Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)

CSL Behring

Status and phase: Not yet enrolling; Phase 2

Conditions: Sickle Cell Disease

Treatments: Biological: Anumigilimab; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT07224360

CSL324_2002

2025-521154-42-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD.

The primary aim of the study is to assess the safety of anumigilimab in participants with SCD. Participants will be treated for 64 weeks: for 12 weeks in the dose escalation period, where the dose will be escalated to each participant's individual MTD; and for 52 weeks at the MTD in the maintenance period.

Enrollment: 63 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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63 participants in 2 patient groups, including a placebo group

Anumigilimab

Experimental group

Description:

Safety of Anumigilimab (CSL324) in Adults with Sickle Cell Disease (SCD)

Treatment:

Biological: Anumigilimab

Placebo

Placebo Comparator group

Description:

Participants will receive matching volume of placebo.

Treatment:

Drug: Placebo