Evaluating CSL889 for treating vaso-occlusive crises in sickle cell disease
A Phase 2, Multicenter, Randomized, Multiple-Dose, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis
This study is testing a new treatment called CSL889 to see if it can help adults and teens with sickle cell disease who are having painful vaso-occlusive crises feel better.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 70 (estimated) |
| Ages | 12 Years and up |
| Sex | All |
| Sponsor | CSL Behring Industry-sponsored |
| Locations | 17 sites (Oakland, California and 16 other locations) |
| Trial ID | NCT06699849 on ClinicalTrials.gov |
What this trial studies
This clinical trial is designed to assess the safety, efficacy, and pharmacokinetics of CSL889, a human hemopexin, administered intravenously to adults and adolescents with sickle cell disease experiencing vaso-occlusive crises. The study consists of two parts: a phase 2 component focusing on initial safety and efficacy, followed by a phase 3 component to further evaluate these aspects in a larger population. Participants will be monitored for the resolution time of vaso-occlusive crises and the overall tolerability of the treatment. The trial aims to provide valuable insights into a potential new therapeutic option for managing this painful condition.
Who should consider this trial
Good fit: Ideal candidates for this study are adults and adolescents aged 12 and older diagnosed with sickle cell disease who are experiencing a new acute vaso-occlusive crisis.
Not a fit: Patients who have had a history of more than five vaso-occlusive crises requiring hospitalization in the past six months or those with acute kidney injury may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce the duration and severity of vaso-occlusive crises in patients with sickle cell disease.
How similar studies have performed: While this approach is novel in the context of CSL889, similar studies targeting vaso-occlusive crises in sickle cell disease have shown promise with other interventions.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Oakland, California and 16 other locations
- Univ. of California, San Francisco Health Care — Oakland, California, United States (Recruiting)
- University of California Irvine — Orange, California, United States (Not_yet_recruiting)
- Golisano Children's Hospital — Fort Myers, Florida, United States (Recruiting)
- The Foundation for Sickle Cell Disease — Hollywood, Florida, United States (Recruiting)
- University of Maryland — Baltimore, Maryland, United States (Recruiting)
- Detroit Medical Center — Detroit, Michigan, United States (Recruiting)
- Henry Ford Health System — Detroit, Michigan, United States (Recruiting)
- Mount Sinai Medical Center — New York, New York, United States (Recruiting)
- Jacobi Medical Center — The Bronx, New York, United States (Recruiting)
- University of Cincinnati — Cincinnati, Ohio, United States (Recruiting)
- The Ohio State University — Columbus, Ohio, United States (Recruiting)
- University of Pittsburgh — Pittsburgh, Pennsylvania, United States (Recruiting)
- Hillman Cancer Center — Pittsburgh, Pennsylvania, United States (Recruiting)
- Virginia Commonwealth University — Richmond, Virginia, United States (Recruiting)
- Hacettepe Universitesi — Ankara, Turkey (Türkiye) (Not_yet_recruiting)
- Istanbul Universitesi — Istanbul, Turkey (Türkiye) (Not_yet_recruiting)
- Özel Acibadem Adana Hastanesi — Seyhan, Turkey (Türkiye) (Not_yet_recruiting)
Study contacts
- Study coordinator: Trial Registration Coordinator
- Email: clinicaltrials@cslbehring.com
- Phone: +1 610-878-4697
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.