Gemcitabine in Newly-Diagnosed Diffuse Midline Glioma

NCT04051047 | Withdrawn Early Phase 1 FDA Drug

• 2 other identifiers: UMCC 2018.159HUM00135182
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The primary aim of this study is to determine the presence of gemcitabine in childhood diffuse midline gliomas (DMG) (previously classified as diffuse intrinsic pontine glioma \[DIPG\]) after systemic treatment with the drug.

Conditions

Glial Tumor of Brain; Diffuse Intrinsic Pontine Glioma; Glioma

Keywords

Glioma

Outcome Measures

Primary Outcomes (1)

• Number of participants with detectable intratumoral gemcitabine after systemic treatment with the drug

  Gemcitabine will be considered to be detectable in a participant's tumor sample if gemcitabine or one of its metabolites (difluorodeoxyuridine \[dFdU\] or gemcitabine triphosphate \[dFdCTP\]) is detected at a measurable level in any of the participant's tumor samples. Undetectable gemcitabine will be defined as gemcitabine, dFdU, and dFdCTP levels below the detectable limits for all of an individual participant's samples, in the setting of detectable gemcitabine, dFdU, or dFdCTP in the plasma and/or cerebrospinal fluid (CSF) for that patient, and the pathologic review showing viable tumor tissue.

  Day 1

Secondary Outcomes (1)

• Concentration of gemcitabine in patient tumor sample

  Day 1

Study Arms (1)

Gemcitabine

EXPERIMENTAL

Gemcitabine will be given at 2100 mg/m2 IV over 30 minutes within 4 hours of planned surgical procedure. The surgical procedure is standard of care.

Drug: Gemcitabine; Procedure: Tumor biopsy and blood draw

Interventions

Gemcitabine DRUG

Participants receive a one time IV dose of gemcitabine prior to having standard of care surgery.

Also known as: Gemzar

Gemcitabine

Tumor biopsy and blood draw PROCEDURE

As part of standard of care, all participants will undergo tumor biopsy and collection of peripheral blood.

Gemcitabine

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age greater than or equal to 3 years and less than 18 years at the time of enrollment.
• Patients must meet both of the first two conditions, OR the third:
• Clinical findings consistent with a presumed new diagnosis of diffuse midline glioma (DMG) in the opinion of the treating neuro-oncologist, AND
• Brain MRI findings consistent with a new diagnosis of DMG based on multidisciplinary consensus after review of imaging
• OR, recurrent DMG requiring tumor resection or biopsy
• Adequate bone marrow, liver, renal and metabolic function (per protocol)
• Adequate coagulation defined as Prothrombin time (PT) and activated partial thromboplastin time (aPTT) ≤ upper limit of normal (ULN ) for age
• Patients must meet one of the following performance scores:
• ECOG performance status scores of 0, 1, or 2; 8
• Karnofsky score of ≥ 60 for patients \> 16 years of age; or
• Lansky score of ≥ 60 for patients ≤ 16 years of age
• DMG biopsy/resection is planned for the clinical care of the patient independent of study participation by the treating pediatric neurosurgeon and neuro-oncologist.
• Informed consent and assent obtained as appropriate.

You may not qualify if:

• Pregnant or breastfeeding patients are not eligible due to teratogenic effects seen in animal/human studies
• Patients who have received any tumor-directed therapy prior to biopsy are not eligible. Concurrent treatment with corticosteroids is allowed.
• Any tumor-specific or clinical features that make surgical intervention unsafe in the opinion of the treating neurosurgeon.
• Patients with personal or family history of bleeding disorders are not eligible.
• Patients with uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements are not eligible.
• Patients with known hypersensitivity to gemcitabine

Sponsors & Collaborators

• University of Michigan Rogel Cancer Center: lead
• University of Colorado, Denver: collaborator

Study Sites (1)

The University of Michigan Rogel Cancer Center

Ann Arbor, Michigan, 48109, United States

Location

MeSH Terms

Conditions

Diffuse Intrinsic Pontine Glioma; Glioma

Interventions

Gemcitabine; Blood Specimen Collection

Condition Hierarchy (Ancestors)

Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Brain Stem Neoplasms; Infratentorial Neoplasms; Brain Neoplasms; Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Heterocyclic Compounds; Deoxycytidine; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Study Officials

• Carl Koschmann, M.D.

  University of Michigan

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2019
• First Posted: August 9, 2019
• Study Start: November 30, 2019
• Primary Completion: December 1, 2021
• Study Completion: December 1, 2021
• Last Updated: November 27, 2019

Record last verified: 2019-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)