SarcomaPhase 1/2NCT07725380
Domatinostat With Sirolimus for Relapsed, Refractory Sarcoma and Osteosarcoma
A Phase 1/2 study testing Domatinostat and Sirolimus in people with Sarcoma and Osteosarcoma, run by H. Lee Moffitt Cancer Center and Research Institute at 4 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 40 years
- Conditions the study is for
- Sarcoma, Osteosarcoma
- What is being tested?
- Domatinostat and Sirolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 74 participants
- Where
- 4 sitesUnited States
- Study dates
- 15 Jul 2026to Aug 2031
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Nationwide Children's Hospital
Columbus, Ohio, 43205
Bhuvana Setty, MD
- Not yet recruiting
Washington University in St. Louis
St Louis, Missouri, 63110
Amy Armstrong, MD
- Recruiting
Moffitt Cancer Center
Tampa, Florida, 33612
Jonathan Metts, MD
Andrew Brohl, MD
- Not yet recruiting
Montefiore Medical Center
The Bronx, New York, 10467
Alice Lee, MD
About this study
Official title: 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
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.
- Conditions
- Sarcoma, Osteosarcoma
- Being tested
- Domatinostat, Sirolimus
- Medical subject headings
- Sarcoma, Osteosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DomatinostatDrug
Tablet
SirolimusDrug
Tablet
| Study group | Role | What they receive |
|---|---|---|
| Phase 1 Dose Escalation Cohort | Experimental | Participants with relapsed or refractory sarcoma will receive domatinostat plus sirolimus. Domatinostat dose levels will be escalated to determine the MTD and/or RP2D. Receives: Drug: Domatinostat; Drug: Sirolimus |
| Phase 2 Arm A (Surgical Complete Remission Osteosarcoma) | Experimental | Patients with relapsed/refractory osteosarcoma who achieve complete surgical remission before enrollment will receive domatinostat plus sirolimus at the RP2D. Receives: Drug: Domatinostat; Drug: Sirolimus |
| Phase 2 Arm B (Measurable Osteosarcoma) | Experimental | Patients with unresectable measurable relapsed/refractory osteosarcoma will receive domatinostat plus sirolimus at the RP2D. Receives: Drug: Domatinostat; Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1: Maximum Tolerated Dose (MTD)
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.
Measured: 28 days
Phase 2: Arm A Event Free Surival
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.
Measured: 12 months
Phase 2: Arm B Event Free Surival
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.
Measured: 4 months
Secondary outcomes (1)
Phase 2: Overall Survival
Overall Survival (OS) of phase 2 participants.
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2026
- Study start15 Jul 2026
- Primary completionAug 2031 (estimated)
- Study completionAug 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SarcomaStudies of the same condition, open and under way.
- More trials run by H. Lee Moffitt Cancer Center and Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 74 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- National Pediatric Cancer Foundation, Healx USA, Inc
- Responsible party
- Sponsor
- Investigators
- Amy Armstrong, MD, Principal investigator, Washington University School of MedicineJonathan Metts, MD, Principal investigator, Moffitt Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07725380
- Sponsor's protocol ID
- MCC-23906
- Status last verified
- Sep 2026
- First submitted
- 21 Jul 2026
- Last updated
- 3 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07725380
- Acronym / sponsor ID
- MCC-23906
- Conditions
- Sarcoma, Osteosarcoma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 74 participants (estimated)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- National Pediatric Cancer Foundation, Healx USA, Inc
- Study start
- 15 Jul 2026 (actual)
- Primary completion
- Aug 2031 (estimated)
- Study completion
- Aug 2031 (estimated)
- First posted
- 24 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Phase 1 Dose Escalation Cohort | Experimental | Drug: Domatinostat, Drug: SirolimusParticipants with relapsed or refractory sarcoma will receive domatinostat plus sirolimus. Domatinostat dose levels will be escalated to determine the MTD and/or RP2D. |
| Phase 2 Arm A (Surgical Complete Remission Osteosarcoma) | Experimental | Drug: Domatinostat, Drug: SirolimusPatients with relapsed/refractory osteosarcoma who achieve complete surgical remission before enrollment will receive domatinostat plus sirolimus at the RP2D. |
| Phase 2 Arm B (Measurable Osteosarcoma) | Experimental | Drug: Domatinostat, Drug: SirolimusPatients with unresectable measurable relapsed/refractory osteosarcoma will receive domatinostat plus sirolimus at the RP2D. |
Who may qualify
- Ages
- 12 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (15 listed)
- Receiving another investigational agent.
- Receiving concurrent anticancer therapy.
- Known hypersensitivity to domatinostat or sirolimus.
- Bone marrow-only disease.
- CNS metastatic disease.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DomatinostatTablet | Drug | Phase 1 Dose Escalation Cohort, Phase 2 Arm A (Surgical Complete Remission Osteosarcoma), Phase 2 Arm B (Measurable Osteosarcoma) |
| SirolimusTablet | Drug | Phase 1 Dose Escalation Cohort, Phase 2 Arm A (Surgical Complete Remission Osteosarcoma), Phase 2 Arm B (Measurable Osteosarcoma) |
Primary outcome
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.
1 secondary outcome
Phase 2: Overall Survival
Time frame: Up to 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Amy Armstrong, MD principal investigator
Washington University School of Medicine
Jonathan Metts, MD principal investigator
Moffitt Cancer Center
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Domatinostat and Sirolimus in people with Sarcoma and Osteosarcoma, run by H. Lee Moffitt Cancer Center and Research Institute at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jessica Crimella (813-745-6250, Jessica.Crimella@moffitt.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase 1: Maximum Tolerated Dose (MTD) (time frame: 28 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2026.