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一项研究 Crizanlizumab (5 mg/kg) 与安慰剂相比，对经常发生血管闭塞危象的青少年和成人镰状细胞病患者的疗效和安全性 (SPARKLE) (SPARKLE)

2026年11月9日 更新者：Novartis Pharmaceuticals

一项 III 期、多中心、随机、安慰剂对照、双盲研究，旨在评估 Crizanlizumab (5 mg/kg) 与安慰剂相比，在有或没有羟基脲/羟基脲治疗的情况下，在患有频繁血管收缩的青少年和成人镰状细胞病患者中的疗效和安全性-闭塞危机

A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.

研究概览

地位

招聘中

条件

干预/治疗

详细说明

Study CSEG101A2303 (SPARKLE) is a Phase III, multicenter, randomized, double-blind study to assess efficacy and safety of crizanlizumab 5 mg/kg versus placebo, with or without hydroxyurea/ hydroxycarbamide therapy (HU/HC), in Sickle Cell Disease patients aged 12 years and older with frequent vaso-occlusive crises (4-12 events in 12 months prior to the screening visit).

Participants will be randomized in a 2:1 ratio to the crizanlizumab 5 mg/kg or placebo treatment arm. Central randomization will be stratified by concomitant HU/HC usage (yes/no) and region (South America, North America, and sub-Saharan Africa) at baseline.

研究类型

介入性

注册 (估计的)

354

阶段

  • 第三阶段

扩展访问

可用的 查看扩展访问记录。

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

研究联系人备份

  • 姓名：Novartis Pharmaceuticals
  • 电话号码：+41613241111

学习地点

  • 乌干达
      • Kampala、乌干达、101
        • 招聘中
        • Novartis Investigative Site
      • Masaka、乌干达、001
        • 招聘中
        • Novartis Investigative Site
      • Tororo、乌干达、10102
        • 招聘中
        • Novartis Investigative Site
  • 哥伦比亚
      • Montería、哥伦比亚、230001
        • 招聘中
        • Novartis Investigative Site
    • Antioquia
      • Medellín、Antioquia、哥伦比亚、050001
        • 招聘中
        • Novartis Investigative Site
    • Valle del Cauca Department
      • Cali、Valle del Cauca Department、哥伦比亚、760046
        • 招聘中
        • Novartis Investigative Site
      • Cali、Valle del Cauca Department、哥伦比亚、760032
        • 招聘中
        • Novartis Investigative Site
  • 巴西
    • Estado de Bahia
      • Salvador、Estado de Bahia、巴西、41253-190
        • 招聘中
        • Novartis Investigative Site
    • Maranhão
      • São Luís、Maranhão、巴西、65020-070
        • 招聘中
        • Novartis Investigative Site
    • São Paulo
      • Campinas、São Paulo、巴西、13083-970
        • 招聘中
        • Novartis Investigative Site
      • Ribeirão Preto、São Paulo、巴西、14048-900
        • 招聘中
        • Novartis Investigative Site
      • Sao Jose Rio Preto、São Paulo、巴西、15090-000
        • 招聘中
        • Novartis Investigative Site
      • São Paulo、São Paulo、巴西、08270-070
        • 招聘中
        • Novartis Investigative Site
      • São Paulo、São Paulo、巴西、01232-010
        • 招聘中
        • Novartis Investigative Site
  • 美国
    • Alabama
      • Birmingham、Alabama、美国、35233
        • 招聘中
        • University Of Alabama
        • 首席研究员：
          • Chibuzo Churchill Ilonze
        • 接触：
    • California
      • Orange、California、美国、92868
        • 招聘中
        • Ctr for Inherited Blood Disorders
        • 接触：
        • 首席研究员：
          • Vanessa Salinas
    • District of Columbia
      • Washington D.C.、District of Columbia、美国、20010
        • 招聘中
        • Childrens National Hospital
        • 首席研究员：
          • Andrew Campbell
        • 接触：
    • Florida
      • Jacksonville、Florida、美国、32209
    • Georgia
      • Augusta、Georgia、美国、30912
        • 招聘中
        • Augusta University Georgia
        • 首席研究员：
          • Girindra Raval
        • 接触：
      • Riverdale、Georgia、美国、30274
        • 招聘中
        • WCG Sonar Clinical Research
        • 首席研究员：
          • Anthony Onyegbula
        • 接触：
    • Illinois
      • Chicago、Illinois、美国、60612
        • 招聘中
        • Uni of Illinois Hospital and HSC
        • 接触：
        • 首席研究员：
          • Santosh Saraf
    • Kentucky
      • Louisville、Kentucky、美国、40202
    • Maryland
      • Baltimore、Maryland、美国、21205
        • 招聘中
        • The Johns Hopkins University School of Medicine
        • 首席研究员：
          • Lydia Pecker
        • 接触：
    • Mississippi
      • Flowood、Mississippi、美国、39232
    • New York
      • The Bronx、New York、美国、10467
        • 招聘中
        • Childrens Hospital at Montefiore
        • 首席研究员：
          • Kaitlin Strumph
        • 接触：
    • North Carolina
      • Greenville、North Carolina、美国、27834
        • 招聘中
        • East Carolina University
        • 首席研究员：
          • Beng Fuh
        • 接触：
      • Winston-Salem、North Carolina、美国、27157
    • Pennsylvania
      • Easton、Pennsylvania、美国、18045
        • 撤销
        • Spoknwrdclinicaltrials
      • Philadelphia、Pennsylvania、美国、19107
    • Texas
      • Houston、Texas、美国、77030
      • Houston、Texas、美国、77030
        • 招聘中
        • Texas Childrens Cancer and Hematology Center
        • 首席研究员：
          • Venee Tubman
        • 接触：
  • 肯尼亚
      • Eldoret、肯尼亚、30100
        • 招聘中
        • Novartis Investigative Site
      • Kisumu、肯尼亚、40100
        • 招聘中
        • Novartis Investigative Site
      • Kisumu、肯尼亚、40103
        • 招聘中
        • Novartis Investigative Site
      • Siaya、肯尼亚、40600
        • 招聘中
        • Novartis Investigative Site
    • Kisumu County
      • Ahero、Kisumu County、肯尼亚、40100
        • 招聘中
        • Novartis Investigative Site

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

12年 至 100年 (孩子、成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

描述

Key Inclusion Criteria:

  1. Participants must be aged 12 years and older on the day of signing informed consent. Adolescents include participants aged 12 to <18 years old and adults include participants aged 18 years and older.
  2. Confirmed diagnosis of SCD by Hb electrophoresis or high-performance liquid chromatography (HPLC) (performed locally or by central laboratory if not available locally). All SCD genotypes are eligible.
  3. Experienced 4 to 12 VOCs (refer to Section 8.3.1 for study definition of VOC) that are HCP-managed (including VOCs leading to management at a health care facility or those managed via remote consultation) within the 12 months prior to the screening visit. Baseline VOCs are determined by medical history and are required to be documented at source.
  4. If the participant is on HU/HC, they must be taking it for at least 6 months and at stable dose for at least 3 months prior to the Screening visit and plan to continue taking it at the same dose and schedule until at least the participant has reached 52 weeks of the planned study treatment. Participants who have initiated HU/HC 6-12 months prior to the screening visit must have evidence of insufficient control of acute pain despite initiation. These participants must have a cumulative of 4-12 VOCs in the 12 months prior to the screening period, with at least 2 during the last 6 months while on HU/HC. If receiving erythropoietin stimulating agent, the participant must have been receiving the drug for at least 6 months prior to screening visit and plan to continue taking the drug at the same dose and schedule until the participant has reached 52 weeks of the planned study treatment.

Participants who have not been receiving HU/HC, and/or erythropoietin stimulating agent must not have received it for at least 6 months prior to screening visit.

Key Exclusion Criteria:

  1. Fewer than 4 or more than 12 VOCs that are HCP-managed (including VOCs leading to management at a health care facility or those managed via remote consultation) within the 12 months prior to screening visit as determined by medical history and documented at source.
  2. History of stem cell transplant and/or gene therapy.
  3. Received blood products within 30 days prior to Week 1 Day 1 dosing.
  4. Any documented history of a clinical stroke or intracranial hemorrhage, or an uninvestigated neurologic finding within the past 12 months before screening visit. Silent infarct only present on imaging is not excluded.
  5. Participating in a chronic transfusion program (pre-planned series of transfusions for prophylactic purposes) and/or planning to undergo an exchange transfusion during the duration of the study; episodic transfusion in response to worsened anemia or VOC is permitted.
  6. Contraindication or hypersensitivity to any drug or metabolites from similar class as study drug or to any excipients of the study drug formulation. History of severe hypersensitivity reaction to other monoclonal antibodies, which in the opinion of the investigator may pose an increased risk of serious infusion reaction.

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：并行分配
  • 屏蔽：四人间

武器和干预

参与者组/臂
干预/治疗
实验性的：Crizanlizumab (SEG101) at 5.0 mg/kg

Participants receive Crizanlizumab (SEG101) at 5.0 mg/kg and standard of care.
生物：Crizanlizumab

Crizanlizumab is supplied in single use 10 mL glass vials at a concentration of 10 mg/mL. One vial contains 100 mg of crizanlizumab. This is a concentrate for solution for IV infusion.

其他名称：
  • SEG101、Adakveo®、Ryverna®
安慰剂比较：Placebo

Participants receive the placebo drug and standard of care.
药品：Placebo

Placebo is supplied in single use 10 mL glass vials at a concentration of 0 mg/mL. This is a concentrate for solution for IV infusion.

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Annualized rate of VOCs that are healthcare professional (HCP)-managed (including VOCs leading to management at a health care facility or those managed via remote consultation) in each treatment arm
大体时间：1 year

Vaso oclusive crisis (VOC) is defined as a pain crisis (acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) lasting for at least 4 hours which is treated as per local guidelines with standard of care therapy used to treat VOC. Acute chest syndrome (ACS), priapism and hepatic or splenic sequestration will be considered VOC in this study.

VOCs included are those HCP-managed in a healthcare facility and HCP-managed via remote consultation.

Annualized rate of VOC events = (Number of VOC events * 365)/(number of days in the observation period).

Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days).
1 year

次要结果测量

结果测量
措施说明
大体时间
The annualized rate of all VOCs including VOCs that are HCP-managed (either at a health care facility or via remote consultation) as well as those that are self-managed without recommendations from HCP during the event in each treatment arm
大体时间：1 year

VOCs can be categorized as those HCP-managed in a healthcare facility, HCP-managed via remote consultation, or self-managed without recommendations from HCP during the event.

Annualized rate of VOC events = (Number of VOC events * 365)/(number of days in the observation period).

To capture VOC events that are self-managed, and in order to avoid VOC recall bias and to make sure the pain events are captured in real-time, a cloud-based application will be used and setup an account for each participant.
1 year
Annualized rate of VOC by subtype of management in each treatment arm over the planned 52-week period.
大体时间：1 year

Vaso oclusive crisis (VOC) is defined as a pain crisis lasting for at least 4 hours which is treated as per local guidelines with standard of care therapy. Acute chest syndrome (ACS), priapism and hepatic or splenic sequestration will be considered VOC in this study.

VOCs can be categorized as those HCP-managed in a healthcare facility, HCP-managed via remote consultation, or self-managed without recommendations from HCP during the event.

Annualized rate of VOC is the number of VOC events during a year period.
1 year
The time to first VOC that is HCP-managed (including VOCs leading to management at a health care facility or those managed via remote consultation) between treatment arms.
大体时间：1 year

VOC is defined as a pain crisis lasting for at least 4 hours which is treated as per local guidelines with standard of care therapy. ACS, priapism and hepatic or splenic sequestration will be considered VOC in this study.

VOCs included are those HCP-managed in a healthcare facility and HCP-managed via remote consultation.

Time to first occurrence of VOC that is HCP-managed (either at a health care facility or via remote consultation) is defined as the time from the date of randomization to the date of the first occurrence of the VOC.
1 year
Proportion of participants free from VOCs that are HCP-managed (including VOCs leading to management at a health care facility or those managed via remote consultation) in each treatment arm over the planned 52-week treatment period.
大体时间：1 year

To assess the number of participants free from VOCs leading to healthcare visit.

VOC is defined as a pain crisis lasting for at least 4 hours which is treated as per local guidelines with standard of care therapy. ACS, priapism and hepatic or splenic sequestration will be considered VOC in this study. A participant is free from VOC if they do not have a VOC crisis.
1 year
Duration of VOCs that are HCP-managed (including VOCs leading to management at a health care facility or those managed via remote consultation) in each treatment arm over the planned 52-week treatment period.
大体时间：1 year

VOC is defined as a pain crisis lasting for at least 4 hours which is treated as per local guidelines with standard of care therapy. ACS, priapism and hepatic or splenic sequestration will be considered VOC in this study.

Duration of HCP-managed VOC is defined as end date of the VOC - start date of the VOC + 1 day.
1 year
Number of participants with anti-SEG101 (crizanlizumab) antibodies (any time)
大体时间：2 years

Immunogenicity: measurement of anti-drug antibodies (ADA) to crizanlizumab.
2 years
Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
大体时间：2 years

Incidence and severity of AEs and SAEs by treatment group, including changes in vital signs, electrocardiograms (ECGs), and laboratory results qualifying and reported as AEs.
2 years
Absolute change from baseline in hemoglobin
大体时间：Baseline, 2 years

Assessment of safety of SEG101
Baseline, 2 years

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Novartis Pharmaceuticals

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2024年10月24日

初级完成 (估计的)

2029年7月2日

研究完成 (估计的)

2030年7月29日

研究注册日期

首次提交

2024年5月27日

首先提交符合 QC 标准的

2024年5月27日

首次发布 (实际的)

2024年6月3日

研究记录更新

最后更新发布 (实际的)

2026年11月10日

上次提交的符合 QC 标准的更新

2026年11月9日

最后验证

2026年11月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • CSEG101A2303

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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