Myelodysplastic Syndrome (MDS)Phase 4MD-CHINANCT03335943
Myelodysplastic Syndrome--CDA-2 Hematological Improvement National Affirmation Study
A Phase 4 study testing CDA-2 (Cell Differentiation Agent 2) in people with Myelodysplastic Syndrome (MDS), run by Chinese Society of Hematology.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Myelodysplastic Syndrome (MDS)
- What is being tested?
- CDA-2 (Cell Differentiation Agent 2)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 800 participants
- Where
- No sites listed yet
- Study dates
- 1 Dec 2017to 1 Dec 2020
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
About this 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
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.
Read the 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.
- Conditions
- Myelodysplastic Syndrome (MDS)
- Being tested
- CDA-2 (Cell Differentiation Agent 2)
- Keywords
- CDA-2, MDS
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CDA-2 (Cell Differentiation Agent 2)Drug
Also called Uroacitides (a compound mixed of peptides and organic acids)
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.
| Study group | Role | What they receive |
|---|---|---|
| CDA-2 (Cell Differentiation Agent 2) | Experimental | Patients will be given CDA-2 therapy. Receives: Drug: CDA-2 (Cell Differentiation Agent 2) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 12 weeks
Secondary outcomes (3)
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
Measured: 12 weeks
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
Measured: 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.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2017
- Study start1 Dec 2017 (estimated)
- Primary completion1 Dec 2019 (estimated)
- Study completion1 Dec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Myelodysplastic Syndrome (MDS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 800 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chinese Society of Hematology
- Sponsor type
- University, hospital or other
- Collaborators
- Harbin Institute of Hematology & Oncology
- Responsible party
- Principal investigator, Wu Depei, Chinese Society of Hematology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03335943
- Sponsor's protocol ID
- CDA-2 MDS-2017
- Acronym
- MD-CHINA
- Status last verified
- Nov 2017
- First submitted
- 17 Oct 2017
- Last updated
- 8 Nov 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Ma X. Epidemiology of myelodysplastic syndromes. Am J Med. 2012 Jul;125(7 Suppl):S2-5. doi: 10.1016/j.amjmed.2012.04.014.
PubMed 22735748Fenaux 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.
PubMed 25185242
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03335943
- Acronym / sponsor ID
- MD-CHINA / CDA-2 MDS-2017
- Conditions
- Myelodysplastic Syndrome (MDS)
- Keywords
- CDA-2, MDS
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 800 participants (estimated)
- Lead sponsor
- Chinese Society of HematologyUniversity, hospital or other
- Collaborators
- Harbin Institute of Hematology & Oncology
- Study start
- 1 Dec 2017 (estimated)
- Primary completion
- 1 Dec 2019 (estimated)
- Study completion
- 1 Dec 2020 (estimated)
- First posted
- 8 Nov 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CDA-2 (Cell Differentiation Agent 2) | Experimental | Drug: CDA-2 (Cell Differentiation Agent 2)Patients will be given CDA-2 therapy. |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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).
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CDA-2 (Cell Differentiation Agent 2)Also: Uroacitides (a compound mixed of peptides and organic acids)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. | Drug | CDA-2 (Cell Differentiation Agent 2) |
Primary outcome
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.
3 secondary outcomes
Response Rate of The Therapy at 12 Weeks
Time frame: 12 weeks
Red Blood Cell Transfusion Independence (RBC-TI) in 24 weeks
Time frame: 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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2017.
- Background: PubMed 22735748Ma X. Epidemiology of myelodysplastic syndromes. Am J Med. 2012 Jul;125(7 Suppl):S2-5. doi: 10.1016/j.amjmed.2012.04.014.
- Background: PubMed 25185242Fenaux 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.
- Related InfoLink provided by the sponsor.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing CDA-2 (Cell Differentiation Agent 2) in people with Myelodysplastic Syndrome (MDS), run by Chinese Society of Hematology.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 8 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hematological Improvement (HI) at 12 Weeks (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03335943, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Nov 2017.