In short
Clinical trials investigating Varegacestat are studying whether it can help people with progressing desmoid tumors. The trials are designed to evaluate safety, tolerability, and effects on disease progression in affected patients. They include different study parts and compare treatment with placebo in some phases.
Key points
- Clinical trials of Varegacestat are studying people with progressing desmoid tumors. The trial data show a Phase 4 study with different parts, including Phase II and Phase III sections, plus an open-label extension. Researchers are looking at safety, tolerability, and whether treatment can slow disease progression. The study also compares Varegacestat with placebo in some parts. Main outcomes include treatment-emergent adverse events, serious adverse events, treatment stopping due to side effects, and progression-free survival.
Trial overview
The available trial data describe one interventional study of Varegacestat in people with progressing desmoid tumors. The study is authorised and includes 198 participants.
The brief summary says the research is designed to look at safety, tolerability, and effects on disease progression in subjects with progressing or progressive desmoid tumors.
Study parts and phase
The trial is listed as Phase 4, but the brief summary also describes separate parts of the study as Phase II and Phase III. This means the source data present the study as a multi-part research program rather than a single simple phase.
Part A is focused on safety and tolerability, Part B is focused on disease progression, and the open-label extension, or OLE, continues safety and tolerability follow-up.
Who the trials are for
The study targets subjects with progressing desmoid tumors or progressive desmoid tumors. In simple terms, this means the tumor is not stable and is continuing to grow or worsen.
The source data do not list the full eligibility rules, such as age limits or prior treatment rules. So, the exact group who can join is not fully described in the provided trial record.
What is being measured
The main safety outcomes are the frequency and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). TEAEs are health problems that start or get worse after treatment begins, while SAEs are more severe problems that may need urgent care.
Another safety-related endpoint is the time to treatment discontinuation because of a TEAE. This shows how long people stay on treatment before side effects lead to stopping the study drug.
The main efficacy, or benefit, outcome listed for Part B is progression-free survival. This means the researchers are measuring how long the disease does not get worse during the study.
Treatment groups and placebo
The intervention list includes AL102 at 4 mg and 1.2 mg by oral use, plus a placebo capsule. The provided trial record does not explain the relationship between AL102 and Varegacestat, so the article only reports the source data as written.
A placebo is a capsule that looks like the study treatment but does not contain the active drug. Researchers use it to help compare results in a fair way.
Study size and status
The study enrollment is listed as 198, which is the planned or total number of participants in the trial record. The trial status is Authorised.
Because the source data only provide one trial, the clinical research picture for Varegacestat in this dataset is focused on progressing desmoid tumors and on measuring safety plus disease control.