jueves, 4 de agosto de 2016

NCT02775383 Clinical Trial - National Cancer Institute

NCT02775383 Clinical Trial - National Cancer Institute

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Tissue Collection and Natural History Study of Patients with Myelodysplastic Syndrome or Myeloproliferative Neoplasms

Basic Trial Information

Phase	Type	Status	Age	Trial IDs
No phase specified	Natural history/Epidemiology, Tissue collection/Repository	Active	18 and over	NHLBI-MDS NCI-2016-00281, NCT02775383

Trial Description

Summary

This research trial collects tissue samples from and studies the history of patients with myelodysplastic syndrome (MDS) or myeloproliferative neoplasms (MPN). Collecting and storing patients' bone marrow, blood, eyebrow hairs, buccal swab, skin, or other tissues to be studied in the laboratory in the future may help doctors learn more about MDS and blood disorders that may lead to MDS. Collecting information about patients and the treatments they receive may allow doctors to better understand how MDS changes over time and this knowledge may lead to better ways to prevent, detect, and treat MDS in the future.

Further Study Information

PRIMARY OBJECTIVES:

I. To develop a high-quality clinical database containing clinical history, including environmental exposure history, presenting signs and symptoms, diagnostic testing results, co-existing diseases, therapies and response to therapies, disease progression, quality of life and survival.

II. To develop a high-quality biorepository linked to the clinical data that will facilitate diverse studies, including genetic, epigenetic, immunologic, proteomic, and cell-functional and cell-phenotypic studies through the development of: central communication with the biorepository to ensure timely and accurate collections and biospecimen data appended to the clinical database; defined standard operating procedures for the collection, processing, storage and distribution, with special emphasis on processing protocols fit-for-purpose to sample requirements for downstream testing; and quality management procedures to ensure minimal numbers of errors in the management of the biospecimens.

III. To facilitate broad use of these linked data and specimens to support studies focused on: improving diagnostic accuracy, risk-stratification and prognostication, and medical decision-making in MDS; understanding quality of life and its relationship to changing disease and treatment status; understanding the pathogenesis of MDS and diverse MDS subtypes, including genetic, epigenetic, immunologic mechanisms; optimizing treatment strategies for specific subtypes of MDS; identifying novel biomarkers for MDS outcomes; and identifying novel targets for therapeutic interventions in MDS.

SECONDARY OBJECTIVES:

I. Detecting improved prognostic factors in low risk MDS. (Potential Studies)

II. Detecting markers of improved response to hypomethylating therapy. (Potential Studies)

III. Quality of life (QOL) and anemia therapy. (Potential Studies)

IV. Genetic factors in patients with complex karyotype. (Potential Studies)

V. Prognostic significance of splicing factor 3b subunit 1 (SF3B1) in MDS patients with refractory anemia with ring sideroblasts (RARS). (Potential Studies)

OUTLINE:

Patients undergo blood, bone marrow, eyebrow hairs, buccal swab and optional skin biopsy. Patients' medical records, baseline laboratory tests, quality of life, and diagnostic information including pathology reports and treatment history are also reviewed.

After the baseline visit, patients are followed up every 6 months. Follow up visits include disease evaluation, physical examination and collection of peripheral blood sample. Bone marrow biopsy and aspiration is performed when clinically necessary. Patients also complete the MDS-specific Qualify of Life in Myelodysplasia Scale (QUALMS), Functional Assessment of Cancer Therapy-General (FACT-G) (version 4), the Patient Reported Outcome Measurement Information System (PROMIS) Short Form version 1.0-Fatigue 7a, and the European Quality of Life-5 Dimensions-5 Levels (EQ-5D-5L).

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) criteria 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

No prior treatment for MDS at entry and through the time of the entry bone marrow aspirate

No treatment with hematopoietic growth factors in prior 6 months

B12 level, serum folate, ferritin, and thyroid-stimulating hormone (TSH) tests performed in prior 6 months

No 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

No treatment with radiation therapy in the two years prior to registration

No non-hormonal treatment for malignancy within the two years prior to registration

No established hereditary bone marrow failure syndrome

No known primary diagnosis of aplastic anemia, classical paroxysmal nocturnal hemoglobinuria, amegakaryocytic thrombocytopenic purpura, or large granular lymphocyte leukemia

Not enrolled in the Connect® MDS/Acute Myeloid Leukemia (AML) Disease Registry

In participants with suspected MDS and prior to registration with subsequent bone marrow evaluation, alternative causes for the cytopenias should be considered (e.g., internal bleeding, autoimmune cytopenias, thyroid disorders, other causes of anemia etc.); in select individuals, the following tests could be performed to assist in the diagnostic work-up; these evaluations are not required by the protocol; however, abnormal results in advance of enrollment may reduce the number of non-MDS cases

Copper, serum level

Iron studies (iron, total iron-binding capacity [TIBC] test, percent saturation)

Direct antiglobulin test

Antinuclear antibody (ANA) test

Based on centralized pathology review, participants will be classified into cases (MDS, MDS/MPN overlap disorders, or idiopathic cytopenia of undetermined significance [ICUS]) and others; in addition to baseline biological samples, longitudinal samples and data will be collected for approximately 2000 cases of MDS or MDS/MPN overlap disorders and 500 cases of ICUS; submitted samples will be reviewed by a central pathologist to determine eligibility for the longitudinal cohort (i.e., an MDS, MDS/MPN, or ICUS diagnosis); should a discrepancy in diagnosis occur between the central review and study site, the study site will be notified to allow for additional information to be submitted to clarify the diagnosis; such notifications will not occur in real time, and are not intended to assist in patient care

Trial Contact Information

Trial Lead Organizations / Sponsors / Collaborators

ECOG-ACRIN Cancer Research Group

National Cancer Institute

Mikkael Aaron Sekeres, Principal Investigator

Trial Sites

U.S.A.

Florida

Jacksonville

Baptist MD Anderson Cancer Center

Edward J. Gorak
Ph: 904-202-7051

Edward J. Gorak
Principal Investigator

Illinois

Bloomington

Illinois CancerCare-Bloomington

James Lloyd Wade
Ph: 217-876-4740
Email: kcheek@dmhhs.org

James Lloyd Wade
Principal Investigator

Galesburg

Illinois CancerCare-Galesburg

James Lloyd Wade
Ph: 217-876-4740
Email: kcheek@dmhhs.org

James Lloyd Wade
Principal Investigator

Peoria

Illinois CancerCare-Peoria

James Lloyd Wade
Ph: 217-876-4740
Email: kcheek@dmhhs.org

James Lloyd Wade
Principal Investigator

Iowa

Des Moines

Iowa Methodist Medical Center

Robert J. Behrens
Ph: 515-282-2921

Robert J. Behrens
Principal Investigator

Medical Oncology and Hematology Associates-Des Moines

Robert J. Behrens
Ph: 515-282-2921

Robert J. Behrens
Principal Investigator