Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis

CSL Behring

Status and phase: Begins enrollment in 2 months; Phase 3; Phase 2

Conditions: Sickle Cell Disease Vaso-occlusive Crisis

Treatments: Biological: CSL889; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT06699849

2024-513440-29-00 (EU Trial (CTIS) Number)

CSL889_2001

Details and patient eligibility

About

This study consists of two parts: phase 2 (Part A) and phase 3 (Part B). It is a multicenter study designed to evaluate the safety, effectiveness, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD, and to evaluate the safety and tolerability of CSL889 in study participants.

Enrollment: 260 estimated patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

•At the time of informed consent: >= 18 years of age (adults); or >= 12 to less than (<) 18 years of age (adolescents, where approved and when enrollment for adolescents has been opened by the sponsor, with the endorsement of the Independent Data Monitoring Committee [IDMC]).

• Diagnosed with SCD (any genotype).
• Presented at the study site with a new acute VOC necessitating treatment with parenteral opioids.

Exclusion criteria

• VOC pain onset greater than or equal to (>=) 72 hours before administration of first parenteral opioid (Part A; may be adjusted for Part B based on prespecified analysis).
• Must not have a history of greater than (>) 5 VOCs requiring hospital admission in the past 6 months; or signs and / or symptoms of ACS; or new neurological symptoms suggestive of acute stroke or transient ischemic attack; or any stage (acute kidney injury) AKI; or been discharged from inpatient hospital admission for VOC or other vaso-occlusive event within 14 days before the current presentation.
• Serum hemoglobin < 6 g/dL, serum ferritin >= 2000 ng/mL, receiving an approved medication for SCD that has not been on a stable, well-tolerated regimen, currently taking methadone or buprenorphine.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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260 participants in 6 patient groups, including a placebo group

Part A: CSL889 Regimen A1

Experimental group

Description:

Participants in this arm will receive CSL889 as per regimen A1.

Treatment:

Biological: CSL889

Part A: CSL889 Regimen A2

Experimental group

Description:

Participants in this arm will receive CSL889 as per regimen A2.

Treatment:

Biological: CSL889

Part A: CSL889 Regimen A3

Experimental group

Description:

Participants in this arm will receive CSL889 as per regimen A3.

Treatment:

Biological: CSL889

Part A: Placebo

Placebo Comparator group

Description:

Participants in this arm will receive placebo matching to CSL889 regimen of Part A.

Treatment:

Drug: Placebo

Part B: CSL889

Experimental group

Description:

Participants in this arm will receive CSL889, as per regimen selected after Part A.

Treatment:

Biological: CSL889

Part B: Placebo

Placebo Comparator group

Description:

Participants in this arm will receive placebo matching the selected CSL889 regimen after Part A.

Treatment:

Drug: Placebo