Advanced CancerPhase 1NCT03900442
Phase 1 Study of PTX-100 in Patients With Advanced Malignancies With PTCL Expansion Cohort
A Phase 1 study testing PTX-100 in people with Advanced Cancer and PTCL, run by Prescient Therapeutics, Ltd. at 1 site in Australia.
Trial summary
This is an open-label, non-randomized study to evaluate the PD, PK, and safety of 500 to 2000 mg/m2 PTX-100 in patients with advanced malignancies. PTX-100 will be administered by IV infusion over 60 minutes on days 1 to 5 of a 14-day cycle for 4 cycles unless toxicity is observed. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Cancer, PTCL
- What is being tested?
- PTX-100
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteAustralia
- Study dates
- 1 Sep 2019to 8 Aug 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Biopsy proven multiple myeloma (MM), peripheral T-cell lymphoma (CTCL, AITL or PTCL-NOS), colo-rectal cancer (CRC), pancreas cancer (PANC), or diffuse gastric cancer (DGC)
- Must have a relapsed or refractory advanced malignancy for which no standard therapy exists.
- Must have at least 6 unstained slides of archived formalin-fixed, paraffin-embedded tumor tissue available for genotyping studies; if insufficient or no archived tissue is available, a fresh tumor biopsy within 30 days prior to Cycle 1/Day 1 is mandatory.
- Age ≥ 18 years
- ECOG performance status ≤ 2
- Adequate hematological function: absolute neutrophil count (ANC) ≥ 1000/mm3, platelet count ≥ 50,000 mm3
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- Adequate hepatic function: total bilirubin ≤ 1.5 times the upper limit of normal (ULN), and alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 times ULN (patients with liver metastases may be enrolled with elevated hepatic function based on Medical Monitor and Investigator review and agreement)
- Adequate renal function: measured, calculated or estimated creatinine clearance of ≥ 50 mL/min
- Female patients of childbearing potential must have a negative serum pregnancy test at screening and agree to use dual methods of contraception. Male patients must use an effective barrier method of contraception if sexually active with a female of childbearing potential. Acceptable methods of contraception are condoms with contraceptive foam, oral, implantable or injectable contraceptives, contraceptive patch, intrauterine device, diaphragm with spermicidal gel, or a sexual partner who is surgically sterilized or postmenopausal. For both male and female patients, effective methods of contraception must be used throughout the study and for 3 months following the last dose.
- Informed consent (current IRB approved version) must be obtained from the patient or legally authorized representative prior to any study-related procedures.
Who cannot join
- Radiation, chemotherapy, immunotherapy, or any other approved anticancer therapy ≤ 2 weeks prior to study treatment
- Participation in another interventional investigational drug study within 4 weeks prior to enrollment
- Concurrent radiation, chemotherapy, immunotherapy, or any other approved or investigational anticancer therapeutic (Patients are allowed to receive ≤ 10 mg/day corticosteroids for the treatment of non malignant disorders.)
- Myocardial infarction within 6 months before screening, New York Heart Association Class II or greater heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, clinically significant pericardial disease, or electrocardiographic evidence of acute ischemic or active conduction system abnormalities
- Uncontrolled infection requiring parenteral antibiotics, antivirals, or antifungals within 1 week prior to first dose (Patients with controlled infection or on prophylactic antibiotics are permitted in the study.)
- Known to be HIV seropositive
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- Known active hepatitis A, B, or C infection or known to be positive for HCV RNA or HBsAg (HBV surface antigen)
- Grade > 2 peripheral neuropathy at screening
- Previous allogeneic transplant within the past 6 months or evidence of clinically significant graft-versus-host disease (if prior bone marrow transplant)
- Any history of malignancy, other than that treated in this study, unless the patient has remained free of the disease for over 3 years (except for properly treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix or breast)
- Any active medical or psychiatric illness that, in the judgement of the investigator, may interfere with adherence to the protocol
- Any malignancy with CNS involvement
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to PTX-100
- Female patients who are pregnant or lactating
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase 1 Pharmacodynamic and Pharmacokinetic Study of the Geranylgeranyltransferase I Inhibitor PTX-100 (GGTI-2418) in Patients With Advanced Malignancies
This is an open-label, non-randomized study to evaluate the PD, PK, and safety of 500 to 2000 mg/m2 PTX-100 in patients with advanced malignancies.
PTX-100 will be administered by IV infusion over 60 minutes on days 1 to 5 of a 14-day cycle for 4 cycles unless toxicity is observed.
Dose escalation is complete and the expansion is open and actively recruiting PTCL patients.
- Conditions
- Advanced Cancer, PTCL
- Being tested
- PTX-100
- Keywords
- PTCL
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PTX-100Drug
Doses of 500 to 2000 mg/m2 will be administered.
| Study group | Role | What they receive |
|---|---|---|
| PTX-100 | Experimental | IV infusion over 60 minutes on days 1 to 5 of a 14-day cycle for 4 cycles Receives: Drug: PTX-100 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Investigate the time- and dose-dependent PD of multiple doses of PTX-100 in patients with advanced malignancies (RAP-1)
PD endpoints will be based on analysis of level of RAP-1 geranylgeranylation proteins in PBMNCs and tumor biopsies:
Measured: One Cycle (cycle = 14 days)
Investigate the time- and dose-dependent PD of multiple doses of PTX-100 in patients with advanced malignancies (PD endpoints)
PD endpoints will be based on analysis of the level of DJ2 farnesylation proteins in PBMNCs and tumor biopsies:
Measured: One Cycle (cycle = 14 days)
Investigate the time- and dose-dependent PK of multiple doses of PTX-100 in patients with advanced malignancies (cmax)
Pharmacokinetics will be evaluated by PK parameters calculated from plasma concentrations of PTX 100. Individual and mean plasma concentrations of PTX-100 versus time as well as individual and mean PK parameters will be determined, for the following:
- Maximum observed plasma concentration (Cmax)
Measured: One Cycle (cycle = 14 days)
Investigate the time- and dose-dependent PK of multiple doses of PTX-100 in patients with advanced malignancies (Tmax)
Pharmacokinetics will be evaluated by PK parameters calculated from plasma concentrations of PTX 100. Individual and mean plasma concentrations of PTX-100 versus time as well as individual and mean PK parameters will be determined, for the following:
- Time to maximum observed plasma concentration (Tmax)
Measured: One Cycle (cycle = 14 days)
Investigate the time- and dose-dependent PK of multiple doses of PTX-100 in patients with advanced malignancies (half life)
Pharmacokinetics will be evaluated by PK parameters calculated from plasma concentrations of PTX 100. Individual and mean plasma concentrations of PTX-100 versus time as well as individual and mean PK parameters will be determined, for the following:
- Apparent elimination half-life (t1/2)
Measured: One Cycle (cycle = 14 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2019
- Study start1 Sep 2019
- Primary completion31 Jul 2025
- Study completion8 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prescient Therapeutics, Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Terrence Chew, MD, Study director, Prescient Therapeutics, Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03900442
- Sponsor's protocol ID
- PTX-100-PD-012017
- Status last verified
- Aug 2025
- First submitted
- 13 Mar 2019
- Last updated
- 2 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Pharmacodynamic and Pharmacokinetic Study of the Geranylgeranyltransferase I Inhibitor PTX-100 (GGTI-2418) in Patients With Advanced Malignancies
- ClinicalTrials.gov ID
- NCT03900442
- Acronym / sponsor ID
- PTX-100-PD-012017
- Conditions
- Advanced Cancer, PTCL
- Keywords
- PTCL
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Prescient Therapeutics, Ltd.Industry (pharma, biotech, devices)
- Study start
- 1 Sep 2019 (actual)
- Primary completion
- 31 Jul 2025 (actual)
- Study completion
- 8 Aug 2025 (actual)
- First posted
- 3 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label, non-randomized study to evaluate the PD, PK, and safety of 500 to 2000 mg/m2 PTX-100 in patients with advanced malignancies.
PTX-100 will be administered by IV infusion over 60 minutes on days 1 to 5 of a 14-day cycle for 4 cycles unless toxicity is observed.
Dose escalation is complete and the expansion is open and actively recruiting PTCL patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PTX-100 | Experimental | Drug: PTX-100IV infusion over 60 minutes on days 1 to 5 of a 14-day cycle for 4 cycles |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Biopsy proven multiple myeloma (MM), peripheral T-cell lymphoma (CTCL, AITL or PTCL-NOS), colo-rectal cancer (CRC), pancreas cancer (PANC), or diffuse gastric cancer (DGC)
- Must have a relapsed or refractory advanced malignancy for which no standard therapy exists.
- Must have at least 6 unstained slides of archived formalin-fixed, paraffin-embedded tumor tissue available for genotyping studies; if insufficient or no archived tissue is available, a fresh tumor biopsy within 30 days prior to Cycle 1/Day 1 is mandatory.
- Age ≥ 18 years
- ECOG performance status ≤ 2
Key exclusion criteria (14 listed)
- Radiation, chemotherapy, immunotherapy, or any other approved anticancer therapy ≤ 2 weeks prior to study treatment
- Participation in another interventional investigational drug study within 4 weeks prior to enrollment
- Concurrent radiation, chemotherapy, immunotherapy, or any other approved or investigational anticancer therapeutic (Patients are allowed to receive ≤ 10 mg/day corticosteroids for the treatment of non malignant disorders.)
- Myocardial infarction within 6 months before screening, New York Heart Association Class II or greater heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, clinically significant pericardial disease, or electrocardiographic evidence of acute ischemic or active conduction system abnormalities
- Uncontrolled infection requiring parenteral antibiotics, antivirals, or antifungals within 1 week prior to first dose (Patients with controlled infection or on prophylactic antibiotics are permitted in the study.)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PTX-100Doses of 500 to 2000 mg/m2 will be administered. | Drug | PTX-100 |
Primary outcome
Investigate the time- and dose-dependent PD of multiple doses of PTX-100 in patients with advanced malignancies (RAP-1)
Time frame: One Cycle (cycle = 14 days)
PD endpoints will be based on analysis of level of RAP-1 geranylgeranylation proteins in PBMNCs and tumor biopsies:
Investigate the time- and dose-dependent PD of multiple doses of PTX-100 in patients with advanced malignancies (PD endpoints)
Time frame: One Cycle (cycle = 14 days)
PD endpoints will be based on analysis of the level of DJ2 farnesylation proteins in PBMNCs and tumor biopsies:
Investigate the time- and dose-dependent PK of multiple doses of PTX-100 in patients with advanced malignancies (cmax)
Time frame: One Cycle (cycle = 14 days)
Pharmacokinetics will be evaluated by PK parameters calculated from plasma concentrations of PTX 100. Individual and mean plasma concentrations of PTX-100 versus time as well as individual and mean PK parameters will be determined, for the following: • Maximum observed plasma concentration (Cmax)
Investigate the time- and dose-dependent PK of multiple doses of PTX-100 in patients with advanced malignancies (Tmax)
Time frame: One Cycle (cycle = 14 days)
Pharmacokinetics will be evaluated by PK parameters calculated from plasma concentrations of PTX 100. Individual and mean plasma concentrations of PTX-100 versus time as well as individual and mean PK parameters will be determined, for the following: • Time to maximum observed plasma concentration (Tmax)
Investigate the time- and dose-dependent PK of multiple doses of PTX-100 in patients with advanced malignancies (half life)
Time frame: One Cycle (cycle = 14 days)
Pharmacokinetics will be evaluated by PK parameters calculated from plasma concentrations of PTX 100. Individual and mean plasma concentrations of PTX-100 versus time as well as individual and mean PK parameters will be determined, for the following: • Apparent elimination half-life (t1/2)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Terrence Chew, MD study director
Prescient Therapeutics, Ltd.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PTX-100 in people with Advanced Cancer and PTCL, run by Prescient Therapeutics, Ltd. at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Investigate the time- and dose-dependent PD of multiple doses of PTX-100 in patients with advanced malignancies (RAP-1) (time frame: One Cycle (cycle = 14 days)). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Sep 2025.