Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3
Trial statusNot yet recruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age6+
SponsorBiocodex
About this trial
Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3.
Eligibility criteria
Qualifiers
1. Male or female patients aged ≥ 6 years at the time of consent signature
2. Diagnosed with primary hyperoxaluria (type 1, 2 or 3) documented as per standard methods
3. With last estimated Glomerular Filtration Rate ≥ 45 mL/min/1.73 m2
4. Able to understand and willing to comply with study requirements and to provide written informed consent. In case of patient under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent as per local and national requirements
Disqualifiers
1. Any relevant change in the use of any component of the standard of care (fluid intake, vitamin B6, potassium citrate) in the 4 weeks prior to inclusion or if such change is planned to occur during the first 6 months of the study
2. If under approved targeted medications (e.g., lumasiran), treatment should have been administered for at least 6 months, with no change in dose or regimen in the 3 months prior to inclusion or ifsuch change is planned it should not occur during the first 12 months of the study
3. History of kidney or liver transplant
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST)> 2 × upper limit of normal (ULN)
Trial design
Treatments tested in this trial
- Stiripentol Oral Capsule
- Placebo Oral Capsule
- Urine samples collect
- Blood samples collect
- Kidney imaging
- Quality of Life questionnaires
Treatment groups
42 Participants
are divided into 2 treatment groupsLocations
Sponsors and collaborators
Biocodex
Lead sponsor
Exystat
Collaborator
Source ClinicalTrials.gov