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

NCT06699849 · tracked via the Priya Life Science UK tracker

Sponsor

CSL Behring

Phase

Phase 2

Started

2025-08-07

Last updated

2026-08-14

Condition(s) studied

Sickle Cell Disease Vaso-occlusive Crisis

Investigational drug(s) / intervention(s)

CSL889Placebo

CSL889: CSL889 is a solution for infusion to be administered by the IV route.

Placebo: Volume and regimen matched to CSL889 will be administered.

Study summary

This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, 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 evaluate the safety and tolerability of CSL889 in study participants, and to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD.

Eligibility

Sex

ALL

Min age

12 Years

Max age

—

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 (\>) 72 hours before administration of first parenteral opioid. * Must not have a history of \> 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.

Primary outcome measure(s)

Number of participants with treatment-emergent adverse events (TEAEs) — Up to Day 28 (End of study [EOS] Visit)
Percentage of participants with TEAEs — Up to Day 28 (EOS Visit)
Number of participants with detectable treatment emergent (TE) anti-CSL889 antibodies — Up to Day 28 (EOS Visit)
Percentage of participants with detectable TE anti-CSL889 antibodies — Up to Day 28 (EOS Visit)
Time to resolution of VOC (time to discontinuation of parenteral opioids) — Up to Day 28 (EOS Visit)

Trial sites (26)

Facility	City	Region	Status
Univ. of California, San Francisco Health Care	Oakland	California	Recruiting
University of California Irvine	Orange	California	Not Yet Recruiting
Golisano Children's Hospital	Fort Myers	Florida	Recruiting
The Foundation for Sickle Cell Disease	Hollywood	Florida	Recruiting
Arthur M. Blank Hospital-Children's Healthcare of Atlanta	Atlanta	Georgia	Recruiting
University of Illinois at Chicago	Chicago	Illinois	Recruiting
University of Louisville Hospital	Louisville	Kentucky	Recruiting
University of Maryland	Baltimore	Maryland	Recruiting
Detroit Medical Center	Detroit	Michigan	Recruiting
Henry Ford Health System	Detroit	Michigan	Recruiting
St. Louis Children's Hospital	St Louis	Missouri	Recruiting
Mount Sinai Medical Center	New York	New York	Recruiting
Jacobi Medical Center	The Bronx	New York	Recruiting
East Carolina University	Greenville	North Carolina	Recruiting
University of Cincinnati	Cincinnati	Ohio	Recruiting
The Ohio State University	Columbus	Ohio	Recruiting
University of Pittsburgh	Pittsburgh	Pennsylvania	Recruiting
Hillman Cancer Center	Pittsburgh	Pennsylvania	Recruiting
Virginia Commonwealth University	Richmond	Virginia	Recruiting
Baskent University School of Medicine	Adana	Turkey (Türkiye)	Recruiting
Hacettepe Universitesi	Ankara	Turkey (Türkiye)	Not Yet Recruiting
Istanbul Universitesi	Istanbul	Turkey (Türkiye)	Not Yet Recruiting
Medical Park Mersin Hospital	Mezitli-Mersin	Turkey (Türkiye)	Recruiting
Özel Acibadem Adana Hastanesi	Seyhan	Turkey (Türkiye)	Not Yet Recruiting
Özel Acibadem Adana Hastanesi	Seyhan/Adana	Turkey (Türkiye)	Recruiting
Guy's Hospital	London	United Kingdom	Recruiting

More CSL Behring trials in the UK

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

Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222) Enrolling by invitation

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B Recruiting

A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE) Recruiting

Combined Dose-Finding and CV Outcomes Study With CSL300 (Clazakizumab) in Adult Subjects With ESKD Undergoing Dialysis (POSIBIL6ESKD) Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06699849 on ClinicalTrials.gov ↗ ← All trials in the UK