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Purpose

This is a parallel-group, Phase 3, double-blind, 2-arm study to investigate the efficacy, safety, PK and PD of oral rilzabrutinib in achieving durable Hb response (DHR) compared with placebo in approximately 90 male and female participants ≥ 18 years of age with a confirmed diagnosis of primary wAIHA. Following a 4-week screening period, eligible participants will be randomized in a 2:1 ratio to receive rilzabrutinib or placebo in primary analysis period (PAP) for a duration of up to 24 weeks. All participants who completed PAP will then continue in open-label period (OLP) to receive rilzabrutinib for a duration of 28 weeks. Upon the completion of OLP, only participants who demonstrate Hb increase during the last 8 weeks of OLP per specified criteria in the protocol will be eligible to continue in long-term extension (LTE) of the study. The duration of the LTE period will be from the first-participant-in (FPI)-LTE until the last participant completes 52 weeks in LTE. The safety follow-up period of this study following treatment completion or discontinuation will be 2 weeks.

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Male and female participants with a documented (confirmed) diagnosis of primary wAIHA for at least 3 months. - Participants who have previously failed to maintain a sustained response after treatment with CS (CS-resistance [defined as failure to obtain hemoglobin response within 3 weeks on at least 1 mg/kg or 60 mg prednisone or equivalent per day], CS-dependent wAIHA [defined as need to continue on prednisone or equivalent at a dose of >10 mg/day to maintain a response]), or are intolerant or ineligible to CS (defined as with contraindications, pre-existing medical conditions or CS-related complications that may render CS intolerant or ineligible per the best clinical judgement of the investigators). - Participants with Eastern Cooperative Oncology Group (ECOG) performance status Grade 2 or lower. - Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Exclusion Criteria

  • Participants with 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. - 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. - Participants with symptomatic herpes zoster within 3 months prior to screening. - Participants with secondary wAIHA from any cause including drugs, Evans Syndrome, 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. - Participants with history of myelodysplastic syndrome. - Participants with uncontrolled or active HBV infection or Active HCV infection. - HIV infection. - Participants with history of solid organ transplant. - Participants with a history of active or latent tuberculosis (TB). - Concurrent treatment with other experimental/investigational drugs within 30 days or 5 half-lives, whichever is longer, prior to treatment start. Participants who previously received treatment with BTK inhibitors for wAIHA before Day 1 (randomization) are not eligible. - Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study. The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
rilzabrutinib 		Oral rilzabrutinib BID
  • Drug: rilzabrutinib
    Pharmaceutical form:tablet-Route of administration:oral
    Other names:
    • SAR444671
Placebo Comparator
placebo 		Oral placebo BID
  • Drug: placebo
    Pharmaceutical form:tablet-Route of administration:oral

Recruiting Locations

Mayo Clinic in Arizona - Phoenix- Site Number : 8400032
Phoenix 5308655, Arizona 5551752 85054

Noble Clinical Research- Site Number : 8400003
Tucson 5318313, Arizona 5551752 85704

City of Hope National Medical Center- Site Number : 8400023
Duarte 5344147, California 5332921 91010

University of Southern California (USC)- Site Number : 8400007
Los Angeles 5368361, California 5332921 90089

The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center- Site Number : 8400006
Torrance 5403022, California 5332921 90502

Hialeah Hospital- Site Number : 8400009
Hialeah 4158476, Florida 4155751 33013

University of Michigan Health System - Ann Arbor- Site Number : 8400014
Ann Arbor 4984247, Michigan 5001836 48109

Mayo Clinic- Site Number : 8400008
Rochester 5043473, Minnesota 5037779 55905

Ohio State University Hospital East- Site Number : 8400020
Columbus 4509177, Ohio 5165418 43203-1779

Baptist Memorial Hospital- Site Number : 8400018
Memphis 4641239, Tennessee 4662168 38120

More Details

Status
Recruiting
Sponsor
Sanofi

Study Contact

Trial Transparency email recommended (Toll free for US & Canada)
800-633-1610
contact-us@sanofi.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.