In short
Getacatetide, also known by its development code CY-101, is an investigational substance being studied for the treatment of locally advanced or metastatic adrenocortical carcinoma (ACC), a rare cancer of the adrenal glands. One Phase 2 clinical trial is currently authorised in the European Union to evaluate this therapy. The trial plans to enroll 41 participants and is testing two different strengths of the drug, given directly into the tumour.
Key points
- Getacatetide (CY-101) is an investigational substance for adrenocortical carcinoma, a rare cancer of the adrenal glands.
- The only registered trial is a Phase 2 study, currently authorised to begin recruiting.
- The trial plans to enroll 41 participants with locally advanced or metastatic disease.
- Getacatetide is given by intratumoural injection directly into the tumour.
- The study measures optimal dose in the first part and objective response rate in the second part.
What is getacatetide?
Getacatetide is an investigational substance that is also known by its development code CY-101. It is not yet an authorised medicine; it is being studied in clinical trials to find out whether it is safe and effective for people with adrenocortical carcinoma. Adrenocortical carcinoma, often shortened to ACC, is a rare and aggressive cancer that forms in the outer layer of the adrenal glands, which sit on top of the kidneys.
The substance is being developed for use in locally advanced or metastatic ACC, meaning cancer that has grown into nearby tissues or spread to other parts of the body. Because ACC is a rare disease, new treatment options are needed, and getacatetide is one of the substances being investigated in this area.
How does getacatetide work?
The registry data for the authorised trial does not include a detailed description of the mechanism of action of getacatetide. What is known from the trial record is that the substance is given directly into the tumour, a method called intratumoural administration. This approach is designed to deliver the drug exactly where the cancer is, rather than throughout the whole body. The trial is designed to assess both the safety and the anti-tumour activity of this approach in people with adrenocortical carcinoma.
Which conditions is it studied for?
- Locally advanced or metastatic adrenocortical carcinoma (ACC) — a rare cancer of the adrenal glands that has either grown into surrounding tissues or spread to other parts of the body. This is the only condition listed in the current trial record.
How is getacatetide given?
Getacatetide is given as a solution for injection directly into the tumour. This route of administration is called intratumoural, often shortened to IT. The trial tests two different strengths of the injection: 40 mg/ml and 20 mg/ml. Both are given by intratumoural injection, meaning the drug is injected straight into the tumour tissue rather than into a vein or under the skin.
What do the clinical trials show?
At present, one clinical trial of getacatetide is registered in the EU Clinical Trials Information System. This is a Phase 2 trial, which means it is testing the drug in a group of patients with the disease to see how well it works and what side effects it causes. The trial has been authorised, meaning it has received the necessary approvals to begin, and it plans to enroll 41 participants with locally advanced or metastatic adrenocortical carcinoma.
The trial is divided into two parts. In the first part, called Phase IIa, researchers will determine the optimal dose of getacatetide by reviewing all clinically relevant data, including toxicity, efficacy, quality of life and pharmacokinetic parameters, which describe how the drug moves through the body. In the second part, called Phase IIb, they will evaluate the anti-tumour activity of the drug by measuring the objective response rate, or ORR, which is the proportion of participants who achieve a complete or partial response according to itRECIST, a set of criteria used to assess how tumours respond to treatment.
The trial is interventional, meaning participants receive the study drug as part of the research. Because the trial is still in the authorised stage, no results are available yet. The information in the registry describes the design and aims of the study, not its findings.
What side effects are known?
The registry record for this trial does not list specific side effects for getacatetide. Because the substance is still investigational and the trial has not reported results, the side effect profile is not yet known. One of the purposes of the Phase 2 trial is to gather information about the safety and tolerability of the drug, including any side effects that occur during treatment.