PDGFR Inhibitor Crenolanib in Children/Young Adults With Diffuse Intrinsic Pontine Glioma or Recu… (NCT01393912) | Clinical Trial Compass
CompletedPhase 1
PDGFR Inhibitor Crenolanib in Children/Young Adults With Diffuse Intrinsic Pontine Glioma or Recurrent High-Grade Glioma
United States55 participantsStarted 2011-07
Plain-language summary
This is a Phase I clinical trial evaluating crenolanib (CP-868,596), an inhibitor of Platelet Derived Growth Factor Receptor (PDGFR)-kinase in children and young adults with newly diagnosed diffuse intrinsic pontine glioma (DIPG) (Stratum A) or in recurrent, progressive or refractory High Grade Glioma (HGG) including DIPG (Stratum B). This study drug targets the most commonly amplified region of genome found in DIPG and pediatric high grade glioma (HGG) which encodes for the PDGF receptor kinase. An oral investigational agent crenolanib will be administered daily during and after local radiation therapy (RT) in Diffuse Intrinsic Pontine Glioma DIPG (Stratum A), or daily for children with recurrent/refractory HGG (Stratum B).
Who can participate
Age range
18 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:
* Age must be ≥ 18 months and \< or equal to 21 years
* Body surface Area (BSA) ≥ 0.55 m\^2
* Lansky (for research participants ≤ 16 years) or Karnofsky (for research participants \> 16 years) performance score ≥ 40 at the time of study enrollment
* Adequate organ function at the time of study enrollment as follows:
* Bone marrow: Absolute neutrophil count (ANC) ≥ 1,000/μL, platelet count ≥ 75,000/μL (transfusion independent), hemoglobin concentration ≥ 8g/dL (may be transfused)
* Renal: Normal serum creatinine concentration based on age as shown below or glomerular filtration rate (GFR) \> 70 ml/min/1.73m\^2
* Age (years): \< or equal to 5 and the maximum serum creatinine (mg/dL) is 0.8;
* 5 \< age \< or equal to 10 and the maximum serum creatinine (mg/dL) is 1.0;
* 10\< age \< or equal to 15 and the maximum serum creatinine (mg/dL) is 1.2;
* \>15 and the maximum serum creatinine (mg/dL) is 1.5;
* Hepatic: Total bilirubin concentration \< or equal to 1.5 times the institutional upper limit of normal for age; SGPT \< or equal to 3 times the institutional upper limit of normal
* Pancreatic: Serum amylase \< or equal to 3 times the institutional upper limit of normal for age; lipase \< or equal to 3 times the institutional upper limit of normal
* Female research participants of childbearing age must not be pregnant as confirmed by a serum or urine pregnancy test within 1 week of start of treatment. Participants must not be breast-f…
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
1
Estimate the maximum tolerated dose (MTD) of crenolanib in pediatric research participants with newly diagnosed DIPG
Timeframe: 2.5 years
2
Estimate the MTD of crenolanib in children and young adults with recurrent/refractory HGG including DIPG
Timeframe: 2.5years
3
Characterize the pharmacokinetics of crenolanib in pediatric patients and relate drug disposition to toxicity