Clinical trial · Interventional
PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
Phase I and Pharmacokinetic Trial of PTC299 in Pediatric Patients With Refractory or Recurrent CNS Tumors
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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 patients with recurrent or refractory primary central nervous system tumors.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Brain and Central Nervous System Tumors | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| VEGF inhibitor PTC299 | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (2)
- measure
- Maximum-tolerated dose
- timeFrame
- First four weeks of treatment
- measure
- Adverse events
- timeFrame
- From the first day of treatment until 30 days after the last dose
Secondary outcomes (3)
- measure
- Percentage of study participants with complete response or partial response to the study treatment
- timeFrame
- Every 8 weeks
- description
- Brain images to assess partial or complete response are performed every 8 weeks after the first dose of the study drug.
- measure
- Pharmacokinetics
- timeFrame
- Day 1 and day 28 of course 1
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 3 Years
- Maximum age
- 21 Years
Show eligibility criteria text
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 therapyReferences
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