An Open Label Dose Finding Study of PTT-4256 in Patients With Solid Tumours (RAISIC-1).

NCT06634849 | Recruiting Phase 1 DMC

• 1 other identifier: PTT-4256-01
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 5

Brief Summary

This open label, dose escalation module will evaluate the safety, tolerability, PK, PD, and preliminary efficacy of PTT-4256 in participants with solid tumours using a combination of accelerated dose titration (ADT) and Bayesian Optimal Interval (BOIN) design.

Conditions

Advanced Solid Tumor

Keywords

PTT-4256, GPR65, oncology, cancer, RAISIC-1

Outcome Measures

Primary Outcomes (1)

• Number of participants with Treatment emergent adverse events (TEAEs), Adverse events and Serious Adverse events as assessed by CTCAE V5.0

  First dose IMP to 21 to 28 days after last IMP dose.

Secondary Outcomes (19)

• To determine the maximum tolerated dose (MTD)

  Baseline to 21 to 28 days after last IMP dose.

• To determine OBD

  Baseline to 21 to 28 days after last IMP dose.

• To determine R2PD

  Baseline to 21 to 28 days after last IMP dose.

• Plasma pharmacokinetics at single dose- Maximum observed concentration (Cmax)

  Pre-dose, 2hours, 4hours, 8hours, and 12 hours Day 1, then at 24hours (Day2), 48 hours (Day 3) and 72 hours (Day4)

• Plasma pharmacokinetics at single dose- Time to maximum concentration (Tmax)

  Pre-dose, 2hours, 4hours, 8hours, and 12 hours Day 1, then at 24hours (Day2), 48 hours (Day 3) and 72 hours (Day4)

Study Arms (1)

PTT-4256

EXPERIMENTAL

Oral administration of PTT-4256 tablet with water

Drug: PTT-4256

Interventions

PTT-4256 DRUG

Single oral intake of PTT-4256 followed by treatment-free period of 3 days to assess safety, PK and PD. After 72-hr post dose PK sample, first 21day cycle of once daily PTT-4256 will begin to assess DLTs. Cohorts A1-A5 will receive 10mg, 20mg, 40mg, 80mg \& 160mg. Following review by SRC, Cohort A6 participants will receive 300mg PTT-4256 daily.

PTT-4256

Eligibility Criteria

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

You may qualify if:

• ≥ 18 years of age at the time of consent.
• Participant has given written informed consent to participate in the study and is able and willing to adhere to the study protocol.
• Participant has cytologically or histologically confirmed solid malignancy and has locally advanced or metastatic disease. Melanoma, non-small cell lung cancer, renal cell carcinoma, metastatic castrate-resistant prostate cancer, cervix cancer, triple negative breast cancer, colorectal cancer, gastric cancer are preferred solid tumours.
• Participant must require systemic treatment for their tumour and either:
• be refractory to,
• have progressed on,
• be intolerant to, or
• be not otherwise a candidate - in the opinion of the Investigator - for any of the currently available standard treatments.
• Participant has measurable disease per RECIST v1.1. Participants with non-measurable disease per RECIST v1.1 might be considered eligible upon discussion with the Sponsor.
• Participant has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
• Participant has an estimated life expectancy of at least 3 months in the opinion of the Investigator.
• Adequate haematological (blood or platelet transfusion not allowed within 7 days prior to Screening), liver, and renal function defined below (repeat measurement of borderline values permitted):
• Haemoglobin ≥ 8.5 g/dL,
• Absolute neutrophil count (ANC) ≥ 1.5 × 109/L,
• Platelet count ≥ 90 x 109/L,

You may not qualify if:

• Inability or unwillingness to adhere to the study protocol, including study procedures and oral intake of the IP.
• Active primary central nervous system (CNS) malignancy, active CNS metastases or leptomeningeal disease. Participants with previously treated primary CNS malignancy or CNS metastases are eligible to participate if:
• they have stable and controlled neurological symptoms without deterioration;
• they have stable disease as assessed by imaging (preferably contrast-enhanced MRI) for at least 28 days prior to first IMP administration;
• they have no evidence of new or enlarging brain metastases; and
• they are not using corticosteroids for at least 7 days prior to first IMP administration.
• Unresolved or unstable serious toxic side effects of prior chemotherapy or radiotherapy, ie, ≥ Grade 2 per CTCAE v5.0 except fatigue, alopecia, infertility, or those relating to palliative radiotherapy within 6 weeks prior to first IMP administration. Participants with residual AEs \> Grade 1 considered not clinically significant may be considered eligible on a case-by-case basis, in discussion with the Sponsor.
• Concurrent active or previous history of other malignancy within the past 2 years before first IMP administration except:
• Malignancy (other than in situ) treated with curative intent and with no known active disease present for ≥ 2 years before first IMP administration and felt to be at low risk of recurrence by the Investigator;
• Adequately treated non-melanoma skin cancer or lentigo malignant with no evidence of disease;
• Adequately treated in situ cancer without evidence of disease.
• Received anti-cancer therapy (including chemotherapy, immunotherapy, radiation therapy, biologic therapy, or any investigational therapy) within 28 days or 5 half-lives of the therapeutic agent, whichever is shorter, prior to the first IMP administration. Palliative adiotherapy given within 28 days prior to the first IMP administration may be considered eligible on a case-by-case basis, in discussion with the Sponsor.
• Uncontrolled symptomatic malignant effusion(s) or those requiring recurrent drainage in the opinion of the Investigator.
• Participants with clinically significant active autoimmune or chronic inflammatory disease that is not well controlled with standard therapy in the opinion of the Investigator.
• Grade 3 or higher immunotherapy-induced autoimmune hepatitis.

Sponsors & Collaborators

• Pathios Therapeutics Pty Ltd: lead

Study Sites (5)

Blacktown Hospital

Blacktown, New South Wales, 2148, Australia

RECRUITING

Scientia Clinical Research

Randwick, New South Wales, 2031, Australia

RECRUITING

Southern Oncology Clinical Research Unit (SOCRU)

Adelaide, South Australia, 5042, Australia

RECRUITING

Austin Health

Heidelberg, Victoria, 3084, Australia

RECRUITING

Linear Clinical Research

Nedlands, Western Australia, 6009, Australia

RECRUITING

MeSH Terms

Conditions

Neoplasms

Central Study Contacts

Debbie Macaro

CONTACT

+44 1235 644 96; enquiries@pathios.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 20, 2024
• First Posted: October 10, 2024
• Study Start: November 4, 2024
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): October 30, 2027
• Last Updated: February 10, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

AU (5)