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

Chinese Society of Hematology

Status and phase

Unknown

Phase 4

Conditions

Myelodysplastic Syndrome (MDS)

Treatments

Drug: CDA-2 (Cell Differentiation Agent 2)

Study type

Interventional

Funder types

Other

Identifiers

NCT03335943

CDA-2 MDS-2017

Details and patient eligibility

About

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.

Full 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.

Enrollment

800 estimated patients

Sex

All

Ages

18 to 85 years old

Volunteers

No 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.