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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06973863




Registration number
NCT06973863
Ethics application status
Date submitted
7/05/2025
Date registered
15/05/2025
Date last updated
11/09/2025

Titles & IDs
Public title
A Study of PEP08 in Patients With MTAP-Del Advanced or Metastatic Solid Tumors
Scientific title
A Phase 1a/1b Study Evaluating the Clinical Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Anti-tumor Efficacy of PEP08 as Monotherapy and Combination Therapy in MTAP-Del Advanced or Metastatic Solid Tumors
Secondary ID [1] 0 0
PEP08-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Advanced Solid Tumor 0 0
Solid Tumors 0 0
MTAP-deleted Solid Tumors 0 0
MTAP Deletion 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - PEP08

Experimental: PEP08 monotherapy and expansion in advanced or metastatic solid tumors - This arm of the study involves dose escalation to evaluate the tolerability and response of PEP08 monotherapy. Participants in this arm will receive daily dosing and be regularly monitored. Treatment will continue until disease progression, unacceptable toxicity, withdrawal of consent, or study termination.


Treatment: Drugs: PEP08
PEP08 is an oral, potent, MTA-cooperative PRMT5 inhibitor.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants Experiencing Adverse Events (AE)
Timepoint [1] 0 0
Up to 2 years
Primary outcome [2] 0 0
Number of Participants Experiencing Serious Adverse Events (SAE)
Timepoint [2] 0 0
Up to 2 years
Primary outcome [3] 0 0
Number of Patients who Experience Dose-Limiting Toxicity
Timepoint [3] 0 0
21 days
Secondary outcome [1] 0 0
Area under the plasma concentration versus time curve (AUC)
Timepoint [1] 0 0
Up to 6 days
Secondary outcome [2] 0 0
Maximum observed plasma concentration (Cmax)
Timepoint [2] 0 0
Up to 6 days
Secondary outcome [3] 0 0
Maximum observed plasma concentration (Cmin)
Timepoint [3] 0 0
Up to 6 days
Secondary outcome [4] 0 0
Time to achieve maximal plasma concentration (Tmax)
Timepoint [4] 0 0
Up to 6 days
Secondary outcome [5] 0 0
Terminal elimination half-life (t1/2)
Timepoint [5] 0 0
Up to 6 days
Secondary outcome [6] 0 0
Objective Response Rate (ORR) per RECIST v1.1
Timepoint [6] 0 0
Up to 2 years

Eligibility
Key inclusion criteria
* Participants must be adults (=18 years) and sign informed consent before undergoing any study-related procedures.
* Eligible participants must have advanced or metastatic solid tumors that are not treatable with surgery or radiation, and show evidence of MTAP gene homozygous deletion or MTAP protein loss based on tumor tissue analysis.
* Participants must have previously received standard treatment for their cancer type, and either experienced disease progression, be refractory, or be intolerant to such therapies.
* At least one measurable lesion is required, evaluated by standard imaging criteria (RECIST v1.1).
* Good general physical condition (ECOG performance status 0-1 for dose escalation; broader range allowed for other parts).
* Adequate function in key organs.
* Able to swallow oral medication and comply with study requirements.
* Women of childbearing potential and men with reproductive potential must use effective contraception during and after the study.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Recent cancer treatment, immunotherapy, or investigational drugs are not allowed before starting the study.
* Live vaccines received shortly before treatment are not allowed.
* Previous use of drugs with similar mechanisms to the study treatment is not allowed.
* Active or unstable brain or meningeal metastases, unless previously treated and stable without needing local treatment or high-dose steroids.
* History of other cancers within the last 2 years, unless low-risk and treated (e.g., in situ or certain skin cancers).
* Uncontrolled disease-related complications (e.g., abnormal calcium levels, fluid buildup around organs).
* Active HIV, hepatitis B or C infections that are not well-controlled.
* Ongoing serious infections or systemic conditions requiring isolation.
* Significant heart disease, such as recent heart failure, ischemia, or arrhythmias.
* History of severe digestive conditions or surgeries affecting drug absorption.
* Recent major surgery.
* Unresolved serious side effects from prior cancer treatment.
* Currently pregnant or breastfeeding.
* Poorly controlled blood pressure or lung conditions.
* Other serious illnesses (e.g., severe anemia, psychiatric or social issues affecting study compliance).
* Any condition that may pose a safety risk or interfere with the study, as judged by the investigator.
* Known drug or substance abuse that may affect study participation.
* Allergy to the study drug or any of its components.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
15/09/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/03/2028
Actual
Sample size
Target
40
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Westmead Hospital - Wentworthville
Recruitment postcode(s) [1] 0 0
2145 - Wentworthville

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
PharmaEngine
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Project Manager
Address 0 0
Country 0 0
Phone 0 0
+886 2 2515 8228
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

No information has been provided regarding IPD availability


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/NCT06973863