NCT04184505

Brief Summary

Open-label, randomized multicenter phase III non-inferiority study

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
274

participants targeted

Target at P50-P75 for phase_3

Timeline
Completed

Started Nov 2020

Longer than P75 for phase_3

Geographic Reach
1 country

46 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 29, 2019

Completed
4 days until next milestone

First Posted

Study publicly available on registry

December 3, 2019

Completed
12 months until next milestone

Study Start

First participant enrolled

November 27, 2020

Completed
5.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2026

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2026

Completed
Last Updated

April 29, 2025

Status Verified

April 1, 2025

Enrollment Period

5.3 years

First QC Date

November 29, 2019

Last Update Submit

April 28, 2025

Conditions

Keywords

MDSTransplantAzacitidine

Outcome Measures

Primary Outcomes (1)

  • Feasibility of HSCT in terms of proportion of patients who receive HSCT of the total number of randomized patients

    Split patients in two categories: the feasibility of HSCT (ITT) in patients with HR-MDS with a proportion of bone marrow blasts below 10% and in patients with a proportion of BM blasts equal or greater than 10%.

    4 years

Study Arms (2)

Standard clinical treatment

ACTIVE COMPARATOR

If BM-blasts \>= 10%: Conventional chemotherapy: induction one cycle (3+7 protocol) and one optional consolidation cycle, followed by HSCT if a suitable sibling or unrelated donor is available versus If BM blasts are \<10%: HSCT upfront

Drug: Standard ChemotherapyProcedure: Allogeneic stem cell transplantation

Experimental treatment

EXPERIMENTAL

If BM-blasts \>= 10%: Azacitidine (AZA) 75mg/sqm/day subcutaneously for 7 days every 28 days (1 cycle of 28 days) for at least 4 cycles, followed by HSCT if a suitable sibling or unrelated donor is available If BM blasts are \<10%: Azacitidine (AZA) 75mg/sqm/day subcutaneously for 7 days every 28 days (1 cycle of 28 days) for at least 4 cycles, followed by HSCT if a suitable sibling or unrelated donor is available

Drug: AzacitidineProcedure: Allogeneic stem cell transplantation

Interventions

75mg/mq/day subcutaneously for 7 days every 28 days

Also known as: AZA
Experimental treatment

1. cycle (induction): i.v. 3+7 (Citarabine 200 mg/m2 iv continuous infusion (24 h) for 7 days, Daunorubicine 60 mg/mq iv day 1-3) 2. cycle (consolidation): i.v. 3+7 (Citarabine 200 mg/m2 iv continuous infusion (24 h) for 7 days, Daunorubicine 45 mg/mq iv day 1-3)

Also known as: STD CHT
Standard clinical treatment

Allogeneic stem cell transplantation

Also known as: HSCT
Experimental treatmentStandard clinical treatment

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patients with newly diagnosed higher-risk MDS, including IPSS Intermediate-2 and high, and IPSS-R intermediate to very-high
  • Age 18-70 years
  • Previously untreated for HR-MDS
  • HSCT - eligible
  • Life expectancy ≥3 months;
  • Signed written informed consent according to ICH/EU/GCP and national local laws
  • Eastern Cooperative Oncology Group Performance Status Grade of 0-2

You may not qualify if:

  • Acute myeloid leukaemia with \>20% blasts in BM or peripheral blood (PB);
  • concurrent malignancy diagnosed in the past 12 months (with the exception of skin basalioma);
  • severe renal, cardiac, liver or lung impairment;
  • pregnant or lactating or potentially fertile (both males and females), who have not agreed to avoid pregnancy during the trial period; Women of childbearing potential and men must agree to use effective contraception during and up to 3 months after treatment with azacitidine.
  • HIV infection; active, uncontrolled HCV or HBV infections or liver cirrhosis;
  • clinically relevant neurological or psychiatric diseases;
  • hypersensitivity (known or suspected) to AZA;
  • prior Treatments:
  • prior investigational drugs (within 30 days);
  • radiotherapy, chemotherapy, or cytotoxic therapy for non-MDS conditions within the previous 6 months;
  • growth factors (EPO, G-CSF or GM-CSF) during the previous 21 days;
  • androgenic hormones during the previous 14 days;
  • prior transplantation or cytotoxic therapy, including azacitidine, AZA or chemotherapy, administered to treat MDS (a previous treatment with Lenalidomide is admitted, provided that lenalidomide had been stopped at least 60 days before enrolment).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (46)

058 - Aon Ss. Antonio E Biagio E C. Arrigo - Soc Ematologia

Alessandria, Italy

RECRUITING

Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi - Sod Clinica Ematologica

Ancona, Italy

RECRUITING

Presidio Ospedaliero Av5 Osp. Gen. Prov.Le "C.G.Mazzoni" - Uoc Ematologia

Ascoli Piceno, Italy

RECRUITING

Aou Consorziale Policlinico - Bari - Uo Ematologia Con Trapianto

Bari, Italy

RECRUITING

Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia

Bologna, Italy

RECRUITING

Asst Degli Spedali Civili Di Brescia - Uo Ematologia

Brescia, Italy

RECRUITING

Asl Brindisi, Ospedale 'Perrino'- Uo Ematologia

Brindisi, Italy

RECRUITING

U.O.C. Oncoematologia - Istituto Oncologico Veneto Irccs - Presidio Ospedaliero S. Giacomo Apostolo

Castelfranco Veneto, Italy

RECRUITING

Aou Policlinico Vittorio Emanuele, Po Ospedaliero "G. Rodolico" - Uo Ematologia Con Trapianto Di Midollo Osseo

Catania, Italy

RECRUITING

Aou Arcispedale Sant'Anna - Cona (Fe) - Uoc Ematologia E Fisiopatologia Della Coagulazione

Cona, Italy

RECRUITING

Aso S. Croce E Carle - Sc Ematologia

Cuneo, Italy

RECRUITING

Aou Ospedali Riuniti- Uoc Ematologia

Foggia, Italy

RECRUITING

Asl Latina, Presidio Ospedaliero Nord - Ospedale Santa Maria Goretti - Uoc Ematologia

Latina, Italy

RECRUITING

Asl Lecce, Ospedale 'V. Fazzi' - Uo Ematologia

Lecce, Italy

RECRUITING

Aulss 3 Serenissima, Ospedale Dell'Angelo Uo Ematologia

Mestre, Italy

RECRUITING

Asst Grande Ospedale Metropolitano Niguarda - Milano - Sc Ematologia

Milan, Italy

RECRUITING

Irccs Ospedale S. Raffaele - Milano - Uo Oncoematologia

Milan, Italy

RECRUITING

Aou Di Modena - Sc Ematologia

Modena, Italy

RECRUITING

Ao Di Rilievo Nazionale Antonio Cardarelli - Napoli - Uoc Ematologia Con Trapianto Di Midollo

Napoli, Italy

RECRUITING

Aou San Luigi Gonzaga - Orbassano - Scdu Ematologia Generale E Oncoematologia

Orbassano, Italy

RECRUITING

Asl Salerno, Presidio Ospedaliero Tortora Pagani - Ematologia

Pagani, Italy

RECRUITING

Aou Policlinico P. Giaccone - Uo Ematologia

Palermo, Italy

RECRUITING

Asl Pescara, Presidio Ospedaliero 'Spirito Santo' - Uoc Ematologia Clinica

Pescara, Italy

RECRUITING

Asl Di Piacenza, Ospedale "Guglielmo Da Saliceto" - Ematologia E Centro Trapianti

Piacenza, Italy

RECRUITING

Aou Pisana - Uo Ematologia Universitaria

Pisa, Italy

RECRUITING

Grande Ospedale Metropolitano "Bianchi-Melacrino-Morelli" Po E. Morelli - Reggio Calabria - Uoc Ematologia

Reggio Calabria, Italy

RECRUITING

Arcispedale Santa Maria Nuova, Irccs - Sc Ematologia

Reggio Emilia, Italy

RECRUITING

Ausl Della Romagna, Ospedale "Infermi" - Uo Ematologia

Rimini, Italy

RECRUITING

Ao Complesso Ospedaliero San Giovanni / Addolorata - Roma - Uoc Ematologia

Roma, Italy

RECRUITING

Ao San Camillo Forlanini Uoc Ematologia E Trapianto Cellule Staminali

Roma, Italy

RECRUITING

Aou Policlinico Tor Vergata - Roma - Uoc Trapianto Cellule Staminali

Roma, Italy

RECRUITING

Aou Sant'Andrea - Roma - Uoc Ematologia

Roma, Italy

RECRUITING

Asl Roma 2, Ospedale S. Eugenio- Uoc Ematologia

Roma, Italy

RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli Irccs - Roma - Area Ematologica

Roma, Italy

RECRUITING

Istituti Fisioterapici Ospitalieri - Ifo - Istituto Regina Elena - Roma - Uosd Ematologia

Roma, Italy

RECRUITING

Policlinico Universitario Campus Bio Medico - Roma - Uoc Ematologia E Trapianto Di Cellule Staminali

Roma, Italy

RECRUITING

Università Degli Studi Di Roma "Sapienza" - Dipartimento Di Medicina Traslazionale E Di Precisione - U.O.C. Ematologia

Roma, Italy

RECRUITING

Aou "San Giovanni Di Sio E Ruggi D'Aragona" - Uoc Ematologia E Trapianti Di Cellule Staminali Emopoietiche

Salerno, Italy

RECRUITING

Aou Di Sassari - Cliniche Universitarie - Stabilimento Cliniche Di San Pietro - Uoc Ematologia

Sassari, Italy

RECRUITING

Aou Senese - Uoc Ematologia E Trapianti

Siena, Italy

RECRUITING

Aou Citta' Della Salute E Della Scienza - Osp. S. Giovanni Battista Molinette - Sc Ematologia 2

Torino, Italy

RECRUITING

Aou Città Della Salute E Della Scienza, Ospedale S. Giovanni Battista Molinette - Torino - Sc Ematologia - Università Degli Studi Di Torino

Torino, Italy

RECRUITING

ENTE ECCLESIASTICO CARDINALE G. PANICo - UO EMATOLOGIA

Tricase, Italy

RECRUITING

Asui Di Udine - Presidio Ospedaliero "Santa Maria Della Misericordia" - Clinica Ematologica

Udine, Italy

RECRUITING

Aou Integrata Di Verona, Policlinico G.B. Rossi - Uoc Ematologia

Verona, Italy

RECRUITING

Asl Di Viterbo, Complesso Ospedaliero Di Belcolle - Uoc Ematologia

Viterbo, Italy

RECRUITING

MeSH Terms

Interventions

Azacitidine

Intervention Hierarchy (Ancestors)

Aza CompoundsOrganic ChemicalsCytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsNucleosidesNucleic Acids, Nucleotides, and NucleosidesRibonucleosides

Study Officials

  • Voso

    AOU POLICLINICO TOR VERGATA - ROMA - UOC TRAPIANTO CELLULE STAMINALI

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Enrico Crea

CONTACT

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 29, 2019

First Posted

December 3, 2019

Study Start

November 27, 2020

Primary Completion

March 1, 2026

Study Completion

March 1, 2026

Last Updated

April 29, 2025

Record last verified: 2025-04

Locations