BYL719C2401: PIK3CA Registry
Research type
Research Study
Full title
PIK3CA Registry: A descriptive study of PIK3CA mutations in patients with HR+/Her2- advanced breast cancer
IRAS ID
279186
Contact name
Stuart McIntosh
Contact email
Sponsor organisation
Novartis Pharma AG
Duration of Study in the UK
0 years, 8 months, 7 days
Research summary
Research Summary
This is a non-interventional, observational cohort study of men and women with Hormone Receptor positive (HR+)/Human Epidermal growth factor Receptor 2 negative (HER 2-) advanced breast cancer. Approximately 45% of patients with HR-positive, HER2-negative breast cancer have alterations in a gene called a PIK3CA gene.
To date, PIK3CA testing is not adopted in standard clinical practice, and there are questions that remain unanswered about the true frequency of PIK3CA mutations across different geographic areas and races. Another question, is whether there are differences in the clinical presentation, management of the disease and patient outcomes that can be associated with PIK3CA mutations.
The purpose of this study is to describe the above information in real world practice.Summary of Results
The purpose of this study was to describe the PIK3CA mutations in men and women with HR+ (Hormone Receptor positive) /HER2- (Human Epidermal growth factor Receptor type 2 negative) advanced breast cancer.
Some cancer types have alterations in a gene called a PIK3CA gene. Every cell has the PIK3CA gene but in some cancer cells, this gene has changed. This is called a mutation. We know that these mutations in the PIK3CA gene play a role in the growth and survival of tumours. Some new treatments are targeting this mutation so it is important that it is fully understood.
This study collected and reviewed data on:• The true frequency of PIK3CA mutations across different areas of the world and races of people.
• The location where the mutations can be found in the gene.
• The demographic (age, gender etc), clinical and disease characteristics presented by those patients whose tumours show PIK3CA mutations and those who do not.
• To characterise treatment patterns among HR+/HER2- advanced breast cancer patients, by PIK3CA mutation status.
• To explore the relationship between PIK3CA status and clinical outcomes (how well the disease is managed and for how long) in advanced breast cancer treatment.This was a non–interventional research study, meaning it involved no treatment or physical intervention (e.g. blood samples) and data was gathered only on events that had already happened.
Tissue samples from participants were sent to a central laboratory in order for the PIK3CA mutational status to be studied, and the following data was collected:
• Medical data used in routine care (e.g. disease characteristics)
• Details of medications the participants had taken for their cancer
• Results of examinations used in routine care (e.g. blood tests, scans)The results of study showed:
• PIK3CA mutations were detected in 808 of 1995 participants (40.5%). The most commonly detected hotspot mutations (locations of the mutation on the gene) were H1047R, E545K, and E542K, which is consistent with results from the ‘SOLAR-1’ study.
• Mutation rates were similar whether tested on primary (initial tumour) or metastatic (cancer that has spread) tissue, which supports the existing evidence that PIK3CA testing can be done on any available cancer biopsy tissue.
• PIK3CA mutation rates were consistent across different parts of the world.
• There was no notable difference between demographic and disease characteristics (the physical and disease characteristics of the participants) in the group with the mutation and the group without.
• Clinical presentation (the signs and symptoms of the cancer that the treating doctor observed), treatment and patient outcomes were similar regardless of PIK3CA mutation status. However, this result should be interpreted with caution as these outcomes may be influenced by the fact that this trial did not involve any treatment.REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
20/EE/0163
Date of REC Opinion
28 Jul 2020
REC opinion
Further Information Favourable Opinion