In short
Engasertib is an investigational drug being studied in a Phase 3 clinical trial for adults with moderate to severe hereditary hemorrhagic telangiectasia (HHT). The trial is authorised and plans to enrol 240 participants. Its main goal is to see whether engasertib 40 mg taken once daily by mouth reduces the number of nosebleeds over 28 weeks compared with a placebo.
Key points
- Engasertib is an investigational drug for hereditary hemorrhagic telangiectasia (HHT).
- The only trial in the data is a Phase 3 study with 240 planned participants.
- The trial is currently authorised, meaning it may begin or is preparing to enrol participants.
- Participants receive engasertib 40 mg once daily by mouth or a placebo.
- The main measure is the total number of nosebleeds through week 28, recorded in an electronic diary.
- The study lasts 28 weeks of double-blind treatment.
What is engasertib?
Engasertib is an investigational drug, which means it has not been approved as a medicine for general use. In the clinical trial data provided, it is studied as a treatment for hereditary hemorrhagic telangiectasia (HHT), a rare inherited condition in which blood vessels do not develop properly. The trial record does not state a trade name or development code for engasertib.
How does engasertib work?
The clinical trial record provided does not describe the mechanism of action of engasertib in detail. No supplementary sources are included in the data, so the molecular target of engasertib is not stated on this page. What the trial tells us is that engasertib is being tested to see whether it can reduce the frequency of nosebleeds, which are a common and often serious problem in people with HHT.
Which conditions is engasertib studied for?
- Hereditary hemorrhagic telangiectasia (HHT) — a rare inherited disorder of the blood vessels. People with HHT have abnormal connections between arteries and veins, which can cause frequent nosebleeds and other bleeding problems.
How is engasertib given?
In the clinical trial, engasertib is given as a 40 mg dose taken by mouth once daily. The trial also includes a placebo arm, which is a tablet that looks the same but contains no active drug.
What do the clinical trials show?
The data contains one clinical trial of engasertib: a Phase 3 study in hereditary hemorrhagic telangiectasia (HHT). The trial is currently authorised, meaning it has received approval to begin but may not yet be recruiting participants. It is an interventional study, which means participants receive a treatment and are followed over time.
The study plans to enrol 240 participants. Participants are adults with moderate to severe HHT. The trial compares engasertib 40 mg once daily by mouth with a placebo over 28 weeks of double-blind treatment, meaning neither the participants nor the researchers know who is receiving the active drug during that period.
The main measure in the trial is the total number of nosebleeds, also called epistaxis events, through week 28. Participants record each nosebleed in an electronic diary called the HHT eDiary. The study aims to show whether engasertib reduces the number of nosebleeds compared with placebo.
What side effects are known?
The clinical trial record provided does not list side effects for engasertib. No supplementary sources with safety information are included in the data, so no side effects can be reported on this page.