Seclidemstat Access for Sarcoma

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Salarius Pharmaceuticals, LLC

Must be taking: Seclidemstat

Disqualifiers: Pregnancy, Breastfeeding, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 5 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to provide continued access to seclidemstat, a treatment for sarcoma, for patients who still benefit from it. Researchers are testing two options: seclidemstat alone and in combination with two other drugs, topotecan and cyclophosphamide. The trial suits those already part of a related study, currently benefiting from the treatment, and able to adhere to the study schedule. It specifically targets those who find the treatment beneficial and wish to continue it. As a Phase 1/Phase 2 trial, it focuses on understanding how the treatment works in people and measuring its effectiveness in an initial group, offering patients a chance to contribute to important research.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but it mentions that you cannot receive certain therapies that are not allowed in the parent protocol. It's best to discuss your current medications with the trial team to see if they are permitted.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that seclidemstat, whether used alone or with the chemotherapy drugs topotecan and cyclophosphamide, generally has a manageable safety profile. Most side effects can be treated with standard care and are not too severe for most patients. In patients with advanced sarcoma, the treatment has shown promise and has been well-tolerated.

However, another study reported a serious, unexpected side effect, which temporarily paused that trial. Fortunately, further review lifted this pause. This indicates that while risks exist, they are being carefully monitored.

Overall, the safety data appears promising, but like any treatment, potential risks exist. It's important to discuss these with the trial team or a healthcare provider. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Most treatments for sarcoma focus on traditional chemotherapy agents, but seclidemstat introduces a new approach by targeting enzymes called lysine-specific demethylase 1 (LSD1), which play a role in gene expression. Researchers are excited because this mechanism can potentially halt tumor growth more effectively and with fewer side effects than standard chemotherapy. In the combination therapy arm, seclidemstat is paired with topotecan and cyclophosphamide, potentially enhancing its effectiveness by attacking the cancer cells through multiple pathways. The single-agent arm offers a simpler treatment regimen, which might be beneficial for patients who cannot tolerate combination therapies.

What evidence suggests that this trial's treatments could be effective for sarcoma?

Research has shown that seclidemstat, one of the treatments in this trial, has potential in treating certain types of sarcomas when used alone. In one study, two patients maintained stable disease, meaning their cancer did not worsen, at specific doses. The drug reverses the effects of a gene fusion that causes Ewing sarcoma, a type of cancer affecting bones and soft tissues.

In this trial, some participants will receive seclidemstat as a single agent, while others will receive it in combination with the drugs topotecan and cyclophosphamide. This combination has shown promising results in previous studies. Specifically, in one study, 38% of patients achieved disease control, meaning their tumors did not grow, and another patient experienced tumor shrinkage after four treatment cycles.

These findings offer hope that seclidemstat, whether used alone or in combination, could be an effective treatment option for sarcoma patients. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

This trial is for patients who have been part of previous Salarius studies and are still benefiting from Seclidemstat, either alone or with other treatments. They must follow the study rules, use contraception if they can have children, and not be pregnant or breastfeeding. People can't join if they were taken off the original study treatment for any reason except to join this new one.

Inclusion Criteria

I am currently in a Salarius study and taking seclidemstat, with no more than a 14-day break between studies.

I am responding well to my current seclidemstat treatment.

I agree to use birth control during the study and will report any pregnancy immediately.

Exclusion Criteria

Pregnant and breastfeeding women

Patient permanently discontinued from parent study treatment for any reason other than entering this rollover study

Receiving prohibited concomitant therapy as described in the rollover protocol or therapy not allowed in the parent protocol

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants continue to receive seclidemstat as a single agent or in combination with topotecan and cyclophosphamide, as assigned per parent protocol

Up to 5 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Seclidemstat

Trial Overview The trial provides ongoing access to Seclidemstat (SP-2577) for those already seeing positive effects from it in past trials by Salarius. It's a rollover protocol which means patients continue with the same medication without interruption.

How Is the Trial Designed?

Group I: TC Combination Experimental Treatment 1 Intervention

Combination of seclidemstat with topotecan and cyclophosphamide, as assigned per parent protocol

Group II: Single agent Experimental Treatment 1 Intervention

Single agent seclidemstat, as assigned per parent protocol

Who Is Running the Clinical Trial?

Salarius Pharmaceuticals, LLC

Lead Sponsor

Trials

5

Recruited

160+

Published Research Related to This Trial

In a study of 806 patients with localized soft tissue sarcoma, those who received perioperative chemotherapy had a 5-year metastasis-free survival rate of 58%, indicating that this treatment can be effective in real-world settings.

While the long-term outcomes of patients receiving perioperative chemotherapy in routine clinical practice were similar to those in clinical trials, there were notable risks of secondary malignancies and cardiac toxicity, highlighting the importance of considering these potential side effects in treatment decisions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-world evidence on perioperative chemotherapy in localized soft tissue sarcoma of the extremities and trunk wall; a population-based study. Boye, K., Lobmaier, I., Kobbeltvedt, MR., et al.[2022]

Using patient-derived tumor organoids (PDTOs) from 194 specimens of 126 sarcoma patients, researchers were able to identify effective treatment options for 59% of the cases, highlighting the potential of PDTOs in personalizing therapy for rare sarcoma subtypes.

The study demonstrated that drug sensitivity in PDTOs correlated with various clinical factors, such as tumor subtype and patient treatment history, suggesting that organoid drug screening can guide treatment decisions and improve patient outcomes in sarcoma.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The landscape of drug sensitivity and resistance in sarcoma. Al Shihabi, A., Tebon, PJ., Nguyen, HTL., et al.[2023]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03600649

Clinical Trial of SP-2577 (Seclidemstat) in Patients With ... To evaluate the safety and tolerability of seclidemstat (SP-2577) as a single agent and in combination with topotecan and cyclophosphamide in patients with ...

2. ascopubs.org ascopubs.org/doi/10.1200/JCO.2021.39.15_suppl.11514

Phase 1 trial of seclidemstat (SP-2577) in patients with ... One pt at 600 mg BID achieved a reduction in target lesions starting at end of C2 with further target lesion tumor shrinkage through end of C4 ...

3. investors.salariuspharma.com investors.salariuspharma.com/news-releases/news-release-details/salarius-pharmaceuticals-announces-interim-results-phase-12

Press Releases Of these 13 patients, five patients (38%) achieved confirmed disease control with no tumor progression observed while treated with seclidemstat ...

4. ascopubs.org ascopubs.org/doi/10.1200/JCO.2021.39.15_suppl.TPS11577

Phase 1 expansion trial of the LSD1 inhibitor seclidemstat ... Seclidemstat has shown in in vitro ES cell lines near additivity efficacy when added to TC. In in vitro studies of other FET-translocated ...

5. clin.larvol.com clin.larvol.com/trial-detail/NCT03600649

Clinical Trial of SP-2577 (Seclidemstat) in Patients With ... In this heavily pretreated population, a patient dosed at 600 mg BID achieved a reduction in three target lesions after 58 days (2 cycles) with further tumor ...

6. investors.salariuspharma.com investors.salariuspharma.com/news-releases/news-release-details/salarius-pharmaceuticals-achieves-dosing-milestone-ongoing-phase

Salarius Pharmaceuticals Achieves Dosing Milestone in ... The Phase 1/2 Sarcoma trial is an open label study exploring the safety and efficacy of seclidemstat in three patient groups. Patients with ...

7. biospace.com biospace.com/fda-removes-partial-clinical-hold-on-salarius-pharmaceuticals-phase-1-2-clinical-trial-with-seclidemstat-in-patients-with-ewing-sarcoma

FDA Removes Partial Clinical Hold on Salarius ... The US Food and Drug Administration (FDA) has removed its partial clinical hold on Salarius' Phase 1/2 Ewing sarcoma clinical trial evaluating seclidemstat.