Myelodysplastic SyndromesPhase 2NCT00915785
Azacytidine for the Treatment of Myelodysplastic Syndromes/Acute Myeloid Leukemia (MDS/AML) With High Risk (Chromosome 7 and or Complex) Cytogenetic Abnormalities
A Phase 2 study testing 5 azacytidine in people with Myelodysplastic Syndromes and Leukemia, Myeloid, Acute, run by King's College London at 1 site in United Kingdom.
Trial summary
The purpose of this study is to assess the hematological and cytogenetic responses with 5 azacytidine in patients over 55 years of age with MDS/AML due to chromosome 7 abnormalities and to assess the hematological and cytogenetic response rates in patients with relapsed AML and chromosome 7 abnormality. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute
- What is being tested?
- 5 azacytidine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Nov 2005to Apr 2013
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Abnormalities to chromosome 7, including monosomy 7 either alone or as part of a complex clone.
- Be > 55 years of age; younger if first or subsequent relapse in patient less < 55 years but with a chromosome 7 abnormality alone or as part of a complex clone.
- Have an International Prognostic Scoring System (IPSS) score of INT 1.5 and a diagnosis of RAEB or RAEB-T per French-American-British (FAB) classification criteria or a diagnosis of Myelodysplastic CMMoL per modified FAB criteria meeting the following:
- Monocytosis in peripheral blood > 1x109/L;
- Dysplasia in one or more myeloid cell lines;
- 10% to 29% blasts in the BM;
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- White blood cell (WBC) < 13,000 x109/L;
- Have a life expectancy of at least 3 months;
- Have an Eastern Cooperative Oncology Group (ECOG) Performance Status Grade of 0-2.
- Have serum bilirubin levels of at least 1.5 x the upper limit of the normal range for the laboratory (ULN). Higher levels are acceptable if these can be attributed to:
- active hemolysis (as indicated by positive direct Coombs' testing);
- decreased or absent haptoglobin level;
- elevated indirect bilirubin and/or lactate dehydrogenase [LDH]); or
- ineffective erythropoiesis (as indicated by bone marrow findings).
- Have serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or serum glutamic-pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) levels of at least 2 x ULN.
- Have serum creatinine levels of at least 1.5 x ULN.
- Women of childbearing potential may participate, providing they meet the following conditions:
- must agree to use at least 2 effective contraceptive methods throughout the study and for 3 months following the date of the last dose of study medication;
- must have a negative serum pregnancy test obtained within 24 hours prior to Day 1.
- Males with female partners of childbearing potential must agree to use at least 2 effective contraceptive methods throughout the study and should avoid fathering a child for 6 months following the date of the last dose of study medication.
- Be able to provide written informed consent.
Who cannot join
- Prior treatment with azacitidine.
- Diagnosis of malignant disease within the previous 12 months (excluding basal cell carcinoma with no complications).
- Diagnosis of metastatic disease.
- Previous diagnosis of hepatic tumors.
- Radiation therapy, chemotherapy, or cytotoxic therapy, given to treat conditions other than MDS/AML and administered within the previous 12 months prior to the first day of treatment (Day 1).
- Known or suspected hypersensitivity to azacitidine or mannitol.
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- Prior or active disease that, in the opinion of the Investigator, may interfere with the procedures or evaluations to be conducted in the study.
- Serious medical illness likely to limit survival to under or equal to 12 months after screening or likely to prevent granting of informed consent (e.g., history of severe congestive heart failure, clinically unstable cardiac disease, or pulmonary disease).
- Psychiatric illness that would prevent granting of informed consent;
- Treatment with erythropoietin or myeloid growth factors (granulocyte colony-stimulating factor [G-CSF] or granulocyte-macrophage colony-stimulating factor [GM-CSF]) during the previous 21 days prior to Day 1.
- Treatment with androgenic hormones during the previous 14 days prior to Day 1.
- Active viral infection with known human immunodeficiency virus (HIV) or viral hepatitis type B or C.
- Treatment with other investigational drugs within the previous 30 days prior to Day 1, or ongoing adverse events from previous treatment with investigational drugs, regardless of the time period.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
King's College Hospital NHS Foundation Trust
London, SE5 9RS
About this study
Official title: Pilot Study of 5 Azacytidine in the Treatment of Myelodysplastic Syndromes/Acute Myeloid Leukaemia With High-risk (Chromosome 7 and or Complex) Cytogenetic Abnormalities
The purpose of this study is to assess the hematological and cytogenetic responses with 5 azacytidine in patients over 55 years of age with MDS/AML due to chromosome 7 abnormalities and to assess the hematological and cytogenetic response rates in patients with relapsed AML and chromosome 7 abnormality.
- Being tested
- 5 azacytidine
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
5 azacytidineDrug
Patients will receive azacytidine 75 mg/m2/day SC for 7 days every 28 days for up to 6 cycles, unless they are discontinued from the treatment.
| Study group | Role | What they receive |
|---|---|---|
| 5 azacytidine | Experimental | Not described Receives: Drug: 5 azacytidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
The rate of haematological and cytogenetic response in patients with MDS/AML with a chromosome 7 abnormality either alone or as part of a complex clone
Measured: Blood - Weekly for first 2 cycles and then fortnightly. Bone marrow - Day 7 of first cycle and then 16 weekly thereafter or ealier if clinically indicated.
Secondary outcomes (4)
Time to relapse after complete remission (CR) or partial remission (PR), or disease progression (per IWG criteria), censored at death
Measured: Blood - Weekly for first 2 cycles and then fortnightly. Bone marrow - Day 7 of first cycle and then 16 weekly thereafter or ealier if clinically indicated.
Duration of response and duration of improvement
Measured: Blood - Weekly for first 2 cycles and then fortnightly. Bone marrow - Day 7 of first cycle and then 16 weekly thereafter or ealier if clinically indicated.
Time to AML transformation or death from any cause
Measured: Blood - Weekly for first 2 cycles and then fortnightly. Bone marrow - Day 7 of first cycle and then 16 weekly thereafter or ealier if clinically indicated.
Additional cytogenetic markers: DNA methylation status, assessment of markers of apoptosis. Change in gene expression and single nucleotide polymorphism profiles.
Measured: Baseline, Day 7 of the first treatment cycle and then 16 weekly (or ealier if clinically indicated).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2009 (estimated)
- Study startNov 2005
- Primary completionApr 2013
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.
- More trials run by King's College LondonThe sponsor's other registered studies.
- 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
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- King's College London
- Sponsor type
- University, hospital or other
- Investigators
- Ghulam J Mufti, MB, DM, FRCP, FRCPath, Principal investigator, King's College London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00915785
- Sponsor's protocol ID
- 05CC12
- Other IDs
- REC - 05/Q0703/168, EudraCT - 2005-003732-22
- Status last verified
- Apr 2013
- First submitted
- 5 Jun 2009
- Last updated
- 24 Apr 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of 5 Azacytidine in the Treatment of Myelodysplastic Syndromes/Acute Myeloid Leukaemia With High-risk (Chromosome 7 and or Complex) Cytogenetic Abnormalities
- ClinicalTrials.gov ID
- NCT00915785
- Acronym / sponsor ID
- 05CC12
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- King's College LondonUniversity, hospital or other
- Study start
- Nov 2005
- Primary completion
- Apr 2013 (actual)
- First posted
- 8 Jun 2009 (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.
The purpose of this study is to assess the hematological and cytogenetic responses with 5 azacytidine in patients over 55 years of age with MDS/AML due to chromosome 7 abnormalities and to assess the hematological and cytogenetic response rates in patients with relapsed AML and chromosome 7 abnormality.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 5 azacytidine | Experimental | Drug: 5 azacytidine |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- Abnormalities to chromosome 7, including monosomy 7 either alone or as part of a complex clone.
- Be > 55 years of age; younger if first or subsequent relapse in patient less < 55 years but with a chromosome 7 abnormality alone or as part of a complex clone.
- Have an International Prognostic Scoring System (IPSS) score of INT 1.5 and a diagnosis of RAEB or RAEB-T per French-American-British (FAB) classification criteria or a diagnosis of Myelodysplastic CMMoL per modified FAB criteria meeting the following:
- Have a life expectancy of at least 3 months;
- Have an Eastern Cooperative Oncology Group (ECOG) Performance Status Grade of 0-2.
Key exclusion criteria (13 listed)
- Prior treatment with azacitidine.
- Diagnosis of malignant disease within the previous 12 months (excluding basal cell carcinoma with no complications).
- Diagnosis of metastatic disease.
- Previous diagnosis of hepatic tumors.
- Radiation therapy, chemotherapy, or cytotoxic therapy, given to treat conditions other than MDS/AML and administered within the previous 12 months prior to the first day of treatment (Day 1).
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 |
|---|---|---|
| 5 azacytidinePatients will receive azacytidine 75 mg/m2/day SC for 7 days every 28 days for up to 6 cycles, unless they are discontinued from the treatment. | Drug | 5 azacytidine |
Primary outcome
The rate of haematological and cytogenetic response in patients with MDS/AML with a chromosome 7 abnormality either alone or as part of a complex clone
Time frame: Blood - Weekly for first 2 cycles and then fortnightly. Bone marrow - Day 7 of first cycle and then 16 weekly thereafter or ealier if clinically indicated.
4 secondary outcomes
Time to relapse after complete remission (CR) or partial remission (PR), or disease progression (per IWG criteria), censored at death
Time frame: Blood - Weekly for first 2 cycles and then fortnightly. Bone marrow - Day 7 of first cycle and then 16 weekly thereafter or ealier if clinically indicated.
Duration of response and duration of improvement
Time frame: Blood - Weekly for first 2 cycles and then fortnightly. Bone marrow - Day 7 of first cycle and then 16 weekly thereafter or ealier if clinically indicated.
Time to AML transformation or death from any cause
Time frame: Blood - Weekly for first 2 cycles and then fortnightly. Bone marrow - Day 7 of first cycle and then 16 weekly thereafter or ealier if clinically indicated.
Additional cytogenetic markers: DNA methylation status, assessment of markers of apoptosis. Change in gene expression and single nucleotide polymorphism profiles.
Time frame: Baseline, Day 7 of the first treatment cycle and then 16 weekly (or ealier if clinically indicated).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2013.
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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.
Ghulam J Mufti, MB, DM, FRCP, FRCPath principal investigator
King's College London
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 5 azacytidine in people with Myelodysplastic Syndromes and Leukemia, Myeloid, Acute, run by King's College London at 1 site in United Kingdom.
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 21 inclusion and 13 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 24 Apr 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The rate of haematological and cytogenetic response in patients with MDS/AML with a chromosome 7 abnormality either alone or as part of a complex clone (time frame: Blood - Weekly for first 2 cycles and then fortnightly. Bone marrow - Day 7 of first cycle and then 16 weekly thereafter or ealier if clinically indicated.). 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 24 Apr 2013.