Open-Label, Cohort-Sequential Dose-Escalation and Dose-Confirmation Phase 1/2 Clinical Trial to Evaluate the Safety and Efficacy of Domatinostat in Combination With Sirolimus in Adolescents and Adults With Relapsed, Refractory Sarcoma and Osteosarcoma
Status: Recruiting
Location: See all (4) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
This is a multicenter, open-label, Phase 1/2 study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and antitumor activity of domatinostat in combination with sirolimus in adolescents and adults with relapsed or refractory sarcoma and osteosarcoma.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Maximum Age: 40
Healthy Volunteers: f
View:
• Age ≥12 and ≤40 years.
• Histologically confirmed relapsed or refractory sarcoma (Phase 1) or osteosarcoma (Phase 2).
• At least one prior standard systemic therapy regimen.
• Not candidates for available therapies known to provide survival benefit.
• Karnofsky/Lansky performance score ≥60.
• Life expectancy ≥16 weeks.
• Adequate organ function: ANC ≥1,000/mcL, Platelets ≥100,000/uL, Hemoglobin ≥8 g/dL, Adequate hepatic and renal function.
• Ability to swallow tablets.
• Negative pregnancy test for females of childbearing potential.
• Agreement to use effective contraception.
• Ability to provide informed consent/assent.
Locations
United States
Florida
Moffitt Cancer Center
RECRUITING
Tampa
Missouri
Washington University in St. Louis
NOT_YET_RECRUITING
St Louis
New York
Montefiore Medical Center
NOT_YET_RECRUITING
The Bronx
Ohio
Nationwide Children's Hospital
RECRUITING
Columbus
Contact Information
Primary
Jessica Crimella
Jessica.Crimella@moffitt.org
813-745-6250
Time Frame
Start Date:2026-07-15
Estimated Completion Date:2031-08
Participants
Target number of participants:74
Treatments
Experimental: Phase 1 Dose Escalation Cohort
Participants with relapsed or refractory sarcoma will receive domatinostat plus sirolimus. Domatinostat dose levels will be escalated to determine the MTD and/or RP2D.
Experimental: Phase 2 Arm A (Surgical Complete Remission Osteosarcoma)
Patients with relapsed/refractory osteosarcoma who achieve complete surgical remission before enrollment will receive domatinostat plus sirolimus at the RP2D.
Experimental: Phase 2 Arm B (Measurable Osteosarcoma)
Patients with unresectable measurable relapsed/refractory osteosarcoma will receive domatinostat plus sirolimus at the RP2D.