Active — Not RecruitingPhase 1

A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma

United States64 participantsStarted 2018-08-01

Plain-language summary

The goal of this study is to evaluate the safety of the study drug PTC596 (Unesbulin) taken in combination with radiotherapy (RT) when given to pediatric patients newly diagnosed with High-Grade Glioma (HGG) including diffuse intrinsic pontine glioma (DIPG). The main aims of the study are to: * Find the safe dose of the study drug PTC596that can be given without causing serious side effects. * Find out the amount of drug that enters blood (in all patients) and tumor (in patients who receive drug prior to a planned surgery for removal of their brain tumor) During the first cycle (6-7weeks), patients will receive drug orally twice a week in combination with daily RT. During subsequent cycles (4 weeks each), they will receive only the study drug orally twice a week. Funding Source - FDA OOPD

Who can participate

Age range

12 Months – 21 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Newly-diagnosed DIPG who are amenable to undergo biopsy at the recommendation of their treating physician
. Newly-diagnosed HGG for whom a second surgical resection is warranted for further debulking or to achieve a near-total or gross total resection after initial diagnosis has been made but prior to start of therapy.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Establish MTD and RP2D of PTC596

Timeframe: At the end of Cycle 1 (42-49 days)

Number of participants with treatment-related adverse events as assessed by CTCAE v5.0

Timeframe: From Day 1 of treatment through 30 days following end of protocol treatment

Maximum Plasma Concentration [Cmax] of PTC596 (A, B, C, D)

Timeframe: Days1 through 29

Tumor Concentration of PTC596 (B)

Timeframe: Day 4 of surgical cycle

Protein levels of BMI1 in tumor

Timeframe: Day 4 of surgical cycle

Trial details

NCT IDNCT03605550

SponsorNationwide Children's Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-03-01

View on ClinicalTrials.gov More High Grade Glioma trials

Plain-language summary

Who can participate

Age range

12 Months – 21 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Newly-diagnosed DIPG who are amenable to undergo biopsy at the recommendation of their treating physician
. Newly-diagnosed HGG for whom a second surgical resection is warranted for further debulking or to achieve a near-total or gross total resection after initial diagnosis has been made but prior to start of therapy.

What they're measuring

Establish MTD and RP2D of PTC596

Timeframe: At the end of Cycle 1 (42-49 days)

Number of participants with treatment-related adverse events as assessed by CTCAE v5.0

Timeframe: From Day 1 of treatment through 30 days following end of protocol treatment

Maximum Plasma Concentration [Cmax] of PTC596 (A, B, C, D)

Timeframe: Days1 through 29

Tumor Concentration of PTC596 (B)

Timeframe: Day 4 of surgical cycle

Protein levels of BMI1 in tumor

Timeframe: Day 4 of surgical cycle