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Trial registered on ANZCTR

Registration number
Ethics application status
Date submitted
Date registered
Date last updated
Date data sharing statement initially provided
Date results information initially provided
Type of registration
Prospectively registered

Titles & IDs
Public title
A single centre, open label, first in human, phase 1 dose escalating study to evaluate the safety, tolerability and preliminary efficacy of an autologous cancer vaccine, RGSH4K, administered intradermally in patients with advanced cancers.
Scientific title
In patients with advanced solid tumours, is an autologous cancer vaccine (RGSH4K), safe and tolerable, and can a biologically active dose be identified?
Secondary ID [1] 286636 0
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Advanced solid tumours 294959 0
Condition category
Condition code
Cancer 295221 295221 0 0
Any cancer

Study type
Description of intervention(s) / exposure
RGSH4K is a vaccine formulated from a patient's own tumour material. Briefly, the tumour cells are lysed or burst to release the proteins and tumour associated antigens. The proteins are further processed in a proprietary procedure and combined with streptavidin, a novel immunostimulant. No live, viable cells remain in the vaccine. Vaccines are prepared on study entry, are stored frozen, and provided individually for injection at Weeks 1, 4 and 7.

Three (3) doses of streptavidin are being assessed in this study: 100 ug, 250 ug, and 500 ug. Each set of vaccines manufactured is unique to each patient and will therefore be a unique batch.

The vaccine is admixed with 200 uL of Freund's Incomplete Adjuvant immediately before administration. Three (3) vaccines in total are administered: Week 1, Week 4, and Week 7. Each vaccine is given as a single, small volume injection intradermally.
Intervention code [1] 291778 0
Treatment: Other
Comparator / control treatment
This is an open label, first in human study in patients with advanced cancer. There is no comparator or control treatment.
Control group

Primary outcome [1] 294976 0
Safety of RGSH4K as measured by the incidence and nature of adverse events using the CTCAE grading scale for adverse events.
Timepoint [1] 294976 0
Adverse events are collected from the time of informed consent and at each study visit: Weeks 1, 4, 7, 10, 12, and 24.
Primary outcome [2] 294977 0
Safety of RGSH4K as measured by vital signs, physical examinations, ECG and laboratory assessments.
Timepoint [2] 294977 0
Vital signs, ECG, physical examinations and laboratory assessments are measured at the following visits: Weeks 1, 4, 7, 10, 12, 24. At week 12, laboratory assessments are not done.
Secondary outcome [1] 314495 0
Determination of a biologically active dose of RGSH4K as measured by CT/MRI (whichever method is most appropriate for the cancer type)
Timepoint [1] 314495 0
CT/MRI is done at screening, week 12, and at week 24.
Secondary outcome [2] 314496 0
Determination of a biologically active dose of RGSH4K as measured by immunology biomarkers: white cell differential, mast cells, B cells (CD20+, CD40+, CD80+) and cytokines (VEGF, IL-6, IFN, TNF)
Timepoint [2] 314496 0
Immunology biomarkers are assessed at weeks 1, 4, 7, 12, and 24.
Secondary outcome [3] 314497 0
Determination of a biologically active dose of RGSH4K as measured by blood cancer markers relevant to the type of cancer a patient has.
Timepoint [3] 314497 0
Relevant blood cancer markers will be assessed at weeks 1, 7, 12, and 24.

Key inclusion criteria
- aged 18 years or over
- has banked approximately 1 gram of fresh frozen tumour in the Regeneus tumour bank
- have advanced solid tumours which are inoperable or refractory to treatment, or chemoradiotherapy is contraindicated, or where standard treatments have failed or do not exist, or in a palliative setting where the patient has refused chemotherapy or it is contraindicated.
Minimum age
18 Years
Maximum age
No limit
Both males and females
Can healthy volunteers participate?
Key exclusion criteria
- Rapidly progressing malignancy
- History of organ transplant and/or current immunosuppressive therapy, current systemic corticosteroids
- concurrent anticancer therapy, or within 4 weeks of vaccine
- Autoimmune disease
- known positive HIV or tests positive to HIV

Study design
Purpose of the study
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
Other design features
Phase 1
Type of endpoint(s)
Statistical methods / analysis

Recruitment status
Stopped early
Data analysis
Data collected is being analysed
Reason for early stopping/withdrawal
Participant recruitment difficulties
Date of first participant enrolment
Date of last participant enrolment
Date of last data collection
Sample size
Accrual to date
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 291213 0
Commercial sector/Industry
Name [1] 291213 0
Regeneus Ltd
Address [1] 291213 0
25 Bridge St
Pymble 2073 NSW
Country [1] 291213 0
Primary sponsor type
Commercial sector/Industry
Regeneus Ltd
25 Bridge St
Pymble 2073 NSW
Secondary sponsor category [1] 289892 0
Name [1] 289892 0
Address [1] 289892 0
Country [1] 289892 0

Ethics approval
Ethics application status
Ethics committee name [1] 292776 0
Bellberry Ltd
Ethics committee address [1] 292776 0
129 Glen Osmond Road
South Australia 5063
Ethics committee country [1] 292776 0
Date submitted for ethics approval [1] 292776 0
Approval date [1] 292776 0
Ethics approval number [1] 292776 0

Brief summary
This study aims to evaluate the safety, tolerability and preliminary efficacy of 3 different doses of a cancer vaccine, manufactured from a patient's own tumour.

Who is it for?
You may be eligible to join this study if you are aged 18 years or above, have banked a tumour sample in the Regeneus Tumour Bank (ACTRN12615000476538) and your tumour is inoperable and non-treatable or current treatment is refractory.

Study details
All patients enrolled in this safety and tolerability study will have their banked tumour samples used to develop a tumour specific vaccine. In brief, the vaccine is manufactured from the tumour, which is homogenised, processed and combined with streptavidin, an immunostimulant. Tumour cells are burst open during this process so that only the tumour proteins remain, i.e. no viable tumour cells remain in the vaccine. At the bedside, the vaccine is mixed with Freund’s Incomplete Adjuvant (FIA), which assists in immune recognition. Patients are given a single, small volume vaccine into the skin on Weeks 1, 4, and 7. Three different dose levels of streptavidin will be assessed (100, 250 or 500 ug) to identify the dose that is biologically active.

Safety and tolerability will be assessed over a 24 week period by recording adverse event information, vital signs, physical examinations and various laboratory assessments. CT or MRI scans and blood samples will also be taken over this period for determination of your response to the vaccine.

It is hoped that this study will aid in the treatment of solid tumours by harnessing a patient's own immune system to target and destroy cancer proteins.
Trial website
Trial related presentations / publications
Public notes

Principal investigator
Name 56966 0
Prof Stephen Clarke
Address 56966 0
Northern Cancer Institute
Level 1
38 Pacific Highway
Country 56966 0
Phone 56966 0
+61 2 9437 1900
Fax 56966 0
Email 56966 0
Contact person for public queries
Name 56967 0
Ms Iona Nicolson-Bowles
Address 56967 0
Northern Cancer Institute
Level 1
38 Pacific Highway
Country 56967 0
Phone 56967 0
+61 2 8037 4100
Fax 56967 0
Email 56967 0
Contact person for scientific queries
Name 56968 0
Ms Janet Wilson
Address 56968 0
Regeneus Ltd
25 Bridge St
Pymble 2073 NSW
Country 56968 0
Phone 56968 0
+61 2 9499 8010
Fax 56968 0
Email 56968 0

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No/undecided IPD sharing reason/comment
Aggregate data will be presented in a publication first.
What supporting documents are/will be available?
No other documents available
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary
The single centre, open label, first-in-human Phase 1 study was designed to evaluate the safety and tolerability of RGSH4K and to identify the biologically active dose(s) to take into future trials. In this study,12 patients, heavily pretreated with chemotherapy or radiotherapy, with various advanced solid tumours received RGSH4K in 3 dose cohorts. A total of 3 vaccines were administered in the treatment phase, given at 3-week intervals, and patients had the option to continue dosing in an extension phase. All dose levels were safe and well tolerated, achieving the safety primary endpoint. There were no dose limiting toxicities and no serious adverse events related to the vaccine. Injection site reactions were the most common adverse event related to RGSH4K administration.
RGSH4K also showed encouraging signs of immune stimulation in some patients, as demonstrated by changes in cancer markers, immune cells and cytokines. This immune stimulation was seen in one or more patients at all three dose levels. Preliminary indications of anti-tumour activity were seen in some patients.