Phase I dose-finding study for melatonin in pediatric oncology patients with relapsed solid tumors

Area of Science:

• Pediatric Oncology
• Pharmacology
• Clinical Trials

Background:

• Melatonin is a natural product used for sleep disturbances.
• Preliminary adult studies suggest melatonin may reduce anorexia and weight loss in advanced cancer.
• High-dose melatonin has not been previously studied in pediatric oncology patients.

Purpose of the Study:

• To determine the safety and tolerability of escalating doses of melatonin in pediatric oncology patients with relapsed solid tumors.
• To establish a safe and effective dose range for melatonin in this population.

Main Methods:

• A multicenter, single-arm, phase I dose-escalation study.
• Melatonin was administered for 8 weeks at three dose levels: 0.075 mg/kg (max 5 mg), 0.15 mg/kg (max 10 mg), and 0.3 mg/kg (max 20 mg).
• Utilized a 3+3 design to assess safety and tolerability.

Main Results:

• Melatonin was well tolerated across all dose levels with no dose-limiting toxicities.
• The only grade 3/4 toxicities observed were myelosuppression, attributed to concurrent chemotherapy.
• Seven out of nine patients experienced weight gain, with a median increase of 3.4%.

Conclusions:

• Melatonin is well-tolerated at a dose of 0.3 mg/kg (maximum 20 mg) in pediatric oncology patients.
• This study supports further investigation of high-dose melatonin for managing symptoms in pediatric cancer patients.