A Phase 2 Study of PTX 100 in Patients With Relapsed/Refractory CTCL
ConditionCTCL
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorPrescient Therapeutics, Ltd.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Parallel
Treatments tested in this trial
PTX-100
DrugPeptidomimetic inhibitor of GGTase 1
Treatment groups
115 Participants
are divided into 3 treatment groupsGroup A: Phase 2a PTX-100 500mg/m2Experimental treatment 1 intervention
Group B: Phase 2a PTX-100 1000mg/m2Experimental treatment 1 intervention
Group C: Phase 2b PTX-100 Recommended Optimal Dose (ROD).Experimental treatment 1 intervention
Trial outcomes
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
Time frame
18 months from day of first treatment until disease progression, unacceptable toxicity, participant or Investigator decision.Secondary outcomes
1
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.2
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.3
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.4
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.Other outcomes
1
To evaluate the effects of Health-related Quality of Life (QoL)
To collect the health related questionnaire for CTCL whihc includes Functional Assessment of Cancer Therapy -General (FACT-G), Skindex-29, European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3L) and ItchyQoL
Time frame
From Baseline till end of study2
Exploratory Biomarkers
Assessing Biomarker downstream signature of PTX-100 treatment: measurement value (and the change from baseline and throughout the study) of gene/ protein expression in blood samples for each subject using Olink Explore HT high-throughput platform
Time frame
From Baseline till end of studySponsors and contacts
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Source ClinicalTrials.gov