Идёт набор NCT06699849

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

Фаза II С лечением Sickle Cell Disease Vaso-occlusive Crisis

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: CSL889, Placebo.
Кому может быть актуально: Состояния в реестре: Sickle Cell Disease Vaso-occlusive Crisis. Базовые параметры: от 12 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: США, Turkey (Türkiye)
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

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

Обзор

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.

Вмешательства

Биопрепарат CSL889
CSL889 is a solution for infusion to be administered by the IV route.
Препарат Placebo
Volume and regimen matched to CSL889 will be administered.

Первичные конечные точки

Number of participants with treatment-emergent adverse events (TEAEs) [Срок оценки: Up to Day 28 (End of study [EOS] Visit)]
Percentage of participants with TEAEs [Срок оценки: Up to Day 28 (EOS Visit)]
Number of participants with detectable treatment emergent (TE) anti-CSL889 antibodies [Срок оценки: Up to Day 28 (EOS Visit)]
Percentage of participants with detectable TE anti-CSL889 antibodies [Срок оценки: Up to Day 28 (EOS Visit)]
Time to resolution of VOC (time to discontinuation of parenteral opioids) [Срок оценки: Up to Day 28 (EOS Visit)]

Вторичные конечные точки (12)

Hospital admission rate [Срок оценки: Up to Day 28 (EOS Visit)]
Length of hospital stay (If hospitalized) [Срок оценки: Up to Day 28 (EOS Visit)]
Percentage of participants experiencing acute chest syndrome (ACS), acute kidney injury (AKI), or stroke [Срок оценки: From the start of investigational product (IP) administration up to Day 8]
Length of acute care stay [Срок оценки: Up to Day 28 (EOS Visit)]
Total Length of acute care and hospital stay [Срок оценки: Up to Day 28 (EOS Visit)]
Re-presentation rate to an acute care facility for VOC or ACS after discharge [Срок оценки: From discharge up to Day 28]
Hospital admission rate for VOC or ACS after discharge [Срок оценки: From discharge up to Day 28]
Opioid consumption [Срок оценки: 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 [Срок оценки: Within 4 hours after the start of CSL889 infusion]
Percentage of participants with ≥ 30% pain reduction by NRS score [Срок оценки: Within 4 hours after the start of CSL889 infusion]
Maximum observed concentration (Cmax) after Doses 1 and 3 of CSL889 [Срок оценки: Before dosing, and up to 12 hours after Doses 1 and 3]
Area under the concentration (AUCtau) after Doses 1 and 3 of CSL889 [Срок оценки: Before dosing, and up to 12 hours after Doses 1 and 3]

Критерии участия

Критерии включения

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.

Критерии исключения

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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет