Pure Global
NCT03696355

Study of GDC-0084 in Pediatric Patients With Newly Diagnosed Diffuse Intrinsic Pontine Glioma or Diffuse Midline Gliomas

Phase I Study of GDC-0084, a Brain-Penetrant PI3 Kinase/mTOR Inhibitor, in Pediatric Patients With Newly Diagnosed Diffuse Intrinsic Pontine Glioma or Diffuse Midline Gliomas After Radiation Therapy
St. Jude Children's Research Hospital·Phase 1·ClinicalTrials.gov·drugNot yet recruiting
View on ClinicalTrials.gov →
Study focus
Disease/Condition
Brain and Central Nervous System Tumors
Drug/Device/Intervention
GDC-0084
Study type
Interventional
Intervention type
drug
Sponsor & location
Primary sponsor
St. Jude Children's Research Hospital
Location
Memphis, United States of America
Timeline & enrollment
Phase
Phase 1
Start date
Nov 19, 2018
End date
Jun 01, 2024
Enrollment
27 participants
Primary outcome

Estimate the maximum tolerated dose (MTD)/recommended phase 2 dose (RP2D) of GDC-0084 after standard-of-care radiation therapy (RT),Incidence of adverse events at least possibly associated with GDC-0084 after RT by stratum,Pharmacokinetics of GDC-0084 by stratum

Summary

Pediatric high-grade gliomas are highly aggressive and treatment options are limited. The
 purpose of this first-in-pediatrics study is to examine the safety, tolerability, and
 pharmacokinetics of GDC-0084 and to estimate its maximum tolerated dose (MTD) when
 administered to pediatric patients with diffuse intrinsic pontine glioma (DIPG) or other
 diffuse midline H3 K27M-mutant gliomas after they have received radiation therapy (RT).
 GDC-0084 is a brain-penetrant inhibitor of a growth-promoting cell signaling pathway that is
 dysregulated in the majority of diffuse midline glioma tumor cells. This study is also
 designed to enable a preliminary assessment of the antitumor activity of single-agent
 GDC-0084, in the hope of enabling rational combination therapy with systemic therapy and/or
 radiation therapy (RT) in this patient population, which is in desperate need of therapeutic
 advances.
 
 Primary Objectives
 
 1. To estimate the maximum tolerated dose (MTD) and/or the recommended phase 2 dosage
 (RP2D) of GDC-0084 in pediatric patients with newly diagnosed diffuse midline glioma,
 including diffuse intrinsic pontine glioma (DIPG)
 
 2. To define and describe the toxicities associated with administering GDC-0084 after
 radiation therapy (RT) in a pediatric population
 
 3. To characterize the pharmacokinetics of GDC-0084 in a pediatric population
 
 Secondary Objectives
 
 1. To estimate the rate and duration of radiographic response in patients with newly
 diagnosed DIPG or other diffuse midline glioma treated with RT followed by GDC-0084
 
 2. To estimate the progression-free survival (PFS) and overall survival (OS) distributions
 for patients with newly diagnosed DIPG or other diffuse midline glioma treated with RT
 followed by GDC-0084

ICD-10 classifications
Malignant neoplasm: Overlapping lesion of brain and other parts of central nervous systemMalignant neoplasm: Central nervous system, unspecifiedBenign neoplasm of brain and other parts of central nervous systemMalignant neoplasm of brainSecondary malignant neoplasm of other and unspecified parts of nervous system
Data source
Registry
ClinicalTrials.gov
Trial ID
NCT03696355
Type
Non-Device Trial
About this record

Access comprehensive clinical trial information for NCT03696355 through Pure Global AI's free database. This Phase 1 trial is sponsored by St. Jude Children's Research Hospital and is currently Not yet recruiting. The study focuses on Brain and Central Nervous System Tumors. Target enrollment is 27 participants.

This page provides complete trial specifications, intervention details, outcomes, and location information. Pure Global AI offers free access to ClinicalTrials.gov data, helping medical device and pharmaceutical companies navigate clinical research efficiently.