A multicentre, adaptive, randomised, multidomain, platform trial for dose optimization in the treatment of adult patients with haematological diseases (BLOOD-dose): core protocol

2026-525658-13-00 Protocol BLOOD-dose-001 Therapeutic use (Phase IV) Authorised, recruitment pending

Status Authorised, recruitment pending · 1 EU/EEA countries · 5 sites · Protocol BLOOD-dose-001

Overview

Sponsor-declared trial summary

Phase Therapeutic use (Phase IV)
Status Authorised, recruitment pending
Participants planned 200
Countries 1
Sites 5

Haematological diseases; any disorder that primarily affects the blood, bone marrow, the lymphatic system and/or blood-forming organs.

The overall aim of the BLOOD-dose platform trial is to identify the optimal treatment intensity for patients with haematological diseases. Given the heterogeneity of haematological diseases, objectives, endpoints and estimands will differ across domains. The objectives for each domain will be described in the Domain-Sp…

Key facts

Sponsor
Region Hovedstaden
Participant type
Patients
Age range
18-64 years, 65+ years
Gender
Male and Female
Therapeutic area
Diseases [C] - Hemic and Lymphatic Diseases [C15], Diseases [C] - Neoplasms [C04]
Decision date (initial)
2026-06-25
Transition trial
No
Low-intervention
No
Rare-disease indication
Yes
Vulnerable population
No
Funding sources
Rigshospitalet Platform Trial Unit

External identifiers

EU CT number
2026-525658-13-00
WHO UTN
U1111-1334-9059
ClinicalTrials.gov
NCT07474961

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

The overall aim of the BLOOD-dose platform trial is to identify the optimal treatment intensity for patients with haematological diseases. Given the heterogeneity of haematological diseases, objectives, endpoints and estimands will differ across domains. The objectives for each domain will be described in the Domain-Specific Appendix.
A core outcome set (COS) for haematological conditions consisting of in total 6 core outcome measurements has been established. 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. Thus, the main objective of a Domain-Specific Appendix could be to compare survival beween the interventions with the endpoint overall survival (OS), with the other 5 core outcome measures listed below as secondary objectives.

Secondary objectives 5

  1. To compare progression free survival (PFS) between the interventions
  2. To compare patient-reported health-related quality of life between the interventions
  3. To assess safety and tolerability of the interventions
  4. To compare the impact of treatment and disease on healthcare resource use between the interventions
  5. To compare the cost of the interventions

Conditions and MedDRA coding

Haematological diseases; any disorder that primarily affects the blood, bone marrow, the lymphatic system and/or blood-forming organs.

VersionLevelCodeTermSystem organ class
25.0 LLT 10086466 Relapsed/refractory multiple myeloma 100000004848
28.1 PT 10047801 Waldenstrom´s macroglobulinaemia 100000004864

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

  1. Participants of any sex who are at least 18 years of age at the time of providing informed consent.
  2. 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.
  3. Eligible for participation in at least one of the currently active domains.
  4. 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 2

  1. The participant is expected to live less than 3 months, as judged by the investigator.
  2. 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. Overall survival (OS)

Secondary endpoints 5

  1. Progression free survival (PFS)
  2. Patient-reported health-related quality of life (HRQoL)
  3. Number and type, seriousness and relationship to study treatments of AEs, vital signs, and clinical laboratory results, as indicated in the DSA.
  4. Planned hospital admission with no emergency room visit; Unplanned hospital admission with no emergency room visit and with emergency room visit; Length of hospital stay; Length of any time spent in an intensive care unit; mergency room visit only; Outpatient/physician office attendance; Physician/nurse telephone contact visit
  5. Exposure to trial medicinal products; Treatment duration

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 6

TECVAYLI 10 mg/mL solution for injection

PRD9891549 · Product

Active substance
Teclistamab
Substance synonyms
BCMAxCD3, JNJ 64007957
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS INJECTION
Max daily dose
1.5 mg/Kg milligram(s)/kilogram
Max total dose
504 mg/kg milligram(s)/kilogram
Max treatment duration
12 Month(s)
Authorisation status
Authorised
ATC code
L01FX24 — -
Marketing authorisation
EU/1/22/1675/001
MA holder
JANSSEN-CILAG INTERNATIONAL NV
MA country
EU
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/20/2331
Modified vs. Marketing Authorisation
No

LYNOZYFIC 5 mg concentrate for solution for infusion

PRD12371732 · Product

Active substance
Linvoseltamab
Substance synonyms
REGN5458
Pharmaceutical form
SOLUTION FOR INFUSION
Route of administration
INTRAVENIOUS INFUSION
Max daily dose
200 mg milligram(s)
Max total dose
33600 mg milligram(s)
Max treatment duration
12 Month(s)
Authorisation status
Authorised
ATC code
NOTASSIGN — -
Marketing authorisation
EU/1/25/1917/001
MA holder
REGENERON IRELAND D.A.C.
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Zanubrutinib

SCP144382924 · ATC

Active substance
Zanubrutinib
Substance synonyms
7-(1-acryloyl-4-piperidinyl)-2-(4-phenoxyphenyl)-4,5,6,7-tetrahydropyrazolo(1,5-a)pyrimidine-3-carboxamide, (7S)-2-(4-PHENOXYPHENYL)-7-(1-(PROP-2-ENOYL)PIPERIDIN-4-YL)-4,5,6,7-TETRAHYDROPYRAZOLO(1,5-A)PYRIMIDINE-3-CARBOXAMIDE, BGB-3111
Route of administration
ORAL
Max daily dose
320 mg milligram(s)
Max total dose
115200 mg milligram(s)
Max treatment duration
12 Month(s)
Authorisation status
Authorised
ATC code
L01EL03 — ZANUBRUTINIB
Marketing authorisation
-
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/19/2167 +1
Modified vs. Marketing Authorisation
No

ELREXFIO 40 mg/mL solution for injection

PRD10988293 · Product

Active substance
Elranatamab
Substance synonyms
PF-06863135, Humanised IgG2k Fc-modified bispecific monoclonal antibody against CD3 and BCMA, RN613
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS INJECTION
Max daily dose
76 mg milligram(s)
Max total dose
25536 mg milligram(s)
Max treatment duration
12 Month(s)
Authorisation status
Authorised
ATC code
L01FX32 — -
Marketing authorisation
EU/1/23/1770/002
MA holder
PFIZER EUROPE MA EEIG
MA country
EU
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/21/2471
Modified vs. Marketing Authorisation
No

TALVEY 2 mg/mL solution for injection

PRD10770266 · Product

Active substance
Talquetamab
Substance synonyms
JNJ-64407564, Anti-GPRC5D x anti-CD3E IgG4 humanised and chimeric monoclonal antibody, Anti-(G protein-coupled receptor class C group 5 member D) x anti-CD3E IgG4 humanised and chimeric monoclonal antibody, JNJ-63483043
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS
Max daily dose
0.8 mg/Kg milligram(s)/kilogram
Max total dose
230.4 mg/Kg milligram(s)/kilogram
Max treatment duration
12 Month(s)
Authorisation status
Authorised
ATC code
NOTASSIGN, L01FX29 — -, -
Marketing authorisation
EU/1/23/1748/001
MA holder
JANSSEN-CILAG INTERNATIONAL NV
MA country
EU
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/21/2486
Modified vs. Marketing Authorisation
No

Ibrutinib

SCP31316403 · ATC

Active substance
Ibrutinib
Substance synonyms
1-((3R)-3-(4-AMINO-3-(4-PHENOXYPHENYL)-1H-PYRAZOLO(3,4-D)PYRIMIDIN-1-YL)PIPERIDIN-1- YL)PROP-2-EN-1-ONE
Route of administration
ORAL USE
Max daily dose
420 mg milligram(s)
Max total dose
151200 mg milligram(s)
Max treatment duration
12 Month(s)
Authorisation status
Authorised
ATC code
L01EL01 — IBRUTINIB
Marketing authorisation
-
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/13/1212 +3
Modified vs. Marketing Authorisation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Region Hovedstaden

45 Total trials 25 Recruiting 7 Ended
Academic / Non-commercial
Sponsor organisation
Region Hovedstaden
Address
Nordre Fasanvej 57
City
Frederiksberg
Postcode
2000
Country
Denmark

Scientific contact point

Organisation
Region Hovedstaden
Contact name
Anne Louise Tølbøll Sørensen

Public contact point

Organisation
Region Hovedstaden
Contact name
Anne Louise Tølbøll Sørensen

Third parties 1

OrganisationCity, countryDuties
Region Hovedstaden
ORG-100003705
 Frederiksberg, Denmark On site monitoring

Locations

1 EU/EEA country · 5 investigational sites

By country

CountryMS statusPlanned subjectsSites
Denmark Authorised, recruitment pending 200 5
Rest of world — 0 —

Investigational sites

Denmark

5 sites · Authorised, recruitment pending
Region Syddanmark
Hæmatologisk Forskningsenhed, J.B. Winsloews Vej 18, 5000, Odense C
Region Sjaelland
Hæmatologisk Afdeling, Vestermarksvej 6, 4000, Roskilde
Region Midtjylland
AUH Blodsygdomme, Palle Juul-Jensens Boulevard 99, 8200, Aarhus N
Aalborg University Hospital
Afdeling for Blodsygdomme, Hobrovej 18-22, 9000, Aalborg
Rigshospitalet
Department of Haematology, Blegdamsvej 9, 2100, Copenhagen Oe

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 16 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) D5_ Subprotocol 2026-525658-13-00_BELLIS 1.2
Protocol (for publication) D5_ Subprotocol_2026-525658-13-00_ElasTEC 1.2
Protocol (for publication) D5_Master protocol 2026-525658-13-00 BLOOD-dose 1.2
Recruitment arrangements (for publication) K1_ Recruitment arrangements 1.1
Subject information and informed consent form (for publication) L1_ SIS_2026-525658-13-00 BLOOD-dose 1.1
Subject information and informed consent form (for publication) L1_SIS and ICF adults_ 2026-525658-13-00_BELLIS 1.1
Subject information and informed consent form (for publication) L1_SIS and ICF adults_2026-525658-13-00_ElasTEC 1.1
Summary of Product Characteristics (SmPC) (for publication) G2_SmPC Elranatamab 1
Summary of Product Characteristics (SmPC) (for publication) G2_SmPC Ibrutinib 1
Summary of Product Characteristics (SmPC) (for publication) G2_SmPC Linvoseltamab 1
Summary of Product Characteristics (SmPC) (for publication) G2_SmPC Talquetamab 1
Summary of Product Characteristics (SmPC) (for publication) G2_SmPC Teclistamab 1
Summary of Product Characteristics (SmPC) (for publication) G2_SmPC Zanubrutinib 1
Synopsis of the protocol (for publication) D5_ Subprotocol synopsis_ENG 2026-525658-13-00_BELLIS 1.1
Synopsis of the protocol (for publication) D5_ Subprotocol synopsis_ENG 2026-525658-13-00_ElasTEC 1.1
Synopsis of the protocol (for publication) D5_Master protocol synopsis_ENG 2026-525658-13-00 BLOOD-dose 1.1

Application history

1 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2026-03-24 Denmark Acceptable
2026-06-25
 2026-06-25