LeukemiaPhase 3NCT00411281
Low-Dose Cytarabine in Treating Infants With Down Syndrome and Transient Myeloproliferative Disorder
A Phase 3 study testing cytarabine and observation in people with Leukemia, run by Children's Oncology Group.
Trial summary
RATIONALE: Drugs used in chemotherapy, such as cytarabine, work in different ways to stop the growth of abnormal cells, either by killing the cells or by stopping them from dividing. Giving low-doses of cytarabine may be an effective treatment for Down syndrome and transient myeloproliferative disorder. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Leukemia
- What is being tested?
- cytarabine and observation
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Mar 2006to Nov 2007
- Phase
- Phase 3
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
DISEASE CHARACTERISTICS:
- Diagnosis of transient myeloproliferative disorder (TMD)
- Diagnosis of Down syndrome or Down syndrome mosaicism (confirmed by karyotype analysis within the past 3 weeks) AND 1 of the following:
- Nonerythroid and nonlymphoid blasts (any amount) in the peripheral blood with verification of a second sample
- Trisomy 21-positive leukemic blasts documented by biopsy of any organ (including > 5% nonerythroid/nonlymphoid blasts documented by bone marrow aspirate or biopsy)
- Immunophenotype characterization required
- High-, intermediate-, or low-risk TMD, as defined by the following:
- High-risk TMD, meeting 1 of the following criteria:
- Life-threatening cardio-respiratory compromise due to complications of TMD (e.g., organomegaly or effusions)
- Life-threatening cardio-respiratory compromise is defined as cardiovascular grade 4 edema, grade 4 pericardial effusions, or grade 4 pleural effusions
- Hyperleukocytosis, defined as a WBC > 100,000/mm³
- Any degree of hepatomegaly (palpable on physical exam) combined with life-threatening hepatic dysfunction
- Life-threatening hepatic dysfunction is defined as grade 4 disseminated intravascular coagulation, grade 4 ascites, grade 4 bilirubin (> 10.0 times upper limit of normal [ULN]), or grade 4 AST or ALT (> 20.0 times ULN)
- Intermediate-risk TMD, meeting all of the following criteria:
- Hepatomegaly (palpable on physical exam) combined with non life-threatening hepatic dysfunction (i.e., grade 1-3 hepatic dysfunction [AST or ALT ≤ 2.5 times ULN] and/or a total or direct bilirubin ≤ 1.5 times ULN)
- No evidence of life-threatening cardiovascular, respiratory, or hepatic compromise due to complications of TMD
- Low-risk TMD, meeting all of the following criteria:
- No palpable hepatomegaly on physical exam OR hepatomegaly is present without hepatic dysfunction (i.e., grade 0 hepatic dysfunction)
- No evidence of life-threatening cardiovascular, respiratory, or hepatic compromise due to complications of TMD
PATIENT CHARACTERISTICS:
- See Disease Characteristics
- No biliary atresia by hepatic ultrasound for patients with bilirubin 3.0-10.0 times ULN
PRIOR CONCURRENT THERAPY:
- No prior antileukemic therapy (except for leukapheresis or exchange transfusion)
About this study
Official title: Treatment of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)
RATIONALE: Drugs used in chemotherapy, such as cytarabine, work in different ways to stop the growth of abnormal cells, either by killing the cells or by stopping them from dividing. Giving low-doses of cytarabine may be an effective treatment for Down syndrome and transient myeloproliferative disorder. Sometimes the disease may not need treatment until it progresses. In this case, observation may be sufficient.
PURPOSE: This phase III trial is studying low-dose cytarabine to see how well it works in treating infants with Down syndrome and transient myeloproliferative disorder.
Read the detailed description
OBJECTIVES:
Primary
- Determine whether very low-dose cytarabine can improve event-free survival (EFS) rates in infants with high-risk transient myeloproliferative disorder (TMD), using high-risk TMD patients from clinical trial COG-A2971 for historic comparison, and in infants with intermediate-risk TMD, using intermediate-risk TMD patients from clinical trial COG-A2971 for historic comparison.
- Maintain the current high overall EFS rate in low-risk TMD patients.
Secondary
- Assess the toxicity of this regimen in these patients.
OUTLINE: This is a nonrandomized, multicenter, crossover study. Patients are stratified according to disease risk (high or intermediate vs low).
- Group I (patients with high- or intermediate-risk transient myeloproliferative disorder [TMD]): Patients receive very low-dose cytarabine subcutaneously twice daily on days 1-7. Treatment repeats every 14 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving stable disease or complete or hepatic clinical remission undergo observation.
- Group II (patients with low-risk TMD): Patients are observed. If symptoms of intermediate- or high-risk disease develop, patients may crossover to group I.
After completion of study treatment, patients are followed periodically for 10 years.
PROJECTED ACCRUAL: A total of 180 patients will be accrued for this study.
- Conditions
- Leukemia
- Being tested
- cytarabine, observation
- Keywords
- acute myeloid leukemia/transient myeloproliferative disorder
- Medical subject headings
- Leukemia, Leukemia, Myeloid, Acute, Myeloproliferative Syndrome, Transient
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cytarabineDrug
Given subcutaneously
observationProcedure or surgery
No intervention
| Study group | Role | What they receive |
|---|---|---|
| Group I | Experimental | Patients receive very low-dose cytarabine subcutaneously twice daily on days 1-7. Treatment repeats every 14 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving stable disease or complete or hepatic clinical remission undergo observation. Receives: Drug: cytarabine |
| Group II | Other | Patients are observed. If symptoms of intermediate- or high-risk disease develop, patients may crossover to group I. Receives: Procedure: observation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free survival
Secondary outcomes (4)
Overall survival
Disease-related mortality
Percentage of patients experiencing grade 3-4 toxicity
Incidence of subsequent leukemia in patients for whom transient myeloproliferative disorder is resolved
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2006 (estimated)
- Study startMar 2006
- Primary completionNov 2007
- Study completionNov 2007
Why it stopped: Per Group Chair: This study will not move forward.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by Children's Oncology GroupThe 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 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Oncology Group
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- April D. Sorrell, MD, Study chair, Rutgers Cancer Institute of New JerseyJeffrey Taub, MD, Study chair, Children's Hospital of Michigan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00411281
- Sponsor's protocol ID
- AAML0532
- Other IDs
- COG-AAML0532, CDR0000518352
- Status last verified
- Aug 2013
- First submitted
- 11 Dec 2006
- Last updated
- 30 Sep 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)
- ClinicalTrials.gov ID
- NCT00411281
- Acronym / sponsor ID
- AAML0532
- Conditions
- Leukemia
- Keywords
- acute myeloid leukemia/transient myeloproliferative disorder
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Per Group Chair: This study will not move forward.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Children's Oncology GroupResearch network
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Mar 2006
- Primary completion
- Nov 2007 (actual)
- Study completion
- Nov 2007 (actual)
- First posted
- 13 Dec 2006 (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.
RATIONALE: Drugs used in chemotherapy, such as cytarabine, work in different ways to stop the growth of abnormal cells, either by killing the cells or by stopping them from dividing. Giving low-doses of cytarabine may be an effective treatment for Down syndrome and transient myeloproliferative disorder. Sometimes the disease may not need treatment until it progresses. In this case, observation may be sufficient.
PURPOSE: This phase III trial is studying low-dose cytarabine to see how well it works in treating infants with Down syndrome and transient myeloproliferative disorder.
Detailed description
OBJECTIVES:
Primary
- Determine whether very low-dose cytarabine can improve event-free survival (EFS) rates in infants with high-risk transient myeloproliferative disorder (TMD), using high-risk TMD patients from clinical trial COG-A2971 for historic comparison, and in infants with intermediate-risk TMD, using intermediate-risk TMD patients from clinical trial COG-A2971 for historic comparison.
- Maintain the current high overall EFS rate in low-risk TMD patients.
Secondary
- Assess the toxicity of this regimen in these patients.
OUTLINE: This is a nonrandomized, multicenter, crossover study. Patients are stratified according to disease risk (high or intermediate vs low).
- Group I (patients with high- or intermediate-risk transient myeloproliferative disorder [TMD]): Patients receive very low-dose cytarabine subcutaneously twice daily on days 1-7. Treatment repeats every 14 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving stable disease or complete or hepatic clinical remission undergo observation.
- Group II (patients with low-risk TMD): Patients are observed. If symptoms of intermediate- or high-risk disease develop, patients may crossover to group I.
After completion of study treatment, patients are followed periodically for 10 years.
PROJECTED ACCRUAL: A total of 180 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group I | Experimental | Drug: cytarabinePatients receive very low-dose cytarabine subcutaneously twice daily on days 1-7. Treatment repeats every 14 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving stable disease or complete or hepatic clinical remission undergo observation. |
| Group II | Other | Procedure: observationPatients are observed. If symptoms of intermediate- or high-risk disease develop, patients may crossover to group I. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| cytarabineGiven subcutaneously | Drug | Group I |
| observationNo intervention | Procedure or surgery | Group II |
Primary outcome
Event-free survival
4 secondary outcomes
Overall survival
Disease-related mortality
Percentage of patients experiencing grade 3-4 toxicity
Incidence of subsequent leukemia in patients for whom transient myeloproliferative disorder is resolved
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2015.
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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.
April D. Sorrell, MD study chair
Rutgers Cancer Institute of New Jersey
Jeffrey Taub, MD study chair
Children's Hospital of Michigan
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing cytarabine and observation in people with Leukemia, run by Children's Oncology Group.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 30 Sep 2015. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 was withdrawn before enrolling participants, so there are no results.
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?
Event-free survival. 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 30 Sep 2015.