Proton Beam Therapy for Chordoma

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: M.D. Anderson Cancer Center

Disqualifiers: Previous irradiation, Metastatic disease, Concurrent malignancy, others

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

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 35

Duration: 7 weeks

19 Participants Needed

Trial Summary

What is the purpose of this trial?

This study is evaluating whether proton beam therapy is effective in the treatment of skull base chordoma.

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.

What data supports the effectiveness of the treatment Proton Beam Therapy for Chordoma?

Research shows that proton beam therapy (PBT) is effective for treating chordomas, a type of bone cancer, by improving local control and survival rates. Studies on different types of chordomas, such as clival, sacral, and skull-base, have demonstrated positive outcomes with this treatment. ^{1 2 3 4 5}

Is Proton Beam Therapy safe for humans?

Proton Beam Therapy (PBT) is generally considered safe and is used for treating various cancers, including those in children, with the potential to reduce side effects compared to traditional radiation. However, more research is needed to fully understand its safety across different conditions. ^{6 7 8 9 10}

How is proton beam therapy different from other treatments for chordoma?

Proton beam therapy (PBT) is unique because it precisely targets tumors with minimal damage to surrounding healthy tissues, making it especially beneficial for chordomas located near critical structures like nerves and blood vessels. This precision helps improve tumor control and survival rates compared to traditional radiation therapies. ^{1 2 3 5 11}

Research Team

David R. Grosshans

Principal Investigator

M.D. Anderson Cancer Center

Eligibility Criteria

This trial is for individuals with a confirmed diagnosis of chordoma at the base of the skull, who have had surgery to remove as much of the tumor as possible. They should be able to perform daily activities without significant assistance (Karnofsky Performance status ≥60) and must not have had previous radiation therapy in that area or any other cancer (except skin cancer) in the past three years.

Inclusion Criteria

I've had a detailed brain scan within the last 3 months.

Signed informed consent

My cancer is a confirmed chordoma located at the base of my skull.

Exclusion Criteria

I have not had any cancer other than non-melanoma skin cancer in the last 3 years.

I have had radiation therapy on the base of my skull.

My cancer has spread to other parts of my body.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive proton beam therapy once a day for about 35 treatments over 7 weeks, possibly combined with photon beam therapy

7 weeks

35 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment with annual follow-up till disease progression or death

1 year

Annual visits (in-person or virtual)

Treatment Details

Interventions

• Photon Beam Therapy
• Proton Beam Therapy

Trial Overview The study is examining whether proton beam therapy alone or combined with photon beam radiation therapy can effectively treat skull base chordoma. The research will also assess how safe these treatments are for patients.

Participant Groups

Group I: Photon Beam Therapy Experimental Treatment 2 Interventions

Proton beam therapy combined with photon radiation therapy where combination improves final dose distribution.

Group II: 70 Gray (Gy) Proton Beam Therapy Experimental Treatment 1 Intervention

Participants treated to 70 cobalt Gray equivalent (CGE) only (the standard treatment).

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

Approved in United States as Proton Beam Therapy for:

• Various cancers including prostate, breast, lung, liver, and head and neck cancers

Approved in European Union as Proton Therapy for:

• Various cancers including ocular melanoma, chordomas, chondrosarcomas, and certain pediatric cancers

Approved in Japan as Proton Beam Therapy for:

• Various cancers including prostate, liver, and ocular melanoma

Approved in Canada as Proton Therapy for:

• Various cancers including ocular melanoma and certain pediatric cancers

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

Findings from Research

A study of 48 children with malignant brain tumors treated with proton beam therapy (PBT) showed that acute toxicities were generally low-grade and manageable, with fatigue, alopecia, and dermatitis being the most common side effects.

The research indicates that PBT is well tolerated in pediatric patients, and acute toxicities can be effectively managed with supportive care, suggesting a favorable safety profile for this treatment approach.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Acute toxicity of proton beam radiation for pediatric central nervous system malignancies. Suneja, G., Poorvu, PD., Hill-Kayser, C., et al.[2013]

Proton beam therapy (PBT) usage in the US increased from 0.4% in 2004 to 1.2% in 2018, particularly among patients with cancers recommended for PBT by the American Society of Radiation Oncology, indicating growing acceptance and application of this treatment.

While PBT use for prostate cancer temporarily decreased from 2011 to 2014, it rebounded by 2018, suggesting fluctuations in treatment preferences or insurance coverage that may influence patient access to this advanced therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment of Proton Beam Therapy Use Among Patients With Newly Diagnosed Cancer in the US, 2004-2018. Nogueira, LM., Jemal, A., Yabroff, KR., et al.[2022]

Proton beam therapy (PBT) is feasible for treating infants with central nervous system tumors, showing promising short-term outcomes with a 3-year overall survival rate of 76.5% among 51 infants studied.

However, the treatment is associated with significant logistical challenges and potential for higher-grade toxicities, including late effects like endocrinopathy and hearing loss, highlighting the need for careful monitoring and longer follow-up to assess long-term impacts.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Feasibility of Proton Beam Therapy for Infants with Brain Tumours: Experiences from the Prospective KiProReg Registry Study. Jazmati, D., Steinmeier, T., Ahamd Khalil, D., et al.[2021]

Proton beam therapy (PBT) shows potential benefits for treating certain cancers, such as pediatric CNS tumors and large ocular melanomas, but current evidence does not support its superiority over traditional photon-based therapies for most other cancer types.

The American Society of Radiation Oncology (ASTRO) recommends further robust clinical trials to better define the appropriate use of PBT, as existing data is insufficient to endorse its routine application in many malignancies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An evidence based review of proton beam therapy: the report of ASTRO's emerging technology committee. Allen, AM., Pawlicki, T., Dong, L., et al.[2018]

References

1. England pubmed.ncbi.nlm.nih.gov

Hyperfractionated high-dose proton beam radiotherapy for clival chordomas after surgical removal. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Outcomes of Patients With Primary Sacral Chordoma Treated With Definitive Proton Beam Therapy. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Clinical Outcomes Following Dose-Escalated Proton Therapy for Skull-Base Chordoma. [2022]

4. Korea (South) pubmed.ncbi.nlm.nih.gov

Clinical outcome of proton therapy for patients with chordomas. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

Extracranial chordoma: Outcome in patients treated with function-preserving surgery followed by spot-scanning proton beam irradiation. [2019]

6. United States pubmed.ncbi.nlm.nih.gov

Patterns of Care in Proton Radiation Therapy for Pediatric Central Nervous System Malignancies. [2018]

7. United States pubmed.ncbi.nlm.nih.gov

Acute toxicity of proton beam radiation for pediatric central nervous system malignancies. [2013]

8. United States pubmed.ncbi.nlm.nih.gov

Assessment of Proton Beam Therapy Use Among Patients With Newly Diagnosed Cancer in the US, 2004-2018. [2022]

9. England pubmed.ncbi.nlm.nih.gov

Feasibility of Proton Beam Therapy for Infants with Brain Tumours: Experiences from the Prospective KiProReg Registry Study. [2021]

10. Ireland pubmed.ncbi.nlm.nih.gov

An evidence based review of proton beam therapy: the report of ASTRO's emerging technology committee. [2018]

11. Germany pubmed.ncbi.nlm.nih.gov

Proton beam therapy for skull base chordomas: a systematic review of tumor control rates and survival rates. [2022]