In short
Clinical trials are investigating Sebetralstat in people with hereditary angioedema, a condition that causes swelling attacks. These studies look at safety, tolerability, and how well the treatment works over time in adults, adolescents, and children with type I or II disease.
Key points
- Sebetralstat is being studied in clinical trials for hereditary angioedema type I or II, a condition that causes swelling attacks. The available studies are Phase 3 trials, which means they are later-stage studies with larger patient groups. One trial looks at long-term safety in adolescents and adults, while another studies safety and tolerability in children aged 2 to less than 12 years. The trials measure adverse events, serious adverse events, lab results, and vital signs. Together, these studies help show how Sebetralstat performs in different age groups with the same condition.
Trial overview
The clinical trials provided are studying Sebetralstat in people with hereditary angioedema type I or II.
Both studies are interventional trials, which means patients receive the study treatment so researchers can observe the results.
The main goal of these trials is to learn about safety, tolerability, and how well the treatment may work in different age groups.
Who can participate
One study includes adolescent and adult patients with hereditary angioedema type I or II.
The other study includes pediatric patients aged 2 to less than 12 years with hereditary angioedema type I or II.
This means the trials are looking at both older patients and younger children, but they are still focused on the same condition.
Trial phases and status
Both trials are Phase 3 studies, which are later-stage clinical trials that usually involve more patients than early studies.
NCT05505916 is listed as authorised and plans to include 150 patients.
NCT06467084 is listed as completed and included 48 patients.
What the trials measure
The long-term study measures the number and percentage of patients with adverse events, serious adverse events, and adverse events that caused treatment to stop early.
It also measures laboratory results and vital signs at scheduled visits to check patient safety over time.
The pediatric study measures the proportion of children who have any adverse event during the study, including fatal adverse events if they occur.
In simple terms, these outcomes help researchers see whether the treatment is safe and how children and adults respond during the study.
Key trial details
NCT05505916 is a long-term study designed to evaluate whether Sebetralstat is safe and effective for treating attacks in patients with hereditary angioedema.
The brief summary says the study is assessing the safety of long-term administration in adolescent and adult patients with HAE type I or II.
The trial uses oral study drug doses listed as 900 mg and 1200 mg.
NCT06467084, called KONFIDENT-KID, is a pediatric Phase 3 study focused on safety and tolerability in children aged 2 to less than 12 years.
The trial uses oral study drug doses listed as 450 mg and 900 mg.
What these studies mean for patients
These trials are important because they test Sebetralstat in different age groups with hereditary angioedema type I or II.
They focus on safety data such as side effects, lab tests, and vital signs, which helps researchers understand how the treatment performs in real study settings.
The pediatric study adds information for younger children, while the long-term study adds information for adolescents and adults.
Based on the trial data provided, the research program is centered on hereditary angioedema and does not list other diseases.