Clinical trial NCT04184505: Feasibility of Allogeneic Stem Cell Transplantation in Higher-risk-MDS (ACROBAT)
Official title: Prospective Randomized Study on the Feasibility of Allogeneic Stem Cell Transplantation in Higher-risk-myelodysplastic Syndromes, Performed Upfront or Preceded by Azacitidine or Conventional Chemotherapy According to the BM-blast Proportion
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: High-risk MDS
Keywords: MDS
Transplant
Azacitidine
Lead sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor class: OTHER
First posted: 2019-12-03
Enrollment: 274
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Open-label, randomized multicenter phase III non-inferiority study

Detailed description: Open-label, randomized, prospective multicenter phase III study to compare the role of HMT followed by HSCT vs HSCT upfront in HR-MDS with \<10% of BM blasts and of CHT vs HMT followed by HSCT in HR-MDS with \>10% BM blasts in terms of feasibility of HSCT (non-inferiority trial).

Eligibility criteria: Inclusion Criteria:

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

Exclusion Criteria:

1. Acute myeloid leukaemia with \>20% blasts in BM or peripheral blood (PB);
2. concurrent malignancy diagnosed in the past 12 months (with the exception of skin basalioma);
3. severe renal, cardiac, liver or lung impairment;
4. 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.
5. HIV infection; active, uncontrolled HCV or HBV infections or liver cirrhosis;
6. clinically relevant neurological or psychiatric diseases;
7. hypersensitivity (known or suspected) to AZA;
8. prior Treatments:

   1. prior investigational drugs (within 30 days);
   2. radiotherapy, chemotherapy, or cytotoxic therapy for non-MDS conditions within the previous 6 months;
   3. growth factors (EPO, G-CSF or GM-CSF) during the previous 21 days;
   4. androgenic hormones during the previous 14 days;
   5. 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).

Primary outcomes:
measure: Feasibility of HSCT in terms of proportion of patients who receive HSCT of the total number of randomized patients
timeFrame: 4 years
Secondary outcomes:
Other outcomes:
Locations:
city: Alessandria
country: Italy
facility: 058 - Aon Ss. Antonio E Biagio E C. Arrigo - Soc Ematologia
city: Ancona
country: Italy
facility: Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi - Sod Clinica Ematologica
city: Ascoli Piceno
country: Italy
facility: Presidio Ospedaliero Av5 Osp. Gen. Prov.Le "C.G.Mazzoni" - Uoc Ematologia
city: Bari
country: Italy
facility: Aou Consorziale Policlinico - Bari - Uo Ematologia Con Trapianto
city: Bologna
country: Italy
facility: Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia
city: Brescia
country: Italy
facility: Asst Degli Spedali Civili Di Brescia - Uo Ematologia
city: Brindisi
country: Italy
facility: Asl Brindisi, Ospedale 'Perrino'- Uo Ematologia
city: Castelfranco Veneto
country: Italy
facility: U.O.C. Oncoematologia - Istituto Oncologico Veneto Irccs - Presidio Ospedaliero S. Giacomo Apostolo
city: Catania
country: Italy
facility: Aou Policlinico Vittorio Emanuele, Po Ospedaliero "G. Rodolico" - Uo Ematologia Con Trapianto Di Midollo Osseo
city: Cona
country: Italy
facility: Aou Arcispedale Sant'Anna - Cona (Fe) - Uoc Ematologia E Fisiopatologia Della Coagulazione
city: Cuneo
country: Italy
facility: Aso S. Croce E Carle - Sc Ematologia
city: Foggia
country: Italy
facility: Aou Ospedali Riuniti- Uoc Ematologia
city: Latina
country: Italy
facility: Asl Latina, Presidio Ospedaliero Nord - Ospedale Santa Maria Goretti - Uoc Ematologia
city: Lecce
country: Italy
facility: Asl Lecce, Ospedale 'V. Fazzi' - Uo Ematologia
city: Mestre
country: Italy
facility: Aulss 3 Serenissima, Ospedale Dell'Angelo Uo Ematologia
city: Milano
country: Italy
facility: Asst Grande Ospedale Metropolitano Niguarda - Milano - Sc Ematologia
city: Milano
country: Italy
facility: Irccs Ospedale S. Raffaele - Milano - Uo Oncoematologia
city: Modena
country: Italy
facility: Aou Di Modena - Sc Ematologia
city: Napoli
country: Italy
facility: Ao Di Rilievo Nazionale Antonio Cardarelli - Napoli - Uoc Ematologia Con Trapianto Di Midollo
city: Orbassano
country: Italy
facility: Aou San Luigi Gonzaga - Orbassano - Scdu Ematologia Generale E Oncoematologia
city: Pagani
country: Italy
facility: Asl Salerno, Presidio Ospedaliero Tortora Pagani - Ematologia
city: Palermo
country: Italy
facility: Aou Policlinico P. Giaccone - Uo Ematologia
city: Pescara
country: Italy
facility: Asl Pescara, Presidio Ospedaliero 'Spirito Santo' - Uoc Ematologia Clinica
city: Piacenza
country: Italy
facility: Asl Di Piacenza, Ospedale "Guglielmo Da Saliceto" - Ematologia E Centro Trapianti
city: Pisa
country: Italy
facility: Aou Pisana - Uo Ematologia Universitaria
city: Reggio Calabria
country: Italy
facility: Grande Ospedale Metropolitano "Bianchi-Melacrino-Morelli" Po E. Morelli - Reggio Calabria - Uoc Ematologia
city: Reggio Emilia
country: Italy
facility: Arcispedale Santa Maria Nuova, Irccs - Sc Ematologia
city: Rimini
country: Italy
facility: Ausl Della Romagna, Ospedale "Infermi" - Uo Ematologia
city: Roma
country: Italy
facility: Ao Complesso Ospedaliero San Giovanni / Addolorata - Roma - Uoc Ematologia
city: Roma
country: Italy
facility: Ao San Camillo Forlanini Uoc Ematologia E Trapianto Cellule Staminali
city: Roma
country: Italy
facility: Aou Policlinico Tor Vergata - Roma - Uoc Trapianto Cellule Staminali
city: Roma
country: Italy
facility: Aou Sant'Andrea - Roma - Uoc Ematologia
city: Roma
country: Italy
facility: Asl Roma 2, Ospedale S. Eugenio- Uoc Ematologia
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli Irccs - Roma - Area Ematologica
city: Roma
country: Italy
facility: Istituti Fisioterapici Ospitalieri - Ifo - Istituto Regina Elena - Roma - Uosd Ematologia
city: Roma
country: Italy
facility: Policlinico Universitario Campus Bio Medico - Roma - Uoc Ematologia E Trapianto Di Cellule Staminali
city: Roma
country: Italy
facility: Università Degli Studi Di Roma "Sapienza" - Dipartimento Di Medicina Traslazionale E Di Precisione - U.O.C. Ematologia
city: Salerno
country: Italy
facility: Aou "San Giovanni Di Sio E Ruggi D'Aragona" - Uoc Ematologia E Trapianti Di Cellule Staminali Emopoietiche
city: Sassari
country: Italy
facility: Aou Di Sassari - Cliniche Universitarie - Stabilimento Cliniche Di San Pietro - Uoc Ematologia
city: Siena
country: Italy
facility: Aou Senese - Uoc Ematologia E Trapianti
city: Torino
country: Italy
facility: Aou Citta' Della Salute E Della Scienza - Osp. S. Giovanni Battista Molinette - Sc Ematologia 2
city: Torino
country: Italy
facility: Aou Città Della Salute E Della Scienza, Ospedale S. Giovanni Battista Molinette - Torino - Sc Ematologia - Università Degli Studi Di Torino
city: Tricase
country: Italy
facility: ENTE ECCLESIASTICO CARDINALE G. PANICo - UO EMATOLOGIA
city: Udine
country: Italy
facility: Asui Di Udine - Presidio Ospedaliero "Santa Maria Della Misericordia" - Clinica Ematologica
city: Verona
country: Italy
facility: Aou Integrata Di Verona, Policlinico G.B. Rossi - Uoc Ematologia
city: Viterbo
country: Italy
facility: Asl Di Viterbo, Complesso Ospedaliero Di Belcolle - Uoc Ematologia
Study officials:
affiliation: AOU POLICLINICO TOR VERGATA - ROMA - UOC TRAPIANTO CELLULE STAMINALI
name: Voso
role: PRINCIPAL_INVESTIGATOR
References: