Decentrialz logo
  • HIPAA Compliant
  • ISO 27001 Certified
Find Trials
About UsContact
Become a Volunteer+1 877 705 1914

Status:

COMPLETED

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

Lead Sponsor:

Pediatric Brain Tumor Consortium

Collaborating Sponsors:

National Cancer Institute (NCI)

PTC Therapeutics

Conditions:

Brain and Central Nervous System Tumors

Eligibility:

All Genders

3-21 years

Phase:

PHASE1

Brief Summary

RATIONALE: PTC299 may stop the growth of tumor cells by blocking blood flow to the tumor. PURPOSE: This phase I trial is studying the side effects and the best dose of PTC299 in treating young patien...

Detailed Description

OBJECTIVES: Primary * To estimate the maximum-tolerated dose and the recommended phase II dose of VEGF inhibitor PTC299 (PTC299) in pediatric patients with recurrent or progressive primary central n...

Eligibility Criteria

Inclusion

  • 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:
  • 8 mg/dL (≤ 5 years of age)
  • 0 mg/dL (\> 5 to ≤ 10 years of age)
  • 2 mg/dL (\> 10 to ≤ 15 years of age)
  • 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

Exclusion

    Key Trial Info

    Start Date :

    November 1 2010

    Trial Type :

    INTERVENTIONAL

    Allocation :

    ACTUAL

    End Date :

    January 1 2015

    Estimated Enrollment :

    28 Patients enrolled

    Trial Details

    Trial ID

    NCT01158300

    Start Date

    November 1 2010

    End Date

    January 1 2015

    Last Update

    May 4 2015

    Active Locations (8)

    Enter a location and click search to find clinical trials sorted by distance.

    Page 1 of 2 (8 locations)

    1

    UCSF Cancer Center and Cancer Research Institute

    San Francisco, California, United States, 94143-0128

    2

    Children's National Medical Center

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

    3

    Children's Memorial Hospital - Chicago

    Chicago, Illinois, United States, 60614

    4

    Duke Comprehensive Cancer Center

    Durham, North Carolina, United States, 27710