A Phase 2 Study of PTX 100 in Patients With Relapsed/Refractory CTCL

NCT06854653 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: PTX-100-02-2024
• Study Type: interventional
• Target: 115
• Locations: 4 countries
• Sites: 15

Brief Summary

This is an open-label, phase 2 randomized study to evaluate the efficacy, safety, pharmacokinetics (PK) and pharmadynamics (PD), of PTX-100 monotherapy at 500 or 1000 mg/m2 in patients with relapsed/refractory Cutaneous T-Cell Lymphoma (CTCL). PTX-100 will be 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. Subjects will be treated or followed up, if subjects discontinue treatment, for up to 18 months.

Conditions

CTCL

Keywords

Relapsed or refractory Cutaneous T Cell Lymphoma; CTCL; PTX-100; Mycosis Fungoides; Sezary Syndrome; T Cell Lymphoma; Non-Hodgkin Lymphoma; Cutaneous Lymphoma

Outcome Measures

Primary Outcomes (1)

• To determine the efficacy of PTX-100 as determined by ORR.

  Objective response rate (ORR): ORR is defined as the proportion of patients who achieved CR or PR as their best response

  18 months from day of first treatment until disease progression, unacceptable toxicity, participant or Investigator decision.

Secondary Outcomes (10)

• To further characterize the efficacy of PTX-100

  Within 18 months after PTX-100 initial dose.

• Progression-free survival (PFS)

  Within 18 months after PTX-100 initial dose.

• Time to response (TTR)

  Within 18 months after PTX-100 initial dose.

• Overall survival (OS)

  Within 18 months after PTX-100 initial dose.

• Duration of response (DoR)

  Within 18 months after PTX-100 initial dose.

Other Outcomes (2)

• To evaluate the effects of Health-related Quality of Life (QoL)

  From Baseline till end of study

• Exploratory Biomarkers

  From Baseline till end of study

Study Arms (3)

Phase 2a PTX-100 500mg/m2

EXPERIMENTAL

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.

Drug: PTX-100

Phase 2a PTX-100 1000mg/m2

EXPERIMENTAL

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.

Drug: PTX-100

Phase 2b PTX-100 Recommended Optimal Dose (ROD).

EXPERIMENTAL

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.

Drug: PTX-100

Interventions

PTX-100 DRUG

Peptidomimetic inhibitor of GGTase 1

Phase 2a PTX-100 1000mg/m2; Phase 2a PTX-100 500mg/m2; Phase 2b PTX-100 Recommended Optimal Dose (ROD).

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult patient ≥18 years of age at the time of signing the informed consent.
• Patient is capable of giving adequate signed informed consent
• Have a confirmed diagnosis of CTCL with histological confirmation
• Patients must have greater than or equal to Stage Ib disease.
• Has received and failed (or intolerant of) at least 2 prior lines of prior systemic therapy for their disease.
• Has measurable disease defined by at least one of the following, within 28 days prior to start of study treatment: by evaluable by mSWAT or quantifiable by flow cytometry or morphology in blood or measurable by Lugano Criteria.
• On a stable dose of systemic corticosteroid (\< 10 mg prednisone or equivalent) are permitted. Participants on a stable dose of topical corticosteroids are permitted.
• Washout period- must be 2 weeks (4 weeks for monoclonal antibodies) or 5 -half-lives (whichever is longer) since any prior anti-cancer therapy.
• Must be human T-cell lymphotropic virus type 1 (HTLV1) negative.
• Has an ECOG PS of 0 to 2.
• Life expectancy of 3 months or greater
• Has adequate bone marrow function.
• Has adequate hepatic function.
• Has adequate Renal function.
• Has adequate coagulation function.

You may not qualify if:

• Patients with known central nervous system involvement.
• Patients who require the use of strong inhibitors or inducers of CYP enzymes or transporters (e.g., CYP3A4, 2D6, 2C19) or (P-gp, BCRP, OATP1B1, OATP1B3, OAT1. OAT3, OCT2, MATE1 and MATE2-K). Patients who are receiving these medications at Screening can be enrolled into the trial if they discontinue them for at least 14 days or 5 half-lives, whichever is longer, before they commence PTX-100. An alternative pharmacological treatment should be instituted by the treating clinician based on clinical judgement.
• Significant cardiovascular disease. A history of, or concurrent interstitial lung disease or severely impaired lung function.
• \. Active viral, bacterial, fungal infection or other serious infection requiring ongoing systemic treatment. Routine antimicrobial prophylaxis is permitted.
• \. Medical history of another malignant tumor within the past 5 years. Exceptions are patients with basal cell carcinoma of the skin, squamous cell carcinoma of the skin or carcinoma in situ who have undergone curative therapy with no evidence of disease.
• \. On an immunomodulatory drug for concomitant or intercurrent conditions or who have received any of these agents within 4 weeks of baseline.
• \. Patients with active viral (any etiology) hepatitis are excluded. However, patients with serologic evidence of chronic hepatitis B virus (HBV) infection (defined by a positive hepatitis B surface antigen test and a positive anti-hepatitis core antigen antibody test) who have a viral load below the limit quantification (HBV deoxyribose nucleic acid titer \< 1000 cps/mL or 200 IU/mL) and are not currently on viral suppressive therapy may be eligible and should be discussed with the Medical Monitor. Patients with a history of hepatitis C virus infection who have completed curative antiviral treatment and have a viral load below the limit of quantification may be eligible and should be discussed with the Medical Monitor.
• \. 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.
• \. Prior allogeneic or autologous hematopoietic transplantation 11. Has a known psychiatric disorder that would interfere with compliance with the requirements 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.

Sponsors & Collaborators

• Prescient Therapeutics, Ltd.: lead

Study Sites (15)

City of Hope Comprehensive Cancer Cente

Duarte, California, 91010, United States

RECRUITING

University of California Irvine

Irvine, California, 92697, United States

RECRUITING

Yale Cancer Center

New Haven, Connecticut, 06510, United States

RECRUITING

Dana-Farber Cancer Institute

Boston, Massachusetts, 022155450, United States

RECRUITING

Rochester Skin Lymphoma Medical Group. PLLC

Rochester, New York, 14642, United States

RECRUITING

Virginia Commonwealth University Massey Comprehensive Cancer Cente

Richmond, Virginia, 980037, United States

RECRUITING

Westmead Hospital

Westmead, New South Wales, 2145, Australia

RECRUITING

Epworth Healthcare

Melbourne, Victoria, 3002, Australia

RECRUITING

Linear Clinical Research

Nedlands, Western Australia, 6009, Australia

RECRUITING

CHU de Bordeaux - Hopital Saint André

Bordeaux, Bordeaux, 33000, France

NOT YET RECRUITING

Hopital Lyon Sud

Lyon, Pierre-Benite, 69310, France

NOT YET RECRUITING

Hopital Saint Louis

Paris, Île-de-France Region, 75010, France

NOT YET RECRUITING

IRCCS Azienda Ospedaliero Universitaria di Bologna - Policlinico S. Orsola-Malpighi

Bologna, Bologna, 40138, Italy

RECRUITING

Universita degli Studi Di Brescia-Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia

Brescia, Brescia, 25123, Italy

RECRUITING

Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele)

Milan, Milano, 20132, Italy

RECRUITING

MeSH Terms

Conditions

Recurrence; Lymphoma, T-Cell, Cutaneous; Mycosis Fungoides; Sezary Syndrome; Lymphoma, T-Cell; Lymphoma, Non-Hodgkin

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Central Study Contacts

Upaly Bahadure

CONTACT

+61 3 9692 7222; upaly@ptxtherapeutics.com

David Wong

CONTACT

david.wong@ptxtherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Randomised parallel recruitment into 2 treatment arms, 500mg/m2 and 1000-mg/m2 for Phase 2a. Phase 2b will be non-randomised and are single arm treatment group. The recommended optimal dose which was determined in Phase 2a will be used in Phase 2b.

Study Record Dates

• First Submitted: February 18, 2025
• First Posted: March 3, 2025
• Study Start: March 28, 2025
• Primary Completion (Estimated): January 1, 2028
• Study Completion (Estimated): June 1, 2028
• Last Updated: April 29, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

AU (3); FR (3); US (6); IT (3)