SJDAWN: St. Jude Children's Research Hospital Phase 1 Study Evaluating Molecularly-Driven Doublet Therapies for Children and Young Adults With Recurrent Brain Tumors
Public ClinicalTrials.gov record NCT03434262. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Molecularly-Driven Doublet Therapy for All Children With Refractory or Recurrent CNS Malignant Neoplasms and Young Adults With Refractory or Recurrent SHH Medulloblastoma
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Approximately 90% of children with malignant brain tumors that have recurred or relapsed after receiving conventional therapy will die of disease. Despite this terrible and frustrating outcome, continued treatment of this population remains fundamental to improving cure rates. Studying this relapsed population will help unearth clues to why conventional therapy fails and how cancers continue to resist modern advances. Moreover, improvements in the treatment of this relapsed population will lead to improvements in upfront therapy and reduce the chance of relapse for all. Novel therapy and, more importantly, novel approaches are sorely needed. This trial proposes a new approach that evaluates rational combination therapies of novel agents based on tumor type and molecular characteristics of these diseases. The investigators hypothesize that the use of two predictably active drugs (a doublet) will increase the chance of clinical efficacy. The purpose of this trial is to perform a limited dose escalation study of multiple doublets to evaluate the safety and tolerability of these combinations followed by a small expansion cohort to detect preliminary efficacy. In addition, a more extensive and robust molecular analysis of all the participant samples will be performed as part of the trial such that we can refine the molecular classification and better inform on potential response to therapy. In this manner the tolerability of combinations can be evaluated on a small but relevant population and the chance of detecting antitumor activity is potentially increased. Furthermore, the goal of the complementary molecular characterization will be to eventually match the therapy with better predictive biomarkers. PRIMARY OBJECTIVES: * To determine the safety and tolerability and estimate the maximum tolerated dose/recommended phase 2 dose (MTD/RP2D) of combination treatment by stratum. * To characterize the pharmacokinetics of combination treatment by stratum. SECONDARY OBJECTIVE: * To estimate the rate and duration of objective response and progression free survival (PFS) by stratum.
Study identification
- NCT ID
- NCT03434262
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1
- Enrollment
- 68 participants
Conditions and interventions
Conditions
- Anaplastic Astrocytoma
- Anaplastic Ependymoma
- Anaplastic Ganglioglioma
- Anaplastic Meningioma
- Anaplastic Oligodendroglioma
- Pleomorphic Xanthoastrocytoma, Anaplastic
- Atypical Teratoid/Rhabdoid Tumor
- Brain Cancer
- Brain Tumor
- Central Nervous System Neoplasms
- Choroid Plexus Carcinoma
- CNS Embryonal Tumor With Rhabdoid Features
- Ganglioneuroblastoma of Central Nervous System
- CNS Tumor
- Embryonal Tumor of CNS
- Ependymoma
- Glioblastoma
- Glioma
- Glioma, Malignant
- Medulloblastoma
- Medulloblastoma; Unspecified Site
- Medulloepithelioma
- Neuroepithelial Tumor
- Neoplasms
- Neoplasms, Neuroepithelial
- Papillary Tumor of the Pineal Region (High-grade Only)
- Pediatric Brain Tumor
- Pineal Parenchymal Tumor of Intermediate Differentiation (High-grade Only)
- Pineoblastoma
- Primitive Neuroectodermal Tumor
- Recurrent Medulloblastoma
- Refractory Brain Tumor
- Neuroblastoma. CNS
- Glioblastoma, IDH-mutant
- Glioblastoma, IDH-wildtype
- Medulloblastoma, Group 3
- Medulloblastoma, Group 4
- Glioma, High Grade
- Neuroepithelial Tumor, High Grade
- Medulloblastoma, SHH-activated and TP53 Mutant
- Medulloblastoma, SHH-activated and TP53 Wildtype
- Medulloblastoma, Chromosome 9q Loss
- Medulloblastoma, Non-WNT Non-SHH, NOS
- Medulloblastoma, Non-WNT/Non-SHH
- Medulloblastoma, PTCH1 Mutation
- Medulloblastoma, WNT-activated
- Ependymoma, Recurrent
- Glioma, Recurrent High Grade
- Glioma, Recurrent Malignant
- Embryonal Tumor, NOS
- Glioma, Diffuse Midline, H3K27M-mutant
- Embryonal Tumor With Multilayered Rosettes (ETMR)
- Ependymoma, NOS, WHO Grade III
- Ependymoma, NOS, WHO Grade II
- Medulloblastoma, G3/G4
- Ependymoma, RELA Fusion Positive
Interventions
- Gemcitabine Drug
- ribociclib Drug
- sonidegib Drug
- trametinib Drug
- filgrastim Biological
Drug · Biological
Eligibility (public fields only)
- Age range
- 1 Year to 39 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Mar 4, 2018
- Primary completion
- Sep 29, 2022
- Completion
- May 23, 2024
- Last update posted
- May 30, 2024
2018 – 2024
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
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About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03434262, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted May 30, 2024 · Synced Sep 4, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03434262 live on ClinicalTrials.gov.