FORMAT Study : Use of fibrinogen in the treatment of bleeding in thrombopenic patients after intensive chemotherapy refractory to platelet transfusion - evaluation by rotem viscoelastometry (pilot single-center study).

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Centre Hospitalier Universitaire De Saint Etienne

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

10 Feb 2022 → 17 Dec 2025

Decision date (initial)

2024-08-29

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

ARMOE (Aide à la Recherche Médicale Ondaine et Environs)

External identifiers

EU CT number

2024-516494-73-00

EudraCT number

2021-000990-10

ClinicalTrials.gov

NCT05091684

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To assess the efficacy of the administration of fibrinogen associated with a platelet transfusion on the relevant visco-elastometric parameters (ROTEM method) in patients with malignant hemopathy, thrombocytopenics, refractory to platelet transfusions, with grade ≥ I hemorrhagic signs according to the WHO - WHO classification, modified according to Slichter.

Secondary objectives 9

1. Evaluate the effect of each treatment administered on the ROTEM viscoelastometry results and the persistence of this effect at 24 hours
2. Evaluate the hemostatic response to platelet transfusion according to the Corrected Count Increment and the cause in the refractory state
3. Evaluate the visco-elastometric response according to the characteristics of the transfused platelet concentrates
4. Evaluate the hemostatic response to fibrinogen as a function of the dose received relative to the patient's weight and the plasma concentration of fibrinogen obtained
5. Evaluate the incidence of hemorrhagic and thrombotic events
6. Evaluate the impact of hemorrhagic and thrombotic risk factors
7. Evaluate the time taken to implement anti-haemorrhagic treatment
8. Compare the availability of the results of the usual coagulation tests and of the blood count with that of the results of the visco-elastometric tests
9. Evaluate the incidence of adverse events

Conditions and MedDRA coding

Hematological malignancy

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Major patient
2. Patient with malignant hemopathy requiring intensive chemotherapy, autologous or allogeneic hematopoietic stem cell transplantation
3. Grade ≥ 1 hemorrhagic symptom according to WHO - WHO classification
4. Failure or impossibility of transfusion of HLA-matched platelet concentrates
5. Body weight between 38 and 78 kg
6. Patient presenting with a platelet refractory state defined according to the Corrected Count Increment (CCI)

Exclusion criteria 15

1. Patient who expressed his opposition to participating in the study
2. Pregnant or breastfeeding woman
3. Legal incapacity or limited legal capacity. Medical or psychological conditions that do not allow the subject to understand the study and sign the consent
4. Patient with non-malignant hematological involvement
5. Patient with a high plasma fibrinogen concentration (> 5g / L)
6. Patients who received fibrinogen administration within 20 days prior to study entry
7. Patient allergic to fibrinogen
8. Patient with disseminated intravascular coagulopathy
9. Patients at known risk of thrombophilia
10. Indication for the use of anti-thrombotic treatment (anti-platelet, anticoagulant)
11. Patients with a thromboembolic history
12. Patients treated with L-Asparaginase or with hypofibrinogenemia secondary to L-Asparaginase treatment
13. Patient with hyperthermia ≥ 38.5°C
14. Patient hospitalized for an invasive procedure
15. Patients with acute leukemia during the induction chemotherapy phase

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Effect of the administration of fibrinogen and the transfusion of a platelet concentrate on the visco-elastometric parameter 'maximum clot elasticity' (MCE) deduced from the MCF "maximum clot firmness" of the EXTEM curve (ROTEM - initiation of coagulation with tissue factor) between the 1st and 3rd laboratory investigation (1st before administration of fibrinogen, and 3rd after administration of fibrinogen and platelets).

Secondary endpoints 9

1. Comparison of the ROTEM viscoelastometric parameters before and after treatment, under EXTEM and FIBTEM conditions (determination of the contribution of fibrinogen) and with all the other ROTEM parameters between the 1st, 2nd and 3rd laboratory investigation.
2. Comparison of all parameters in ROTEM viscoelastometry under EXTEM and FIBTEM conditions before and after platelet transfusion as a function of CCI and the cause in the refractory state.
3. Comparison of parameters in ROTEM viscoelastometry before and after platelet transfusion as a function of the characteristics of the platelet concentrates.
4. Comparison of the ROTEM viscoelastometry parameters before and after fibrinogen as a function of the dose received relative to the patient's weight and the plasma concentration of fibrinogen
5. Incidence of hemorrhagic events and thrombotic events
6. Collection of hemorrhagic and thrombotic risk factors.
7. Time from diagnosis of bleeding to administration of treatment; delay between the first ROTEM results and the administration of treatments; and ROTEM results before and after procedures.
8. Time to get the first ROTEM result and the result of conventional coagulation tests and complete blood count after blood collection.
9. Collection of adverse events and serious adverse events.

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Centre Hospitalier Universitaire De Saint Etienne

Sponsor organisation

Centre Hospitalier Universitaire De Saint Etienne

Address

Avenue Albert Raimond

City

Saint Priest En Jarez

Postcode

42270

Country

France

Scientific contact point

Organisation

Centre Hospitalier Universitaire De Saint Etienne

Contact name

project manager

Public contact point

Organisation

Centre Hospitalier Universitaire De Saint Etienne

Contact name

project manager

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 5 files

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

Application history

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