A Phase 2a, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety of Anumigilimab in Adults with Sickle Cell Disease

The primary objective is to evaluate the safety of anumigilimab in adults diagnosed with sickle cell disease. The secondary objectives include:

• Assessment of the pharmacokinetics of anumigilimab.
• Evaluation of the effect of anumigilimab on the frequency of vaso-occlusive crises.

This study involves 51 participants diagnosed with sickle cell disease. The study population consists of adults of both sexes who are considered a vulnerable population. Eligible individuals must have a confirmed diagnosis of any genotype of the disease and have experienced between 1 and 12 vaso-occlusive crises requiring medical facility visits and treatment with parenteral opioids or parenteral nonsteroidal anti-inflammatory drugs within the 12 months prior to screening. Regarding pharmacological management, participants must be on a stable and well-tolerated hydroxyurea regimen for at least 30 days or have discontinued or refused such treatment.

This Phase 2a, multicenter, randomized, double-blind, placebo-controlled study is designed to assess the safety of anumigilimab in adults diagnosed with sickle cell disease. Participants will be assigned to receive either 50 mg of anumigilimab or saline 0.9% via subcutaneous injection. The study involves a screening visit to confirm eligibility based on age, genotype, and history of vaso-occlusive crises. The clinical assessment includes monitoring for treatment-emergent adverse events, adverse events of special interest, and changes in vital signs or laboratory assessments. Following the initial treatment phase, a 52-week maintenance period is conducted to evaluate the annualized rate of crises and serum drug levels. Total participant involvement is determined by the study protocol, which includes periodic follow-up visits and an end-of-study visit.

The experimental treatment consists of anumigilimab, which is administered as a solution for injection. The specified dosage is 50 mg, delivered via subcutaneous injection.

The control group receives a placebo consisting of 0.9% saline.

The efficacy assessment in this study of sickle cell disease involves specific parameters measured during the trial period. The secondary endpoints include the measurement of serum anumigilimab levels from the initial dose through week 65. During the 52-week maintenance period, the annualized rate of vaso-occlusive crisis occurring in a medical facility will be evaluated.