Clinical trial NCT03751917: Long-term Safety Study of Arsenic Trioxide in Newly Diagnosed, Low-to-intermediate Risk Acute Promyelocytic Leukemia
Official title: A Long-Term Retrospective and Prospective Safety Study of Arsenic Trioxide in Patients With Newly Diagnosed, Low- to Intermediate-Risk Acute Promyelocytic Leukemia (APL)
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Acute Promyelocytic Leukemia
Keywords: Acute promyelocytic leukemia
Adults
Arsenic trioxide
Lead sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor class: OTHER
First posted: 2018-11-23
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The Source Population for the study is patients included in existing multinational disease registries for APL in Europe. APL registries collect real-world data on APL demographics and provide information on disease characteristics, treatment patterns and long-term effects. Participating physicians are not subject to any instructions with regard to the diagnosis and therapy of their patients. All patient treatment is carried out within routine clinical practice at the discretion of the physician and according to existing treatment guidelines.

Data will be extracted from the registries for all patients meeting the above inclusion and exclusion criteria to provide the study population.

Brief summary: The therapeutic advantage of the association of ATRA + Arsenic Trioxide is more favorable and manageable as compared to ATRA + chemotherapy. Nevertheless, at present, there is not enough information on the incidence of long-term side effects.

This study, as well as other similar studies conducted around Europe, will focus on following patients treated with this therapy on a long-term basis.

Once all studies in Europe will be concluded, all data will be analyzed together.

Detailed description: Considering the clear therapeutic advantage associated with ATRA+ATO combination therapy and the more favorable and overall manageable safety profile compared to ATRA+chemotherapy, the benefits of the combination in the first-line indication do appear to overweigh the risks.

However, no information regarding the actual adverse event (AE) incidence and the long-term toxicity of ATRA+ATO is available at present and therefore, as a postmarketing commitment, TEVA (the Company holding the Marketing Authorisation of Trisenox® (Arsenic trioxide)) is setting up a long-term safety cohort study of Trisenox in newly diagnosed low- to intermediate-risk APL patients retrospectively analyzing data from APL disease registries all around Europe.

As a result, this observational study is part of the retrospective PASS Study (A Post-Authorisation Long-Term Retrospective Safety Cohort Study of Arsenic Trioxide in First Line Low-to Intermediate-Risk Acute Promyelocytic Leukaemia Patients) that will use data from multinational APL disease registries in Europe. The present study will focus on Italian patients.

Eligibility criteria: Inclusion Criteria:

* APL diagnosis based on cytological criteria and confirmed by the presence of the (15;17) translocation and/or the presence of the PML/RARA rearrangement (with the determination of the breakpoint subtype).
* Newly diagnosed low- to intermediate-risk APL (white blood cells \[WBC\] count ≤10x103/µL)
* First line treatment with ATRA+ATO
* Aged 18 years or above
* Signed informed consent, if applicable

Exclusion Criteria:

* High risk APL (WBC count \> 10x103/µL)
* APL relapse

Primary outcomes:
measure: Number of grade III/IV (Common Terminology Criteria for Adverse Events (CTCAE) v4.03) adverse events of special interest (AESI).
timeFrame: At a maximum of 5 years from study entry
Secondary outcomes:
measure: Number of unexpected serious adverse events (SAEs).
timeFrame: At a maximum of 5 years from study entry
measure: Number of patients developing secondary malignancies.
timeFrame: At a maximum of 5 years from study entry
measure: Number of patients developing therapy-related myelodysplastic syndrome (tMDS).
timeFrame: At a maximum of 5 years from study entry
measure: Number of patients developing acute myeloid leukemia (tAML).
timeFrame: At a maximum of 5 years from study entry
measure: Number of patients who die.
timeFrame: At a maximum of 5 years from study entry
Other outcomes:
Locations:
city: Alessandria
country: Italy
facility: Aon Ss. Antonio E Biagio E C. Arrigo - Alessandria - Soc Ematologia
city: Bari
country: Italy
facility: Aou Consorziale Policlinico - Bari - Uo Ematologia Con Trapianto
city: Bari
country: Italy
facility: UO Ematologia con trapianto-Universita' degli Studi di Bari Aldo Moro
city: Bergamo
country: Italy
facility: Azienda Ospedaliera - Papa Giovanni XXIII
city: Brescia
country: Italy
facility: Asst Degli Spedali Civili Di Brescia - Uo Ematologia
city: Cagliari
country: Italy
facility: Ao Brotzu, Presidio Ospedaliero A. Businco - Cagliari - Sc Ematologia E Ctmo
city: Cagliari
country: Italy
facility: CTMO - Ematologia - Ospedale "Binaghi"
city: Castelfranco Veneto
country: Italy
facility: U.O.C. Oncoematologia - Istituto Oncologico Veneto Irccs, Presidio Ospedaliero S. Giacomo Apostolo
city: Catanzaro
country: Italy
facility: A.O. Pugliese Ciaccio - Presidio Ospedaliero A.Pugliese - U.O. di Ematologia
city: Genova
country: Italy
facility: Irccs Aou San Martino - Genova - Uo Ematologia E Trapianti
city: Genova
country: Italy
facility: U.O.C. di Ematologia 1 e 2 IRCCS AOU San Martino-IST
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Granda Osp. Maggiore Policlinico UOC Oncoematologia
city: Milano
country: Italy
facility: Irccs Ospedale S. Raffaele - Milano - Uo Oncoematologia
city: Milano
country: Italy
facility: Ospedale Niguarda " Ca Granda" - SC Ematologia
city: Monza
country: Italy
facility: Azienda Ospedaliera "S.Gerardo"
city: Naples
country: Italy
facility: Napoli Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"
city: Palermo
country: Italy
facility: Ospedali Riuniti "Villa Sofia-Cervello"
city: Pescara
country: Italy
facility: U.O. Ematologia Clinica - Azienda USL di Pescara
city: Roma
country: Italy
facility: Aou Policlinico Tor Vergata - Roma - Uoc Trapianto Cellule Staminali
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli Irccs - Roma - Area Ematologica
city: Roma
country: Italy
facility: Universita' "Sapienza" - Dip Biotecnologie Cellulari - Divisione di Ematologia
city: Roma
country: Italy
facility: Universita' Cattolica del Sacro Cuore - Policlinico A. Gemelli
city: Roma
country: Italy
facility: Università Degli Studi Di Roma "Sapienza" - Dipartimento Di Medicina Traslazionale E Di Precisione - U.O.C. Ematologia
city: San Giovanni Rotondo
country: Italy
facility: Ente Ecclesiastico Casa Sollievo Della Sofferenza - San Giovanni Rotondo - Ematologia
city: Torino
country: Italy
facility: Aou Città Della Salute E Della Scienza, Ospedale S. Giovanni Battista Molinette - Torino - Sc Ematologia 2
city: Torino
country: Italy
facility: Dipartimento di Oncologia Ematologia 2 A.O. Citta' della Salute S. G. Battista
city: Vicenza
country: Italy
facility: Aulss 8 Berica - Ospedale Di Vicenza - Uoc Ematologia
city: Vicenza
country: Italy
facility: ULSS N.6 Osp. S. Bortolo
Study officials:
References: