Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ended
Participants planned
40
Countries
1
Sites
16
Patients with high risk myelodysplastic syndrome or acute myeloid leukemia
Achieving a disease-free survival of 35% (expected 12%), 12 months after transplant
Key facts
- Sponsor
- Groupe Francophone Des Myelodysplasies
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Neoplasms [C04]
- Trial duration
- 22 Jun 2021 → 22 Apr 2025
- Decision date (initial)
- 2024-10-18
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- ASTEX
External identifiers
- EU CT number
- 2024-515353-24-00
- EudraCT number
- 2019-003685-40
- ClinicalTrials.gov
- NCT04857645
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy, Therapy
Achieving a disease-free survival of 35% (expected 12%), 12 months after transplant
Secondary objectives 7
- Proportion of patients receiving allo SCT
- Proportion of patients eligible for post-transplant oral decitabine
- Proportion of patients eligible for post-transplant DLI
- Grade III/IV toxicities
- Non-relapse mortality
- Proportion of patients completing treatment schedule
- Overall survival at 12 and 24 months, DFS at 24 months after transplant
Conditions and MedDRA coding
Patients with high risk myelodysplastic syndrome or acute myeloid leukemia
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.0 | LLT | 10028534 | Myelodysplastic syndrome NOS | 10029104 |
| 21.0 | LLT | 10000886 | Acute myeloid leukemia | 10029104 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 7
- Patients aged from 18 to 70 years
- MDS, CMML or AML should have at least one of those criteria: 1) For MDS: Revised IPSS poor or very poor ; For AML: ELN adverse risk ; 2) Unfavorable genetics defined as follow: 4 or more cytogenetic abnormalities or 3 cytogenetic abnormalities and TP53 or other unfavorable mutations (ASXL1, RUNX1) or 3 cytogenetic abnormalities and monosomal karyotype or mutations involving EVI1 ; 3) AML post MDS or MPN, or AML relapsing less than 2 years after first CR
- AML patients should have received chemotherapy
- Marrow blast < 20% for MDS and < 10% for AML post chemotherapy before HSCT
- Non-proliferative disease
- A donor is available (HLA matched or mismatched)
- Adequate contraception in women < 50 years and for men. Subjects must agree to use, and to be able to comply with, effective contraception without interruption, at least the first six months after transplant, throughout the entire duration of study drug therapy and for 3 months after the last dose of study drug therapy.
Exclusion criteria 11
- ECOG 3 or more
- Cancer less than 2 years before inclusion or cancer not in remission the last 2 years before inclusion (except in situ cancer or baso cellular cancer)
- Cardiac failure with EF < 50%
- Creatininemia level > 150 µmol/L
- Liver enzyme > 3 N
- Conjugated bilirubinemia > 25 µmol/L
- MDS occurring in patients with Fanconi anemia or congenital dyskeratosis
- Proliferative disease in patients not in remission: WBC > 15 G/L or use of continuous cytotoxic to maintain WBC < 15 G/L
- AML with marrow or peripheral blast count higher than 10% after chemotherapy
- No contraception
- Pregnant or breastfeeding women
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- DFS at 1 year post transplant
Secondary endpoints 2
- Overall survival from the date of transplantation and from the date of inclusion at 1 year and 2 years
- Risk factors for DFS, OS NRM at 1 year and 2 years
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD11224174 · Product
- Active substance
- Decitabine
- Substance synonyms
- 5-AZA-2'-DEOXYCYTIDINE, 4-AMINO-1-(2-DEOXY-.BETA.-D-ERYTHRO-PENTOFURANOSYL)-S-TRIAZIN-2(1H)-ONE
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 1 Other
- Max total dose
- 1 Other
- Max treatment duration
- 10 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- TAIHO ONCOLOGY, INC.
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Groupe Francophone Des Myelodysplasies
- Sponsor organisation
- Groupe Francophone Des Myelodysplasies
- Address
- Opital St Louis Hemato Seniors T4, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux
- City
- Paris
- Postcode
- 75010
- Country
- France
Scientific contact point
- Organisation
- Groupe Francophone Des Myelodysplasies
- Contact name
- Marie ROBIN
Public contact point
- Organisation
- Groupe Francophone Des Myelodysplasies
- Contact name
- Marie ROBIN
Locations
1 EU/EEA country · 16 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ended | 40 | 16 |
| Rest of world | — | 0 | — |
Investigational sites
Centre Hospitalier Universitaire D'Angers
Service des maladies du sang, 4 Rue Larrey, 49100, Angers
Centre Henri Becquerel
Département d'hématologie, 1 Rue D Amiens, 76000, Rouen
Centre Hospitalier Universitaire De Bordeaux
Hôpital de Haut-Lévêque - Service des maladies du sang, Avenue De Magellan, 33600, Pessac
Assistance Publique Hopitaux De Paris
Hôpital Pitié Salpétrière - Service d'hématologie clinique, 47 Boulevard De L Hopital, 75651, Paris Cedex 13
Centre Hospitalier Universitaire De Montpellier
Hôpital Saint Eloi - Service d'hématologie clinique, 80 Avenue Augustin Fliche, 34295, Montpellier Cedex 5
Centre Hospitalier Universitaire De Nantes
Hôtel Dieu - Service d'hématologie clinique, 1 Place Alexis Ricordeau, 44000, Nantes
Centre Hospitalier Universitaire De Toulouse
IUCT Oncopole - Département d'hématologie, 1 Avenue Irene Joliot Curie, 31059, Toulouse Cedex 9
Assistance Publique Hopitaux De Paris
Hôpital Saint Louis - Service d'hématologie-greffe, 1 Avenue Claude Vellefaux, 75010, Paris
Assistance Publique Hopitaux De Paris
Hôpital Necker - Service d'hématologie adulte, 149 Rue De Sevres, 75015, Paris
Hospices Civils De Lyon
CH Lyon sud - Service d'hématologie clinique, 165 Chemin Du Grand Revoyet, 69310, Pierre Benite
Centre Hospitalier Universitaire De Saint Etienne
ICLN - Hématologie clinique-Thérapie cellulaire, Avenue Albert Raimond, 42270, Saint Priest En Jarez
Centre Hospitalier Universitaire Amiens Picardie
Service d'hématologie clinique et thérapie cellulaire, 30 Avenue De La Croix Jourdain, 80054, Amiens Cedex 1
Centre Hospitalier Universitaire Grenoble Alpes
Clinique universitaire d'hématologie, Boulevard De La Chantourne, Cs 10217, Grenoble Cedex 9
CHRU De Nancy
CHU Brabois - Service d'hématologie clinique, Rue Du Morvan, 54500, Vandoeuvre Les Nancy
Centre Hospitalier Et Universitaire De Limoges
Hôpital Dupuytren - Service d'hématologie clinique et thérapie cellulaire, 2 Avenue Martin Luther King, 87042, Limoges Cedex 1
University Hospital Of Clermont-Ferrand
CHU Estaing - Service d'hématologie clinique et de thérapie cellulaire, 58 Rue Montalembert, 63003, Clermont Ferrand Cedex 1
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| France | 2021-06-22 | 2025-04-22 | 2021-06-22 | 2023-02-23 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Summary of results Art. 37(4) CTR
| Title | Submission date | Status | Type |
|---|---|---|---|
| Summary of results_2024-515353-24-00 SUM-115950
|
2026-01-22T11:23:38 | Submitted | Summary of Results |
Layperson summary Annex V
| Title | Submission date | Status | Type |
|---|---|---|---|
| Lay Person Summary_2024-515353-24-00 | 2026-01-22T11:25:29 | Submitted | Laypersons Summary of Results |
Documents 8 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Laypersons summary of results (for publication) | Lay Person Summary_2024-515353-24-00_ENG | 1 |
| Laypersons summary of results (for publication) | Lay Person Summary_2024-515353-24-00_FR | 1 |
| Protocol (for publication) | D1_Protocol 2024-515353-24-00 | 4.1 |
| Recruitment arrangements (for publication) | 2024-515353-24-00_document_additionnel_V1_20240819_GFM | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_Blank document | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF | 4 |
| Summary of results (for publication) | Summary of results_2024-515353-24-00 | 1 |
| Synopsis of the protocol (for publication) | D1_ Protocol synopsis_FR 2024-515353-24-00 | 4 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-09-30 | France | Acceptable 2024-10-14
|
2024-10-18 |