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

Not Applicable

Not yet recruiting

• Conditions: Sickle Cell Disease
• Interventions: Biological: Anumigilimab; Drug: Placebo

• Registration Number: NCT07224360
• Lead Sponsor: CSL Behring

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-08
• Study First Submitted: 2025-10-31
• Study First Submitted QC: 2025-10-31
• Last Update Submitted: 2025-10-31
• Study First Posted: 2025-11-04
• Last Update Posted: 2025-11-04
• Study Start: 2025-11-15
• Primary Completion: 2028-06-29
• Study Completion: 2028-06-29

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 63

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Anumigilimab	Anumigilimab	Safety of Anumigilimab (CSL324) in Adults with Sickle Cell Disease (SCD)
Placebo	Placebo	Participants will receive matching volume of placebo.

Primary Outcome Measures

Name	Time	Method
Number of participants with Treatment Emergent Adverse Events (TEAEs), Overall and by severity, seriousness and relationship to Investigational Product (IP)	From baseline up to Week 75
Percentage of participants with TEAEs Overall and by severity, seriousness and relationship to IP	From baseline up to Week 75
Number of participants with Adverse Events of Special Interest (AESI)	From baseline up to Week 75	The following AEs are to be reported as AESI: neutropenia (Common Terminology Criteria for Adverse Events \[CTCAE\] Grade 3 or 4), severe infection and neutropenic fever.
Percentage of participants with AESI	From baseline up to Week 75	The following AEs are to be reported as AESI: neutropenia (CTCAE Grade 3 or 4), severe infection and neutropenic fever.
Number of participants with clinically relevant changes from baseline in laboratory assessments and vital signs	From baseline up to Week 75
Percentage of participants with clinically relevant changes from baseline in laboratory assessments and vital signs	From baseline up to Week 75

Secondary Outcome Measures

Name	Time	Method
Serum concentration of Anumigilimab	From the first dose of anumigilimab to Week 65
Annualized rate of Vaso-occlusive crisis (VOC) (medical facility)	During the maintenance period from Week 13 to Week 65	VOC (medical facility) is defined as VOC that results in a visit to a medical facility and treatment with parenteral opioids or a parenteral nonsteroidal anti-inflammatory drug.