Using Temozolomide to Treat Advanced Pheochromocytoma and Paraganglioma Before Surgery

A Study on the Safety and Effectiveness of Temozolomide for Neoadjuvant Treatment of Pheochromocytoma or Paraganglioma Patients

This study is testing if the drug temozolomide can shrink advanced pheochromocytoma or paraganglioma tumors to make surgery safer for patients who are at high risk.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 10 Years to 70 Years
• Sex: All
• Sponsor: Peking Union Medical College Hospital (Academic / other)
• Locations: 1 site (Beijing)
• Trial ID: NCT05885386 on ClinicalTrials.gov

What this trial studies

This phase II trial evaluates the effectiveness of temozolomide as a neoadjuvant therapy for patients with locally advanced or unresectable pheochromocytoma or paraganglioma. The study aims to make these tumors resectable and reduce surgical risks by administering temozolomide orally in a controlled regimen. Patients will receive the drug for a specified duration, followed by imaging assessments to determine the possibility of surgery. The trial focuses on patients who are at high surgical risk or have severe complications that prevent surgery.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 10-70 diagnosed with unresectable pheochromocytoma or paraganglioma, or those with severe complications that prevent surgery.

Not a fit: Patients with resectable tumors or those who do not meet the eligibility criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could enable surgical removal of tumors that are currently deemed unresectable, improving patient outcomes.

How similar studies have performed: While temozolomide has shown promise in treating metastatic pheochromocytoma and paraganglioma, this specific neoadjuvant approach is novel and has not been extensively tested.

Eligibility criteria

Inclusion Criteria:

* Provide written informed consent.
* Age 10-70 years old
* Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2.
* The patient is diagnosed as pheochromocytoma or paraganglioma which is unresectable with R0 surgery, or extensive and thus maybe requiring resection of important organs, or Inoperable due to heart and other complications, or with very high surgical risk.
* Estimated life expectancy longer than 6 months.
* Confirmed non-pregnancy and lactation. During the entire study period and within 6 months after the last administration, the subjects and their spouses are willing to use efficient contraceptive measures.
* Laboratory requirements:

  * Absolute granulocyte count (AGC) greater than 1.5 x 109/L;
  * Platelet count greater than 80 x 109/L; 3) Hemoglobin greater than 90g/L;
  * Serum bilirubin less than 1.5 x upper limit of normal (ULN);
  * Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) less than 2.5 x ULN; Serum creatinine less than 1.5 x ULN or creatinine clearance (CCr)≥60ml/min;
  * Doppler ultrasound assessment: left ventricular ejection fraction (LVEF) ≥ lower limit of normal value (50%).

Exclusion Criteria:

* Have other tumors.
* Patients were treated with other antitumor agents.
* Pregnant or nursing women.
* A history of allergic reactions to temozolomide or dacarbazine.
* Severe myelosuppression or abnormal coagulation.
* Severe liver and kidney insufficiency.
* Bowel obstruction or other conditions that interfere with taking medication.

Where this trial is running

Beijing

• Peking Union Medical College Hospital — Beijing, China (Recruiting)

Study contacts

• Principal investigator: Anli Tong — Peking Union Medical College Hospital
• Study coordinator: Anli Tong

Conditions Pheochromocytoma; Paraganglioma