Actively Recruiting

Phase 1
Age: 2Years - 25Years
All Genders
ID06624371

Atovaquone Combined With Radiation in Children With Malignant Brain Tumors

Led by Emory University · Updated on 2025-07-22

18

Participants Needed

2

Research Sites

N/A

Total Duration

On this page

Sponsors

E

Emory University

Lead Sponsor

M

Morningside Foundation/Peach Bowl LegACy Fund

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and tolerability of atovaquone combined with standard radiation therapy in children and young adults aged 2 to 25 years who have newly diagnosed pediatric high-grade glioma, diffuse midline glioma, or diffuse intrinsic pontine glioma. The study also aims to assess longer-term use of atovaquone in patients with relapsed or progressed tumors, including medulloblastoma, following radiation therapy. Atovaquone is an approved antiparasitic drug being studied for its potential to improve cancer treatment by inhibiting a protein involved in tumor survival and immune suppression. Participants receive oral atovaquone doses adjusted by age and weight alongside radiation therapy. Newly diagnosed patients begin atovaquone treatment for about two weeks before starting approximately six weeks of combined atovaquone and MRI-guided proton radiation therapy. Those with relapsed or progressive disease or who have completed radiation previously receive atovaquone for up to six months if tolerated, beginning 2 to 4 weeks after relapse, progression, or radiation completion. Throughout the study, participants undergo assessments for drug-related toxicities at baseline and study end, with additional monitoring of tumor progression and survival over one year. Safety evaluations include performance status, liver and organ function tests, and seizure stability. The research team measures progression-free survival, overall survival, and tumor response rates to understand atovaquone’s tolerability and potential impact on these aggressive brain tumors.

CONDITIONS

Brief Title

Atovaquone Combined With Radiation in Children With Malignant Brain Tumors

Who Can Participate

Age: 2Years - 25Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Newly diagnosed pediatric high-grade glioma or diffuse midline glioma patients with confirmed diagnosis or DIPG defined by MRI
  • Relapsed or progressive pediatric high-grade glioma, diffuse midline glioma, diffuse intrinsic pontine glioma, or medulloblastoma after radiation therapy
  • Age between 2 and 25 years
  • Weight greater than 10 kg
  • Karnofsky or Lansky performance score above 50%
  • Stable seizures without recent medication changes
  • Adequate liver, organ, and marrow function as defined by study parameters
  • For relapsed patients, recovery from prior treatments and timing since last therapies met
Not Eligible

You will not qualify if you...

  • Chronic systemic illness or concurrent illness
  • Receipt of anti-cancer therapy other than radiation for newly diagnosed patients
  • Presence of metastatic tumor in newly diagnosed patients
  • Uncontrolled seizures or need for increased anti-epileptic drugs
  • Incomplete recovery from prior surgery or acute treatment effects
  • History of allergic reactions to atovaquone or similar compounds
  • Symptomatic or large intratumoral hemorrhage
  • Pregnant or breastfeeding females; reproductive-age participants must use effective contraception during and 3 months after treatment
  • Insufficient recovery time from prior therapies including chemotherapy, immunotherapy, radiation, stem cell transplant as specified for relapsed patients

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Run-in Period

Duration - 2 weeks (+/- 7 days)

Participants newly diagnosed with pediatric brain tumors receive Atovaquone alone for 2 weeks to assess tolerance before combined treatment.

Visits as needed for dosing and safety monitoring

Treatment

Duration - Approximately 6 weeks

Participants receive combined Atovaquone and MRI-guided proton radiation therapy to treat malignant brain tumors.

Daily radiation treatments and regular visits for medication administration and monitoring

Treatment

Duration - Up to 6 months

Participants with relapsed or progressive tumors receive Atovaquone for up to 6 months in combination with or following radiation therapy, depending on their treatment history.

Regular visits for medication dosing and monitoring

Follow-up

Duration - Up to 12 months

Participants are monitored for drug safety, tumor progression, and survival outcomes after completion of treatment.

Periodic follow-up visits for assessments

Trial Site Locations

Total: 2 locations

1

Arthur M Blank Hospital

Atlanta, Georgia, United States, 30329

Actively Recruiting

2

Children's Healthcare of Atlanta: Scottish Rite

Atlanta, Georgia, United States, 30342

Actively Recruiting

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Research Team

T

Tobey MacDonald, MD

A

Amber Kaminski

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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