NCT07725380 · H. Lee Moffitt Cancer Center and Research Institute
Domatinostat With Sirolimus for Relapsed, Refractory Sarcoma and Osteosarcoma
What this study is about
This is a conducted at multiple hospitals, where both patients and doctors know the treatment given, Phase 1/2 study evaluating the safety, tolerability, how the drug moves through the body, how the drug affects the body, and antitumor activity of domatinostat in combination with sirolimus in adolescents and adults with relapsed or refractory sarcoma and osteosarcoma.
View original scientific description
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.
Interventions
DRUG
Domatinostat
Tablet
DRUG
Sirolimus
Tablet
Primary outcome measures
Phase 1: Maximum Tolerated Dose (MTD)
Time frame: 28 days
For the phase 1 portion, a standard 3+3 design will be used to determine the MTD and/or RP2D for the Phase 2 portion of this study.
Phase 2: Arm A Event Free Surival
Time frame: 12 months
Event free survival (EFS) is defined as the duration of time from start of treatment to time of disease progression, secondary malignancy, or death, whichever occurs first.
Phase 2: Arm B Event Free Surival
Time frame: 4 months
Event free survival (EFS) is defined as the duration of time from start of treatment to time of disease progression, secondary malignancy, or death, whichever occurs first.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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.
Exclusion criteria
- Receiving another investigational agent.
- Receiving concurrent anticancer therapy.
- Known hypersensitivity to domatinostat or sirolimus.
- Bone marrow-only disease.
- CNS metastatic disease.
- Major surgery within 2 weeks prior to treatment.
- Active malignancy within the previous 3 years (with protocol-specified exceptions).
- History of solid organ transplantation.
- Significant recent cardiac disease.
- Concurrent prohibited CYP3A4/P-gp interacting medications.
- Uncontrolled infection or other serious uncontrolled illness.
- Pregnancy or breastfeeding.
- HIV infection on antiretroviral therapy.
- Weight \<40 kg.
- Inability to comply with study requirements.
Where
- Tampa, Florida
- St Louis, Missouri
- The Bronx, New York
- Columbus, Ohio
Collaborators
National Pediatric Cancer Foundation, Healx USA, Inc
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 24, 2026 · Source of record for eligibility and locations