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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04907851


Additional trial details provided through ANZCTR are available at the end of this record.


Registration number
NCT04907851
Ethics application status
Date submitted
25/05/2021
Date registered
1/06/2021
Date last updated
29/09/2021

Titles & IDs
Public title
A Study to Assess RXC004 Efficacy in Advanced Solid Tumours After Progression on Standard of Care (SoC) Therapy (PORCUPINE2)
Scientific title
A Modular, Phase II, Open-Label, Multicentre Study to Assess the Preliminary Efficacy and Safety of RXC004, in Patients With Advanced Solid Tumours That Have Progressed Following Therapy With Current Standard of Care
Secondary ID [1] 0 0
RXC004/0003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Advanced Solid Tumours 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - RXC004
Other interventions - Denosumab

Experimental: Module 1 - RNF43 Mutated Advanced (unresectable)/Metastatic Pancreatic Cancer (Stage III/IV) - Patients (Karnofsky performance status =70) will be recruited and dosed with RXC004 (2 mg once daily [QD], orally) within 6 weeks of progression following 1st line SoC treatment.

Experimental: Module 2 -Advanced (unresectable)/Metastatic Biliary Tract Cancer (Stage III/IV) - Patients (Eastern Cooperative Oncology Group [ECOG] performance status 0-1) will be recruited and dosed with RXC004 (2 mg QD, orally) within 6 weeks of progression, following 1st line SoC treatment.


Treatment: Drugs: RXC004
RXC004 will be administered orally, 2 mg QD Dose Formulation: 0.5 mg or 1 mg capsules.

Other interventions: Denosumab
Denosumab will be administered via subcutaneous (SC) injection, 120 mg once every month. Use: Prophylactic

Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Progression free survival (percent) rate at 6 months using Investigator assessment according to Response Evaluation Criteria in Solid Tumours, version 1.1 (RECIST 1.1) - To assess the anti-tumour activity of RXC004. Progression free survival rate at 6 months is defined as the proportion of patients who remain alive and free of progression at 6 months.
Timepoint [1] 0 0
At 6 months
Secondary outcome [1] 0 0
Objective Response Rate (ORR) using Investigator assessments according to RECIST 1.1 - To further assess the preliminary efficacy of RXC004. ORR is defined as the proportion of patients with a best overall response of complete response (CR) or partial response (PR), based on local Investigator assessment, as defined in RECIST 1.1.
Timepoint [1] 0 0
Up to 20.5 months
Secondary outcome [2] 0 0
Disease Control Rate (DCR) using Investigator assessments according to RECIST 1.1 - To further assess the preliminary efficacy of RXC004. DCR is defined as the proportion of patients with a best overall response of either CR, PR or stable disease (SD) for at least 6 weeks.
Timepoint [2] 0 0
Up to 20.5 months
Secondary outcome [3] 0 0
PFS using Investigator assessments according to RECIST 1.1 - To further assess the preliminary efficacy of RXC004. PFS is defined as the time from first dose of study treatment until the date of disease progression or death (by any cause in the absence of progression), regardless whether the patient withdraws from the assigned study treatment or receives another anticancer prior to progression.
Timepoint [3] 0 0
Up to 20.5 months
Secondary outcome [4] 0 0
Percentage change in the sum of target lesions using Investigator assessments according to RECIST 1.1 - To further assess the preliminary efficacy of RXC004. Percentage change in tumour size will be derived at each visit by the percentage change from baseline in the sum of diameters of target lesions. The best percentage change in tumour size will be the patients value representing the largest decrease (or smallest increase) from baseline in tumour size.
Timepoint [4] 0 0
Up to 20.5 months
Secondary outcome [5] 0 0
Overall survival (OS) - To further assess the preliminary efficacy of RXC004. OS is defined as the time from first day of study treatment until death due to any cause.
Timepoint [5] 0 0
Up to 20.5 months
Secondary outcome [6] 0 0
Maximum observed plasma concentration (Cmax) - To assess the pharmacokinetic (PK) of RXC004.
Timepoint [6] 0 0
At Cycle 0 and Cycle 1 (Each cycle is 21 days in length)
Secondary outcome [7] 0 0
Time to Cmax (tmax) - To assess the PK of RXC004.
Timepoint [7] 0 0
At Cycle 0 and Cycle 1 (Each cycle is 21 days in length)
Secondary outcome [8] 0 0
Minimum observed concentration across the dosing interval (Cmin) - To assess the PK of RXC004.
Timepoint [8] 0 0
At each treatment cycle (Each cycle is 21 days in length), up to 20.5 months
Secondary outcome [9] 0 0
Terminal rate constant (?z) - To assess the PK of RXC004.
Timepoint [9] 0 0
At Cycle 0 and Cycle 1 (Each cycle is 21 days in length)
Secondary outcome [10] 0 0
Terminal half-life (t½) - To assess the PK of RXC004.
Timepoint [10] 0 0
At Cycle 0 and Cycle 1 (Each cycle is 21 days in length)
Secondary outcome [11] 0 0
Area under the plasma concentration-time curve from zero to infinity (AUC0-8) - To assess the PK of RXC004.
Timepoint [11] 0 0
At Cycle 0 and Cycle 1 (Each cycle is 21 days in length)
Secondary outcome [12] 0 0
Total plasma clearance after oral administration (CL/F) - To assess the PK of RXC004.
Timepoint [12] 0 0
At Cycle 0 and Cycle 1 (Each cycle is 21 days in length)
Secondary outcome [13] 0 0
Apparent volume of distribution after oral administration (Vz/F) - To assess the PK of RXC004.
Timepoint [13] 0 0
At Cycle 0 and Cycle 1 (Each cycle is 21 days in length)
Secondary outcome [14] 0 0
Number of patients with adverse events (AEs) - To assess the safety and tolerability profile of RXC004.
Timepoint [14] 0 0
From time of signature of main study informed consent form throughout the treatment period and until the 30 days after last dose of RXC004 (Up to 20.5 months)

Eligibility
Key inclusion criteria
Core

- At least one lesion that is measurable by RECIST 1.1 at baseline (within 28 days prior
to start of study treatment).

- Mandatory paired biopsies; Patients must have at least one lesion suitable for biopsy
at screening

- Adequate organ and marrow function

- Female patients of childbearing potential must have a negative pregnancy test prior to
start of dosing

- Female patients of childbearing potential and male patients with female partners of
childbearing potential must agree to use a highly effective method of contraception
during the study and for at least 5 months after the last dose of study drug.

Module 1 (PDAC) Specific Inclusion Criteria

- Histological documentation of advanced (unresectable)/metastatic (Stage III/IV) PDAC,
with documented loss of function tumour mutation in RNF43

- Patients must have received one prior systemic treatment for advanced
(unresectable)/metastatic PDAC (Stage III/IV), with clear evidence of radiological
disease progression

- Patients must be enrolled and receive first dose of study treatment within 6 weeks of
radiologically confirmed RECIST1.1 progression

- Karnofsky performance status =70.

Module 2 (BTC) Specific Inclusion Criteria

- Histological documentation of advanced (unresectable)/metastatic (Stage III/IV) BTC
(intrahepatic or extrahepatic cholangiocarcinoma, ampulla of Vater, or gallbladder
cancer)

- Patients must have received one prior systemic treatment for advanced
(unresectable)/metastatic BTC, with clear evidence of radiological disease progression

- Patients must be enrolled and receive first dose of study treatment within 6 weeks of
radiologically confirmed RECIST1.1 progression

- ECOG status 0 or 1.

Core
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Prior therapy with a compound of the same mechanism of action as RXC004

- Patients at higher risk of bone fractures

- Any known uncontrolled inter-current illness or persistent clinically significant
toxicity related to prior anti-cancer treatment

- Patients who have any history of an active (requiring treatment) other malignancy
within 2 years of study entry

- Patients with known or suspected brain metastases

- Use of anti-neoplastic agents and other investigational drugs within 4 weeks prior to
the first dose of study treatment

- Patients with a known hypersensitivity to any RXC004 excipients

- Patients with a contra-indication for denosumab treatment

- Patients who are pregnant or breast-feeding

- Known active human immunodeficiency viruses (HIV), hepatitis B (HBV), or hepatitis C
(HCV) infections

- Use of any live vaccines against infectious diseases (e.g., influenza, varicella)
within 4 weeks (28 days) of initiation of study treatment.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
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
Parallel
Other design features
Phase
Phase 2
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
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Wollongong Hospital - Wollongong
Recruitment postcode(s) [1] 0 0
2500 - Wollongong
Recruitment outside Australia
Country [1] 0 0
United Kingdom
State/province [1] 0 0
Aberdeen
Country [2] 0 0
United Kingdom
State/province [2] 0 0
Belfast
Country [3] 0 0
United Kingdom
State/province [3] 0 0
Birmingham
Country [4] 0 0
United Kingdom
State/province [4] 0 0
Cambridge
Country [5] 0 0
United Kingdom
State/province [5] 0 0
Glasgow
Country [6] 0 0
United Kingdom
State/province [6] 0 0
London
Country [7] 0 0
United Kingdom
State/province [7] 0 0
Manchester
Country [8] 0 0
United Kingdom
State/province [8] 0 0
Oxford
Country [9] 0 0
United Kingdom
State/province [9] 0 0
Sheffield
Country [10] 0 0
United Kingdom
State/province [10] 0 0
Surrey
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Wirral

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Redx Pharma Plc
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study is to evaluate the preliminary efficacy and safety of RXC004 monotherapy in
advanced solid tumours that have progressed following SoC treatment.
Trial website
https://clinicaltrials.gov/show/NCT04907851
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
Craig Tilston
Address 0 0
Country 0 0
Phone 0 0
+44(0) 1625 469908
Fax 0 0
Email 0 0
c.tilston@redxpharma.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04907851

Additional trial details provided through ANZCTR
Accrual to date
Recruiting in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 45
Wollongong Hospital
Recruitment postcode(s) [1] 50
2500
Funding & Sponsors
Funding source category [1] 63
Charities/Societies/Foundations
Name [1] 63
Australian Genomic Cancer Medicine Centre Ltd t/a Omico
Address [1] 63
Level 7, 370 Victoria Street Darlinghurst NSW 2010
Country [1] 63
Australia
Funding source category [2] 64
Commercial sector/Industry
Name [2] 64
Redx Pharma Plc
Address [2] 64
Block 33 Mereside, Alderley Park Alderley Edge, Cheshire SK10 4TG
Country [2] 64
United Kingdom
Primary sponsor
Commercial sector/Industry
Primary sponsor name
Redx Pharma Plc
Primary sponsor address
Block 33
Mereside, Alderley Park
Alderley Edge, Cheshire
SK10 4TG
Primary sponsor country
United Kingdom
Secondary sponsor category [1] 62
Charities/Societies/Foundations
Name [1] 62
Australian Genomic Cancer Medicine Centre Ltd t/a Omico
Address [1] 62
Level 7, 370 Victoria Street Darlinghurst NSW 2010
Country [1] 62
Australia
Ethics approval
Ethics application status
Approved
Ethics committee name [1] 40
St Vincent’s Hospital HREC
Address [1] 40
Research Office St Vincent’s Hospital Translational Research Centre 97-105 Boundary Street Darlinghurst NSW 2010
Country [1] 40
Australia
Date submitted for ethics approval [1] 40
Approval date [1] 40
18/08/2021
Ethics approval number [1] 40
 
Public notes

Contacts
Principal investigator
Title 297 0
Dr
Name 297 0
Lorraine Chantrill
Address 297 0
Head of Department Medical Oncology L3 Illawarra Cancer Care Centre Wollongong Hospital New Dapto Rd Wollongong NSW 2500
Country 297 0
Australia
Phone 297 0
+61 2 4222 5260
Fax 297 0
Email 297 0
Lorraine.chantrill@health.nsw.gov.au
Contact person for public queries
Title 298 0
Dr
Name 298 0
Enmoore Lin
Address 298 0
The George Institute for Global Health Level 5, 1 King St Newtown NSW 2042
Country 298 0
Australia
Phone 298 0
+61 2 8052 4511
Fax 298 0
Email 298 0
ELin@georgeinstitute.org.au
Contact person for scientific queries
Title 299 0
Mr
Name 299 0
Craig Tilston
Address 299 0
Redx Pharma Plc Block 33 Mereside Alderley Park Alderley Edge, Cheshire SK10 4TG
Country 299 0
United Kingdom
Phone 299 0
+44 1625 469908
Fax 299 0
Email 299 0
c.tilston@redxpharma.com