Myelodysplastic SyndromePhase 2NCT00744757
An Efficacy and Safety Study of Decitabine in Participants With Myelodysplastic Syndrome (MDS)
A Phase 2 study testing Decitabine in people with Myelodysplastic Syndrome, run by Johnson & Johnson Taiwan Ltd at 6 sites in Taiwan.
Trial summary
The purpose of this study is to evaluate the response rate of decitabine in previously treated and untreated Taiwanese participants with Myelodysplastic Syndrome (MDS - a disease associated with decreased production of blood cells, blood cells are produced but do not mature normally). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome
- What is being tested?
- Decitabine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 37 participants
- Where
- 6 sitesTaiwan
- Study dates
- Aug 2008to Aug 2012
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants with documented pathological (bone marrow, no longer than 30 days before first dosing in study) evidence of Myelodysplastic syndromes (MDS) or of chronic myelomonocytic leukemia (CMML) by World health organisation classification
- Participants with international prognostic scoring system (IPSS) score equal to 0.5 or more (only for participants for whom IPSS is applicable)
- Participants with an Eastern oncology cooperative group (ECOG) performance status of 0-2
- Participants with adequate hepatic (liver) and renal (kidney) function as measured by pre-treatment laboratory criteria within 21 days of starting treatment with decitabine
- Participants must have recovered from toxic effects of previous therapy and not receiving any chemotherapy for a minimum of 4 weeks (6 weeks if the participants has been treated with a nitrosoureas) before to the first dose of study drug
Who cannot join
- Participants with a diagnosis of acute myeloid leukemia (AML) (greater than 30 percent bone marrow blasts)
- Participants with AML with multilineage dysplasia (abnormal development or cell growth) following MDS (20-30 percent bone marrow blasts) can be enrolled. For these latter participants an observation period of 1 month is necessary to exclude those participants with rapid progression to full blown AML
- Participants with previous treatment with azacitadine or decitabine or hematopoietic stem cell transplantation less than 1 year prior to study enrollment
- Participants with past history of malignancy and received any treatment for this before malignancy within the last 3 years, except for superficial bladder cancer, basal cell or squamous cell carcinoma of the skin, cervical intraepithelial neoplasia (CIN) or prostate intraepithelial neoplasia (PIN)
- Participants with known hepatitis B (surface antigen-positive) or active hepatitis C infection
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Taiwan6 sites
Study site
Changhua
Study site
Kaohsiung City
Study site
Taichung
Study site
Tainan
Study site
Taipei
Study site
Tau-Yuan County 333
About this study
Official title: A Phase II Multi-center Study of 5-AZA-2'-Deoxycytidine (Decitabine) Single Agent in Taiwanese Patients With Myelodysplastic Syndrome (MDS)
The purpose of this study is to evaluate the response rate of decitabine in previously treated and untreated Taiwanese participants with Myelodysplastic Syndrome (MDS - a disease associated with decreased production of blood cells, blood cells are produced but do not mature normally).
Read the detailed description
This is an open-label (a medical research study in which participants and researchers are told which treatments the participants are receiving, "unblinded"), multi-center (when more than 1 hospital or medical school team work on a medical research study), single-arm study of decitabine. The study will consist of 5 phases: Pre-treatment phase (before 30 days of first dose), Treatment phase (consist of 8 cycles, each cycle of 28 days), End-of-treatment phase (consist of 30-42 days after the last dose of cycle 8, or at time of discontinuation), Extension phase (1 cycle of 4 weeks) and Post-study phase or follow-up phase (every 2 months until 1 year, lost to follow-up or death). Participants who achieve a complete remission (when a medical problem gets better or goes away at least for a while) will be treated for at least 2 more cycles after first documentation of complete response (CR), after which treatment can be discontinued. Decitabine in a dose of 20 milligram per square meter (mg per m^2) will be administered intravenously over 1 hour infusion, 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity. The primary objective is to evaluate the best response rate (complete response and partial response). Participants' safety will be monitored throughout the study.
- Conditions
- Myelodysplastic Syndrome
- Being tested
- Decitabine
- Keywords
- 5-aza-2'-deoxycytidine, decitabine, dacogen, 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 Dacogen, 5-aza-2'-deoxycytidine
Decitabine 20 mg per m^2 will be administered intravenous infusion over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
| Study group | Role | What they receive |
|---|---|---|
| Decitabine | Experimental | Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity. Receives: Drug: Decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Response
Percentage of participants with response: complete response (CR) or partial response (PR) according to International Working Group (IWG) 2006 criteria was evaluated. CR in bone marrow is defined as<=to 5% myeloblasts with normal maturation of all cell lines and persistent dysplasia was noted in peripheral blood hemoglobin >=11 gram (g) per deciliter (dl), platelets >=100*10^9 liter (l), neutrophils >=1.0*10^9 l, Blasts 0%. Partial response is defined as all CR criteria if abnormal before treatment except: bone marrow blasts decreased by >=50% over pre-treatment but still >=5%.
Measured: Day 1 of Cycle 2, 4, 6, 8; each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)
Secondary outcomes (9)
Percentage of Participants With Hematologic Treatment Response
Hematologic treatment response was assessed as per IWG 2006 criteria. This is measured in erythroid(HI-E), platelet(HI-P) and neutrophil(HI-N) lineages. HI-E response(pre-treatment<11 gram per deciliter [g/dl]):hemoglobin increase by>=1.5 g/dl and relevant reduction in RBC transfusions by 4 RBC transfusions/8week. HI-P response (pre-treatment<100*109/l):absolute increase of >=30*10^9/l for participants starting with>20*10^9/l and increase from <20*10^9/l to>20*10^9/l and by at least 100%. HI-N response (pre-treatment<1.0*10^9/l): at least 100% increase and an absolute increase >0.5*10^9/l.
Measured: Day 1 of Cycle 1, 2, 3, 4, 5, 6, 7 and 8, each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)
Percentage of Participants With Cytogenetic Response
Cytogenetic responses was assessed as per IWG 2006 criteria which define complete response as disappearance of the chromosomal abnormality without appearance of new ones and partial response as at least 50 percent reduction of the chromosomal abnormality.
Measured: Day 1 of Cycle 2, 4, 6, 8; each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)
Time to Acute Myeloid Leukemia (AML) Progression or Death
Time to AML is defined as greater than 30 percent of blasts in bone marrow or the time to death was calculated from the date of treatment start until disease progression to AML or until death, which ever occurred first. Participants who were still alive and did not progress to AML were censored at the moment of last visit.
Measured: Start of treatment until disease progression or death (whichever occur first) or up to 736 days
Overall Survival
Overall survival was defined as the time from the date of treatment start until death (whatever the cause). It was calculated from Kaplan-Meier estimates. Participants still alive were censored at the moment of last visit or contact.
Measured: Start of treatment until disease progression or death (whichever occur first) or up to 736 days
Percentage of Participants With Transfusion Dependency
Transfusion requirements for both red blood cells as well as platelets were recorded for each participant.
Measured: 8 weeks before first dose until disease progression or death (whichever occur first) or up to 736 days
Percentage of Participants With Transfusion Independency
Transfusion independent participants were calculated from all the participants who required transfusion in the duration of 8 weeks before first dose until disease progression or death or up to 736 days. Transfusion independence was defined as lack of requirement for transfusions for at least 8 weeks.
Measured: 8 weeks before first dose and 736 days of treatment
Duration for Hospitalization
Duration of hospitalization was calculated for each participant, using the sum of all hospital days by subtracting the date of discharge from the date of admission.
Measured: Cycle 1 up to Cycle 8, each cycle of 28 days.
Number of Events Which Led to Hospitalization
The events (reasons) for hospitalizations such as infection, transfusion, acute choleycystitis, allergic transfusion reaction, dyspnoea with right pleural effusion, febrile neutropenia, fever, for decitabine, Myelodysplastic Syndrome (MDS) hematuria, paronychia, pneumonia, heart failure, peri-anal abscess (PAA), pancytopenia,fluctuated neutropenia fever, right dorsal foot cellulitis, Right lower (Rt.Lw) lung pneumonia with impending respiratory (resp) failure, Serious adverse event (SAE)+schedule hospitalization for decitabine, septic shock and not available were reported.
Measured: Start of treatment until disease progression or death or up to Cycle 8, each cycle of 28 days
Quality of Life Assessment
The quality of life was assessed by the questionnaire QLQ C-30 designed by European Organization for the Research and Treatment of Cancer (EORTC). EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer participants. Most questions used 4-point scale (1=Not at All' to 4=Very Much') and 2 questions: Q29 on overall health and Q30 on overall quality of life uses 7-point scale ranging from 1=Very Poor to 7=Excellent. Higher score indicates better quality of life.
Measured: Day 1 of Cycle 1 and Cycle 8 (each cycle of 28 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2008 (estimated)
- Study startAug 2008
- Primary completionSep 2010
- Study completionAug 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myelodysplastic SyndromeStudies 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
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johnson & Johnson Taiwan Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Johnson & Johnson Taiwan, Ltd. Clinical Trial, Study director, Johnson & Johnson Taiwan Ltd
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00744757
- Sponsor's protocol ID
- CR014785
- Other IDs
- DACOGENMDS2001
- Status last verified
- Aug 2013
- First submitted
- 29 Aug 2008
- Last updated
- 9 Sep 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Multi-center Study of 5-AZA-2'-Deoxycytidine (Decitabine) Single Agent in Taiwanese Patients With Myelodysplastic Syndrome (MDS)
- ClinicalTrials.gov ID
- NCT00744757
- Acronym / sponsor ID
- CR014785
- Conditions
- Myelodysplastic Syndrome
- Keywords
- 5-aza-2'-deoxycytidine, decitabine, dacogen, Myelodysplastic Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Johnson & Johnson Taiwan LtdIndustry (pharma, biotech, devices)
- Study start
- Aug 2008
- Primary completion
- Sep 2010 (actual)
- Study completion
- Aug 2012 (actual)
- First posted
- 1 Sep 2008 (estimated)
- Results
- Posted 15 Aug 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the response rate of decitabine in previously treated and untreated Taiwanese participants with Myelodysplastic Syndrome (MDS - a disease associated with decreased production of blood cells, blood cells are produced but do not mature normally).
Detailed description
This is an open-label (a medical research study in which participants and researchers are told which treatments the participants are receiving, "unblinded"), multi-center (when more than 1 hospital or medical school team work on a medical research study), single-arm study of decitabine. The study will consist of 5 phases: Pre-treatment phase (before 30 days of first dose), Treatment phase (consist of 8 cycles, each cycle of 28 days), End-of-treatment phase (consist of 30-42 days after the last dose of cycle 8, or at time of discontinuation), Extension phase (1 cycle of 4 weeks) and Post-study phase or follow-up phase (every 2 months until 1 year, lost to follow-up or death). Participants who achieve a complete remission (when a medical problem gets better or goes away at least for a while) will be treated for at least 2 more cycles after first documentation of complete response (CR), after which treatment can be discontinued. Decitabine in a dose of 20 milligram per square meter (mg per m^2) will be administered intravenously over 1 hour infusion, 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity. The primary objective is to evaluate the best response rate (complete response and partial response). Participants' safety will be monitored 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 |
|---|---|---|
| Decitabine | Experimental | Drug: DecitabineDecitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Participants with documented pathological (bone marrow, no longer than 30 days before first dosing in study) evidence of Myelodysplastic syndromes (MDS) or of chronic myelomonocytic leukemia (CMML) by World health organisation classification
- Participants with international prognostic scoring system (IPSS) score equal to 0.5 or more (only for participants for whom IPSS is applicable)
- Participants with an Eastern oncology cooperative group (ECOG) performance status of 0-2
- Participants with adequate hepatic (liver) and renal (kidney) function as measured by pre-treatment laboratory criteria within 21 days of starting treatment with decitabine
- Participants must have recovered from toxic effects of previous therapy and not receiving any chemotherapy for a minimum of 4 weeks (6 weeks if the participants has been treated with a nitrosoureas) before to the first dose of study drug
Key exclusion criteria (5 listed)
- Participants with a diagnosis of acute myeloid leukemia (AML) (greater than 30 percent bone marrow blasts)
- Participants with AML with multilineage dysplasia (abnormal development or cell growth) following MDS (20-30 percent bone marrow blasts) can be enrolled. For these latter participants an observation period of 1 month is necessary to exclude those participants with rapid progression to full blown AML
- Participants with previous treatment with azacitadine or decitabine or hematopoietic stem cell transplantation less than 1 year prior to study enrollment
- Participants with past history of malignancy and received any treatment for this before malignancy within the last 3 years, except for superficial bladder cancer, basal cell or squamous cell carcinoma of the skin, cervical intraepithelial neoplasia (CIN) or prostate intraepithelial neoplasia (PIN)
- Participants with known hepatitis B (surface antigen-positive) or active hepatitis C infection
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: Dacogen, 5-aza-2'-deoxycytidineDecitabine 20 mg per m^2 will be administered intravenous infusion over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity. | Drug | Decitabine |
Primary outcome
Percentage of Participants With Response
Time frame: Day 1 of Cycle 2, 4, 6, 8; each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)
Percentage of participants with response: complete response (CR) or partial response (PR) according to International Working Group (IWG) 2006 criteria was evaluated. CR in bone marrow is defined as<=to 5% myeloblasts with normal maturation of all cell lines and persistent dysplasia was noted in peripheral blood hemoglobin >=11 gram (g) per deciliter (dl), platelets >=100*10^9 liter (l), neutrophils >=1.0*10^9 l, Blasts 0%. Partial response is defined as all CR criteria if abnormal before treatment except: bone marrow blasts decreased by >=50% over pre-treatment but still >=5%.
9 secondary outcomes
Percentage of Participants With Hematologic Treatment Response
Time frame: Day 1 of Cycle 1, 2, 3, 4, 5, 6, 7 and 8, each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)
Percentage of Participants With Cytogenetic Response
Time frame: Day 1 of Cycle 2, 4, 6, 8; each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)
Time to Acute Myeloid Leukemia (AML) Progression or Death
Time frame: Start of treatment until disease progression or death (whichever occur first) or up to 736 days
Overall Survival
Time frame: Start of treatment until disease progression or death (whichever occur first) or up to 736 days
Percentage of Participants With Transfusion Dependency
Time frame: 8 weeks before first dose until disease progression or death (whichever occur first) or up to 736 days
Percentage of Participants With Transfusion Independency
Time frame: 8 weeks before first dose and 736 days of treatment
Duration for Hospitalization
Time frame: Cycle 1 up to Cycle 8, each cycle of 28 days.
Number of Events Which Led to Hospitalization
Time frame: Start of treatment until disease progression or death or up to Cycle 8, each cycle of 28 days
Quality of Life Assessment
Time frame: Day 1 of Cycle 1 and Cycle 8 (each cycle of 28 days)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Johnson & Johnson Taiwan, Ltd. Clinical Trial study director
Johnson & Johnson Taiwan Ltd
6 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 Syndrome, run by Johnson & Johnson Taiwan Ltd at 6 sites in Taiwan.
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 5 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: finished, as of the sponsor's last update on 9 Sep 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Taiwan. 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?
Yes. Results are posted in the registry (first posted 15 Aug 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Participants With Response (time frame: Day 1 of Cycle 2, 4, 6, 8; each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)). 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 9 Sep 2013.