Myelodysplastic SyndromesPhase 2NCT03045510
Efficacy and Safety of Ultra Small Dose Decitabine for the Lower Risk MDS Patients With Transfusion Dependent
A Phase 2 study testing decitabine in people with Myelodysplastic Syndromes, run by Shandong University at 2 sites in China.
Trial summary
Myelodysplastic syndrome (MDS) is widely recognized as a clonal hematopoietic stem cell disorder. Decitabine has been approved for the treatment of all subtypes of myelodysplastic syndrome (MDS). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- decitabine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 2 sitesChina
- Study dates
- 1 Dec 2016to 30 Jul 2020
- Phase
- Phase 2
- 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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- diagnosis of MDS
- The IPSS [17] score ≤ 1
- patients with transfusion dependent
- Adequate hepatic and renal function (aspartate aminotransferase [AST] ≤ 2.5 x upper normal limit, alanine aminotransferase [ALT] ≤ 2.5 x upper normal limit, bilirubin ≤ 1.5 x upper normal limit and creatinine < 2 x upper normal limit, Ccr > 60ml/min ).
Who cannot join
- Decitabine and Arsenic trioxide allergy
- Pregnancy and lactation
- Cardiovascular disease
- ECOG score > 2
- HCV, HIV, HBsAg seropositive status
Trial locations and contacts
2 sites in 1 country.
Hou Ming contact
China2 sites, 2 recruiting
- Recruiting
Qilu hospital, Shandong University
Jinan, Shandong, 250012
Ming Hou, houming@medmail.com.cn
Ming Hou
- Recruiting
Shandong University Qilu Hospital
Jinan, Shandong, 250012
Ming Lv
Ming Hou, Dr
About this study
Official title: A Single-center Prospective Clinical Trial of the Efficacy and Safety of Ultra Small Dose Decitabine for the Lower Risk Myelodysplastic Syndrome Patients With Transfusion Dependent
Myelodysplastic syndrome (MDS) is widely recognized as a clonal hematopoietic stem cell disorder. Decitabine has been approved for the treatment of all subtypes of myelodysplastic syndrome (MDS). However, the use of decitabine is often limited by its severe toxicity represented by myelosuppression even at relatively low doses. In lower-risk patients (including IPSS low and int-1 risk groups), treatment mainly aims at improving cytopenias, especially anemia. However, although several drugs may improve anemia, sometimes durably, most of lower risk MDS eventually require red blood cell (RBC) transfusions during their disease course. Long term RBC transfusions lead to iron overload mainly due to an increase in reticulo-endothelial iron recycling.Cardiac, liver and endocrine (diabetes mellitus) dysfunction due to iron overload and often leading to fatal outcome has been reported in heavily transfused lower risk MDS patients.
To date, the optimal regimen for decitabine treatment is not well established. In this study, we perform a prospective analysis to explore the decitabine schedule for the treatment of lower risk myelodysplastic syndrome patients with transfusion dependent.
Read the detailed description
The investigators are undertaking a single-center, single-arm study of 50 lower risk myelodysplastic syndrome patients with transfusion dependent from Shandong University Qilu Hospital . All the participants are selected to receive ultra small dose decitabine treatment (given intravenously at a dose of 3.5mg/m2, qd x 5d, every four weeks for one cycle). A routine blood examination is performed twice every week. Bone marrow (BM) is examined with routine aspirate smear and G-banding analysis every 1-2 treatment courses to evaluate responses.Adverse events are also recorded throughout the study.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- decitabine
- Keywords
- Decitabine Myelodysplastic syndrome
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
decitabineDrug
Also called ultra small dose decitabine
Decitabine 3.5mg/m2，ivdrip，qd x 5d, every four weeks for one cycle. It will be given six cycles.
| Study group | Role | What they receive |
|---|---|---|
| Ultra small dose decitabine | Experimental | Decitabine 3.5mg/m2，ivdrip，qd x 5d, every four weeks for one cycle. It will be given six cycles. Receives: Drug: decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
complete response
Bone marrow blasts not more than 5%, absolute neutrophil count more than 1*10^9/L, HgB more than 100g/L, and platelet count more than 100*10^9/L.
Measured: 30 days from the emrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2017 (estimated)
- Study start1 Dec 2016
- Primary completion30 Dec 2018 (estimated)
- Study completion30 Jul 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shandong University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ming Hou, Shandong University
- Investigators
- Ming Lv, Doctor, Principal investigator, Shandong University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03045510
- Sponsor's protocol ID
- lower risk MDS-decitabine
- Status last verified
- Jan 2017
- First submitted
- 3 Feb 2017
- Last updated
- 7 Feb 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-center Prospective Clinical Trial of the Efficacy and Safety of Ultra Small Dose Decitabine for the Lower Risk Myelodysplastic Syndrome Patients With Transfusion Dependent
- ClinicalTrials.gov ID
- NCT03045510
- Acronym / sponsor ID
- lower risk MDS-decitabine
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Decitabine Myelodysplastic syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Shandong UniversityUniversity, hospital or other
- Study start
- 1 Dec 2016 (actual)
- Primary completion
- 30 Dec 2018 (estimated)
- Study completion
- 30 Jul 2020 (estimated)
- First posted
- 7 Feb 2017 (estimated)
- 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.
Myelodysplastic syndrome (MDS) is widely recognized as a clonal hematopoietic stem cell disorder. Decitabine has been approved for the treatment of all subtypes of myelodysplastic syndrome (MDS). However, the use of decitabine is often limited by its severe toxicity represented by myelosuppression even at relatively low doses. In lower-risk patients (including IPSS low and int-1 risk groups), treatment mainly aims at improving cytopenias, especially anemia. However, although several drugs may improve anemia, sometimes durably, most of lower risk MDS eventually require red blood cell (RBC) transfusions during their disease course. Long term RBC transfusions lead to iron overload mainly due to an increase in reticulo-endothelial iron recycling.Cardiac, liver and endocrine (diabetes mellitus) dysfunction due to iron overload and often leading to fatal outcome has been reported in heavily transfused lower risk MDS patients.
To date, the optimal regimen for decitabine treatment is not well established. In this study, we perform a prospective analysis to explore the decitabine schedule for the treatment of lower risk myelodysplastic syndrome patients with transfusion dependent.
Detailed description
The investigators are undertaking a single-center, single-arm study of 50 lower risk myelodysplastic syndrome patients with transfusion dependent from Shandong University Qilu Hospital . All the participants are selected to receive ultra small dose decitabine treatment (given intravenously at a dose of 3.5mg/m2, qd x 5d, every four weeks for one cycle). A routine blood examination is performed twice every week. Bone marrow (BM) is examined with routine aspirate smear and G-banding analysis every 1-2 treatment courses to evaluate responses.Adverse events are also recorded throughout the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ultra small dose decitabine | Experimental | Drug: decitabineDecitabine 3.5mg/m2，ivdrip，qd x 5d, every four weeks for one cycle. It will be given six cycles. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- diagnosis of MDS
- The IPSS [17] score ≤ 1
- patients with transfusion dependent
- Adequate hepatic and renal function (aspartate aminotransferase [AST] ≤ 2.5 x upper normal limit, alanine aminotransferase [ALT] ≤ 2.5 x upper normal limit, bilirubin ≤ 1.5 x upper normal limit and creatinine < 2 x upper normal limit, Ccr > 60ml/min ).
Key exclusion criteria (5 listed)
- Decitabine and Arsenic trioxide allergy
- Pregnancy and lactation
- Cardiovascular disease
- ECOG score > 2
- HCV, HIV, HBsAg seropositive status
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 |
|---|---|---|
| decitabineAlso: ultra small dose decitabineDecitabine 3.5mg/m2，ivdrip，qd x 5d, every four weeks for one cycle. It will be given six cycles. | Drug | Ultra small dose decitabine |
Primary outcome
complete response
Time frame: 30 days from the emrollment
Bone marrow blasts not more than 5%, absolute neutrophil count more than 1*10^9/L, HgB more than 100g/L, and platelet count more than 100*10^9/L.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Feb 2017.
- Individual participant dataSharing planned: Undecided
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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.
Hou Ming contact
Ming Lv, Doctor principal investigator
Shandong University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing decitabine in people with Myelodysplastic Syndromes, run by Shandong University at 2 sites in China.
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 4 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 7 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Contact Hou Ming (houming@medmail.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
complete response (time frame: 30 days from the emrollment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 7 Feb 2017.