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

Official Summary

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.

Eligibility Requirements

• Minimum Age: 18 Years

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: NONE
• Enrollment: 400 participants

Study Arms

• Standard dose of teclistamab OR talquetamab OR elranatamab OR linvoseltamab (ACTIVE_COMPARATOR)
  Standard dose of teclistamab OR talquetamab OR elranatamab OR linvoseltamab in patients with relapsed/refractory multiple myeloma
• Reduced dose of teclistamab OR talquetamab OR elranatamab OR linvoseltamab (EXPERIMENTAL)
  Reduced frequency of teclistamab OR talquetamab OR elranatamab OR linvoseltamab in patients with relapsed/refractory multiple myeloma
• Standard-dose BTK inhibitors in patients with Waldenström´s macroglobulinemia (ACTIVE_COMPARATOR)
  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
• Reduced dose of BTK inhibitors in patients with Waldenström´s macroglobulinemia (EXPERIMENTAL)
  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

Interventions

• DRUG: teclistamab OR talquetamab OR elranatamab OR linvoseltamab — ElasTEC: A phase 4, open-label, parallel-group, two-arm domain on the BLOOD-dose platform trial to evaluate the non-inferiority, safety, and effectiveness of reduced-frequency bispecific antibody treatments (teclistamab, talquetamab, elranatamab and linvoseltamab) compared with standard-frequency treatment in patients with relapsed/refractory multiple myeloma.
• DRUG: BTK inhibitors (ibrutinib and zanubrutinib) — BELLIS: 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

Primary Outcomes

• Overall survival (OS is defined as the time from randomization until the time of death due to any cause, assessed up to 5 years.)

Secondary Outcomes

• 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.)

Eligibility Criteria

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.

Trial Locations

• Copenhagen University Hospital - Rigshospitalet, Copenhagen, Greater Copenhagen Area, Denmark
• Aalborg University Hospital, Aalborg, Denmark
• Aarhus University Hospital, Aarhus, Denmark
• Odense University Hospital, Odense, Denmark
• Roskilde University Hospital, Roskilde, Denmark

Contact Information

• Anne Louise Tølbøll Sørensen, Ass. Prof. — CONTACT
  Phone: +45 35451864
  Email: anne.louise.toelboell.soerensen@regionh.dk
• Troels Hammer, Ass. Prof. — CONTACT
  Phone: +45 35455198
  Email: troels.hammer@regionh.dk

Study Officials

• Anne Louise Tølbøll Sørensen, Ass. Prof. — STUDY_CHAIR
  Rigshospitalet, Denmark

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