Recruiting NCT06699849
Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: CSL889, Placebo.
- Who it may be relevant to
- Registry conditions: Sickle Cell Disease Vaso-occlusive Crisis. Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Turkey (Türkiye)
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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.
Interventions
- Biological CSL889
CSL889 is a solution for infusion to be administered by the IV route. - Drug Placebo
Volume and regimen matched to CSL889 will be administered.
Primary outcome measures
- Number of participants with treatment-emergent adverse events (TEAEs) [Time frame: Up to Day 28 (End of study [EOS] Visit)]
- Percentage of participants with TEAEs [Time frame: Up to Day 28 (EOS Visit)]
- Number of participants with detectable treatment emergent (TE) anti-CSL889 antibodies [Time frame: Up to Day 28 (EOS Visit)]
- Percentage of participants with detectable TE anti-CSL889 antibodies [Time frame: Up to Day 28 (EOS Visit)]
- Time to resolution of VOC (time to discontinuation of parenteral opioids) [Time frame: Up to Day 28 (EOS Visit)]
Secondary outcome measures (12)
- Hospital admission rate [Time frame: Up to Day 28 (EOS Visit)]
- Length of hospital stay (If hospitalized) [Time frame: Up to Day 28 (EOS Visit)]
- Percentage of participants experiencing acute chest syndrome (ACS), acute kidney injury (AKI), or stroke [Time frame: From the start of investigational product (IP) administration up to Day 8]
- Length of acute care stay [Time frame: Up to Day 28 (EOS Visit)]
- Total Length of acute care and hospital stay [Time frame: Up to Day 28 (EOS Visit)]
- Re-presentation rate to an acute care facility for VOC or ACS after discharge [Time frame: From discharge up to Day 28]
- Hospital admission rate for VOC or ACS after discharge [Time frame: From discharge up to Day 28]
- Opioid consumption [Time frame: From the time of enrollment to discharge (up to Day 28 [EOS Visit])]
- Number of participants with ≥ 30% pain reduction by Numeric Rating Scale (NRS) score [Time frame: Within 4 hours after the start of CSL889 infusion]
- Percentage of participants with ≥ 30% pain reduction by NRS score [Time frame: Within 4 hours after the start of CSL889 infusion]
- Maximum observed concentration (Cmax) after Doses 1 and 3 of CSL889 [Time frame: Before dosing, and up to 12 hours after Doses 1 and 3]
- Area under the concentration (AUCtau) after Doses 1 and 3 of CSL889 [Time frame: Before dosing, and up to 12 hours after Doses 1 and 3]
Eligibility 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 16 centers
- Univ. of California, San Francisco Health Care — Oakland
- University of California Irvine — Orange
- Golisano Children's Hospital — Fort Myers
- The Foundation for Sickle Cell Disease — Hollywood
- University of Louisville Hospital — Louisville
- University of Maryland — Baltimore
- Detroit Medical Center — Detroit
- Henry Ford Health System — Detroit
- … and 8 more centers
Turkey (Türkiye) · 3 centers
- Hacettepe Universitesi — Ankara
- Istanbul Universitesi — Istanbul
- Özel Acibadem Adana Hastanesi — Seyhan
Identifiers
NCT: NCT06699849 · CSL889_2001 · 2024-513440-29-00