Screening Gene Mutations in Myeloid Cancers by Next Generation Sequencing to Improve Treatment Results

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Brief Title

Screening Gene Mutations in Myeloid Cancers by Next Generation Sequencing to Improve Treatment Results

Official Title

Screening Gene Mutations in Myeloid Cancers by Next Generation Sequencing to Improve Treatment Results

Brief Summary

      Genetic mutations have closely linked to the pathogenesis and prognostication of myeloid
      cancers. In addition, a number of molecularly targeted agents have been developed in recent
      years. With the advent of next generation sequencing (NGS), we now are able to detect a wide
      range of mutations more rapidly, accurately, and economically. In this study, the
      investigators will use NGS to screen and analyze myeloid-associated gene mutations in the
      participants, and aim to build up the mutational landscapes of the various myeloid cancers,
      and investigate how these mutations are linked to clinical outcome.
    

Detailed Description

      Genetic mutations have closely linked to the pathogenesis and prognostication of myeloid
      cancers (including acute myeloid leukemia, myelodysplastic syndromes, and myeloproliferative
      neoplasms). In recent years, a number of novel therapeutic agents targeting various genetic
      mutations have been developed. For instance, patients with FLT3-ITD and IDH2 mutations have
      been shown to derive benefits from midostaurin and enasidenib, respectively. Furthermore, the
      TP53-mutated patients once categorized in the very high risk group, have been found to
      respond favorably to the hypomethylating agent, decitabine. Detecting a wide array of cancer
      mutations nowadays by the traditional Sanger method has become not only time-consuming but
      also not as cost-effective. With the advent of next generation sequencing (NGS), we now are
      able to detect a panel gene mutations more rapidly, accurately, and economically.

      In this study, the investigators will screen and analyze myeloid-associated gene mutations in
      participants with myeloid cancers, with an in-house designed targeted NGS panel. The total
      nucleic acid from patients' blood or bone marrow specimens will be extracted, and then
      subjected to the library preparation procedure based on multiplex PCR amplification.
      Sequencing will be performed on Illumina MiSeq sequencer, and the results will be analyzed
      using our in-house developed bioinformatic workflow. Briefly, the sequenced reads will be
      aligned to human reference genome hg19 with BWA-mem, and somatic mutations called with
      Mutect2. The variants will be annotated via SnpEff with RefSeq, dbSNP, 1000 Genome Project,
      COSMIC and ClinVar databases. The investigators aim to build up the mutational landscapes of
      the above mentioned myeloid cancers, and investigate how these mutations are linked to
      clinical outcome.
    


Study Type

Observational


Primary Outcome

Incidence of various genetic mutations in myeloid cancers


Condition

Acute Myeloid Leukemia



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

1000

Start Date

August 19, 2019

Completion Date

December 31, 2024

Primary Completion Date

August 31, 2024

Eligibility Criteria

        Inclusion criteria:

          1. Patients ≥ 18 years of age diagnosed with myeloid cancers (including acute myeloid
             leukemia, myelodysplastic syndromes, and myeloproliferative neoplasms).

          2. Healthy Volunteers ≥ 18 years of age.

          3. Participants must be willing to provide voluntary written informed consent before
             study related procedures.

        Exclusion criteria:

          1. Patients ≥ 18 years of age diagnosed with non-myeloid cancers.

          2. Patients <18 years of age diagnosed with myeloid cancers (including acute myeloid
             leukemia, myelodysplastic syndromes, and myeloproliferative neoplasms).
      

Gender

All

Ages

18 Years - 120 Years

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

Wen-Chien Chou, MD, PhD, +886-972651701, [email protected]

Location Countries

Taiwan

Location Countries

Taiwan

Administrative Informations


NCT ID

NCT04060485

Organization ID

201803002RIPD


Responsible Party

Sponsor

Study Sponsor

National Taiwan University Hospital


Study Sponsor

Wen-Chien Chou, MD, PhD, Principal Investigator, National Taiwan University Hospital


Verification Date

August 2019