A Multicentre, Adaptive, Randomised, Multidomain, Platform Trial for Dose Optimization in the Treatment of Adult Patients With Haematological Diseases (BLOOD-dose): Core Protocol
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 400
- 试验地点
- 5
- 主要终点
- Overall survival
研究概览
简要总结
BLOOD-dose is a multicentre, adaptive, randomized, multidomain platform trial designed to optimize treatment dosing strategies in adult patients with haematological diseases.
The BLOOD-dose core protocol outlines the overall clinical trial design that applies to all included interventions, while domain-specific appendices (DSA) detail the unique characteristics of each domain and specify domain-specific interventions.
New domains will be incorporated over time to address distinct dose-optimization research questions across different haematological conditions and interventions.
详细描述
Background:
Approved dosing regimens in haematology are largely derived from clinical trials conducted in relatively homogeneous patient populations, which may not reflect the diversity encountered in routine clinical practice. Many new anticancer and haematological treatments are developed using early phase trial designs that define dose selection primarily based on dose-limiting toxicity, often aiming to establish a maximum tolerated dose. While this approach supports regulatory approval, it may not identify the optimal biological or clinically effective dose for long-term treatment. This uncertainty may contribute to overtreatment, increased toxicity, impaired quality of life, and unnecessary healthcare costs. Furthermore, established long-term or life-long treatment regimens represent important opportunities for dose optimization, especially as therapeutic strategies and patient needs evolve over time.
Platform trials provide an efficient framework to evaluate multiple interventions within a single disease area under a unified master protocol. In domain-based platform trials, interventions are grouped into predefined domains, enabling efficient comparisons, rapid progress, and the addition of new research domains over time.
Objectives:
The BLOOD-dose platform trial aims to determine the optimal treatment intensity for patients with haematological diseases. Due to disease heterogeneity, objectives, endpoints, and estimands will vary across domains.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants of any sex who are at least 18 years of age at the time of providing informed consent.
- •Participant diagnosed with a haematological disease, i.e. any disorder that primarily affects the blood, bone marrow, the lymphatic system and/or blood-forming organs.
- •Should be eligible for participation in at least one of the currently active domains.
- •Capable of giving signed informed consent for each applicable DSA(s). By consenting to a domain, participants also consent to participation in BLOOD-dose.
排除标准
- •The participant tient is expected to live less than 3 months, as judged by the investigator.
- •Any condition that, in the opinion of the investigator, impairs the participant's ability to understand trial procedures, provide informed consent and/or interfere with participation and/or compliance in the trial.
- •Domain eligibility criteria: each domain has its own specific eligibility criteria detailed in each DSA.
研究组 & 干预措施
Standard dose of teclistamab OR talquetamab OR elranatamab OR linvoseltamab
Standard dose of teclistamab OR talquetamab OR elranatamab OR linvoseltamab in patients with relapsed/refractory multiple myeloma
干预措施: teclistamab OR talquetamab OR elranatamab OR linvoseltamab (Drug)
Reduced dose of teclistamab OR talquetamab OR elranatamab OR linvoseltamab
Reduced frequency of teclistamab OR talquetamab OR elranatamab OR linvoseltamab in patients with relapsed/refractory multiple myeloma
干预措施: teclistamab OR talquetamab OR elranatamab OR linvoseltamab (Drug)
Standard-dose BTK inhibitors in patients with Waldenström´s macroglobulinemia
A phase 4, open-label, parallel-group, two-arm domain to assess the effectiveness and safety of reduced-dose BTK inhibitors (ibrutinib and zanubrutinib) compared to standard-dose in male and female patients with Waldenström´s macroglobulinemia
干预措施: BTK inhibitors (ibrutinib and zanubrutinib) (Drug)
Reduced dose of BTK inhibitors in patients with Waldenström´s macroglobulinemia
Phase 4, open-label, parallel-group, two-arm domain to assess the effectiveness and safety of reduced-dose BTK inhibitors (ibrutinib and zanubrutinib) compared to standard-dose in male and female patients with Waldenström´s macroglobulinemia
干预措施: BTK inhibitors (ibrutinib and zanubrutinib) (Drug)
结局指标
主要结局
Overall survival
时间窗: OS is defined as the time from randomization until the time of death due to any cause, assessed up to 5 years.
To compare survival between the interventions. The interventions will be defined in the DSA. The end of period follow-up will be defined in the DSA.
次要结局
- Progression free survival(PFS is defined as the time from randomization until clinical progression or death from any cause, assessed up to 5 years.)
- Patient-reported health-related quality of life(1 year)
- Number of Participants with Treatment Emergent Adverse Events as Assessed by CTCAE v6.0(Through study completion, an average of 1 year)
- Hospital Admission(From Time of randomization to end of follow-up, assessed up to 2 years.)
- Cost of intervention(From first dose to last recorded date of dosing OR From randomization to last recorded date of dosing or end of study, whichever occurs first, assessed up to 2 years.)
研究者
Anne Louise Tølbøll Sørensen
Clinical Associate Professor
Rigshospitalet, Denmark