Combination immunotherapy for newly diagnosed DIPG and DMG
A Phase I Clinical Trial of Neo-antigen Heat Shock Protein Vaccine (rHSC-DIPGVax) in Combination With Checkpoint Blockade for the Treatment of Diffuse Intrinsic Pontine Glioma (DIPG) and Diffuse Midline Glioma in Childhood
This study is testing a new combination of a cancer vaccine and other treatments to see if it can help children with newly diagnosed DIPG and DMG fight their tumors better after finishing radiation therapy.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 36 (estimated) |
| Ages | 12 Months to 18 Years |
| Sex | All |
| Sponsor | Ann & Robert H Lurie Children's Hospital of Chicago Academic / other |
| Drugs / interventions | radiation, BALSTILIMAB, ZALIFRELIMAB, immunotherapy |
| Locations | 3 sites (Orange, California and 2 other locations) |
| Trial ID | NCT04943848 on ClinicalTrials.gov |
What this trial studies
This phase I clinical trial evaluates the safety and tolerability of rHSC-DIPGVax combined with checkpoint inhibitors Balstilimab and Zalifrelimab in patients with newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) and Diffuse Midline Glioma (DMG). The trial employs a 3+3 design, starting with older children aged 5 to 18 years before including younger patients aged 12 months to 18 years. The study aims to enhance the immune response against these tumors by combining a cancer vaccine with therapies that inhibit negative regulatory pathways in the tumor microenvironment. Participants must have completed radiation therapy six to ten weeks prior to enrollment.
Who should consider this trial
Good fit: Ideal candidates are children aged 12 months to 18 years with newly diagnosed, biopsy-proven DIPG or DMG.
Not a fit: Patients with non-biopsy proven tumors or those outside the specified age range may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could provide a new treatment option for patients with DIPG and DMG, conditions that currently have limited effective therapies.
How similar studies have performed: While this specific combination is novel, similar immunotherapy approaches have shown promise in other studies targeting different cancers.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria:
* Subjects with newly diagnosed typical or non-typical, biopsy-proven DIPG or DMG are eligible for study enrollment. Biopsy is not required for subjects with radiographically typical DIPG meeting imaging criteria. Biopsy is required for DMG's and non-radiographically typical DIPG. Histone mutation must be confirmed by pathology report. Radiographically typical DIPG defined as a tumor with a pontine epicenter and diffuse involvement of more than 2/3 of the pons.
= Subjects ages \> or = to 12 months and \< or = 18 years ("Lead In", Part A, and Part B require first three patients be \> or = to 12 years of age)
* BSA \> or = 0.35m2 at the time of study enrollment
* Performance score: Karnofsky \>50% of subjects \>16 years of age and Lansky \> or = 50 for subjects \< or = 16 years of age. Subjects who are unable to walk because of paralysis but are up in a wheelchair will be considered ambulatory for the purpose of assessing the performance score.
* Must start radiation therapy within 42 days from date of diagnostic imaging. C1D1 must be within 42 days to 70 days post radiation (6-10 weeks). Patients CANNOT receive temozolomide during radiation
* Corticosteroids should be weaned as tolerated after radiation therapy with the goal of \< or = 0.5mg/kg/day for a minimum of 7 days prior to enrollment.
* Subjects must have measurable disease
Exclusion Criteria:
* Patients cannot receive temozolomide during radiation
* Disseminated disease
* Subjects who have received any cancer therapy except for radiation
* Autoimmune or immune disorders
* Active respiratory disorder or infection
* Active viral infection
Where this trial is running
Orange, California and 2 other locations
- Children's Health Orange County (CHOC) — Orange, California, United States (Recruiting)
- Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (Recruiting)
- Dana-Farber Boston Children's Cancer and Blood Disorders Center — Boston, Massachusetts, United States (Recruiting)
Study contacts
- Principal investigator: Ashley Plant-Fox, MD — Ann and Robert H. Lurie Children's Hospital
- Study coordinator: Monica Newmark, BS, RN
- Email: MNewmark@luriechildrens.org
- Phone: 312-227-4847
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.