Sickle Cell Disease
Conditions
Keywords
Anemia, Genetic Disorder, Vaso-occlusive Crises
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.
Interventions
BIOLOGICALAnumigilimab
Participants will receive anumigilimab, escalated to a maximum dose guided by absolute neutrophil count (ANC) and safety concerns.
DRUGPlacebo
Volume matched saline will be administered SC.
Sponsors
CSL Behring
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Intervention model description
This is a double blind, placebo controlled study.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Belgium, France, Italy, United Kingdom, United States
Contacts
CONTACTTrial Registration Coordinator
STUDY_DIRECTORStudy Director
CSL Behring
Outcome results
None listed