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PTC596 + Radiotherapy for Brain Tumor

Phase 1
Waitlist Available
Research Sponsored by Nationwide Children's Hospital
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients with newly-diagnosed non-brainstem high-grade glioma (HGG) are eligible
Patients eligible for the surgical stratum include patients with newly-diagnosed DIPG who are amenable to undergo biopsy at the recommendation of their treating physician
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from date of treatment until date of death due to any cause or date of last follow-up, assessed up to 60 months
Awards & highlights

Study Summary

This trial is investigating the safety and efficacy of PTC596 in children and young adults with brain tumors. PTC596 is a novel drug that is being studied for its ability to improve the efficacy of radiation therapy. The trial is divided into two parts: the first part is designed to assess the maximum tolerated dose of PTC596 that can be given with radiation therapy, and the second part is a surgical study to assess the amount of the drug that is taken up by the tumor and surrounding tissue. The goals of the trial are to learn more about the safety and efficacy of PTC596 in this population, as well as to assess the quality of

Who is the study for?
This trial is for children and young adults aged between 1 and 21 with newly diagnosed high-grade gliomas, including DIPG. They must be able to swallow pills, have not had cancer treatment before, and their organs must function well. Those with certain other health issues or who can't follow the study rules are excluded.Check my eligibility
What is being tested?
The safety of PTC596 (Unesbulin) combined with radiotherapy is being tested in pediatric patients with brain tumors like DIPG. The study aims to find a safe drug dose that doesn't cause serious side effects and to measure how much drug gets into the blood and tumor.See study design
What are the potential side effects?
Specific side effects aren't listed here, but generally, participants may experience reactions related to the medication or radiotherapy such as nausea, fatigue, skin changes from radiation, or potential bone marrow suppression due to the investigational drug.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been recently diagnosed with a high-grade glioma that is not in my brainstem.
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I have a new diagnosis of DIPG and my doctor recommends a biopsy.
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I have a high-grade glioma and need more surgery to remove as much of it as possible before starting treatment.
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I am between 12 and 21 years old.
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My brain tumor is confirmed high-grade glioma or diffuse astrocytoma through biopsy.
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I am between 1 and 21 years old.
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My brain tumor is a type known as high-grade glioma.
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I can swallow whole capsules.
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I have not received any cancer treatment before.
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I can swallow pills.
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I have been newly diagnosed with a brain tumor known as DIPG without needing a biopsy.
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My heart, lungs, kidneys, liver, bones, and nervous system are all functioning well.
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I am mostly able to care for myself, regardless of my age.
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I do not have widespread DIPG or HGG cancer.
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I have been recently diagnosed with a high-grade glioma that is not in my brainstem.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from date of treatment until date of death due to any cause or date of last follow-up, assessed up to 60 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and from date of treatment until date of death due to any cause or date of last follow-up, assessed up to 60 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Establish MTD and RP2D of PTC596
Maximum Plasma Concentration [Cmax] of PTC596 (A, B, C, D)
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0
+2 more
Secondary outcome measures
Evaluate Overall survival
Evaluate Progression Free survival

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment (PTC596)Experimental Treatment2 Interventions
PTC596 administered orally twice weekly (M/Th or T/F schedule) concomitantly with RT for 6 -7 weeks. Each subsequent cycle is defined as 28 days. Post RT patients will continue to receive PTC596 twice weekly for up to 26 cycles at RP2D of 200mg/m2 with a maximum dose capped at 400mg for patients with BSA ≥2.0
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
PTC596
2016
Completed Phase 1
~40
Radiotherapy
2017
Completed Phase 3
~2610

Find a Location

Who is running the clinical trial?

PTC TherapeuticsIndustry Sponsor
74 Previous Clinical Trials
6,166 Total Patients Enrolled
Nationwide Children's HospitalLead Sponsor
337 Previous Clinical Trials
5,220,090 Total Patients Enrolled
5 Trials studying Diffuse Intrinsic Pontine Glioma
750 Patients Enrolled for Diffuse Intrinsic Pontine Glioma
Margot Lazow, MDStudy ChairNationwide Children's Hospital
2 Previous Clinical Trials
550 Total Patients Enrolled
2 Trials studying Diffuse Intrinsic Pontine Glioma
550 Patients Enrolled for Diffuse Intrinsic Pontine Glioma

Media Library

PTC596 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT03605550 — Phase 1
Diffuse Intrinsic Pontine Glioma Research Study Groups: Treatment (PTC596)
Diffuse Intrinsic Pontine Glioma Clinical Trial 2023: PTC596 Highlights & Side Effects. Trial Name: NCT03605550 — Phase 1
PTC596 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03605550 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

To what extent has the population size for this medical test grown?

"Affirmative. The information on clinicaltrials.gov indicates that this medical experiment is actively searching for participants, which it posted and edited in August 2018 and September 2022 respectively. To complete the trial, 64 individuals are sought-after from 10 distinct sites."

Answered by AI

Is there an opportunity to take part in this medical experiment?

"This medical trial is open to 64 individuals between the ages of one year and 21 years old who have been diagnosed with diffuse intrinsic pontine glioma. Additionally, those taking part must meet specific criteria: Age wise, they must be no younger than 12 months and no older than 21; Neurologically-speaking, patients ought to be able to ingest oral medications; Performance Level should stand at Karnofsky ≥ 50 for people over 16 years of age or Lansky ≥ 50 if under that age bracket (those unable to walk because of paralysis will still qualify as ambulatory); Diagnosis-wise it needs to either be a newly-diagn"

Answered by AI

May individuals who are under the age of seventy partake in this research?

"The terms of participation for this medical trial stipulate that the minimum participant age must be 12 Months and the maximum is 21."

Answered by AI

Are there available vacancies for potential participants in this experiment?

"Indeed, the clinicaltrials.gov website confirms that recruitment for this trial is still ongoing since its inception on August 1st 2018. It requires 64 patients to be sourced from 10 medical centres."

Answered by AI

Has PTC596 acquired the endorsement of the Food and Drug Administration?

"The team at Power rated PTC596 a 1 on their safety scale, as this study is only in Phase 1. Thus far, there has been limited data collected to support the drug's efficacy and safety."

Answered by AI

How many healthcare locations are currently undertaking this experiment?

"Currently, this experiment is being conducted in 10 clinical centres situated across the United States. These include Columbus, Chicago and Washington D.C., with other sites scattered around the nation. To reduce travel strain for participants, it can be beneficial to select a trial site located as close to you as possible."

Answered by AI

Who else is applying?

What state do they live in?
South Dakota
How old are they?
< 18
What site did they apply to?
Dana-Farber Cancer Institute
What portion of applicants met pre-screening criteria?
Met criteria
~10 spots leftby Mar 2025