Clinical trial NCT02192619: National Acute Promyelocytic Leukemia (APL) Observational Study NAPOLEON-Registry of the German AML Intergroup
Official title: National Acute Promyelocytic Leukemia (APL) Observational Study NAPOLEON-Registry of the German AML Intergroup
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Newly-diagnosed APL (de Novo or Therapy-related)
Relapsed APL
Keywords: APL
acute promyelocytic leukemia
AML M3
Lead sponsor: Gesellschaft fur Medizinische Innovation - Hamatologie und Onkologie mbH
Sponsor class: OTHER
First posted: 2014-07-17
Enrollment: 500
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with newly-diagnosed or relapsed APL (de novo or therapy-related) who require a therapeutic intervention according to their disease state are the study population within this registry.

Brief summary: The registry aims to document epidemiologic data, treatment and long-term outcome as well as quality of life of patients with APL. Additionally, a biobanking project for further translational studies is integrated.

Prospective population-based non-interventional and non-randomized multicenter registry.

Detailed description: * collection of epidemiological data for APL: age distribution, prognostic factors, distribution of subgroups, incidence
* documentation of efficacy and safety of the first line and salvage therapy in APL including
* documentation of minimal residual disease (MRD)
* correlation of clinical outcomes with chosen therapy
* collection and evaluation of quality of life
* validation of published prognostic factors / new potential prognostic factors
* acquisition of bone marrow, peripheral blood and buccal swab samples for biobanking and translational studies under the umbrella of the specific study-group biobanking concepts

Eligibility criteria: Inclusion Criteria:

* newly-diagnosed APL (either de novo or therapy-related), within 12 months of diagnosis
* or relapsed APL, within 12 months of diagnosis of relapse

  1. confirmed by the presence of the translocation t(15; 17)
  2. and / or confirmed by the detection of the fusion transcript of PML/RARa

Exclusion Criteria:

* none

Primary outcomes:
measure: epidemiological parameters
timeFrame: yearly follow up for 5 years
measure: diagnostic quality indicators
timeFrame: yearly follow up for 5 years
measure: type of therapy
timeFrame: yearly follow up for 5 years
measure: response, recurrence and time of death and resulting outcomes RFS and OS
timeFrame: yearly follow up for 5 years
Secondary outcomes:
measure: complete remission (CR) and CRm
timeFrame: yearly follow up for 5 years
measure: treatment related mortality (TRM)
timeFrame: yearly follow up for 5 years
measure: cumulative incidence of relapse (CIR)
timeFrame: yearly follow up for 5 years
measure: grade IV toxicities
timeFrame: yearly follow up for 5 years
measure: quality of life (QoL): EORTC QLQ-C30
timeFrame: yearly follow up for 5 years
Other outcomes:
Locations:
city: Dresden
country: Germany
facility: Prof. Dr. U. Platzbecker
Study officials:
affiliation: Universitätsklinikum Ulm, for the AML-SG group
name: Richard F. Schlenk, Prof. Dr.
role: PRINCIPAL_INVESTIGATOR
affiliation: Universitätsklinikum Mannheim
name: Eva Lengfelder, PD Dr.
role: PRINCIPAL_INVESTIGATOR
affiliation: Universitätsklinikum Leipzig
name: Dietger Niederwieser, Prof. Dr.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Kayser S, Schlenk RF, Lebon D, Carre M, Gotze KS, Stolzel F, Berceanu A, Schafer-Eckart K, Peterlin P, Hicheri Y, Rahme R, Raffoux E, Chermat F, Krause SW, Aulitzky WE, Rigaudeau S, Noppeney R, Berthon C, Gorner M, Jost E, Carassou P, Keller U, Orvain C, Braun T, Saillard C, Arar A, Kunzmann V, Wemeau M, De Wit M, Niemann D, Bonmati C, Schwanen C, Abraham J, Aljijakli A, Haiat S, Kramer A, Reichle A, Gnadler M, Willekens C, Spiekermann K, Hiddemann W, Muller-Tidow C, Thiede C, Rollig C, Serve H, Bornhauser M, Baldus CD, Lengfelder E, Fenaux P, Platzbecker U, Ades L. Characteristics and outcome of patients with low-/intermediate-risk acute promyelocytic leukemia treated with arsenic trioxide: an international collaborative study. Haematologica. 2021 Dec 1;106(12):3100-3106. doi: 10.3324/haematol.2021.278722.