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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
115
Peptidomimetic inhibitor of GGTase 1
City of Hope Comprehensive Cancer Cente
Duarte, California, United States
RECRUITINGTo 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.
To further characterize the efficacy of PTX-100
Skin response as per Modified Severity-Weighted Assessment Tool (mSWAT). The mSWAT uses body surface area in 12 body areas to calculate involvement. A weighting factor is also used: Patches, which are flat, earned a multiplier of one. Plaques, which are raised, earned a multiplier of two. Tumors, which are larger and solid, earned a multiplier of three. Lesion BSA multiplied with the weighting factor with the sum of regions affected gives a final mSWAT score. The SWAT result is a number is monitored over time. A stable number suggested a stable condition. A lower number suggested less active CTCL and conversely, a higher number suggested more active CTCL.
Time frame: Within 18 months after PTX-100 initial dose.
Progression-free survival (PFS)
Defined as the time from the date of PTX-100 initiation to the date of first documented progression or death.
Time frame: Within 18 months after PTX-100 initial dose.
Time to response (TTR)
TTR: defined as the time from the date of PTX-100 initiation to first confirmed objective response (CR or PR)
Time frame: Within 18 months after PTX-100 initial dose.
Overall survival (OS)
OS: defined as the time interval between the date of PTX-100 initiation and the date of death due to any cause
Time frame: Within 18 months after PTX-100 initial dose.
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University of California Irvine
Irvine, California, United States
RECRUITINGYale Cancer Center
New Haven, Connecticut, United States
RECRUITINGDana-Farber Cancer Institute
Boston, Massachusetts, United States
RECRUITINGRochester Skin Lymphoma Medical Group. PLLC
Rochester, New York, United States
RECRUITINGVirginia Commonwealth University Massey Comprehensive Cancer Cente
Richmond, Virginia, United States
RECRUITINGWestmead Hospital
Westmead, New South Wales, Australia
RECRUITINGEpworth Healthcare
Melbourne, Victoria, Australia
RECRUITINGLinear Clinical Research
Nedlands, Western Australia, Australia
RECRUITINGCHU de Bordeaux - Hopital Saint André
Bordeaux, Bordeaux, France
NOT_YET_RECRUITING...and 5 more locations
Duration of response (DoR)
DoR: defined as the time interval between the first confirmed objective response (CR or PR) and the first occurrence of objective progression (PD) or death from any cause.
Time frame: Within 18 months after PTX-100 initial dose.
Complete response rate (CRR)
Complete response rate (CRR) evaluated according to the Lugano2014 criteria.
Time frame: Within 18 months after PTX-100 initial dose.
Pharmacokinetics (PK) of PTX-100: Cmax
PTX-100 peak plasma concentration (Cmax) in plasma
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 to peak drug concentration in plasma (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
Cmin for the minimum blood plasma concentration reached by PTX-100 during the time interval between administration of two doses
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
AUCtau, area under the curve from time 0 to time tau (the dosing interval) at steady state. AUCtau is typically expressed in units of concentration multiplied by time (e.g., ng·h/mL)
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.