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Observational Study for the Evaluation of Incidence of Familial AML/MDSs in Patients With Myeloid Neoplasms

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ClinicalTrials.gov Identifier: NCT04460950
Recruitment Status : Recruiting
First Posted : July 8, 2020
Last Update Posted : April 20, 2021
Sponsor:
Information provided by (Responsible Party):
Gruppo Italiano Malattie EMatologiche dell'Adulto

Tracking Information
First Submitted Date June 25, 2020
First Posted Date July 8, 2020
Last Update Posted Date April 20, 2021
Actual Study Start Date December 16, 2020
Estimated Primary Completion Date March 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: July 1, 2020)
Incidence of familial AML/MDSs [ Time Frame: at 1 year ]
Evaluation of incidence of familial AML/MDSs in patients with de novo MDSs or AML with almost one relative affected by hematologic neoplasms and/or other cancers at young age (< 40 years). The incidence of familial AML/MDSs will be evaluated by means of number of diagnosis of familial AML/MDSs on the number of all diagnosis of AML/MDS between January 2014 and December 2022.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: July 1, 2020)
  • Hematological characteristics of familial AML/MDSs [ Time Frame: at 1 year ]
    Evaluation of hematological characteristics of patients with familial AML/MDS.
  • Distribution of familial hematologic neoplasms and/or other cancers [ Time Frame: at 1 year ]
    Type and rate of familial hematologic neoplasms and/or other cancers
  • Incidence of transplantation [ Time Frame: at 1 year ]
    Outcome of transplant in patients with familial AML/MDS
  • Survival Distribution [ Time Frame: at 1 year ]
    Overall Survival (OS) and Progression Free Survival (PFS)
  • Standardized Mortality Ratio (SMR) [ Time Frame: at 1 year ]
    SMR to compare the survival risk in patients with familial AML/MDS with the risk in the Italian general population
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Observational Study for the Evaluation of Incidence of Familial AML/MDSs in Patients With Myeloid Neoplasms
Official Title A Retrospective and Prospective Multicentre Observational Study for the Evaluation of Incidence of Familial AML/MDSs in Patients With Myeloid Neoplasms (AML/MDS)
Brief Summary Observational study aimed at evaluating the incidence of familial AML/MDSs in patients with de novo MDSs or AML with almost one relative affected by hematologic neoplasms and/or other cancers at young age (< 40 years)
Detailed Description

This is a multicenter, retrospective and prospective, observational study aims to collect clinical information on patients with familial AML/MDSs from January 2014 to December 2022. No intervention is expected.

The purpose of this study is to identify and characterize the patients with familial MDSs or AML (i.e. with relatives affected by hematologic neoplasms and/or other cancers at young age (< 40 years)) or presence of signs, symptoms or laboratory tests compatible with one of the known syndromes with germinal susceptibility to AML/MDSs.

In order to estimate the incidence of familial AML/MDSs, a survey will be sent every year to all participating sites to collect only the number of the all diagnosis of AML/MDS. All patients will be followed until December 2023 in order to have at least 1 year of observation.

Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population All patients diagnoses with AML and MDSs from January 2014 to December 2022 will be registered. Study population will include patients with MDSs or AML with at least one family member of first or second degree affected by myeloid neoplasm or other hematological malignancy, or by solid tumors with onset at age < 40 years.
Condition
  • Acute Myeloid Leukemia
  • Familial Acute Myeloid Leukemia
  • Myelodysplastic Syndromes
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: July 1, 2020)
237
Original Estimated Enrollment Same as current
Estimated Study Completion Date March 2024
Estimated Primary Completion Date March 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Patient age > 18 years old
  • Diagnosis of AML or MDSs from January 2014 to December 2022
  • Suspected familiarity for myeloid neoplasm: at least one family member of first or second degree affected by myeloid neoplasm or other hematological malignancy, or by solid tumors with onset at age < 40 years or presence of signs, symptoms or laboratory tests compatible with one of the known syndromes with germinal susceptibility to AML/MDSs
  • Signed written informed consent according to ICH/EU/GCP and national local laws.

Exclusion Criteria:

  • none
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Paola Fazi 0670390528 p.fazi@gimema.it
Contact: Enrico Crea 0670390514 e.crea@gimema.it
Listed Location Countries Italy
Removed Location Countries  
 
Administrative Information
NCT Number NCT04460950
Other Study ID Numbers AML2120
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Gruppo Italiano Malattie EMatologiche dell'Adulto
Study Sponsor Gruppo Italiano Malattie EMatologiche dell'Adulto
Collaborators Not Provided
Investigators Not Provided
PRS Account Gruppo Italiano Malattie EMatologiche dell'Adulto
Verification Date April 2021