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Atovaquone with Radiation for Pediatric Brain Tumors

This study is testing a drug called Atovaquone along with standard radiation therapy in children and young adults (ages 2 to 25) who have certain types of brain tumors, including high-grade glioma, diffuse midline glioma, diffuse intrinsic pontine glioma, and medulloblastoma. Atovaquone is an existing drug that may help radiation therapy work better by affecting cancer cells and the body's immune response. The main goal is to see if this combination treatment is safe and tolerable. Researchers are also looking at the safety of Atovaquone over a longer period for those whose tumors have returned or progressed. This study aims to enroll 18 participants, but its current status is unclear.

Study design
This is an interventional study planning to enroll 18 participants. It is testing the combination of Atovaquone and radiation therapy.
What's involved
Participants will receive Atovaquone oral suspension twice daily with meals. They will also undergo MRI-guided proton radiation therapy for 54-60 Gy in daily fractions.
Compensation
Not stated in the trial record.
Follow-up
Safety will be measured at baseline and at the end of the study, which is 10 weeks for Stratum 1 and 7 months for Stratum 2.

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NCT06624371

Atovaquone Combined With Radiation in Children With Malignant Brain Tumors

Recruiting
PHASE1Ages 2–25InterventionalTreatment
Emory University
~18 participants
Updated 2026-08-03 on ClinicalTrials.gov
What's tested:AtovaquoneRadiation Therapy

At a glance

Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Drug Limiting toxicities (DLT) in Stratum 1
Measured over Baseline, end of study (10 weeks)
+1 more outcome measured

Conditions

High-grade Glioma
Medulloblastoma
Diffuse Intrinsic Pontine Glioma
Diffuse Midline Glioma, H3 K27M-Mutant

Explore related trials

Diffuse Intrinsic Pontine GliomaDiffuse Midline GliomaMalignant GliomaMedulloblastomaH3 K27M Variant

Where it's being run

2 sites across 1 states
Georgia2

Study leadership

  • Tobey MacDonald, MD · PRINCIPAL_INVESTIGATOR · Emory University

Who to contact

Tobey MacDonald, MD
[email protected]
404-727-1447
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Stratum 1
Newly diagnosed pHGG/DMG/DIPG Patients must have histologically confirmed pediatric high-grade glioma (pHGG, WHO Grade 3 or 4) or diffuse midline glioma with altered H3K27 (DMG, WHO Grade 4). Primary pHGG or DMG spinal tumors are eligible. Diffuse intrinsic pontine glioma (DIPG) defined by MRI does not require histological confirmation.
Weight \> 10kg
Karnofsky and Lansky performance score \> 50%
Patients with stable seizures (e.g., no seizures for ≥ 7 days and not requiring escalation or addition of anti-epileptic drugs) will be eligible.
Adequate liver function defined as:
Total bilirubin ≤ 2x upper limit of normal (ULN) and
AST (SGOT) and ALT (SGPT) ≤ 225 U/L (5x the ULN). The ULN for AST and ALT will be 45 U/L.
Patients must have normal organ and marrow function as defined below:
absolute neutrophil count \> 1,000/mcL
platelets \> 100,000/mcL
hemoglobin \> 8g/dL
Total bilirubin within normal institutional limits
AST(SGOT)/ALT(SGPT) \< 5 x (\<10 x if taking steroids) the institutional upper limit of normal
creatinine within normal institutional limits for age 2 OR
creatinine clearance \> 60mL/min/1.73 m for patients with creatinine levels above institutional normal
Relapsed, progressive pHGG/DMG/DIPG and medulloblastoma (MB) or pHGG/DMG/DIPG after completion of standard radiation therapy without prior atovaquone exposure and before progression. Patients with metastatic disease are allowed for Stratum 2 only.
Measurable disease is not necessary for enrollment study.
Patients must have previously undergone standard-of-care treatment including surgery, radiation, and/or first-line adjuvant chemotherapy before the experimental treatment (atovaquone).
Patients must have recovered from the acute treatment-related toxicities (defined as \< grade 1 if not defined in eligibility criteria) of all prior chemotherapy, immunotherapy or radiotherapy prior to entering this study. There is no upper limit to the number of prior therapies that is allowed.
Age \> 2 to 25 years
Weight \> 10kg
Karnofsky and Lansky performance score \> 50%
Patients with stable seizures (e.g., no seizures for ≥ 7 days and not requiring escalation or addition of anti-epileptic drugs) will be eligible.
Patients must have normal organ and marrow function as defined above for Stratum 1
Adequate liver function is defined as:

Exclusion

Chronic systemic concurrent illness
Concurrent or history of anti-cancer therapy other than RT
Patients with metastatic tumor are excluded for Stratum 1 only.
Patients with uncontrolled seizures or seizure requiring escalation or addition of anti-epileptic drugs are excluded.
Patients must fully recover from all acute effects of prior surgical intervention.
History of allergic reactions to atovaquone or attributed to compounds of similar chemical or biological composition to atovaquone.
Symptomatic intratumoral hemorrhage, or asymptomatic intratumoral hemorrhage larger than punctate foci, at any time prior to enrollment.
Pregnant or breast-feeding women will not be entered into this study as there may be fetal risks or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method during treatment and for 3 months after stopping treatment. This should be documented in the electronic medical records as part of the consent discussion.
Concurrent illness
Patients must have recovered from all prior therapy as follows:
Patients must fully recover from all acute effects of prior surgical intervention.
History of allergic reactions to atovaquone or attributed to compounds of similar chemical or biological composition to atovaquone.
Pregnant or breast-feeding women will not be entered into this study as there may be fetal risks or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method during treatment and for 3 months after stopping treatment. This should be documented in the electronic medical records as part of the consent discussion.

What this trial measures

  • Drug Limiting toxicities (DLT) in Stratum 1Baseline, end of study (10 weeks)

    Adverse events that meet definition of DLT and tolerability. The outcome will be evaluated by descriptive statistics. Rates of symptomatic and asymptomatic radiation necrosis will also be estimated for Stratum 1 patients.

  • Drug Limiting toxicities (DLT) in Stratum 2Baseline, End of study (Month 7)

    Adverse events that meet definition of DLT and tolerability. The outcome will be evaluated by descriptive statistics. Rates of symptomatic and asymptomatic radiation necrosis will also be estimated for Stratum 2 patients.