NCT03778996

SM-88 Maintenance Therapy for Advanced Ewing's Sarcoma and as Salvage Therapy for Sarcoma

Phase 2 Clinical Trial to Evaluate Efficacy and Safety of SM-88 Used With Methoxsalen, Phenytoin, and Sirolimus (MPS) as Maintenance Therapy Following Standard Treatments for Ewing's Sarcoma or as Salvage Therapy for Advanced Sarcomas

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 31 (actual)
• Sponsor: Sarcoma Oncology Research Center, LLC · Academic / Other
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The primary objective is to evaluate the efficacy of SM-88, a combination metabolic cancer treatment, in two study cohorts: * Clinically advanced Ewing's Sarcoma patients who have not progressed at the conclusion of systemic treatment * Clinically advanced sarcoma patients in the salvage treatment setting Up to 24 efficacy evaluable patients (up to 12 per cohort) will be enrolled. Study patients will receive oral SM-88, with scheduled safety and efficacy evaluations.

Detailed description

This prospective, open-label, two stage, pilot phase 2 trial evaluates the efficacy and safety of SM-88 in two cohorts of patients: 1) as maintenance therapy following standard primary or palliative treatments for Ewing's sarcoma patients with high risk of relapse or disease progression; and 2) as salvage therapy for patients with clinically advanced sarcomas. The primary objective is to evaluate the efficacy of SM-88, a combination metabolic cancer treatment, measured as positive efficacy events, including overall response, maintaining stable disease for ≥ 3 months, or progression free survival at least 1.5 times longer than the last prior line of treatment. Eligible patients will receive daily oral treatment with SM-88, which consists of D,L-alpha-metyrosine, used with methoxsalen, phenytoin, and sirolimus in continuous treatment cycles of 28 days. Treatment will continue until: 1) Symptomatic, clinical progression with radiographic progressive disease; 2) 48 weeks after documented complete response; or 3) evidence of unacceptable toxicity, or other decision to discontinue treatment

Conditions

• Sarcoma, Ewing
• Sarcoma

Interventions

Type	Name	Description
DRUG	Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS)	Daily oral combination therapy for cancer

Timeline

• Start date: 2020-01-03
• Primary completion: 2027-12-19
• Completion: 2028-03-31
• First posted: 2018-12-19
• Last updated: 2025-02-24

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study