ClinicalTrials.gov record NCT07474961

BLOOD-dose: A Platform Trial Evaluating Dose Optimization in Hematological Diseases.

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

A Multicentre, Adaptive, Randomised, Multidomain, Platform Trial for Dose Optimization in the Treatment of Adult Patients With Haematological Diseases (BLOOD-dose): Core Protocol

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 4A study of a treatment that is already approved, to learn more about its long-term effects, safety or use in everyday practice.
Age18 years old and older
SexAll
LocationDenmark
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

BLOOD-dose is a multicentre, adaptive, randomized, open-label platform trial testing dosing strategies in adults with haematological diseases, including Waldenstrom Macroglobulinaemia and Multiple Myeloma. The trial uses domain-specific appendices to study different drugs and questions; examples include reduced versus standard dosing of bispecific antibodies (teclistamab, talquetamab, elranatamab, linvoseltamab) for relapsed/refractory multiple myeloma and reduced versus standard dosing of BTK inhibitors (ibrutinib, zanubrutinib) in Waldenström´s macroglobulinemia. Outcomes measured include overall survival, progression-free survival, patient-reported quality of life, treatment-emergent adverse events, hospital admissions, and cost.

What it is studying. The study compares reduced-dose or reduced-frequency schedules with standard dosing for specified haematology drugs to evaluate their effects on survival, disease progression, quality of life, adverse events, hospital admissions, and cost.

What the researchers hope to learn. Scientific goals: determine optimal treatment intensity (dose or dosing frequency) for different haematological diseases; compare overall survival and progression-free survival across dosing strategies; measure health-related quality of life, treatment-emergent adverse events, hospitalisation rates, and intervention costs. A core outcome set of measures will be used, with each domain selecting at least one core outcome as its primary endpoint.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • teclistamab OR talquetamab OR elranatamab OR linvoseltamab
  • BTK inhibitors (ibrutinib and zanubrutinib)

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants are randomized within a disease-specific domain to a dosing strategy (typically a standard-dose or a reduced-dose) of the study drug(s) defined for that domain (for example teclistamab/talquetamab/elranatamab/linvoseltamab for multiple myeloma or ibrutinib/zanubrutinib for Waldenström). They receive the assigned treatment and are followed over time to measure outcomes including overall survival and progression-free survival (assessed up to 5 years in domains) and patient-reported quality of life (measured at least up to 1 year). The trial also records treatment-emergent side effects, hospital admissions, and exposure/cost measures (with some follow-up periods described up to 1–2 years depending on the domain). Exact visit schedules, tests, dosing details, and domain-specific eligibility are provided in each domain-specific appendix (DSA), and informed consent is required for each applicable DSA.

How the study is structured. This is an interventional, randomized, parallel-group, open-label, adaptive multidomain platform trial (Phase 4, treatment purpose). Each domain (described in a domain-specific appendix) randomizes participants to compare reduced versus standard dosing for a particular drug or class. New domains and interventions can be added over time. Follow-up periods are defined by each domain; overall survival is assessed up to 5 years and other outcomes up to 1–2 years as specified in the domain-specific appendices.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • The listed sites are in Denmark. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Waldenström macroglobulinemia

Clinical trials63 trials listed for Waldenström macroglobulinemiaEvery trial the registry lists for Waldenström macroglobulinemia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Waldenström macroglobulinemia researchLatest monthly update: September 2026. Includes: Upfront treatment outcomes. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
March 2027 (estimated)
Primary completion
December 2036 (estimated)
Study completion
December 2036 (estimated)
Target enrollment
400 participants (estimated)
Last updated on registry
Mar 16, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Anne Louise Tølbøll Sørensen

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (5)

Terms used on this page · explanations by MedicalUpdates.ai

randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
eligibility criteria
The rules the study team uses to decide who may take part. They are usually split into inclusion criteria and exclusion criteria.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
dose-limiting toxicity
A side effect, defined in advance, that is severe enough to limit how far the dose can be raised in a dose-escalation study. How many such events stop escalation is set by the study's rules.
maximum tolerated dose
The highest dose found in a dose-escalation study that can be given without too many dose-limiting side effects.
endpoints
Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.
primary endpoint
The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

← Back to clinical trials