A Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Oral Rilzabrutinib Compared With Placebo in Participants 18 Years of Age and Older With Warm Autoimmune Hemolytic Anemia

Conditions

• Autoimmune Haemolytic Anaemia

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• rilzabrutinib — DRUG
  Pharmaceutical form:tablet-Route of administration:oral
• placebo — DRUG
  Pharmaceutical form:tablet-Route of administration:oral

Study Details

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.

Key Dates

Start date

Aug 18, 2025

Status verified

Jun 2026

Primary completion

May 31, 2028

Completion

Dec 26, 2029

Study Design

Enrollment

90 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: rilzabrutinib
  Oral rilzabrutinib BID
• Placebo Comparator: placebo
  Oral placebo BID

Primary Outcome Measure

Proportion of participants achieving DHR. DHR is defined as an increase of Hb by ≥2 g/dL from baseline on at least two thirds of evaluable scheduled visits between Week 12 and Week 24 (inclusive) in the PAP [ Time Frame: By Week 24 ]

Central Contacts

• Trial Transparency email recommended (Toll free for US & Canada)
  800-633-1610
  [email protected]

Locations (23)

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