RILZABRUTINIB for Warm autoimmune hemolytic anemia

Inclusion criteria

• {"criterion_text":"- Male and female patients with a confirmed diagnosis of primary wAIHA or systemic lupus erythematosus (SLE)-associated wAIHA (without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations)\n- Participants who have previously failed to maintain a sustained response after treatment with corticosteroids\n- Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower\n- Up-to-date vaccination status as per local guideline\n- Body mass index (BMI) >17.5 and <40 kg/m2\n- All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies\n- Core Part B: Evidence of treatment efficacy to rilzabrutinib as defined by achieving overall response during Part A\n- Core Part B: -\tCompletion of Part A treatment period (24 weeks)\n- Extended Part B: Completion of Core Part B period."}

Exclusion criteria

• {"criterion_text":"- 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\n- Part B only: Presence of unacceptable side effect(s) or toxicity associated with rilzabrutinib such that there is an unfavorable risk-benefit assessment for continued treatment with rilzabrutinib in the opinion of the Investigator and/or Sponsor\n- Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years\n- Secondary wAIHA from any cause including drugs, lymphoproliferative disorders (low-count monoclonal B-cell lymphocytosis is allowed), infectious or autoimmune disease, or active hematologic malignancies. Participants with positive antinuclear antibodies but without a definitive diagnosis of an autoimmune disease are allowed\n- Myelodysplastic syndrome\n- Uncontrolled or active HBV infection: Patients with positive HBsAg and/or HBV DNA\n- HIV infection\n- Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 half-lives, whichever is greater, prior to treatment start\n- Known allergy to any of the study medications, their analogues, or excipients in the various formulations of any agent\n- Part B only: Participants who receive any therapy during Part A known to be active in wAIHA"}

Primary endpoints

• {"endpoint_text":"- Proportion of participants with overall hemoglobin response","definition_or_measurement_approach":""}
• {"endpoint_text":"- Proportion of participants who maintain durable response achieved during Part A or achieve a durable response during Part B and have a hemoglobin response","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- Proportion of participants with durable hemoglobin response","definition_or_measurement_approach":""}
• {"endpoint_text":"- Median time from baseline to first hemoglobin response","definition_or_measurement_approach":"Median time measured from baseline to first hemoglobin response (time-to-event)."}
• {"endpoint_text":"- Frequency of rescue therapy (any wAIHA-directed therapy other than predniso[lo]ne or transfusion) received","definition_or_measurement_approach":"Count/frequency of any wAIHA-directed rescue therapy excluding predniso[lo]ne and transfusion."}
• {"endpoint_text":"- Change from baseline in FACIT-Fatigue scale score","definition_or_measurement_approach":"Change from baseline in FACIT-Fatigue questionnaire score."}
• {"endpoint_text":"- Safety assessments","definition_or_measurement_approach":"Standard safety and tolerability assessments as per protocol (not further specified in the record)."}

Denmark

Earliest CTIS Part Ii Submission Date

07-02-2024

Latest Decision Or Authorization Date

27-02-2024

Processing Time Days

20

Number Of Sites

1

Number Of Participants

3

Spain

Earliest CTIS Part Ii Submission Date

07-02-2024

Latest Decision Or Authorization Date

01-03-2024

Processing Time Days

23

Number Of Sites

4

Number Of Participants

6

Italy

Earliest CTIS Part Ii Submission Date

07-02-2024

Latest Decision Or Authorization Date

28-02-2024

Processing Time Days

21

Number Of Sites

3

Number Of Participants

4

Primary sponsor

Full Name

Sanofi-Aventis Recherche & Developpement

Organisation Type

Pharmaceutical company

Country Of Registered Address

France

Contract research organisations

Name

Pharmaceutical Product Development LLC

Responsibilities

sponsorDuties code 4

Name

Nuvisan GmbH

Responsibilities

sponsorDuties code 4

Name

Wuxi Apptec Co. Ltd.

Responsibilities

sponsorDuties code 4

Third parties

• {"country":"Spain","full_name":"Alcura Health Espana S.A.","duties_or_roles":"sponsorDuties code 14","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"sponsorDuties code 4","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Nuvisan GmbH","duties_or_roles":"sponsorDuties code 4","organisation_type":"Pharmaceutical company"}
• {"country":"China","full_name":"Wuxi Apptec Co. Ltd.","duties_or_roles":"sponsorDuties code 4","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"ESMS Global Limited","duties_or_roles":"sponsorDuties code 15 (Centralized 24-Hour Emergency system)","organisation_type":"Pharmaceutical company"}