Clinical Trials /

Myelodysplastic Syndrome--CDA-2 Hematological Improvement National Affirmation Study

NCT03335943

Description:

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.

Related Conditions:
  • Myelodysplastic Syndromes
Recruiting Status:

Unknown status

Phase:

Phase 4

Trial Eligibility

Document

Title

  • Brief Title: Myelodysplastic Syndrome--CDA-2 Hematological Improvement National Affirmation Study
  • 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

Clinical Trial IDs

  • ORG STUDY ID: CDA-2 MDS-2017
  • NCT ID: NCT03335943

Conditions

  • Myelodysplastic Syndrome (MDS)

Interventions

DrugSynonymsArms
CDA-2 (Cell Differentiation Agent 2)Uroacitides (a compound mixed of peptides and organic acids)CDA-2 (Cell Differentiation Agent 2)

Purpose

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.
    

Trial Arms

NameTypeDescriptionInterventions
CDA-2 (Cell Differentiation Agent 2)ExperimentalPatients will be given CDA-2 therapy.
  • CDA-2 (Cell Differentiation Agent 2)

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.
      
Maximum Eligible Age:85 Years
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Hematological Improvement (HI) at 12 Weeks
Time Frame:12 weeks
Safety Issue:
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.

Secondary Outcome Measures

Measure:Response Rate of The Therapy at 12 Weeks
Time Frame:12 weeks
Safety Issue:
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
Measure:Red Blood Cell Transfusion Independence (RBC-TI) in 24 weeks
Time Frame:24 weeks
Safety Issue:
Description:Proportion of subjects who are Red blood cell (RBC) transfusion free over any consecutive 84-day period within 24 weeks
Measure: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
Time Frame:24 weeks
Safety Issue:
Description:The EORTC QLQ will be evaluated for each patients at the beginning and end of the study.

Details

Phase:Phase 4
Primary Purpose:Interventional
Overall Status:Not yet recruiting
Lead Sponsor:Chinese Society of Hematology

Trial Keywords

  • CDA-2, MDS

Last Updated

November 4, 2017