In short
Clinical trials are studying Stiripentol in people age 6 years and older with primary hyperoxaluria types 1, 2, and 3. These studies are looking at whether it can help lower urinary oxalate and how safe it is in this patient group. The main trial is a Phase 3 study.
Key points
- Clinical trials of Stiripentol are focused on primary hyperoxaluria, including PH1, PH2, and PH3. The main study is a Phase 3 interventional trial in patients 6 years and older. It is designed to evaluate efficacy and safety, with a main goal of reducing urinary oxalate excretion. The trial plans to enroll 42 participants. The key measurement is the percent change in 24-hour urinary oxalate excretion corrected for body surface area from baseline to Month 6.
Trial overview
The available trial is an interventional study, which means researchers give a study treatment and compare results with a control group. It is studying Stiripentol in people with primary hyperoxaluria, including PH1, PH2, and PH3.
The study title says it is evaluating the efficacy and safety of Stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2, or 3. The study is authorised and planned for 42 participants.
Who can participate
This trial is for patients who are at least 6 years old and have primary hyperoxaluria type 1, type 2, or type 3. The trial data do not list other entry rules, so the main known target group is children, teens, and adults in this age range with these conditions.
What the study measures
The main endpoint is the percent change in 24-hour urinary oxalate excretion corrected for body surface area from baseline to Month 6. In simple terms, the study checks whether the amount of oxalate in urine changes after treatment over 6 months.
Oxalate is measured in a full-day urine sample, which helps show how much of this substance the body is passing out. The correction for body surface area helps adjust the result for differences in body size.
Treatment and comparison groups
The trial includes Stiripentol given as Diacomit 250 mg hard capsules and Diacomit 500 mg hard capsules. The listed dose in the trial data is 3000 mg by oral use.
The study also uses a matched placebo, which is a look-alike capsule with no active ingredient. Using a placebo helps researchers compare the study treatment with a control group in a fair way.
Study design and phase
This is a Phase 3 trial. Phase 3 studies are later-stage trials that usually involve more participants and focus on whether a treatment works and how safe it is in the target group.
The study status is Authorised, which means it has been approved to take place. The planned enrollment is 42 people.
What this means for patients
For patients and families, this trial is mainly about whether Stiripentol can help lower urinary oxalate in primary hyperoxaluria. The study is not broad; it is focused on people with specific subtypes of the disease and starts at age 6.
Because the main endpoint is measured over 6 months, the trial is looking for a change over time rather than a quick short-term result. The available data show a clear research aim, but they do not provide final results yet.