Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12+
SponsorCSL Behring
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Parallel
Treatments tested in this trial
CSL889
Biological/VaccineCSL889 is a solution for infusion to be administered by the IV route.
Placebo
DrugVolume and regimen matched to CSL889 will be administered.
Treatment groups
70 Participants
are divided into 3 treatment groupsGroup A: CSL889 Regimen 1Experimental treatment 1 intervention
Group B: CSL889 Regimen 2Experimental treatment 1 intervention
Group C: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Number of participants with treatment-emergent adverse events (TEAEs)
Time frame
Up to Day 28 (End of study [EOS] Visit)2
Percentage of participants with TEAEs
Time frame
Up to Day 28 (EOS Visit)3
Number of participants with detectable treatment emergent (TE) anti-CSL889 antibodies
Time frame
Up to Day 28 (EOS Visit)4
Percentage of participants with detectable TE anti-CSL889 antibodies
Time frame
Up to Day 28 (EOS Visit)Secondary outcomes
1
Hospital admission rate
Hospital admission rate in participants with SCD presenting with VOC who received greater than or equal to (≥) 1 dose of CSL889 in the acute care setting.
Time frame
Up to Day 28 (EOS Visit)2
Length of hospital stay (If hospitalized)
Time frame
Up to Day 28 (EOS Visit)3
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 84
Length of acute care stay
Time frame
Up to Day 28 (EOS Visit)Sponsors and contacts
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Source ClinicalTrials.gov