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.

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.

Outcomes:

Given the heterogeneity of haematological diseases, objectives, endpoints, and estimands will differ across domains. A core outcome set (COS) comprising 6 core outcome measurements has been established through a Delphi consensus process. Each domain is expected to include at least one core outcome measure as its primary endpoint, with all other core outcomes included as secondary endpoints.

Design:

BLOOD-dose is an investigator-initiated, multicentre, adaptive, randomized, multidomain platform trial.

Domains and interventions:

Interventions across different haematological diseases will be defined in domain-specific appendices that will be amended over time.

Eligibility:

In addition to meeting the core protocol eligibility criteria, participants must also meet the domain-specific eligibility criteria for at least one domain.

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.

The BLOOD-dose management committee owns the rights to all intellectual property and the combined data collected as part of BLOOD-dose; individual sites retain ownership of data collected at their specific sites.

An anonymised version of the final dataset for each domain may be shared with other researchers following a reasonable request (i.e., a research proposal outlining the objectives, methodologies, and plans for data usage) and subsequent approval by the platform and domain management committees.

Participants will be informed about the possibility of data sharing during the informed consent process.

Analysis code may be shared with other researchers after reasonable request and approval by the platform and domain management committee.

15.01.2030 - 15.01.2055

An anonymised version of the final dataset for each domain may be shared with other researchers upon reasonable request. Requests should include a research proposal outlining the objectives, methodologies, and intended use of the data, and will be subject to review and approval by the platform and relevant domain management committees.

Data sharing will be conducted in accordance with the General Data Protection Regulation (GDPR) and all applicable national and international legislative and regulatory requirements governing the protection of personal data. Only fully anonymised data will be shared, and appropriate safeguards and data governance procedures will be applied to ensure compliance with data protection obligations.

The study protocol, Statistical Analysis Plan (SAP), Informed Consent Form (ICF), Clinical Study Report (CSR), and analytical code may be made available upon request.

A webaddress for finding more information about the IPD sharing plan is not yet available