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.
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
| Drug | Synonyms | Arms |
|---|
| 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
| Name | Type | Description | Interventions |
|---|
| CDA-2 (Cell Differentiation Agent 2) | Experimental | Patients 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: | Unknown status |
| Lead Sponsor: | Chinese Society of Hematology |
Trial Keywords
Last Updated
November 8, 2017
