PTX-100 for Cutaneous T-Cell Lymphoma

Recruiting at 15 trial locations

Overseen By David Wong

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Prescient Therapeutics, Ltd.

Must not be taking: CYP inhibitors, Immunomodulatory drugs

Disqualifiers: CNS involvement, Cardiovascular disease, Active infection, Other malignancy, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

Duration: 18 months

115 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called PTX-100 for individuals with Cutaneous T-Cell Lymphoma (CTCL), a type of skin cancer. Researchers aim to determine the effectiveness and safety of PTX-100 at two different doses. The treatment involves regular IV infusions over several months. Individuals with CTCL that hasn't responded to at least two previous treatments might be eligible to join. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial requires a washout period of 2 weeks (or 4 weeks for monoclonal antibodies) since any prior anti-cancer therapy. If you are on strong inhibitors or inducers of certain enzymes, you must stop them for at least 14 days before starting the trial. It's best to discuss your current medications with the trial team to see if any adjustments are needed.

Is there any evidence suggesting that PTX-100 is likely to be safe for humans?

Research has shown that PTX-100 has been safe in earlier studies. In a past study involving patients with various blood and solid cancers, PTX-100 was generally well-tolerated. Most patients did not experience serious side effects, indicating that while some side effects might occur, they are usually not severe.

The current phase of the study tests PTX-100 in people with Cutaneous T-Cell Lymphoma. Researchers will closely monitor for any new safety issues. This phase aims to gather more detailed information about the treatment's safety for patients. Previous results are promising, but ongoing studies will provide clearer answers. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Cutaneous T-Cell Lymphoma?

PTX-100 is unique because it targets a specific protein involved in cancer cell growth, offering a new mechanism of action compared to standard treatments like chemotherapy or radiation. Unlike other treatments for cutaneous T-cell lymphoma, which generally have broader effects on rapidly dividing cells, PTX-100 specifically inhibits the Ras pathway, a key factor in cancer cell survival. Researchers are excited about PTX-100 because it has the potential to be more targeted, which might reduce side effects and improve outcomes for patients.

What evidence suggests that PTX-100 might be an effective treatment for Cutaneous T-Cell Lymphoma?

Studies have shown that PTX-100 holds promise for treating T-cell lymphomas, including Cutaneous T-Cell Lymphoma (CTCL). In earlier research, PTX-100 demonstrated positive results and was generally safe for patients. The FDA has granted this treatment "orphan drug designation," indicating its potential for treating rare diseases like CTCL. Early research suggests that PTX-100 targets specific parts of cancer cells, offering a new approach to treating these lymphomas. Although this treatment remains under study, the initial results are encouraging. Participants in this trial will receive PTX-100 at varying dosages to determine the optimal treatment regimen. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals with Cutaneous T-Cell Lymphoma (CTCL) that has come back or didn't respond to previous treatments. Specific details about who can join are not provided, but typically participants need to meet certain health standards and may be required to have a particular stage of disease.

Inclusion Criteria

Life expectancy of 3 months or greater

A WOCBP must have a negative serum pregnancy within 72 hours of the first dose of study treatment

My cancer is at least Stage Ib.

Exclusion Criteria

Has a known psychiatric disorder that would interfere with compliance with the requirements of the study

A history or current evidence of any condition, laboratory abnormality or other circumstance that might confound the results of the study or interfere with patient participation for the full duration of the study

Is a consumer of illicit or recreational drugs or has a recent history (within the last year) of drug or alcohol abuse or dependence that in the judgment of the Investigator, would interfere with compliance with the requirements of the study

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

1 visit (in-person)

Treatment

PTX-100 administered by IV infusion over 60 minutes on days 1 to 5 of a 14-day cycle for 4 cycles, then 21-day cycle thereafter

18 months

Multiple visits per cycle for infusion and assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

18 months

What Are the Treatments Tested in This Trial?

Interventions

• PTX-100

Trial Overview The study tests PTX-100 at two different doses (500 or 1000 mg/m2) given through an IV over an hour on the first five days of a cycle. Initially, cycles are 14 days each for four cycles, then extend to 21-day cycles. Participants will be observed or continue treatment up to 18 months.

How Is the Trial Designed?

Group I: Phase 2b PTX-100 Recommended Optimal Dose (ROD). Experimental Treatment 1 Intervention

Phase 2b PTX-100 will be the Recommended Optimal Dose form Phase 2a and follow the same infusion timeline outlined previously. IV infusion of RD over 60 minutes on days 1 to 5 of a 14-day cycle for 4 cycles then followed by IV infusion of RD over 60 minutes on days 1 to 5 of a 21-day cycle up to 18months.

Group II: Phase 2a PTX-100 500mg/m2 Experimental Treatment 1 Intervention

Phase 2a PTX-100 will be 500mg/m2 IV infusion over 60 minutes on days 1 to 5 of a 14-day cycle for 4 cycles. This is then followed by IV infusion over 60 minutes on days 1 to 5 of a 21-day cycle for up to 18months.

Group III: Phase 2a PTX-100 1000mg/m2 Experimental Treatment 1 Intervention

Phase 2a PTX-100 will be 1000mg/m2 IV infusion over 60 minutes on days 1 to 5 of a 14-day cycle for 4 cycles. This is then followed by IV infusion over 60 minutes on days 1 to 5 of a 21-day cycle for up to 18months.

Who Is Running the Clinical Trial?

Prescient Therapeutics, Ltd.

Lead Sponsor

Trials

7

Recruited

260+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06854653

NCT06854653 | A Phase 2 Study of PTX 100 in Patients ... This is an open-label, phase 2 randomized study to evaluate the efficacy, safety, pharmacokinetics (PK) and pharmadynamics (PD), of PTX-100 monotherapy at ...

2. clfoundation.org clfoundation.org/ptx-100

Targeted Therapy: PTX-100 PTX-100 has recently completed a Phase 1b expansion cohort study in T cell lymphomas, where it showed encouraging efficacy and safety. The US FDA has granted ...

3. masseycancercenter.org masseycancercenter.org/news/massey-becomes-first-us-site-to-enroll-patients-in-global-phase-2a-trial-of-first-in-class-therapy-for-t-cell-lymphoma/

Massey becomes first U.S. site to enroll patients in global ... Based on these results, PTX-100 received orphan drug designation from the United States Food and Drug Administration for all T-cell lymphomas, ...

4. ashpublications.org ashpublications.org/blood/article/142/Supplement%201/1705/499254/Phase-1-Pharmacodynamic-and-Pharmacokinetic-Study

Phase 1 Pharmacodynamic and Pharmacokinetic Study of the ... Conclusions: Preliminary data from this phase 1 dose escalation study of PTX-100 indicates promising safety profile in this difficult to treat ...

5. smallcaps.com.au smallcaps.com.au/prescient-therapeutics-ptx-100-phase-2a-trials-outstanding-results

Prescient Therapeutics' PTX-100 Progresses to Phase 2a ... Prescient Therapeutics' PTX-100 Progresses to Phase 2a Trials after Outstanding Phase 1b Results · Success Story · FDA Designations · Painful ...

6. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/580355

A Phase 2 Study of PTX 100 in Patients With Relapsed ... This is an open-label, phase 2 randomized study to evaluate the efficacy, safety, pharmacokinetics (PK) and pharmadynamics (PD), of PTX-100 monotherapy at ...

7. ucihealth.org ucihealth.org/clinical-trials/UCI-24-188

An Open-Label, Phase II Study to Evaluate the Efficacy, ... This is an open-label, phase 2 randomized study to evaluate the efficacy, safety, pharmacokinetics (PK) and pharmadynamics (PD), of PTX-100 ...