临床试验/NCT06699849

NCT06699849

招募中

2 期

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

CSL Behring18 个研究点分布在 2 个国家目标入组 70 人2025年8月7日

适应症Sickle Cell Disease Vaso-occlusive Crisis

干预措施Placebo CSL889

概览

阶段: 2 期
干预措施: Placebo
疾病 / 适应症: Sickle Cell Disease Vaso-occlusive Crisis
发起方: CSL Behring
入组人数: 70
试验地点: 18
主要终点: Number of participants with treatment-emergent adverse events (TEAEs)
状态: 招募中
最后更新: 3天前

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验

概览

简要总结

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.

注册库

clinicaltrials.gov

开始日期

2025年8月7日

结束日期

2027年10月22日

最后更新

3天前

研究类型

Interventional

研究设计

Parallel

性别

All

研究者

发起方

CSL Behring

责任方

Sponsor

入排标准

入选标准

•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.

排除标准

•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.

研究组 & 干预措施

Placebo

Participants in this arm will receive placebo matching to CSL889 regimen.

干预措施: Placebo

CSL889 Regimen 2

Participants in this arm will receive CSL889 as per regimen 2.

干预措施: CSL889

CSL889 Regimen 1

Participants in this arm will receive CSL889 as per regimen 1.

干预措施: CSL889

结局指标

主要结局

Number of participants with treatment-emergent adverse events (TEAEs)

时间窗: Up to Day 28 (End of study [EOS] Visit)

Percentage of participants with TEAEs

时间窗: Up to Day 28 (EOS Visit)

Number of participants with detectable treatment emergent (TE) anti-CSL889 antibodies

时间窗: Up to Day 28 (EOS Visit)

Percentage of participants with detectable TE anti-CSL889 antibodies

时间窗: Up to Day 28 (EOS Visit)

Time to resolution of VOC (time to discontinuation of parenteral opioids)

时间窗: Up to Day 28 (EOS Visit)

次要结局

Trough concentration (Ctrough) after each dose of CSL889(Up to Day 5)
Accumulation ratio (AR) for AUCtau of CSL889 (the ratio between the AUCtau of Doses 3 and 1)(Before dosing and at up to 12 hours after Doses 1 and 3)
AR for Cmax of CSL889 (the ratio between the Cmax of Doses 3 and 1)(Before dosing and at up to 12 hours after Doses 1 and 3)
AR for Ctrough of CSL889 (the ratio between the Ctrough of the last dose and Dose 1)(Before dosing and after Dose 1 and the last dose)
Length of hospital stay (If hospitalized)(Up to Day 28 (EOS Visit))
Percentage of participants experiencing acute chest syndrome (ACS), acute kidney injury (AKI), or stroke(From the start of investigational product (IP) administration up to Day 8)
Length of acute care stay(Up to Day 28 (EOS Visit))
Total Length of acute care and hospital stay(Up to Day 28 (EOS Visit))
Opioid consumption(From the time of enrollment to discharge (up to Day 28 [EOS Visit]))
Maximum observed concentration (Cmax) after Doses 1 and 3 of CSL889(Before dosing, and up to 12 hours after Doses 1 and 3)
Area under the concentration (AUCtau) after Doses 1 and 3 of CSL889(Before dosing, and up to 12 hours after Doses 1 and 3)
Time of maximum concentration (Tmax) after Doses 1 and 3 of CSL889(Before dosing, and up to 12 hours after Doses 1 and 3)
Hospital admission rate for VOC or ACS after discharge(From discharge up to Day 28)
Opioid consumption(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(Within 4 hours after the start of CSL889 infusion)
Hospital admission rate(Up to Day 28 (EOS Visit))
Total Length of acute care and hospital stay(Up to Day 28 (EOS Visit))
Re-presentation rate to an acute care facility for VOC or ACS after discharge(From discharge up to Day 28)
Length of hospital stay (If hospitalized)(Up to Day 28 (EOS Visit))
Percentage of participants experiencing acute chest syndrome (ACS), acute kidney injury (AKI), or stroke(From the start of investigational product (IP) administration up to Day 8)
Length of acute care stay(Up to Day 28 (EOS Visit))
Percentage of participants with ≥ 30% pain reduction by NRS score(Within 4 hours after the start of CSL889 infusion)
Maximum observed concentration (Cmax) after Doses 1 and 3 of CSL889(Before dosing, and up to 12 hours after Doses 1 and 3)
Area under the concentration (AUCtau) after Doses 1 and 3 of CSL889(Before dosing, and up to 12 hours after Doses 1 and 3)
Time of maximum concentration (Tmax) after Doses 1 and 3 of CSL889(Before dosing, and up to 12 hours after Doses 1 and 3)
Trough concentration (Ctrough) after each dose of CSL889(Up to Day 5)
Accumulation ratio (AR) for AUCtau of CSL889 (the ratio between the AUCtau of Doses 3 and 1)(Before dosing and at up to 12 hours after Doses 1 and 3)
AR for Cmax of CSL889 (the ratio between the Cmax of Doses 3 and 1)(Before dosing and at up to 12 hours after Doses 1 and 3)
AR for Ctrough of CSL889 (the ratio between the Ctrough of the last dose and Dose 1)(Before dosing and after Dose 1 and the last dose)
Cmax after each dose in Sparse PK subset of CSL889(Up to Day 5)
Ctrough after each dose in Sparse PK subset of CSL889(Up to Day 5)
AR for Cmax in Sparse PK subset of CSL889(Up to Day 5)
AR for Ctrough in Sparse PK subset of CSL889(Up to Day 5)