Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
ConditionSickle Cell Disease
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorCSL Behring
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
a. On stable and well-tolerated Hydroxyurea (HU) regimen for at least 30 days before Screening.
Disqualifiers
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
Design model
Parallel
Treatments tested in this trial
Anumigilimab
Biological/VaccineParticipants will receive anumigilimab, escalated to a maximum dose guided by absolute neutrophil count (ANC) and safety concerns.
Placebo
DrugVolume matched saline will be administered SC.
Treatment groups
63 Participants
are divided into 2 treatment groupsGroup A: AnumigilimabExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Number of participants with Treatment Emergent Adverse Events (TEAEs), Overall and by severity, seriousness and relationship to Investigational Product (IP)
Time frame
From baseline up to Week 752
Percentage of participants with TEAEs Overall and by severity, seriousness and relationship to IP
Time frame
From baseline up to Week 753
Number of participants with Adverse Events of Special Interest (AESI)
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.
Time frame
From baseline up to Week 754
Percentage of participants with AESI
The following AEs are to be reported as AESI: neutropenia (CTCAE Grade 3 or 4), severe infection and neutropenic fever.
Time frame
From baseline up to Week 75Secondary outcomes
1
Serum concentration of Anumigilimab
Time frame
From the first dose of anumigilimab to Week 652
Annualized rate of Vaso-occlusive crisis (VOC) (medical facility)
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.
Time frame
During the maintenance period from Week 13 to Week 65Sponsors and contacts
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Source ClinicalTrials.gov