Conditions

Keywords

recurrent childhood malignant germ cell tumorrecurrent childhood brain stem gliomarecurrent childhood brain tumorrecurrent childhood central nervous system embryonal tumorrecurrent childhood cerebellar astrocytomarecurrent childhood cerebral astrocytomarecurrent childhood ependymomarecurrent childhood medulloblastomarecurrent childhood pineoblastomarecurrent childhood rhabdomyosarcomarecurrent childhood spinal cord neoplasmrecurrent childhood subependymal giant cell astrocytomarecurrent childhood visual pathway and hypothalamic gliomarecurrent childhood visual pathway gliomachildhood central nervous system choriocarcinomachildhood central nervous system germ cell tumorchildhood central nervous system germinomachildhood central nervous system mixed germ cell tumorchildhood central nervous system teratomachildhood central nervous system yolk sac tumorchildhood astrocytomachildhood medulloepitheliomachildhood meningiomachildhood mixed gliomachildhood oligodendrogliomachildhood pineal parenchymal tumor

Outcome Measures

Primary Outcomes (2)

Maximum-tolerated dose
First four weeks of treatment
Adverse events
From the first day of treatment until 30 days after the last dose

Secondary Outcomes (3)

Percentage of study participants with complete response or partial response to the study treatment
Every 8 weeks
Pharmacokinetics

Interventions

VEGF inhibitor PTC299Drug

This is a dose escalation study. Study participants will receive .6 or 1.2 mg/kg orally twice daily or 1.2, 1.5, or 2.0 mg/kg orally three times daily for four consecutive weeks (a course). In the absence of unacceptable toxicity or disease progression, treatment may continue for up to 12 courses (approximately one year)

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age3 Years - 21 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64)

DISEASE CHARACTERISTICS: * Histologically confirmed diagnosis of primary central nervous system (CNS) malignancy at time of diagnosis or recurrence * Histology verification not required for intrinsic brain stem tumors and optic pathway gliomas * Must have radiographic evidence of progression * Recurrent, progressive, or refractory disease to standard therapy and for which there is no known curative therapy PATIENT CHARACTERISTICS: * Karnofsky performance status (PS) 50-100% (patients \> 16 years of age) OR Lansky PS 50-100% (patients ≤ 16 years of age) * Body weight ≥ 15 kg and ≤ 100 kg * Patients with neurological deficits allowed provided they are stable for ≥ 1 week * Able to swallow capsules * ANC ≥ 1,000/μL (unsupported) * Platelet count ≥ 100,000/μL (unsupported) * Hemoglobin ≥ 8 g/dL (may be supported) * Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥ 70 mL/min/1.73 m\^2 OR serum creatinine normal based on age as follows: * 0.8 mg/dL (≤ 5 years of age) * 1.0 mg/dL (\> 5 to ≤ 10 years of age) * 1.2 mg/dL (\> 10 to ≤ 15 years of age) * 1.5 mg/dL (\> 15 years of age) * Urine protein/creatinine ratio \< 1.0 * Total bilirubin ≤ 1.5 times upper limit of normal (ULN) * ALT and AST ≤ 2.5 times ULN * Albumin ≥ 2.5 g/dL * PT and activated PTT ≤ 1.2 times ULN * Not pregnant or nursing * Negative pregnancy test * Fertile patients must use effective contraception * No clinically significant unrelated systemic illness that would compromise the patient's ability to tolerate protocol therapy, or would likely interfere with the study procedures or results, including any of the following: * Serious infections including ongoing systemic bacterial, fungal, or viral infection * Significant cardiac, pulmonary, hepatic, or other organ dysfunction * Willing and able to comply with schedule visits, drug administration plan, laboratory tests, including pharmacokinetic and pharmacodynamic assessments, or other study procedures * No known coagulopathy or bleeding diathesis * No known history of drug-induced liver injury * No CNS, pulmonary, gastrointestinal, or urinary bleeding within the past month * No uncontrolled systemic hypertension (systolic BP or diastolic BP \> 95% percentile for age) * No alcohol or drug addiction * Able to tolerate periodic MRI scans and gadolinium contrast PRIOR CONCURRENT THERAPY: * See Disease Characteristics * Recovered from the acute toxic of all prior therapy (excluding alopecia and neurotoxicity) * At least 3 weeks since prior myelosuppressive anticancer chemotherapy (6 weeks for nitrosourea) * At least 14 days since prior investigational or biological agent * At least 3 half-lives since prior biological agents that have a prolonged half-life * At least 3 half-lives since prior monoclonal antibody * At least 2 weeks since prior local palliative radiotherapy * At least 6 weeks since prior total-body irradiation, craniospinal radiotherapy, or radiotherapy to ≥ 50% of the pelvis * At least 90 days since prior allogeneic bone marrow transplantation * No active graft-versus-host disease * Concurrent dexamethasone or other corticosteroids allowed provided dose is stable for ≥ 7 days * At least 1 week since prior colony-forming growth factors (e.g., filgrastim, sargramostim, erythropoietin) * At least 14 days since long-acting colony-forming growth factor formulations (e.g., pegfilgrastim) * More than 4 weeks since prior major surgical procedures * More than 2 weeks since prior intermediate surgical procedures * More than 7 days since minor surgical procedures * No other concurrent anticancer or investigational drug therapy

Sponsors & Collaborators

Pediatric Brain Tumor Consortium
Research networklead
National Cancer Institute (NCI)
NIHcollaborator
PTC Therapeutics

Study Sites (8)

UCSF Cancer Center and Cancer Research Institute

San Francisco, California, 94143-0128, United States

Children's National Medical Center

Washington D.C., District of Columbia, 20010-2970, United States

Children's Memorial Hospital - Chicago

Chicago, Illinois, 60614, United States

Duke Comprehensive Cancer Center

Durham, North Carolina, 27710, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104-4318, United States

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

St. Jude Children's Research Hospital

MeSH Terms

Conditions

Central Nervous System NeoplasmsAstrocytomaFamilial ependymomaMedulloblastomaSpinal Cord NeoplasmsOptic Nerve GliomaMeningiomaOligodendroglioma

Interventions

emvododstat

Condition Hierarchy (Ancestors)

Nervous System NeoplasmsNeoplasms by SiteNeoplasmsNervous System DiseasesGlioma

Study Officials

Roger J. Packer, MD
Children's National Research Institute
PRINCIPAL INVESTIGATOR

PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (3)

Interventions

Eligibility Criteria

Sponsors & Collaborators

Study Sites (8)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (3)

Interventions

Eligibility Criteria

Sponsors & Collaborators

Study Sites (8)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

Source record

Locations