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Myelodysplastic Syndrome--CDA-2 Hematological Improvement National Affirmation Study (MD-CHINA)

Primary Purpose

Myelodysplastic Syndrome (MDS)

Status
Unknown status
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
CDA-2 (Cell Differentiation Agent 2)
Sponsored by
Chinese Society of Hematology
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myelodysplastic Syndrome (MDS) focused on measuring CDA-2, MDS

Eligibility Criteria

18 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Documented diagnosis of MDS according to World Health Organization (WHO)/French American British (FAB) classification that meets IPSS-R classification of low, or intermediate-1 risk disease.
  • Subject is 18 to 85years of age the time of signing the informed consent form (ICF).
  • Able to adhere to the study visit schedule and other protocol requirements
  • Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2.
  • Laboratory test results within these ranges: Serum creatinine </=1.5 mg/dL x Upper limit of the normal (ULN),Blood urine nitrogen (BUN)</=1.5 mg/dL x Upper limit of the normal (ULN),Total bilirubin </=1.5 mg/dL x Upper limit of the normal (ULN),Serum glutamic oxaloacetic transaminase/aspartate transaminase (SGOT/AST) and Serum glutamic pyruvic transaminase/alanine transaminase (SGPT/ALT)</=2 x Upper limit of the normal (ULN).
  • No prior intensive combination chemotherapy or dose Azacitidine,Decitabine,and Lenalidomide,etc.
  • Capable of understanding the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent.

Exclusion Criteria:

  • IPSS risk group intermediate-2 or high risk
  • breast feeding and pregnant women
  • MDS associated with del 5q cytogenetic abnormality
  • Patients with history of hepatitis B, C, HIV(+), alcoholic liver disease or evidence of hepatopathy will be excluded.
  • Any significant concurrent disease, illness, or psychiatric disorder that would compromise patient safety or compliance, interfere with consent, study participation, follow up, or interpretation of study results.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    CDA-2 (Cell Differentiation Agent 2)

    Arm Description

    Patients will be given CDA-2 therapy.

    Outcomes

    Primary Outcome Measures

    Hematological Improvement (HI) at 12 Weeks
    Hematologic improvement (HI) per International Working Group (IWG),HI: hemoglobin increase of >= 1.5 g/dL, platelet increase of >= 30,000/mL (starting with > 20,000/mL), neutrophils increase of >= 100% and > 500/μL.

    Secondary Outcome Measures

    Response Rate of The Therapy at 12 Weeks
    IWG 2006 response criteria - CR: bone marrow evaluation shows <= 5% blasts; normal maturation of all cells lines (mCR), peripheral blood evaluation shows hemoglobin >= 11 g/dL, neutrophils >= 1000/mL, platelets >= 100,000/mL, 0% blasts; PR: Same as CR, except blasts decrease >= 50%, still greater than 5% in bone marrow
    Red Blood Cell Transfusion Independence (RBC-TI) in 24 weeks
    Proportion of subjects who are Red blood cell (RBC) transfusion free over any consecutive 84-day period within 24 weeks
    Change From Baseline to that of the 24 weeks of Scores of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire - Core 30 (EORTC QLQ C-30) Physical Functioning Scale
    The EORTC QLQ will be evaluated for each patients at the beginning and end of the study.

    Full Information

    First Posted
    October 17, 2017
    Last Updated
    November 4, 2017
    Sponsor
    Chinese Society of Hematology
    Collaborators
    Harbin Institute of Hematology & Oncology
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03335943
    Brief Title
    Myelodysplastic Syndrome--CDA-2 Hematological Improvement National Affirmation Study
    Acronym
    MD-CHINA
    Official Title
    The Efficacy and Safety of CDA-2 for the Treatment of IPSS Lower/Intermediate-risk Myelodysplastic Syndrome Patients: a Multi-centered Prospective Open Study
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    November 2017
    Overall Recruitment Status
    Unknown status
    Study Start Date
    December 1, 2017 (Anticipated)
    Primary Completion Date
    December 1, 2019 (Anticipated)
    Study Completion Date
    December 1, 2020 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    Chinese Society of Hematology
    Collaborators
    Harbin Institute of Hematology & Oncology

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    This Study aims to evaluate the efficacy and safety of CDA-2 in the treatment of International Prognostic Scoring System (IPSS) Lower/Intermediate-risk myelodysplastic syndrome (MDS) in Chinese patients.
    Detailed Description
    Patients with lower/intermediate-risk myelodysplastic syndrome (MDS) have rare therapeutic options other than supportive care. In pilot studies, CDA-2 showed promising results of hematological improvement in these patients. To date, the optimal regimen for CDA-2 treatment is not well established. The researchers are going to make a multi-centered clinical trial to evaluate the efficacy and safety of CDA-2 in 800 patients with International Prognostic Scoring System(IPSS) Lower/Intermediate-risk myelodysplastic syndrome (MDS). Eligible patients will be given CDA-2 intravenously, with 200 ml each day for 14 consecutive days in every four weeks (one cycle). The treatment will be repeated at least for 3 cycles. The patients will be followed up to 24 weeks. The primary endpoint is hematological improvement (HI) at 12 weeks according to IWG criteria. Full blood counts will be done on all patients every week. Change in bone marrow function as measured by changes in bone marrow morphology and cytogenetics will be assessed before and after 3 cycles of the treatment. The secondary endpoint is the therapy response. Complete remission (CR), partial remission (PR) and response duration, side effects, evaluation of QOL will be evaluated at the end of the treatment in every cycle. Adverse events of the treatment will be recorded for evaluation of the safety.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Myelodysplastic Syndrome (MDS)
    Keywords
    CDA-2, MDS

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 4
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    800 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    CDA-2 (Cell Differentiation Agent 2)
    Arm Type
    Experimental
    Arm Description
    Patients will be given CDA-2 therapy.
    Intervention Type
    Drug
    Intervention Name(s)
    CDA-2 (Cell Differentiation Agent 2)
    Other Intervention Name(s)
    Uroacitides (a compound mixed of peptides and organic acids)
    Intervention Description
    CDA-2 will be given intravenously, with 200 ml each day for 14 consecutive days in every four weeks (one cycle). The treatment will be repeated at least for 3 cycles.
    Primary Outcome Measure Information:
    Title
    Hematological Improvement (HI) at 12 Weeks
    Description
    Hematologic improvement (HI) per International Working Group (IWG),HI: hemoglobin increase of >= 1.5 g/dL, platelet increase of >= 30,000/mL (starting with > 20,000/mL), neutrophils increase of >= 100% and > 500/μL.
    Time Frame
    12 weeks
    Secondary Outcome Measure Information:
    Title
    Response Rate of The Therapy at 12 Weeks
    Description
    IWG 2006 response criteria - CR: bone marrow evaluation shows <= 5% blasts; normal maturation of all cells lines (mCR), peripheral blood evaluation shows hemoglobin >= 11 g/dL, neutrophils >= 1000/mL, platelets >= 100,000/mL, 0% blasts; PR: Same as CR, except blasts decrease >= 50%, still greater than 5% in bone marrow
    Time Frame
    12 weeks
    Title
    Red Blood Cell Transfusion Independence (RBC-TI) in 24 weeks
    Description
    Proportion of subjects who are Red blood cell (RBC) transfusion free over any consecutive 84-day period within 24 weeks
    Time Frame
    24 weeks
    Title
    Change From Baseline to that of the 24 weeks of Scores of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire - Core 30 (EORTC QLQ C-30) Physical Functioning Scale
    Description
    The EORTC QLQ will be evaluated for each patients at the beginning and end of the study.
    Time Frame
    24 weeks

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    85 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Documented diagnosis of MDS according to World Health Organization (WHO)/French American British (FAB) classification that meets IPSS-R classification of low, or intermediate-1 risk disease. Subject is 18 to 85years of age the time of signing the informed consent form (ICF). Able to adhere to the study visit schedule and other protocol requirements Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2. Laboratory test results within these ranges: Serum creatinine </=1.5 mg/dL x Upper limit of the normal (ULN),Blood urine nitrogen (BUN)</=1.5 mg/dL x Upper limit of the normal (ULN),Total bilirubin </=1.5 mg/dL x Upper limit of the normal (ULN),Serum glutamic oxaloacetic transaminase/aspartate transaminase (SGOT/AST) and Serum glutamic pyruvic transaminase/alanine transaminase (SGPT/ALT)</=2 x Upper limit of the normal (ULN). No prior intensive combination chemotherapy or dose Azacitidine,Decitabine,and Lenalidomide,etc. Capable of understanding the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent. Exclusion Criteria: IPSS risk group intermediate-2 or high risk breast feeding and pregnant women MDS associated with del 5q cytogenetic abnormality Patients with history of hepatitis B, C, HIV(+), alcoholic liver disease or evidence of hepatopathy will be excluded. Any significant concurrent disease, illness, or psychiatric disorder that would compromise patient safety or compliance, interfere with consent, study participation, follow up, or interpretation of study results.

    12. IPD Sharing Statement

    Plan to Share IPD
    No
    Citations:
    PubMed Identifier
    22735748
    Citation
    Ma X. Epidemiology of myelodysplastic syndromes. Am J Med. 2012 Jul;125(7 Suppl):S2-5. doi: 10.1016/j.amjmed.2012.04.014.
    Results Reference
    background
    PubMed Identifier
    25185242
    Citation
    Fenaux P, Haase D, Sanz GF, Santini V, Buske C; ESMO Guidelines Working Group. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2014 Sep;25 Suppl 3:iii57-69. doi: 10.1093/annonc/mdu180. Epub 2014 Jul 25. No abstract available.
    Results Reference
    background
    Links:
    URL
    https://www.ncbi.nlm.nih.gov/pubmed/?term=Ma+X.+The+American+journal+of+medicine.+2012%3B125(7+Suppl)%3AS2-5.
    Description
    Related Info
    URL
    https://www.ncbi.nlm.nih.gov/pubmed/?term=Fenaux+P%2C+Haase+D%2C+Sanz+GF%2C+Santini+V%2C+Buske+C%2C+Group+EGW.+Annals+of+oncology+%3A+official+journal+of+the+European+Society+for+Medical+Oncology+%2F+ESMO.
    Description
    Related Info

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