Platelet count >=50 x10^9/L’.
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End point type |
Secondary
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End point timeframe |
From Baseline (Day 1) up to Week 56
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Percentage of Subjects who did not Require Rescue Therapy for an Acute Episode of Thrombocytopenia After Week 3 | ||||||||||||||
End point description |
Data was collected to assess the effect of treatment with SAR445088 (BIVV020) on the requirement for rescue ITP therapy. The ITT population consisted of all exposed subjects.
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End point type |
Secondary
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End point timeframe |
Up to Week 84
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| No statistical analyses for this end point | |||||||||||||||
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End point title |
Number of Subjects With Anti-Drug Antibody (ADAs) Response to SAR445088 (BIVV020) | ||||||||||||||||||||||||
End point description |
Plasma samples were analysed for the presence of ADAs for SAR445088 (BIVV020) using validated assays. Treatment-induced ADA was defined as ADAs that developed during the TE 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 TE period than the baseline. The TE ADA positive was defined as either treatment-induced ADA positive or treatment-boosted ADA positive during the TE period. Inconclusive ADA was defined as the one that could not irrefutably be classified as with or without TE ADA. The ADA population consisted of all enrolled and treated subjects (safety population) with at least 1 post-baseline ADA sample. Negative: -ve; Treatment period: TP.
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End point type |
Secondary
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End point timeframe |
Up to Week 103
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| No statistical analyses for this end point | |||||||||||||||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
The TEAEs were collected from first study treatment administration (Day 1) up to Week 103
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Adverse event reporting additional description |
The Safety population consisted of all enrolled subjects 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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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
25.1
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Reporting groups
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Reporting group title |
SAR445088
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Reporting group description |
Subjects received a loading dose of SAR445088 (BIVV020) 50 mg/kg IV on Day 1, followed by maintenance doses of 600 mg SC weekly starting on Day 8 until the last subject enrolled completed 52 weeks of treatment. The maximum duration of treatment for an individual subject was up to 81 weeks. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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08 Dec 2020 |
Changes in protocol were implemented to address requests received in a Voluntary Harmonisation Procedure communication. Added limit as a safety measure and added further information to support likelihood of long-term safety. Specified additional safety monitoring. Added mention of Data Monitoring Committee to synopsis, renamed “Data Monitoring Committee” subheading as “Study Monitoring Committee,” and added a brief description of the Data Monitoring Committee, which included at least 2 investigators participating in the study. Required contraception extended due to the prolonged half-life of BIVV020. Additional details for investigational medicinal product administration added as per Health Authority request. Added statement referring to the Pharmacy Manual for procedure details. Clarified that thrombopoietin agonists may be restarted by the Investigator after BIVV020 administration if a subject has an insufficient response to BIVV020. Added guidance for thrombocytosis. Clarified that the Declaration of Helsinki guidelines referenced are the most recent guidelines from 2013. Double-barrier contraception removed as a highly effective contraceptive method as per the Clinical Trial Facilitation Group. |
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10 Dec 2020 |
The protocol was amended to address requests received from the United States Food and Drug Administration as well as for additional corrections and clarifications. Pre-dose weight added on Day 1. Clarified that clear titer cutoffs associated with protection do not exist for most of the required vaccines. Clarified an inclusion criterion: For subjects who have not previously received sutimlimab, one of their prior treatments must have been a thrombopoietin receptor agonist. Clarified that vaccinations, such as the seasonal influenza vaccine, are permitted during the study. Clarified that a dose increase in a concomitant ITP medication is considered rescue therapy. Clarified the testing to be performed in case of hypersensitivity/allergic reaction. Additional requirements and considerations for AE/SAE reporting clarified for subjects transitioning from study TDR16218. Addition of a dosing and enrollment hold for toxicity. Thrombopoietin removed from clinical chemistry panel as it was not essential for study and could not be performed through the central laboratory. Additional clarifications and corrections to typography and formatting to maintain consistency with document standards. |
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Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
