Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

The National Myelodysplastic Syndromes (MDS) Study (MDS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02775383
Recruitment Status : Recruiting
First Posted : May 17, 2016
Last Update Posted : May 2, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
National Heart, Lung, and Blood Institute (NHLBI)

Tracking Information
First Submitted Date May 2, 2016
First Posted Date May 17, 2016
Last Update Posted Date May 2, 2019
Study Start Date April 2016
Estimated Primary Completion Date September 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 16, 2016)
Number of patients dying [ Time Frame: Through study completion, an average of 3 years ]
Deaths will be reported on study case report forms
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT02775383 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: May 16, 2016)
Number of participants progressing to Acute Myeloid Leukemia (AML) [ Time Frame: Through study completion, an average of 3 years ]
Progression to AML will be reported on study case report forms
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 The National Myelodysplastic Syndromes (MDS) Study
Official Title The National Myelodysplastic Syndromes Natural History Study
Brief Summary Multi-center study enrolling patients suspected or newly diagnosed with myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS). Participants will be followed long term. Clinical data, blood, and tissue samples will be collected to establish a biorepository to facilitate the study of the natural history of MDS.
Detailed Description

Multi-center, prospective cohort study enrolling patients from centers in the National Cancer Institute (NCI) National Clinical Trials Network (NCTN) and NCI Community Oncology Research Program (NCORP). The accrual period is 5+ years. After central pathology review of registered participants, approximately 2,000 cases of MDS or MDS/MPN overlap disorders, and 500 cases of ICUS will be identified for the longitudinal study cohort and up to 1000 cases will be identified for the cross-sectional cohort. No more than 3500 total participants will be registered. Participants in the longitudinal cohort may be followed for life.

The goal of the National MDS Study is to establish a publicly available resource to facilitate the study of MDS natural history. This will be accomplished through: 1) Creation of a multi-institutional, longitudinal collection of consistently processed and clinically well-annotated blood and tissue specimens collected prospectively from participants with MDS and participants with idiopathic cytopenia of undetermined significance (ICUS); and 2) Support for investigator-initiated studies of MDS that will have high-impact for MDS patients, including basic science, clinical, health outcomes and epidemiological research.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
bone marrow, peripheral blood, serum, plasma, eyebrow hairs with attached follicles, buccal cells, skin biopsy
Sampling Method Non-Probability Sample
Study Population Patients with suspected MDS or MDS/MPN overlap disorder who are undergoing diagnostic workup and bone marrow assessments, and patients diagnosed with MDS within 6-months of enrollment and undergoing clinical evaluation and bone marrow assessments to confirm MDS or evaluate disease status
Condition Myelodysplastic Syndromes (MDS)
Intervention Other: Therapeutic
Observational Study
Study Groups/Cohorts
  • Longitudinal
    Participants with MDS, MDS/MPN overlap disorder, or ICUS who are followed long term
    Intervention: Other: Therapeutic
  • Cross-sectional
    Participants who do not have MDS, MDS/MPN overlap disorder, or ICUS and have the baseline visit only
    Intervention: Other: Therapeutic
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: May 16, 2016)
3500
Original Estimated Enrollment Same as current
Estimated Study Completion Date September 2021
Estimated Primary Completion Date September 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Suspected (e.g., persistent unexplained cytopenia, circulating peripheral blasts etc.) MDS or MDS/MPN overlap disorders and undergoing diagnostic work-up with planned bone marrow assessments, or diagnosed with de novo or therapy-related MDS within 6-months of enrollment per the World Health Organization (WHO) criteria1 and undergoing clinical evaluation and planned bone marrow assessments to confirm MDS or to evaluate disease status
  • Bone marrow aspirate expected to be performed within 1 week of registration, and in all cases must be performed no later than 4 weeks after enrollment
  • Age 18 or older
  • B12 level, serum folate, ferritin, and Thyroid-Stimulating Hormone (TSH) tests performed in prior 6 months

Exclusion Criteria:

  • Prior treatment for MDS at entry and through the time of the entry bone marrow aspirate
  • Treatment with hematopoietic growth factors in prior 6 months
  • Diagnosis of a solid tumor or hematologic malignancy within two years prior to enrollment except for in situ cancer of the skin (basal or squamous cell), cervix, bladder, breast, or prostate
  • Treatment with radiation therapy in the two years prior to registration
  • Non-hormonal treatment for malignancy within the two years prior to registration
  • Established hereditary bone marrow failure syndrome
  • Known primary diagnosis of aplastic anemia, classical paroxysmal nocturnal hemoglobinuria, amegakaryocytic thrombocytopenic purpura, or large granular lymphocyte leukemia
  • Enrolled in the Connect® MDS/AML Disease Registry (NCT01688011)
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Tammy Frank mdscontact@emmes.com
Contact: Jason Thompson jthompson@emmes.com
Listed Location Countries Israel,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02775383
Other Study ID Numbers NHLBI-MDS
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party National Heart, Lung, and Blood Institute (NHLBI)
Study Sponsor National Heart, Lung, and Blood Institute (NHLBI)
Collaborators National Cancer Institute (NCI)
Investigators
Study Chair: Mikkael Sekeres, MD, MS The Cleveland Clinic
Study Chair: Steven Gore, MD Yale University
PRS Account National Heart, Lung, and Blood Institute (NHLBI)
Verification Date April 2019