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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06854653
Registration number
NCT06854653
Ethics application status
Date submitted
18/02/2025
Date registered
3/03/2025
Date last updated
31/05/2025
Titles & IDs
Public title
A Phase 2 Study of PTX 100 in Patients With Relapsed/Refractory CTCL
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Scientific title
An Open-Label, Phase 2 Study of PTX-100 Monotherapy in Patients With Relapsed or Refractory Cutaneous T-Cell Lymphoma.
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Secondary ID [1]
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PTX-100-02-2024
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
CTCL
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Condition category
Condition code
Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - PTX-100
Experimental: Phase 2a PTX-100 500mg/m2 - 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.
Experimental: Phase 2a PTX-100 1000mg/m2 - 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.
Experimental: Phase 2b PTX-100 Recommended Optimal Dose (ROD). - 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.
Treatment: Drugs: PTX-100
Peptidomimetic inhibitor of GGTase 1
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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To determine the efficacy of PTX-100 as determined by ORR.
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Assessment method [1]
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Objective response rate (ORR): ORR is defined as the proportion of patients who achieved CR or PR as their best response
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Timepoint [1]
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18 months from day of first treatment until disease progression, unacceptable toxicity, participant or Investigator decision.
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Secondary outcome [1]
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To further characterize the efficacy of PTX-100
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Assessment method [1]
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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.
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Timepoint [1]
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Within 18 months after PTX-100 initial dose.
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Secondary outcome [2]
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Progression-free survival (PFS)
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Assessment method [2]
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Defined as the time from the date of PTX-100 initiation to the date of first documented progression or death.
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Timepoint [2]
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Within 18 months after PTX-100 initial dose.
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Secondary outcome [3]
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Time to response (TTR)
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Assessment method [3]
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TTR: defined as the time from the date of PTX-100 initiation to first confirmed objective response (CR or PR)
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Timepoint [3]
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Within 18 months after PTX-100 initial dose.
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Secondary outcome [4]
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Overall survival (OS)
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Assessment method [4]
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OS: defined as the time interval between the date of PTX-100 initiation and the date of death due to any cause
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Timepoint [4]
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Within 18 months after PTX-100 initial dose.
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Secondary outcome [5]
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Duration of response (DoR)
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Assessment method [5]
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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.
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Timepoint [5]
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Within 18 months after PTX-100 initial dose.
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Secondary outcome [6]
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Complete response rate (CRR)
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Assessment method [6]
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Complete response rate (CRR) evaluated according to the Lugano2014 criteria.
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Timepoint [6]
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Within 18 months after PTX-100 initial dose.
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Secondary outcome [7]
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Pharmacokinetics (PK) of PTX-100: Cmax
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Assessment method [7]
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PTX-100 peak plasma concentration (Cmax) in plasma
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Timepoint [7]
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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.
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Secondary outcome [8]
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Pharmacokinetics (PK) of PTX-100: Tmax
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Assessment method [8]
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Time to peak drug concentration in plasma (Tmax)
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Timepoint [8]
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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.
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Secondary outcome [9]
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Pharmacokinetics (PK) of PTX-100: Cmin
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Assessment method [9]
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Cmin for the minimum blood plasma concentration reached by PTX-100 during the time interval between administration of two doses
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Timepoint [9]
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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.
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Secondary outcome [10]
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Pharmacokinetics (PK) of PTX-100: AUCtau
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Assessment method [10]
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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)
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Timepoint [10]
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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.
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Eligibility
Key inclusion criteria
1. Adult patient =18 years of age at the time of signing the informed consent.
2. Patient is capable of giving adequate signed informed consent
3. Have a confirmed diagnosis of CTCL with histological confirmation
4. Patients must have greater than or equal to Stage Ib disease.
5. Has received and failed (or intolerant of) at least 2 prior lines of prior systemic therapy for their disease.
6. 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.
7. On a stable dose of systemic corticosteroid (< 10 mg prednisone or equivalent) are permitted. Participants on a stable dose of topical corticosteroids are permitted.
8. Washout period- must be 2 weeks (4 weeks for monoclonal antibodies) or 5 -half-lives (whichever is longer) since any prior anti-cancer therapy.
9. Must be human T-cell lymphotropic virus type 1 (HTLV1) negative.
10. Has an ECOG PS of 0 to 2.
11. Life expectancy of 3 months or greater
12. Has adequate bone marrow function.
13. Has adequate hepatic function.
14. Has adequate Renal function.
15. Has adequate coagulation function.
16. Patients with Human Immunodeficiency virus (HIV) must be on established and stable effective anti-retroviral therapy for at least 4 weeks and have an HIV viral load of less than 400 copies/mL.
17. Male patients are eligible to participate if they agree to use a highly effective contraception during the treatment period and for at least 3 months after the last dose of study treatment and refrain from donating sperm during this period.
18. Female patients are eligible to participate if they are not pregnant, not breastfeeding, and at least one of the following conditions applies:
-Not a woman of childbearing potential (WOCBP).
* OR
* A WOCBP who agrees to use a contraceptive method that is highly effective (with a failure rate of < 1% per year) or be abstinent from heterosexual intercourse as their preferred method and usual lifestyle, beginning the time of informed consent, during the treatment period and for at least 3 months after the last dose of study treatment.
19. A WOCBP must have a negative serum pregnancy within 72 hours of the first dose of study treatment.
20. Must be willing and able to adhere to the study as judged by the Investigator.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Patients with known central nervous system involvement.
2. 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.
3. Significant cardiovascular disease. A history of, or concurrent interstitial lung disease or severely impaired lung function.
5. Active viral, bacterial, fungal infection or other serious infection requiring ongoing systemic treatment. Routine antimicrobial prophylaxis is permitted.
6. 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.
7. On an immunomodulatory drug for concomitant or intercurrent conditions or who have received any of these agents within 4 weeks of baseline.
8. 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.
9. 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.
10. Prior allogeneic or autologous hematopoietic transplantation 11. Has a known psychiatric disorder that would interfere with compliance with the requirements of the study.
12. 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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
28/03/2025
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/06/2028
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Actual
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Sample size
Target
115
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,VIC,Washingto
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Recruitment hospital [1]
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Westmead Hospital - Westmead
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Recruitment hospital [2]
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Epworth Healthcare - Melbourne
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Recruitment hospital [3]
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Linear Clinical Research - Nedlands
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment postcode(s) [2]
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3002 - Melbourne
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Recruitment postcode(s) [3]
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6009 - Nedlands
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Connecticut
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Country [2]
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United States of America
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State/province [2]
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Florida
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Country [3]
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United States of America
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State/province [3]
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Virginia
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Country [4]
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France
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State/province [4]
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Île-de-France
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Country [5]
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Italy
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State/province [5]
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Milano
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Country [6]
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Italy
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State/province [6]
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Bologna
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Country [7]
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Italy
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State/province [7]
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Brescia
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Prescient Therapeutics, Ltd.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/study/NCT06854653
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Address
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Upaly Bahadure
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Address
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Country
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Phone
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+61 3 9692 7222
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Fax
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Email
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upaly@ptxtherapeutics.com
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Contact person for scientific queries
Data sharing statement
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT06854653
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