An Open-Label, Phase 2 Study of PTX-100 Monotherapy in Patients With Relapsed or Refractory Cutaneous T-Cell Lymphoma.
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.
Detailed description
PTX-100 from a Phase I study shown to help some CTCL patients. This Phase II study will be conducted in a larger population size and there will be initially two groups/arms in the first phase called Phase 2a. This phase will randomize and enroll 20 subjects into the 500 mg/m2 and 20 subjects into the 1000 mg/m2 PTX treatment arms. After determining the recommended optimal dose from phase 2a, for Phase 2b, 75 subjects will then be allocated into this single arm part of the study. Once subject has signed the informed consent, subject will undergo a 28 day screening period, where eligibility would be determined. Once subject is eligible, subject will be dosed with IP. Safety bloods will be taken on the first day of every cycle. Pharmacokinetics (PKs) which are blood samples sent to the Sponsors associated laboratory and will be analysed on how PTX-100 interacts biologically. PKs will be taken on Cycle1Day1(C1D1) to C1D5 and C1D8 for the first 4 cycles. Subject will also undergo skin evaluation and safety exams at every Cycle Day 1. Subjects will also complete quality of life questionnaires at every Cycle Day1. Subjects will be on the study for 18months, until disease progression, unacceptable toxicity, participant or Investigator decision, or until study treatment discontinuation criteria are met, whichever occurs first.
Arms & interventions
- DrugPTX-100
Peptidomimetic inhibitor of GGTase 1
Outcome measures
Primary
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
Time frame: 18 months from day of first treatment until disease progression, unacceptable toxicity, participant or Investigator decision.
Secondary
To further characterize the efficacy of PTX-100
Time frame: Within 18 months after PTX-100 initial dose.
Progression-free survival (PFS)
Time frame: Within 18 months after PTX-100 initial dose.
Time to response (TTR)
Time frame: Within 18 months after PTX-100 initial dose.
Overall survival (OS)
Time frame: Within 18 months after PTX-100 initial dose.
Duration of response (DoR)
Time frame: Within 18 months after PTX-100 initial dose.
Complete response rate (CRR)
Time frame: Within 18 months after PTX-100 initial dose.
Pharmacokinetics (PK) of PTX-100: Cmax
Time frame: Cycle 1: Day 1 and Day 5: pre-dose, 5 mins post-infusion, and at 1, 2, 4, 6, and 8 hours after dose. Day 2, 3 and 4: 24 hours after previous dose, bloods taken at Pre and at 5 mins post-infusion. Day 8; 72 hours after the Day 5 dose and prior to dosing.
Pharmacokinetics (PK) of PTX-100: Tmax
Time frame: Cycle 1: Day 1 and Day 5: pre-dose, 5 mins post-infusion, and at 1, 2, 4, 6, and 8 hours after dose. Day 2, 3 and 4: 24 hours after previous dose, bloods taken at Pre and at 5 mins post-infusion. Day 8; 72 hours after the Day 5 dose and prior to dosing.
Pharmacokinetics (PK) of PTX-100: Cmin
Time frame: Cycle 1: Day 1 and Day 5: pre-dose, 5 mins post-infusion, and at 1, 2, 4, 6, and 8 hours after dose. Day 2, 3 and 4: 24 hours after previous dose, bloods taken at Pre and at 5 mins post-infusion. Day 8; 72 hours after the Day 5 dose and prior to dosing.
Pharmacokinetics (PK) of PTX-100: AUCtau
Time frame: Cycle 1: Day 1 and Day 5: pre-dose, 5 mins post-infusion, and at 1, 2, 4, 6, and 8 hours after dose. Day 2, 3 and 4: 24 hours after previous dose, bloods taken at Pre and at 5 mins post-infusion. Day 8; 72 hours after the Day 5 dose and prior to dosing.
Eligibility criteria
Study locations (6)
City of Hope Comprehensive Cancer Cente
Duarte, California, 91010
University of California Irvine
Irvine, California, 92697
Yale Cancer Center
New Haven, Connecticut, 06510
Dana-Farber Cancer Institute
Boston, Massachusetts, 022155450
Rochester Skin Lymphoma Medical Group. PLLC
Rochester, New York, 14642
Virginia Commonwealth University Massey Comprehensive Cancer Cente
Richmond, Virginia, 980037