Myelodysplastic SyndromePhase 1POE07-01NCT00852709
Phase I Dose-Escalation Trial of Clofarabine Followed by Escalating Doses of Fractionated Cyclophosphamide in Children With Relapsed or Refractory Acute Leukemias
A Phase 1 study testing Clofarabine and Cyclophosphamide in people with Myelodysplastic Syndrome and Acute Myeloid Leukemia, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 7 sites in 2 countries.
Trial summary
This is a Phase I study designed to determine the MTD and assess the toxicity associated with clofarabine followed by fractionated cyclophosphamide in patients > 1 year of age or < 21 years of age with relapsed or refractory acute leukemias. There will be 25 to 35 patients enrolled. Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Myelodysplastic Syndrome, Acute Myeloid Leukemia, Myeloproliferative Disorders
- What is being tested?
- Clofarabine and Cyclophosphamide
- How many people
- 35 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 1 Sep 2007to 20 Nov 2009
- Phase
- Phase 1
- 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 1 year to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 for adolescent/young adult patients.
- Lansky Performance Index > 50 for pediatric patients less than age 10 years.
- Laboratory values obtained < 7 days prior to receiving study treatment:
- Total bilirubin < 1.5 mg/dL unless elevated due to hemolysis. The conjugated serum bilirubin prior to study entry must be within the normal range.
- Aspartate transaminase (AST) and alanine transaminase (ALT) < 2.5 × upper limit of normal (ULN)
- Serum creatinine < 1.0 mg/dL in adolescent/young adults (patients 12 to 17 years of age). For pediatric patients with serum creatinine above the ULN, creatinine clearance > 90 ml/min/1.73m2 calculated using the Schwartz formula may be enrolled. Collected creatinine clearance may be substituted.
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- Patients 18 years and older (Adult population): Serum creatinine <1.0 mg/dL; if serum creatinine >1.0 mg/dL, then the estimated glomerular filtration rate (GFR) must be >60 mL/min/1.73 m2
- Cardiac function must be normal per the institution normal as measured by echocardiogram (ECHO) within 7 days.
- Patients should have no evidence of myositis as detected by abnormal serum creatine kinase and/or myoglobin.
Who cannot join
- No chemotherapy, radiation, or major surgery within 2 weeks prior to first dose of study drug except for 5-azacytidine, thalidomide, hydroxyurea, imatinib (Gleevec), and interferon which must be discontinued at least 3 days before study entry and the patient should have recovered from the toxic side effects of such therapy. In the instance of progressive disease, anti-leukemia therapy may have been administered within the 2-week period as long but the subject should have recovered from the toxic effects of that therapy. Also, intrathecal therapy may be administered within the 2-week period for subjects with CNS disease.
- Patients who have had an allogeneic or autologous hematopoietic stem cell transplant.
- Patients must have discontinued all growth factors, except Procrit (epoetin), at least 1 week before study.
- Patients with known HIV positive status or AIDS.
- Patients with known active Hepatitis B, Hepatitis C or cirrhosis.
- History of severe coronary artery disease, including myocardial infarction within the previous 3 months, arrhythmias other than atrial flutter or fibrillation requiring medication, or uncontrolled congestive heart failure.
- Patients with active uncontrolled infection, fever of infection, or evidence for progressive disease by CT scans of the lungs, sinuses, or abdomen. Patients who are on antimicrobial therapy and stable, CT scans must have been stable for 4 weeks, may be enrolled but there must be no evidence of an active infection. Patients with fever due to leukemia may be enrolled.
- Pregnant or lactating patients. Female patients of childbearing potential must have a negative serum pregnancy test within 14 days before study entry.
Trial locations and contacts
7 sites in 2 countries.
7 sites in 2 countries
United States6 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
University of Colorado Health Sciences Center and The Children's Hospital
Aurora, Colorado, 80045
Pediatrix Hematology/Oncology University of Florida College of Medicine
Gainesville, Florida, 32610-0296
M.D. Anderson Comprehensive Cancer Center
Houston, Texas, 77030
Vanderbilt Children's Hospital
Nashville, Tennessee, 37232-6310
Phoenix Children's Hospital
Phoenix, Arizona, 85016-7710
Canada1 site
Southern Alberta Children's Cancer Program
Calgary, Alberta, T3B6A8
About this study
This is a Phase I study designed to determine the MTD and assess the toxicity associated with clofarabine followed by fractionated cyclophosphamide in patients > 1 year of age or < 21 years of age with relapsed or refractory acute leukemias. There will be 25 to 35 patients enrolled. Cohorts of 3 to 6 patients each will receive escalated doses of clofarabine followed by fractionated cyclophosphamide until the MTD is reached. There will be no intra-patient dose escalation. Single-agent cyclophosphamide will be administered by 2-hour IVI on Day 0 of cycle 1. On Days 1, 2, and 3 and Days 8, 9, and 10 clofarabine will be administered by IVI 2 hours before each dose of cyclophosphamide (see the treatment schema below). A cycle is defined as 28 days.
- Conditions
- Myelodysplastic Syndrome, Acute Myeloid Leukemia, Myeloproliferative Disorders, Acute Lymphocytic Leukemia, Acute Promyelocytic Leukemia, Acute Leukemia
- Being tested
- Clofarabine, Cyclophosphamide
- Keywords
- Myelodysplastic syndrome that has transformed to AML (AML/MDS), Treatment-related AML, AML evolving from myeloproliferative disorders (MPD), Acute lymphocytic leukemia (ALL), Acute promyelocytic leukemia (APL) refractory to arsenic therapy or retinoic acid therapy, Relapsed and/or refractory acute leukemia with progressive disease since last therapy, Chronic myelogenous leukemia (CML) in accelerated phase or blast crisis refractory to imatinib, High-risk MPD including myelofibrosis, chronic myelomonocytic leukemia (CMML, and relapsed or refractory juvenile myelomonocytic leukemia (JMML).
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute, Myeloproliferative Disorders, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Promyelocytic, Acute, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Primary Myelofibrosis, Leukemia, Myelomonocytic, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ClofarabineDrug
On Days 1, 2, and 3 and Days 8, 9, and 10 clofarabine will be administered by IVI 2 hours before each dose of cyclophosphamide
CyclophosphamideDrug
Single-agent cyclophosphamide will be administered by 2-hour IVI on Day 0 of cycle 1. On Days 1, 2, and 3 and Days 8, 9, and 10 clofarabine will be administered by IVI 2 hours before each dose of cyclophosphamide
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the feasibility, tolerability, toxicities, and MTD of clofarabine followed by fractionated cyclophosphamide in pediatric patients with relapsed or refractory acute leukemias.
Measured: 1 cycle
Secondary outcomes (1)
To obtain preliminary descriptive data of the biologic and pharmacodynamic effects of clofarabine followed by fractionated cyclophosphamide on marrow and circulating leukemic blasts in pediatric patients with relapsed or refractory acute leukemias.
Measured: 1 cycle
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2009 (estimated)
- Study start1 Sep 2007
- Primary completion20 Nov 2009
- Study completion20 Nov 2009
Why it stopped: Lack of accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
- Sponsor type
- University, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00852709
- Sponsor's protocol ID
- J0715
- Other IDs
- POE07-01
- Acronym
- POE07-01
- Status last verified
- Feb 2018
- First submitted
- 26 Feb 2009
- Last updated
- 15 Feb 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Dose-Escalation Trial of Clofarabine Followed by Escalating Doses of Fractionated Cyclophosphamide in Children With Relapsed or Refractory Acute Leukemias
- ClinicalTrials.gov ID
- NCT00852709
- Acronym / sponsor ID
- POE07-01 / J0715
- Conditions
- Myelodysplastic Syndrome, Acute Myeloid Leukemia, Myeloproliferative Disorders, Acute Lymphocytic Leukemia, Acute Promyelocytic Leukemia, Acute Leukemia
- Keywords
- Myelodysplastic syndrome that has transformed to AML (AML/MDS), Treatment-related AML, AML evolving from myeloproliferative disorders (MPD), Acute lymphocytic leukemia (ALL), Acute promyelocytic leukemia (APL) refractory to arsenic therapy or retinoic acid therapy, Relapsed and/or refractory acute leukemia with progressive disease since last therapy, Chronic myelogenous leukemia (CML) in accelerated phase or blast crisis refractory to imatinib, High-risk MPD including myelofibrosis, chronic myelomonocytic leukemia (CMML, and relapsed or refractory juvenile myelomonocytic leukemia (JMML).
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of accrual
- Enrolment
- 35 participants (estimated)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsUniversity, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Study start
- 1 Sep 2007 (actual)
- Primary completion
- 20 Nov 2009 (actual)
- Study completion
- 20 Nov 2009 (actual)
- First posted
- 27 Feb 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.
This is a Phase I study designed to determine the MTD and assess the toxicity associated with clofarabine followed by fractionated cyclophosphamide in patients > 1 year of age or < 21 years of age with relapsed or refractory acute leukemias. There will be 25 to 35 patients enrolled. Cohorts of 3 to 6 patients each will receive escalated doses of clofarabine followed by fractionated cyclophosphamide until the MTD is reached. There will be no intra-patient dose escalation. Single-agent cyclophosphamide will be administered by 2-hour IVI on Day 0 of cycle 1. On Days 1, 2, and 3 and Days 8, 9, and 10 clofarabine will be administered by IVI 2 hours before each dose of cyclophosphamide (see the treatment schema below). A cycle is defined as 28 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 for adolescent/young adult patients.
- Lansky Performance Index > 50 for pediatric patients less than age 10 years.
- Laboratory values obtained < 7 days prior to receiving study treatment:
- Total bilirubin < 1.5 mg/dL unless elevated due to hemolysis. The conjugated serum bilirubin prior to study entry must be within the normal range.
- Aspartate transaminase (AST) and alanine transaminase (ALT) < 2.5 × upper limit of normal (ULN)
Key exclusion criteria (8 listed)
- No chemotherapy, radiation, or major surgery within 2 weeks prior to first dose of study drug except for 5-azacytidine, thalidomide, hydroxyurea, imatinib (Gleevec), and interferon which must be discontinued at least 3 days before study entry and the patient should have recovered from the toxic side effects of such therapy. In the instance of progressive disease, anti-leukemia therapy may have been administered within the 2-week period as long but the subject should have recovered from the toxic effects of that therapy. Also, intrathecal therapy may be administered within the 2-week period for subjects with CNS disease.
- Patients who have had an allogeneic or autologous hematopoietic stem cell transplant.
- Patients must have discontinued all growth factors, except Procrit (epoetin), at least 1 week before study.
- Patients with known HIV positive status or AIDS.
- Patients with known active Hepatitis B, Hepatitis C or cirrhosis.
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 |
|---|---|---|
| ClofarabineOn Days 1, 2, and 3 and Days 8, 9, and 10 clofarabine will be administered by IVI 2 hours before each dose of cyclophosphamide | Drug | – |
| CyclophosphamideSingle-agent cyclophosphamide will be administered by 2-hour IVI on Day 0 of cycle 1. On Days 1, 2, and 3 and Days 8, 9, and 10 clofarabine will be administered by IVI 2 hours before each dose of cyclophosphamide | Drug | – |
Primary outcome
To determine the feasibility, tolerability, toxicities, and MTD of clofarabine followed by fractionated cyclophosphamide in pediatric patients with relapsed or refractory acute leukemias.
Time frame: 1 cycle
1 secondary outcome
To obtain preliminary descriptive data of the biologic and pharmacodynamic effects of clofarabine followed by fractionated cyclophosphamide on marrow and circulating leukemic blasts in pediatric patients with relapsed or refractory acute leukemias.
Time frame: 1 cycle
Results and safety
The study was stopped early (reason given: Lack of accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Feb 2018.
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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.
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Clofarabine and Cyclophosphamide in people with Myelodysplastic Syndrome and Acute Myeloid Leukemia, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 7 sites in 2 countries.
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 9 inclusion and 8 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: stopped early, as of the sponsor's last update on 15 Feb 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Canada. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 stopped early (reason given: Lack of accrual). 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?
To determine the feasibility, tolerability, toxicities, and MTD of clofarabine followed by fractionated cyclophosphamide in pediatric patients with relapsed or refractory acute leukemias. (time frame: 1 cycle). 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 15 Feb 2018; AzTrials last refreshed its copy on 26 Oct 2026.