Sarcoma, EwingPhase 2HopESNCT03778996
SM-88 Maintenance Therapy for Advanced Ewing's Sarcoma and as Salvage Therapy for Sarcoma
A Phase 2 study testing Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS) in people with Sarcoma, Ewing and Sarcoma, run by Sarcoma Oncology Research Center, LLC at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Sarcoma, Ewing, Sarcoma
- What is being tested?
- Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 31 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Jan 2020to 31 Mar 2028
- Phase
- Phase 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Ability to understand and willingness to provide written informed consent to participate in this study
- ≥12 years of age
- Diagnosis:
- Maintenance treatment cohort: Histologic or cytologic diagnosis of Ewing's sarcoma, including non-resected, locally advanced or metastatic disease
- Salvage treatment cohort: Non-resected, locally advanced or metastatic sarcoma (including Ewing's sarcoma) for which there is no standard of care treatment and no curative other option
- Radiographic disease assessment within 35 days prior to enrollment and planned treatment start with study drug
Show all 17Show fewer
- Prior treatment:
- Maintenance treatment cohort : 1-3 prior lines of systemic treatment (including current treatment)
- Salvage cohort: Any number of prior treatments
- Maintenance treatment cohort only: Patient completed current line of treatment (systemic, surgery, radiation) prior to enrollment, without disease progression as compared to baseline AND has achieved at least one of the following
- CR in response to current second or third line treatment
- PR in response to current line of treatment (after at least 4 cycles, if treatment included systemic therapy)
- SD in response to current line of treatment (after at least 4 cycles, if treatment included systemic therapy)
- Measurable disease, except for patients in Cohort A who have achieved CR at the conclusion of current 2nd or 3rd line of treatment
- ECOG performance status 0-2
- Adequate organ function defined as all laboratory parameters ≤ Grade 2 NCI CTCAE criteria
- Patients must be able to swallow and retain whole capsules
Who cannot join
- Systemic anticancer agents within 14 days prior to treatment on study
- Major surgery within 30 days
- Prior treatment with SM-88
- Any screening laboratory, electrocardiogram (ECG), other clinical finding, comorbidity or clinical history that, in the opinion of the investigator, indicates an unacceptable risk for patient to participate in the study or would limit patient's ability to comply with study requirements
- History of any drug allergies or significant adverse reactions to any of the components of SM-88
- History of light sensitive diseases for which methoxsalen would be contraindicated
- Current or anticipated treatment with a contraindicated medication
- Evidence of viral infections including human immunodeficiency virus (HIV), hepatitis B, and hepatitis C
Trial locations and contacts
1 site in 1 country.
United States1 site
Sarcoma Oncology Research Center
Santa Monica, California, 90403
About this study
Official title: 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
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.
Read the 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
- Being tested
- Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS)
- Keywords
- Ewing's Sarcoma, Locally advanced sarcoma, Metastatic sarcoma, SM-88, D.L-alpha-metyrosine
- Medical subject headings
- Sarcoma, Ewing, Sarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS)Drug
Also called SM-88
Daily oral combination therapy for cancer
| Study group | Role | What they receive |
|---|---|---|
| Maintenance Treatment: Ewing's Sarcoma | Experimental | Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus Receives: Drug: Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS) |
| Salvage Treatment: Sarcoma | Experimental | Combination metyrosine-derivative, low-dose methoxasalen, phenytoin and sirolimus Receives: Drug: Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate
Complete response (CR) + partial response (PR) as evaluated using RECIST 1.1
Measured: Every 3 months for up to 2 years
Stable Disease for at Least 3 Months
Stable disease (SD) as evaluated using RECIST 1.1
Measured: Every 3 months for up to 2 years
Progression Free Survival on Study of at Least 1.5 Times the Duration of PFS for the Last Prior Treatment
From date of enrollment until the date of first documented progression, as evaluated using RECIST 1.1, or date of death, whichever occurs first
Measured: Every 3 months for up to 2 years
Secondary outcomes (4)
Duration of Response
From date of CR or PR until the date of first documented progression, as evaluated using RECIST 1.1
Measured: Every 3 months for up to 2 years
Overall Survival
From date enrollment until the date of death
Measured: Every 3 months for up to 2 years
Clinical Benefit Rate
CR+PR+SD as evaluated using RECIST 1.1
Measured: Every 3 months for up to 2 years
Incidence of Treatment-Emergent Adverse Events as assessed by CTCAE v5
Adverse events will be assessed at each visit and at unscheduled visits as clinically indicated
Measured: From date of enrollment until 28 days after last treatment with SM-88
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2018
- Study start3 Jan 2020
- Primary completion19 Dec 2027 (estimated)
- Study completion31 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sarcoma, EwingStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sarcoma Oncology Research Center, LLC
- Sponsor type
- University, hospital or other
- Collaborators
- Tyme, Inc
- Responsible party
- Sponsor
- Investigators
- Sant P Chawla, MD, Principal investigator, Sarcoma Oncology Research Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03778996
- Sponsor's protocol ID
- SM-88-JAF-16
- Acronym
- HopES
- Status last verified
- Feb 2025
- First submitted
- 6 Dec 2018
- Last updated
- 24 Feb 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT03778996
- Acronym / sponsor ID
- HopES / SM-88-JAF-16
- Conditions
- Sarcoma, Ewing, Sarcoma
- Keywords
- Ewing's Sarcoma, Locally advanced sarcoma, Metastatic sarcoma, SM-88, D.L-alpha-metyrosine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 31 participants (actual)
- Lead sponsor
- Sarcoma Oncology Research Center, LLCUniversity, hospital or other
- Collaborators
- Tyme, Inc
- Study start
- 3 Jan 2020 (actual)
- Primary completion
- 19 Dec 2027 (estimated)
- Study completion
- 31 Mar 2028 (estimated)
- First posted
- 19 Dec 2018 (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.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Maintenance Treatment: Ewing's Sarcoma | Experimental | Drug: Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS)Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus |
| Salvage Treatment: Sarcoma | Experimental | Drug: Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS)Combination metyrosine-derivative, low-dose methoxasalen, phenytoin and sirolimus |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (17 listed)
- Ability to understand and willingness to provide written informed consent to participate in this study
- ≥12 years of age
- Diagnosis:
- Radiographic disease assessment within 35 days prior to enrollment and planned treatment start with study drug
- Prior treatment:
Key exclusion criteria (8 listed)
- Systemic anticancer agents within 14 days prior to treatment on study
- Major surgery within 30 days
- Prior treatment with SM-88
- Any screening laboratory, electrocardiogram (ECG), other clinical finding, comorbidity or clinical history that, in the opinion of the investigator, indicates an unacceptable risk for patient to participate in the study or would limit patient's ability to comply with study requirements
- History of any drug allergies or significant adverse reactions to any of the components of SM-88
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 |
|---|---|---|
| Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS)Also: SM-88Daily oral combination therapy for cancer | Drug | Maintenance Treatment: Ewing's Sarcoma, Salvage Treatment: Sarcoma |
Primary outcome
Overall Response Rate
Time frame: Every 3 months for up to 2 years
Complete response (CR) + partial response (PR) as evaluated using RECIST 1.1
Stable Disease for at Least 3 Months
Time frame: Every 3 months for up to 2 years
Stable disease (SD) as evaluated using RECIST 1.1
Progression Free Survival on Study of at Least 1.5 Times the Duration of PFS for the Last Prior Treatment
Time frame: Every 3 months for up to 2 years
From date of enrollment until the date of first documented progression, as evaluated using RECIST 1.1, or date of death, whichever occurs first
4 secondary outcomes
Duration of Response
Time frame: Every 3 months for up to 2 years
Overall Survival
Time frame: Every 3 months for up to 2 years
Clinical Benefit Rate
Time frame: Every 3 months for up to 2 years
Incidence of Treatment-Emergent Adverse Events as assessed by CTCAE v5
Time frame: From date of enrollment until 28 days after last treatment with SM-88
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 24 Feb 2025.
- Sarcoma Oncology Research CenterLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Sant P Chawla, MD principal investigator
Sarcoma Oncology Research Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS) in people with Sarcoma, Ewing and Sarcoma, run by Sarcoma Oncology Research Center, LLC at 1 site 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 17 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 24 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 phase mean?
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate (time frame: Every 3 months for up to 2 years). 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 24 Feb 2025; AzTrials last refreshed its copy on 26 Oct 2026.