Brain and Central Nervous System TumorsPhase 2NCT00003241
Phenylacetate in Treating Children With Recurrent or Progressive Brain Tumors
A Phase 2 study testing phenylacetate in people with Brain and Central Nervous System Tumors, run by Texas Children's Cancer Center at 6 sites in United States.
Trial summary
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase II trial to study the effectiveness of phenylacetate in treating children with recurrent or progressive brain tumors. Read more about this study
- Who is it for?
- Ages 2 to 21 years
- Conditions the study is for
- Brain and Central Nervous System Tumors
- What is being tested?
- phenylacetate
- How many people
- Not given
- Where
- 6 sitesUnited States
- Study dates
- May 1998to Sep 2004
- 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 2 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS: Histologically confirmed recurrent or progressive brain tumors including the following: Anaplastic astrocytoma and glioblastoma multiforme Brain stem glioma Medulloblastoma or primitive neuroectodermal tumors present in supratentorial or posterior fossa locations Ependymoma Low grade gliomas Other Measurable or evaluable disease by CT or MRI OR Histologically confirmed previously untreated glial tumors including: Brain stem glioma Glioblastoma multiforme Measurable disease after surgery
PATIENT CHARACTERISTICS: Age: 2 to 21 Performance status: Karnofsky 50-100% (over 10 years of age) Lansky 50-100% (under 10 years of age) Life expectancy: At least 8 weeks Hematopoietic: Absolute neutrophil count greater than 1,000/mm3 Platelet count greater than 50,000/mm3 Hemoglobin greater than 7.0 g/dL Transfusion support allowed after bone marrow transplantation or extensive radiation Hepatic: Bilirubin less than 1.5 mg/dL SGPT less than 2 times normal Renal: Creatinine no greater than 1.5 mg/dL OR Creatinine clearance greater than 60 mL/min Other: Not pregnant or nursing Fertile patients must use effective contraception during and for 6 months after study No significant systemic illness including infections No amino acidurias or organic acidemias
PRIOR CONCURRENT THERAPY: Biologic therapy: Recovered from all prior immunotherapy No concurrent prophylactic hematopoietic growth factors Chemotherapy: At least 3 weeks since prior myelosuppressive chemotherapy (6 weeks for nitrosourea) and recovered No other concurrent cancer chemotherapy Endocrine therapy: Stable or decreasing dosage of dexamethasone for intracranial pressure within 2 weeks of study entry No concurrent dexamethasone used as an antiemetic Radiotherapy: At least 8 weeks since prior radiotherapy to evaluable lesions and recovered Surgery: At least 4 months since prior radiosurgery Other: No other concurrent investigational agents
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Pediatric Oncology Branch
Bethesda, Maryland, 20892
Cleveland Clinic Cancer Center
Cleveland, Ohio, 44195
Texas Children's Cancer Center
Houston, Texas, 77030-2399
Children's Hospital Los Angeles
Los Angeles, California, 90027-0700
UCSF Cancer Center and Cancer Research Institute
San Francisco, California, 94115-0128
Children's National Medical Center
Washington D.C., District of Columbia, 20010-2970
About this study
Official title: Phase II Study of Phenylacetate in Pediatric Patients With Central Nervous System Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.
PURPOSE: Phase II trial to study the effectiveness of phenylacetate in treating children with recurrent or progressive brain tumors.
Read the detailed description
OBJECTIVES: I. Determine the efficacy of phenylacetate in terms of response rate and time to progression in children with recurrent or progressive brain tumors, or with previously untreated poor prognosis brain tumors. II. Assess the toxicity of phenylacetate in these patients treated at the maximum tolerated dose. III. Determine the correlation between serum steady state phenylacetate levels and toxicity or response in these patients.
OUTLINE: Patients are stratified by histologic type (anaplastic astrocytoma and glioblastoma multiforme vs brain stem glioma vs medulloblastoma and primitive neuroectodermal tumors vs ependymoma vs low grade glioma vs others). Patients receive phenylacetate as a continuous intravenous infusion on days 1-28. Courses of treatment are given continuously without rest. Treatment continues in the absence of disease progression or unacceptable toxicity. Patients are followed weekly.
PROJECTED ACCRUAL: A total of 9-30 patients per stratum will be accrued for this study in 2 years.
- Conditions
- Brain and Central Nervous System Tumors
- Being tested
- phenylacetate
- Keywords
- recurrent childhood brain tumor, childhood high-grade cerebral astrocytoma, untreated childhood brain stem glioma, recurrent childhood brain stem glioma, recurrent childhood supratentorial primitive neuroectodermal tumor, recurrent childhood cerebellar astrocytoma, recurrent childhood cerebral astrocytoma, recurrent childhood medulloblastoma, recurrent childhood ependymoma
- Medical subject headings
- Central Nervous System Neoplasms, Astrocytoma, Medulloblastoma, Familial ependymoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
phenylacetateDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2004 (estimated)
- Study startMay 1998
- Study completionSep 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Brain and Central Nervous System TumorsStudies 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
Sponsor and investigators
- Lead sponsor
- Texas Children's Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- Lisa Bomgaars, MD, Study chair, Texas Children's Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00003241
- Sponsor's protocol ID
- TCCC-H-5057
- Other IDs
- CDR0000066116, NCI-98-C-0107D, NCI-T96-0070
- Status last verified
- Apr 2000
- First submitted
- 1 Nov 1999
- Last updated
- 26 Jun 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Phenylacetate in Pediatric Patients With Central Nervous System Tumors
- ClinicalTrials.gov ID
- NCT00003241
- Acronym / sponsor ID
- TCCC-H-5057
- Conditions
- Brain and Central Nervous System Tumors
- Keywords
- recurrent childhood brain tumor, childhood high-grade cerebral astrocytoma, untreated childhood brain stem glioma, recurrent childhood brain stem glioma, recurrent childhood supratentorial primitive neuroectodermal tumor, recurrent childhood cerebellar astrocytoma, recurrent childhood cerebral astrocytoma, recurrent childhood medulloblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- Texas Children's Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- May 1998
- Study completion
- Sep 2004 (actual)
- First posted
- 10 Sep 2004 (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 use different ways to stop tumor cells from dividing so they stop growing or die.
PURPOSE: Phase II trial to study the effectiveness of phenylacetate in treating children with recurrent or progressive brain tumors.
Detailed description
OBJECTIVES: I. Determine the efficacy of phenylacetate in terms of response rate and time to progression in children with recurrent or progressive brain tumors, or with previously untreated poor prognosis brain tumors. II. Assess the toxicity of phenylacetate in these patients treated at the maximum tolerated dose. III. Determine the correlation between serum steady state phenylacetate levels and toxicity or response in these patients.
OUTLINE: Patients are stratified by histologic type (anaplastic astrocytoma and glioblastoma multiforme vs brain stem glioma vs medulloblastoma and primitive neuroectodermal tumors vs ependymoma vs low grade glioma vs others). Patients receive phenylacetate as a continuous intravenous infusion on days 1-28. Courses of treatment are given continuously without rest. Treatment continues in the absence of disease progression or unacceptable toxicity. Patients are followed weekly.
PROJECTED ACCRUAL: A total of 9-30 patients per stratum will be accrued for this study in 2 years.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 2 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, 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 |
|---|---|---|
| phenylacetate | Drug | – |
Primary outcome
Not reported in the source record.
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 26 Jun 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.
Lisa Bomgaars, MD study chair
Texas Children's Cancer Center
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 phenylacetate in people with Brain and Central Nervous System Tumors, run by Texas Children's Cancer Center at 6 sites 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?
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 26 Jun 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 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 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.
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 26 Jun 2013.