Proton Therapy for Craniopharyngioma

Overseen By Thomas E. Merchant, DO, PhD

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: St. Jude Children's Research Hospital

Disqualifiers: Prior radiation, Intracystic P-32, Pregnancy

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment for craniopharyngioma, a rare brain tumor, using intensity-modulated proton therapy. This advanced radiation therapy aims to treat the tumor effectively while reducing side effects compared to traditional radiation. Participants will either undergo surgery followed by proton therapy if needed or receive proton therapy directly if surgery isn't possible. The trial seeks children and young adults diagnosed with craniopharyngioma who have not had previous radiation treatments. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to benefit from cutting-edge therapy.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that intensity-modulated proton therapy is generally safe and well-tolerated for treating craniopharyngioma, especially in children and teenagers. Studies have found that it effectively controls the tumor with manageable short-term and long-term side effects. Compared to traditional photon therapy, proton therapy might improve survival rates and cause fewer harmful effects. However, some side effects can appear years after treatment. Considering these potential risks and benefits is important when evaluating participation in a clinical trial. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about proton therapy for craniopharyngioma because it offers a more precise way to target tumors. Unlike traditional radiation, which can affect surrounding healthy tissue, intensity-modulated proton therapy delivers radiation directly to the tumor site, minimizing damage to nearby areas. This precision is especially important in brain tumors, where sparing healthy tissue can lead to better outcomes and fewer side effects. Additionally, for patients who cannot have surgery, proton therapy provides a non-invasive alternative that still effectively targets the tumor.

What evidence suggests that intensity-modulated proton therapy could be an effective treatment for craniopharyngioma?

Research has shown that proton therapy, particularly the advanced form known as intensity-modulated proton therapy (IMPT), effectively treats craniopharyngioma, a type of brain tumor. In this trial, participants whose tumors cannot be surgically removed will undergo 6 weeks of proton therapy. Studies have found that this method effectively controls the tumor and causes fewer side effects than traditional x-ray radiation. IMPT is also linked to better survival rates and fewer harmful effects. Patients generally tolerate it well, making it a promising treatment option for this condition. ^{1 2 3 6 7}

Who Is on the Research Team?

Thomas Merchant

Principal Investigator

St. Jude Children's Research Hospital

Are You a Good Fit for This Trial?

This trial is for individuals aged 0-21 diagnosed with craniopharyngioma, a rare brain tumor. It's open to those who have not had complete surgical removal of the tumor or require observation after radical surgery. Pregnant females and patients previously treated with certain intracystic therapies or radiation are excluded.

Inclusion Criteria

I was diagnosed with my condition before turning 22.

My diagnosis of craniopharyngioma was confirmed through tests or surgery.

Exclusion Criteria

I have had treatment with P-32 or bleomycin directly into a cyst.

I have had radiation therapy in parts before.

Pregnant females are excluded due to the teratogenic or abortifacient effects of radiation.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Surgery

Participants undergo surgery to remove the tumor if eligible. If the entire tumor is not removed, they proceed to proton therapy.

Immediate

Proton Therapy

Participants receive 6 weeks of intensity-modulated proton therapy if the tumor is not completely resected.

6 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, with outcome data collected for comparison to other participant groups.

5 years

What Are the Treatments Tested in This Trial?

Interventions

• Intensity-Modulated Proton Therapy
• Surgery

Trial Overview The study tests intensity-modulated proton therapy (IMPT) for treating craniopharyngioma when surgery doesn't remove all of the tumor, aiming to see if IMPT can be more effective and cause fewer side effects than traditional radiation treatments.

How Is the Trial Designed?

Group I: Tumor-Surgery Active Control 2 Interventions

Participants who are eligible to undergo surgery to remove the tumor will proceed to surgery. If all tumor is removed, they will be followed over 5 years for outcome comparison to the other participant groups. If the entire tumor is not removed by surgery, participants will receive 6 weeks of proton therapy. They will then be followed for 5 years to collect outcome data for comparison to the other participant groups.

Group II: Tumor-No Surgery Active Control 1 Intervention

Participants whose tumor cannot be resected through surgery will receive 6 weeks of proton therapy. They will then be followed over 5 years for outcome comparison to the other participant groups.

Intensity-Modulated Proton Therapy is already approved in United States, European Union, Canada, Japan for the following indications:

Approved in United States as IMPT for:

• Head and neck cancers
• Squamous cell carcinoma
• Nasopharyngeal cancer
• Oropharyngeal cancer
• Thyroid cancer
• Salivary gland cancer

Approved in European Union as IMPT for:

• Head and neck cancers
• Squamous cell carcinoma
• Nasopharyngeal cancer
• Oropharyngeal cancer
• Thyroid cancer
• Salivary gland cancer

Approved in Canada as IMPT for:

• Head and neck cancers
• Squamous cell carcinoma
• Nasopharyngeal cancer
• Oropharyngeal cancer
• Thyroid cancer
• Salivary gland cancer

Approved in Japan as IMPT for:

• Head and neck cancers
• Squamous cell carcinoma
• Nasopharyngeal cancer
• Oropharyngeal cancer
• Thyroid cancer
• Salivary gland cancer

Who Is Running the Clinical Trial?

St. Jude Children's Research Hospital

Lead Sponsor

Trials

451

Recruited

5,326,000+

Published Research Related to This Trial

Intensity Modulated Proton Therapy (IMPT) provided better target coverage for recurrent nasopharyngeal carcinoma (rNPC) compared to Intensity Modulated Radiotherapy (IMRT), achieving higher coverage percentages for both the gross tumor volume and clinical target volume.

While IMPT showed advantages in sparing critical neurological organs, it also resulted in higher maximum doses to the internal carotid artery and nasopharyngeal mucosa, indicating a need for careful monitoring of potential complications when using this treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dosimetric comparison of intensity modulated radiotherapy and intensity modulated proton therapy in the treatment of recurrent nasopharyngeal carcinoma. Hung, HM., Chan, OCM., Mak, CH., et al.[2022]

Proton radiation therapy, particularly intensity-modulated proton therapy (IMPT), offers better dose localization compared to conventional photon radiation therapy, potentially reducing damage to surrounding healthy tissues in patients with head and neck and skull base tumors.

Initial clinical experiences suggest that IMPT may lead to fewer acute and long-term toxicities, and ongoing multi-institutional trials aim to further establish its effectiveness in treating these complex tumors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Proton radiation therapy for head and neck cancer. Chan, AW., Liebsch, NJ.[2018]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11566716/

Proton beam therapy for craniopharyngioma - PubMed Central PBT for craniopharyngioma, especially in children and adolescents, has shown impressive local control and acceptable acute and late toxicities.

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0167814020302334

Pencil beam scanning proton therapy for the treatment of ... Surgery and proton therapy results in excellent disease control for pediatric craniopharyngioma. ... Intensity Modulated Proton Therapy (IMPT). 2021, ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12474669/

Efficacy and safety of proton therapy and photon therapy for ... Compared with photon therapy, proton therapy may be associated with improved survival outcomes and reduced incidence of toxic effects in CP ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S2331518023000252

Review Articles Craniopharyngioma and Proton Therapy Proton therapy is an effective and well-tolerated radiation therapy modality for patients with craniopharyngioma.

5. frontiersin.org frontiersin.org/journals/oncology/articles/10.3389/fonc.2023.1180993/full

Outcome after proton beam therapy versus photon-based ... Proton beam therapy (PBT) is being increas16ingly used to treat residual craniopharyngioma (CP) after hypothalamus-sparing surgery.

6. cureus.com cureus.com/articles/391357-therapeutic-outcomes-and-toxicity-mitigation-of-proton-beam-therapy-in-pediatric-neuro-oncology-a-systematic-review

Therapeutic Outcomes and Toxicity Mitigation of Proton ... Imaging Changes in Pediatric Intracranial Ependymoma Patients Treated With Proton Beam Radiation Therapy Compared to Intensity Modulated ...

7. floridaproton.org floridaproton.org/about-us/research-portfolio

Proton Therapy Research Portfolio A comprehensive look at proton therapy for prostate cancer: The physics, the history, more than 30 years of data and the current research.