Myelodysplastic Syndrome--CDA-2 Hematological Improvement National Affirmation Study (MD-CHINA)
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|ClinicalTrials.gov Identifier: NCT03335943|
Recruitment Status : Not yet recruiting
First Posted : November 8, 2017
Last Update Posted : November 8, 2017
|Condition or disease||Intervention/treatment||Phase|
|Myelodysplastic Syndrome (MDS)||Drug: CDA-2 (Cell Differentiation Agent 2)||Phase 4|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||800 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Estimated Study Start Date :||December 1, 2017|
|Estimated Primary Completion Date :||December 1, 2019|
|Estimated Study Completion Date :||December 1, 2020|
Experimental: CDA-2 (Cell Differentiation Agent 2)
Patients will be given CDA-2 therapy.
Drug: CDA-2 (Cell Differentiation Agent 2)
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.
Other Name: Uroacitides (a compound mixed of peptides and organic acids)
- Hematological Improvement (HI) at 12 Weeks [ Time Frame: 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.
- Response Rate of The Therapy at 12 Weeks [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 24 weeks ]The EORTC QLQ will be evaluated for each patients at the beginning and end of the study.