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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 28 October 2024
Main ID:  EUCTR2020-003880-24-IT
Date of registration: 04/06/2021
Prospective Registration: Yes
Primary sponsor: SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
Public title: Safety, pharmacokinetics, and efficacy of subcutaneous isatuximab in adults with warm autoimmune hemolytic anemia
Scientific title: A multicenter, open-label, non-randomized, Phase 1b/2 study to evaluate the safety, pharmacokinetics,and efficacy of subcutaneous isatuximab in adults with warm autoimmune hemolytic anemia - .
Date of first enrolment: 07/06/2021
Target sample size: 23
Recruitment status: Not Recruiting
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-003880-24
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: yes Other trial design description: Open-label If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1  
Phase:  Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no
Countries of recruitment
Belgium France Germany Hungary Italy Netherlands United Kingdom United States
Contacts
Name: Contact point   
Address:  Viale L. Bodio, 37/B 20158 Milano Italy
Telephone: 0239394168
Email: informazioni.medicoscientifiche@sanofi.com
Affiliation:  Sanofi S.r.l.
Name: Contact point   
Address:  Viale L. Bodio, 37/B 20158 Milano Italy
Telephone: 0239394168
Email: informazioni.medicoscientifiche@sanofi.com
Affiliation:  Sanofi S.r.l.
Key inclusion & exclusion criteria
Inclusion criteria:
- Participant must be >/=18 to years of age, inclusive, at the time of signing the informed consent.
- Males and females with a confirmed diagnosis of primary w AIHA or systemic lupus erythematosus (SLE)-associated w AIHA (without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations) who meet the following criteria:
a) Hemoglobin level <10 g/dL at screening.
b) Hemolysis (haptoglobin c) Positive direct antiglobulin test (DAT) (IgG or IgG + complement C3d pattern or IgM warm autoantibodies (positive dual DAT)).
- Participants who have previously failed to maintain a sustained response after treatment with corticosteroids (corticosteroid-refractory or corticosteroid-dependent primary wAIHA).
- Part A only: Participants who have previously failed to maintain a sustained response after treatment with rituximab (or other anti-CD20 monoclonal antibodies). The last dose of the anti-CD20 antibody must have been administered at least 12 weeks before enrollment.
- Part B: Participants who have had an insufficient response to at least 1 prior therapy in addition to corticosteroids (splenectomy is regarded as a prior therapy).
- Contraceptive use by men and women
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 17
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 6

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 or her participation in the study as determined by the Investigator.
- Serious infection that required hospitalization within 3 months prior to enrollment.
- Secondary wAIHA from any cause including drugs, lymphoproliferative disorders, infectious or autoimmune disease (SLE without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations is allowed), or active hematologic malignancies. Participants with positive antinuclear antibodies but without a definitive diagnosis of an autoimmune disease are allowed.
- History of coagulation or bleeding disorders (Evans Syndrome is allowed).
- Uncontrolled or active HBV or HCV infection
- HIV infection.
- Serum gammaglobulin levels <3 g/L.
- Females who are pregnant, lactating, or considered unreliable with respect to contraceptive practice.
- Concurrent treatment with corticosteroids, unless the participant has been on a stable daily dose for >/= 30 days prior to enrollment.
- Treatment with cyclophosphamide within 4 weeks prior to enrollment.
- Treatment with cytotoxic drugs (other than cyclophosphamide) within 12 weeks prior to enrollment.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Warm autoimmune hemolytic anemia
MedDRA version: 20.0 Level: PT Classification code 10047822 Term: Warm type haemolytic anaemia System Organ Class: 10005329 - Blood and lymphatic system disorders
Intervention(s)

Product Name: Montelukast
Product Code: [.]
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: MONTELUKAST
Current Sponsor code: .
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-

Product Name: Isatuximab
Product Code: [SAR650984]
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: Isatuximab
Current Sponsor code: SAR650984
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 140-

Product Name: Paracetamolo
Product Code: [.]
Pharmaceutical Form: Tablet
INN or Proposed INN: PARACETAMOLO
Current Sponsor code: .
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 500-

Product Name: Famotidina
Product Code: [.]
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: FAMOTIDINA
Current Sponsor code: .
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-

Product Name: Levocitirizina
Product Code: [.]
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: LEVOCETIRIZINA DICLORIDRATO
Current Sponsor code: .
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-

Primary Outcome(s)
Timepoint(s) of evaluation of this end point: Part A: Through Day 169
Part B: Through Day 85
Main Objective: • Part A: To evaluate the safety and tolerability of subcutaneous injections of isatuximab in adults with wAIHA
• Part B: To evaluate the efficacy of the selected dose in adults with wAIHA
Secondary Objective: Part A (Cohorts 2 and 3 only)
-To evaluate the efficacy of isatuximab in adults with wAIHA
-To evaluate the durability of response to isatuximab and time to response
-To evaluate the impact of isatuximab treatment on fatigue
• Part B
-To evaluate the safety and tolerability of isatuximab in adults with wAIHA
-To evaluate the durability of response to isatuximab and time to response
-To evaluate the impact of isatuximab treatment on fatigue
• Parts A (all Cohorts) and B
-To evaluate the effect of isatuximab on markers of hemolysis
-To characterize the pharmacokinetic profile of isatuximab in adults with wAIHA
-To evaluate the immunogenicity of isatuximab
Primary end point(s): Part A: To assess safety and tolerability
Standard clinical and laboratory parameters and adverse events
Part B
-To evaluate overall response rate (R) or complete response (CR) at Day 85.
R is defined as an increase in hemoglobin by >/=2 g/dL from baseline and an absence of transfusion in the last 7 days. Biochemical evidence of hemolysis may still be present.
CR is defined as hemoglobin >/=11 g/dL (women) or >/=12 g/dL (men), no evidence of hemolysis (normal bilirubin, LDH, haptoglobin, and reticulocytes), and absence of transfusion from 6 weeks after the first isatuximab dose.
Secondary Outcome(s)
Secondary end point(s): Part A (Cohorts 2 and 3 Only):
1)To evaluate overall response rate (R) or complete response (CR) at Day 85; R is defined as an increase in hemoglobin by >/=2 g/dL from baseline and an absence of transfusion in the last 7 days. Biochemical evidence of hemolysis may still be present. CR is defined as hemoglobin >/=11 g/dL (women) or >/=12 g/dL (men), no evidence of hemolysis (normal bilirubin, LDH, haptoglobin, and reticulocytes), and absence of transfusion from 6 weeks after the first isatuximab dose.
2)Overall response rate at Day 169, time to R or CR, time to loss of R or CR, proportion of participants requiring rescue therapy (any wAIHA-directed therapy other than prednisone or transfusion) or splenectomy; Overall response rate at Day 169, time to R or CR, time to loss of R or CR (loss of R defined as hemoglobin <10 g/dL at two consecutive visits at least 7 days apart and initiation of new treatment for anemia or increase in steroid dose; loss of CR is defined as hemoglobin <11 g/dL (women) or <12 g/dL (men) at two consecutive visits at least 7 days apart), proportion of participants requiring rescue therapy (any wAIHA-directed therapy other than prednisone or transfusion) or splenectomy
3)-FACIT-fatigue scale score
Part B:
4)-To assess safety and tolerability; Standard clinical and laboratory parameters and adverse events.
5)Overall response rate at Day 169, time to R or CR, time to loss of R or CR, proportion of participants requiring rescue therapy (any wAIHA-directed therapy other than prednisone or transfusion) or splenectomy; Overall response rate at Day 169, time to R or CR, time to loss of R or CR (loss of R defined as hemoglobin <10 g/dL at two consecutive visits at least 7 days apart and initiation of new treatment for anemia or increase in steroid dose; loss of CR is defined as hemoglobin <11 g/dL (women) or <12 g/dL (men) at two consecutive visits at least 7 days apart), proportion of participants requiring rescue therapy (any wAIHA-directed therapy other than prednisone or transfusion) or splenectomy
6)-FACIT-fatigue scale score
Part A (All Cohorts) and B:
7)-Change from baseline in LDH, haptoglobin, reticulocytes, and total Bilirubin
8)-PK parameters after subcutaneous Administrations; Includes maximum concentration received after injection (Cmax) and area under the plasma concentration versus time curve calculated over the dosing interval T (336 h) (AUC0-2week)
9)Incidence and titer (if relevant) of antiisatuximab antibodies.
Timepoint(s) of evaluation of this end point: Part A (Cohorts 2 and 3 Only):
1)Through Day 85
2)-9) Through Day 169
Secondary ID(s)
ACT16832
2020-003880-24-DE
Source(s) of Monetary Support
Sanofi-Aventis Recherche & Développement
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 12/01/2021
Contact:
Results
Results available: Yes
Date Posted: 23/06/2024
Date Completed: 26/06/2023
URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2020-003880-24/results
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