RightFit
RecruitingPhase 2

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

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
12 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests CSL889 (human hemopexin), given through a vein, against a placebo. It is for adults and some teens with sickle cell disease who are having a new pain crisis caused by blocked blood flow. Researchers will check safety, how the body processes the drug, and whether it helps the crisis end sooner.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be an adult, or a teen age 12–17 where teen enrollment is approved and open.
  • Have any type of sickle cell disease.
  • Come to the study site with a new pain crisis that needs opioid pain medicine given by injection or through a vein.

Who can't join

  • Pain from the crisis started more than 72 hours before the first opioid dose given by injection or through a vein.
  • Had more than 5 pain crises that required a hospital stay in the past 6 months.
  • Have signs or symptoms of acute chest syndrome, a serious lung problem.
  • Have new symptoms suggesting a stroke or a brief stroke-like event.
  • Have any stage of sudden kidney injury.
  • Left the hospital within the past 14 days after a stay for a pain crisis or another event caused by blocked blood flow.
  • Have hemoglobin below 6 g/dL or ferritin at least 2,000 ng/mL. Hemoglobin carries oxygen in the blood; ferritin stores iron.
  • Take an approved sickle cell medicine without a steady treatment plan that you tolerate well, or currently take methadone or buprenorphine.

Simplified for readability. The official criteria below are what the study team uses.

Official 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.

Study sites (20)

CA

DC

FL

GA

IL

KY

MD

MI

MO

NC

NY

OH

PA

VA