Blastic Phase Chronic Myelogenous LeukemiaPhase 1NCT00053963
FR901228 in Treating Children With Refractory or Recurrent Solid Tumors or Leukemia
A Phase 1 study testing romidepsin in people with Blastic Phase Chronic Myelogenous Leukemia and Childhood Central Nervous System Germ Cell Tumor, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
This phase I trial is studying the side effects and best dose of FR901228 in treating children with refractory or recurrent solid tumors or leukemia. Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Blastic Phase Chronic Myelogenous Leukemia, Childhood Central Nervous System Germ Cell Tumor, Childhood Choroid Plexus Tumor
- What is being tested?
- romidepsin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2002to Feb 2006
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Histologically confirmed malignancy
- Extracranial solid tumors or brain tumors*
- Diagnosis of leukemia allowed after maximum tolerated dose is determined, including any of the following:
- Acute lymphoblastic leukemia
- Acute myelogenous leukemia
- Chronic myelogenous leukemia in blast crisis
Show all 55Show fewer
- Disease must be refractory to conventional therapy or no effective conventional therapy exists
- CNS tumors resulting in neurological deficits must be stable for 2 weeks before study entry
- Performance status - Karnofsky 60-100% (over 10 years old)
- Performance status - Lansky 60-100% (10 years old and under)
- At least 8 weeks
- Absolute neutrophil count at least 1,000/mm^3 (for solid tumor patients without bone marrow involvement)
- Platelet count at least 100,000/mm^3 (for solid tumor patients without bone marrow involvement; platelet transfusion independent) OR 20,000/mm^3 (for leukemia patients; platelet transfusion allowed)
- Hemoglobin at least 8.0 g/dL (RBC transfusions allowed)
- Bilirubin no greater than 1.5 times upper limit of normal (ULN)
- ALT no greater than 5 times ULN
- Albumin at least 2 g/dL
- Glomerular filtration rate at least 70 mL/min
- Creatinine based on age as follows:
- No greater than 0.8 mg/dL (for patients 5 years of age and under)
- No greater than 1.0 mg/dL (for patients 6 to 10 years of age)
- No greater than 1.2 mg/dL (for patients 11 to 15 years of age)
- No greater than 1.5 mg/dL (for patients over 15 years of age)
- Calcium normal (with or without supplementation)
- Shortening fraction at least 27% by echocardiogram OR ejection fraction at least 50% by MUGA
- No symptomatic congestive heart failure
- No uncontrolled cardiac arrhythmia
- QTc less than 450 msec
- No evidence of dyspnea at rest
- No exercise intolerance
- Pulse oximetry greater than 94%
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception during and for 1 month after completion of study treatment
- Magnesium and potassium normal (with or without supplementation)
- No uncontrolled seizure disorder
- No uncontrolled infection
- No graft-vs-host disease
- No seizure disorder unless well controlled and not on enzyme-inducing anticonvulsants
- At least 1 week since prior growth factors
- At least 3 weeks since prior biologic therapy or immunotherapy and recovered
- At least 6 months since prior allogeneic stem cell transplantation
- No concurrent routine prophylactic growth factors
- At least 3 weeks since prior myelosuppressive chemotherapy (4 weeks for nitrosoureas) and recovered
- No prior FR901228 (depsipeptide)
- No other concurrent anticancer chemotherapy
- Concurrent dexamethasone for CNS tumors allowed if on stable dose or decreasing dose for at least 1 week before study entry
- Recovered from prior radiotherapy
- At least 2 weeks since prior local palliative radiotherapy (small port)
- At least 6 months since prior craniospinal radiotherapy or radiotherapy to at least 50% of pelvis
- At least 6 weeks since other prior substantial bone marrow radiation
- More than a 5 half-life washout period since prior and no concurrent medications associated with prolongation of QTc interval
- No concurrent enzyme-inducing anticonvulsants
- No concurrent hydrochlorothiazide
- No other concurrent investigational drugs
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Oncology Group
Arcadia, California, 91006-3776
About this study
Official title: A PHASE I STUDY OF DEPSIPEPTIDE (NSC#630176, IND# 51810) IN PEDIATRIC PATIENTS WITH REFRACTORY SOLID TUMORS AND LEUKEMIAS
This phase I trial is studying the side effects and best dose of FR901228 in treating children with refractory or recurrent solid tumors or leukemia. Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die
Read the detailed description
PRIMARY OBJECTIVES:
- Determine the maximum tolerated dose (MTD) of FR901228 (depsipeptide) in pediatric patients with refractory or recurrent solid tumors.
II. Determine the dose-limiting toxic effects of this drug in these patients. III. Determine the pharmacokinetics of this drug in these patients. IV. Assess tolerability of this drug at the solid tumor MTD in patients with refractory or recurrent leukemia.
- Determine, preliminarily, the antitumor activity of this drug in these patients.
OUTLINE: This is a dose-escalation, multicenter study.
Patients receive FR901228 (depsipeptide) IV over 4 hours on days 1, 8, and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Cohorts of 3-6 patients with solid tumors receive escalating doses of FR901228 until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.
Cohorts of 3 patients (6 patients total) with leukemia receive FR901228 as above at the MTD.
Patients are followed for survival.
- Conditions
- Blastic Phase Chronic Myelogenous Leukemia, Childhood Central Nervous System Germ Cell Tumor, Childhood Choroid Plexus Tumor, Childhood Chronic Myelogenous Leukemia, Childhood Craniopharyngioma, Childhood Grade I Meningioma
- Being tested
- romidepsin
- Medical subject headings
- Blast Crisis, Choroid Plexus Neoplasms, Astrocytoma, Spinal Cord Neoplasms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Familial ependymoma, Medulloblastoma, Optic Nerve Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
romidepsinDrug
Also called FK228, FR901228, Istodax
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Arm I | Experimental | Patients receive FR901228 (depsipeptide) IV over 4 hours on days 1, 8, and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Receives: Drug: romidepsin |
What is being measured
The primary outcome is the main question the study is designed to answer.
MTD, defined as that dose at which fewer than one-third of patients experience DLT, graded according to the NCI CTC version 2.0
Measured: Up to 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2003 (estimated)
- Study startSep 2002
- Primary completionFeb 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Blastic Phase Chronic Myelogenous LeukemiaStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Maryam Fouladi, Principal investigator, Children's Oncology Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00053963
- Sponsor's protocol ID
- NCI-2012-01803
- Other IDs
- ADVL0212, U01CA097452, CDR0000269671
- Status last verified
- Jan 2013
- First submitted
- 5 Feb 2003
- Last updated
- 16 Jan 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A PHASE I STUDY OF DEPSIPEPTIDE (NSC#630176, IND# 51810) IN PEDIATRIC PATIENTS WITH REFRACTORY SOLID TUMORS AND LEUKEMIAS
- ClinicalTrials.gov ID
- NCT00053963
- Acronym / sponsor ID
- NCI-2012-01803
- Conditions
- Blastic Phase Chronic Myelogenous Leukemia, Childhood Central Nervous System Germ Cell Tumor, Childhood Choroid Plexus Tumor, Childhood Chronic Myelogenous Leukemia, Childhood Craniopharyngioma, Childhood Grade I Meningioma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- Sep 2002
- Primary completion
- Feb 2006 (actual)
- First posted
- 6 Feb 2003 (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 phase I trial is studying the side effects and best dose of FR901228 in treating children with refractory or recurrent solid tumors or leukemia. Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die
Detailed description
PRIMARY OBJECTIVES:
- Determine the maximum tolerated dose (MTD) of FR901228 (depsipeptide) in pediatric patients with refractory or recurrent solid tumors.
II. Determine the dose-limiting toxic effects of this drug in these patients. III. Determine the pharmacokinetics of this drug in these patients. IV. Assess tolerability of this drug at the solid tumor MTD in patients with refractory or recurrent leukemia.
- Determine, preliminarily, the antitumor activity of this drug in these patients.
OUTLINE: This is a dose-escalation, multicenter study.
Patients receive FR901228 (depsipeptide) IV over 4 hours on days 1, 8, and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Cohorts of 3-6 patients with solid tumors receive escalating doses of FR901228 until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.
Cohorts of 3 patients (6 patients total) with leukemia receive FR901228 as above at the MTD.
Patients are followed for survival.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm I | Experimental | Drug: romidepsinPatients receive FR901228 (depsipeptide) IV over 4 hours on days 1, 8, and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (55 listed)
- Histologically confirmed malignancy
- Disease must be refractory to conventional therapy or no effective conventional therapy exists
- CNS tumors resulting in neurological deficits must be stable for 2 weeks before study entry
- Performance status - Karnofsky 60-100% (over 10 years old)
- Performance status - Lansky 60-100% (10 years old and under)
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| romidepsinAlso: FK228, FR901228, IstodaxGiven IV | Drug | Arm I |
Primary outcome
MTD, defined as that dose at which fewer than one-third of patients experience DLT, graded according to the NCI CTC version 2.0
Time frame: Up to 28 days
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 16 Jan 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.
Maryam Fouladi principal investigator
Children's Oncology Group
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing romidepsin in people with Blastic Phase Chronic Myelogenous Leukemia and Childhood Central Nervous System Germ Cell Tumor, run by National Cancer Institute (NCI) at 1 site in United States.
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 55 inclusion and 0 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 16 Jan 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 States. 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 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?
MTD, defined as that dose at which fewer than one-third of patients experience DLT, graded according to the NCI CTC version 2.0 (time frame: Up to 28 days). 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 16 Jan 2013.