Acute Myeloid LeukemiaNCT04060485
Screening Gene Mutations in Myeloid Cancers by Next Generation Sequencing to Improve Treatment Results
An observational study of Acute Myeloid Leukemia and Myelodysplastic Syndromes, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 120 yearsHealthy volunteers can take part
- Conditions the study is for
- Acute Myeloid Leukemia, Myelodysplastic Syndromes, Myeloproliferative Neoplasm
- How many people
- 1,000 participants
- Where
- 1 siteTaiwan
- Study dates
- 19 Aug 2019to 31 Dec 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 120 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Adult myeloid cancer (including acute myeloid leukemia, myelodysplastic syndromes, and myeloproliferative neoplasms) patients (newly diagnosed or relapsed) diagnosed at the National Taiwan University Hospital (NTUH) according to the updated 2016 WHO myeloid disease classification.
Who may be able to join
- Patients ≥ 18 years of age diagnosed with myeloid cancers (including acute myeloid leukemia, myelodysplastic syndromes, and myeloproliferative neoplasms).
- Healthy Volunteers ≥ 18 years of age.
- Participants must be willing to provide voluntary written informed consent before study related procedures.
Who cannot join
- Patients ≥ 18 years of age diagnosed with non-myeloid cancers.
- Patients <18 years of age diagnosed with myeloid cancers (including acute myeloid leukemia, myelodysplastic syndromes, and myeloproliferative neoplasms).
Trial locations and contacts
1 site in 1 country.
Taiwan1 site, 1 recruiting
- Recruiting
National Taiwan University Hospital
Taipei, 100
Wen-Chien Chou, MD, PhD, +886-972651701, wchou@ntu.edu.tw
Chi-Yuan Yao, MD, MSc, +886-972655691, Y06960@ms1.ylh.gov.tw
About this study
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.
Read the 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.
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndromes, Myeloproliferative Neoplasm, Aplastic Anemia
- Keywords
- Next generation sequencing, Acute Myeloid Leukemia, Myelodysplastic Syndromes, Myeloproliferative Neoplasm, Aplastic Anemia, Genetic mutation, Prognosis
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Myeloproliferative Disorders, Anemia, Aplastic
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of various genetic mutations in myeloid cancers
Peripheral blood or marrow blood will be obtained from routine practice blood/marrow sampling specimens (no extra venipuncture or bone marrow aspiration would be required) and sent for NGS analyses.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2019
- Study start19 Aug 2019 (estimated)
- Primary completion31 Aug 2024 (estimated)
- Study completion31 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by National Taiwan University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 1,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Wen-Chien Chou, MD, PhD, Principal investigator, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04060485
- Sponsor's protocol ID
- 201803002RIPD
- Status last verified
- Aug 2019
- First submitted
- 15 Aug 2019
- Last updated
- 19 Aug 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Data sharing and collaboration possibilities will be discussed upon request.
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Screening Gene Mutations in Myeloid Cancers by Next Generation Sequencing to Improve Treatment Results
- ClinicalTrials.gov ID
- NCT04060485
- Acronym / sponsor ID
- 201803002RIPD
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndromes, Myeloproliferative Neoplasm, Aplastic Anemia
- Keywords
- Next generation sequencing, Acute Myeloid Leukemia, Myelodysplastic Syndromes, Myeloproliferative Neoplasm, Aplastic Anemia, Genetic mutation, Prognosis
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Study start
- 19 Aug 2019 (estimated)
- Primary completion
- 31 Aug 2024 (estimated)
- Study completion
- 31 Dec 2024 (estimated)
- First posted
- 19 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 120 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Adult myeloid cancer (including acute myeloid leukemia, myelodysplastic syndromes, and myeloproliferative neoplasms) patients (newly diagnosed or relapsed) diagnosed at the National Taiwan University Hospital (NTUH) according to the updated 2016 WHO myeloid disease classification.
Key inclusion criteria (3 listed)
- Patients ≥ 18 years of age diagnosed with myeloid cancers (including acute myeloid leukemia, myelodysplastic syndromes, and myeloproliferative neoplasms).
- Healthy Volunteers ≥ 18 years of age.
- Participants must be willing to provide voluntary written informed consent before study related procedures.
Key exclusion criteria (2 listed)
- Patients ≥ 18 years of age diagnosed with non-myeloid cancers.
- Patients <18 years of age diagnosed with myeloid cancers (including acute myeloid leukemia, myelodysplastic syndromes, and myeloproliferative neoplasms).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Incidence of various genetic mutations in myeloid cancers
Time frame: 5 years
Peripheral blood or marrow blood will be obtained from routine practice blood/marrow sampling specimens (no extra venipuncture or bone marrow aspiration would be required) and sent for NGS analyses.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2019.
- Individual participant dataSharing planned: Undecided. Data sharing and collaboration possibilities will be discussed upon request.
Related trials for Acute Myeloid Leukemia
RecruitingObservational study
A Long-term Follow-up Study of Patients With ARD103 CAR-T Cell Therapies
Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia, in Relapsed or Refractory
RecruitingPhase 2
MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
RecruitingPhase 1
225Ac-DOTA-Anti-CD38 Daratumumab Monoclonal Antibody With Fludarabine, Melphalan and Total Marrow and Lymphoid Irradiation as Conditioning Treatment for Donor Stem Cell Transplant in Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
RecruitingPhase 2
Testing the Addition of Venetoclax or Gemtuzumab Ozogamicin (GO) to Usual Treatment Regimen (Cytarabine and Daunorubicin, "7+3") for Core Binding Factor Acute Myeloid Leukemia (CBF-AML) to Improve Response (A MYELOMATCH Treatment Trial)
Core Binding Factor Acute Myeloid Leukemia
RecruitingPhase 1/2
Phase I/II Trial: Engineered Donor Graft (Orca Q) for Pediatric Hematopoietic Cell Transplant (HCT)
Acute Myeloid Leukemia, Acute Lymphoid Leukemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wen-Chien Chou, MD, PhD principal investigator
National Taiwan University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acute Myeloid Leukemia and Myelodysplastic Syndromes, run by National Taiwan University Hospital at 1 site in Taiwan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Aug 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Wen-Chien Chou, MD, PhD (+886-972651701, wchou@ntu.edu.tw), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of various genetic mutations in myeloid cancers (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Aug 2019.