Testing Anumigilimab (CSL324) safety in adults with sickle cell disease

Phase 2a, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease

This study will test whether subcutaneous Anumigilimab is safe for adults with sickle cell disease who had 1–12 painful vaso-occlusive crises in the past year.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 63 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: CSL Behring (Industry-sponsored)
• Drugs / interventions: anumigilimab
• Locations: 5 sites (Little Rock, Arkansas and 4 other locations)
• Trial ID: NCT07224360 on ClinicalTrials.gov

What this trial studies

This is a randomized, double-blind, placebo-controlled phase 2a trial that gives Anumigilimab or placebo by subcutaneous injection at multiple international sites. Participants enter a 12-week dose-escalation period to reach an individual maximum tolerated dose (MTD), followed by 52 weeks of maintenance dosing at that MTD, for a total of 64 weeks on treatment. The primary focus is on safety and tolerability in adults with any genotype of sickle cell disease who have had recent vaso-occlusive crises. Study procedures will include regular clinical visits, laboratory monitoring, and recording of adverse events.

Who should consider this trial

Good fit: Adults aged 18 or older with a confirmed diagnosis of sickle cell disease of any genotype who have had 1 to 12 vaso-occlusive crises requiring medical treatment in the past 12 months and who are either on a stable hydroxyurea regimen or have discontinued/refused hydroxyurea are the intended participants.

Not a fit: People with an absolute neutrophil count under 2.5 x 10^9 cells/L at screening, those with unstable doses of SCD preventive medications, or those under 18 years old are unlikely to qualify or benefit from participation.

Why it matters

Potential benefit: If shown to be safe, this could open the way to a new therapeutic option for people with sickle cell disease and support later studies of effectiveness against painful crises and related complications.

How similar studies have performed: Monoclonal antibody approaches in sickle cell disease remain early in development and Anumigilimab's safety in SCD has limited prior data beyond early-phase work.

Eligibility criteria

Inclusion Criteria:

* • Adults aged greater than or equal to (\>=) 18 years on the day of signing the informed consent form.
* • Confirmed diagnosis of SCD of any genotype.
* • Experienced 1 to 12 VOCs requiring a visit to a medical facility and treatment with parenteral opioids or a parenteral nonsteroidal anti-inflammatory drug within the 12 months before Screening.
* • HU Regimen:
* a. On stable and well-tolerated Hydroxyurea (HU) regimen for at least 30 days before Screening.
* or
* b. HU was discontinued or refused (eg, due to concern of side effects or lack of effect).

Exclusion Criteria:

* • Absolute neutrophil count less than (\<) 2.5 ×10\^9 cells/Litre at Screening or Baseline (Week 1 Day 1).
* • If on SCD preventive medication, dose is not stable in the 30 days before Screening.

Where this trial is running

Little Rock, Arkansas and 4 other locations

• UAMS Medical Center — Little Rock, Arkansas, United States (Recruiting)
• The Foundation for Sickle Cell Disease — Hollywood, Florida, United States (Recruiting)
• Center for Cancer and Blood Disorders — Bethesda, Maryland, United States (Recruiting)
• Southern Specialty Research — Flowood, Mississippi, United States (Recruiting)
• Jacobi Medical Center — The Bronx, New York, United States (Recruiting)

Study contacts

• Study coordinator: Trial Registration Coordinator

Conditions Sickle Cell Disease; Anemia; Genetic Disorder; Vaso-occlusive Crises