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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.

Phase 1
Conditions
Advanced solid tumours
Cancer - Any cancer
Registration Number
ACTRN12615000516583
Lead Sponsor
Regeneus Ltd
Brief 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.

Detailed Description

Not available

Recruitment & Eligibility

Status
Stopped early
Sex
All
Target Recruitment
13
Inclusion Criteria

Summary:
- 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.

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

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Safety of RGSH4K as measured by the incidence and nature of adverse events using the CTCAE grading scale for adverse events. [Adverse events are collected from the time of informed consent and at each study visit: Weeks 1, 4, 7, 10, 12, and 24. ];Safety of RGSH4K as measured by vital signs, physical examinations, ECG and laboratory assessments.[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 Measures
NameTimeMethod
Determination of a biologically active dose of RGSH4K as measured by CT/MRI (whichever method is most appropriate for the cancer type)[CT/MRI is done at screening, week 12, and at week 24. ];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)[Immunology biomarkers are assessed at weeks 1, 4, 7, 12, and 24. ];Determination of a biologically active dose of RGSH4K as measured by blood cancer markers relevant to the type of cancer a patient has. [Relevant blood cancer markers will be assessed at weeks 1, 7, 12, and 24. ]
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