ClinicalTrials.gov record NCT06465472
Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3
Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This randomized, double-blind, placebo-controlled phase 3 trial studies stiripentol in people aged 6 years and older with primary hyperoxaluria types 1, 2, or 3. Participants are assigned to stiripentol (target 50 mg/kg/day, up to 3,000 mg/day) or matching placebo for a 6-month blinded period, then enter a 6-month open-label period in which study treatment is provided and blind is maintained on results. After 12 months of study treatment, patients who benefit may be proposed to join a longer open-label extension to continue assessing long-term safety and efficacy.
What it is studying. The study measures whether oral stiripentol affects 24-hour urinary oxalate excretion (corrected for body surface area) compared with placebo at 6 months, and collects safety data. It also follows kidney-related outcomes, kidney stone events, estimated glomerular filtration rate, and quality-of-life measures over time, with additional follow-up in an open-label extension for longer-term assessment.
What the researchers hope to learn. To determine whether stiripentol changes urinary oxalate excretion and to gather safety data; to assess effects on kidney function, kidney stone events, and patient-reported quality of life; and to evaluate longer-term safety and efficacy in an extension.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Stiripentol Oral Capsule
- Placebo Oral Capsule
- Biological
- Urine samples collect
- Blood samples collect
- Other
- Kidney imaging
- Quality of Life questionnaires
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Screening Criteria :
- Male or female patients aged ≥ 6 years at the time of consent signature
- Diagnosed with primary hyperoxaluria (type 1, 2 or 3) documented as per standard methods
- With last estimated Glomerular Filtration Rate ≥ 45 mL/min/1.73 m2
- 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
- Female patients with contraception (intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, surgical sterilization of male partner, established use of oral, implantable, injectable, or transdermal hormonal methods of contraception, a double-barrier method (combination of male condom with cap, diaphragm, or sponge, in conjunction with spermicide) or true sexual abstinence
- Diagnosed with primary hyperoxaluria disease and subtype (type 1, 2 or 3) confirmed by genetic testing
- Receiving optimal management of the disease through standard of care strategies (e.g., increased fluid intake, vitamin B6, potassium citrate) with or without approved target medications (e.g., lumasiran). Patients not receiving lumasiran can only be enrolled if they are not eligible for treatment with lumasiran for the specific following reasons: contraindications, previous treatment discontinued due to lack of efficacy or poor tolerability, not meeting national or regional eligibility criteria for treatment, investigator judgement
- With mean 24-hour urinary oxalate excretion from 2 valid 24-hour urine collections ≥ 0.70 mmol/24h/1.73m²
- With estimated Glomerular Filtration Rate ≥ 45 mL/min/1.73 m2 (Schwartz et al., 2009 in pediatric patients and CKD-EPI in adults)
- Pubescent and adult female patients must have a negative urine or serum pregnancy test within 60 days prior to first dose of study treatment if of childbearing potential. If the urine pregnancy test is positive or cannot be confirmed as negative, a serum pregnancy test will be required. The serum pregnancy test must be negative for the patient to be eligible
- In France, patient affiliated with or who benefits from a social security scheme
Exclusion criteria
- 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
- 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
- History of kidney or liver transplant
- Presenting any of the following liver function tests abnormalities during the screening period:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST)> 2 × upper limit of normal (ULN)
- Total bilirubin > 1.5 x ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert's syndrome are eligible if the total bilirubin is < 2 x ULN
- Recent (4 weeks before the screening visit) or planned change in eating habits
- Intermittent fasting planned during the 6 first months of the study period (e.g., Ramadan)
- Other medical conditions or comorbidities, treatment, which in the opinion of the investigator, would interfere with study compliance or data interpretation
- Presenting any significant biological or clinical anomalies that are not compatible with participation in the study according to the investigator
- History of severe allergy, asthma, skin rashes, intolerance to lactose or hypersensitivity to the study treatments
- Treatment affecting hepatic metabolism (i.e., cimetidine, ketoconazole, fluconazole, itraconazole, phenytoin, rifampicin, rifabutin) that is ongoing or has been taken in the month prior to the selection visit
- Treatment affecting the renal tubule (probenecid, β-lactam, etc.,) that is ongoing or has been taken in the two weeks prior to the start of the study
- Contraindications to stiripentol as defined in the applicable Investigator's Brochure (i.e. patients presenting a hypersensitivity to the active substance or any excipients)
- Patient at risk of pregnancy, pregnant or breastfeeding female
- Patient under guardianship or curatorship
- Patient under the protection of the Court or deprived of liberty
- Patient participating in another interventional clinical trial which could interfere with the trial's results or impact the other trial's results; or within the last 30 days or 5 half-lives of the study investigational treatment, whichever is longer, prior to the urinary sampling during the screening period, or are in follow-up of another clinical study prior to randomization
- Patient whose current state of health does not allow him/her to give consent
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
A multicenter randomized, double-blind, placebo-controlled phase 3 study The study is designed to compare the efficacy of stiripentol to a placebo in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3. The study will be conducted in 2 periods: a 6-month, placebo-controlled, double-blind treatment period (period 1) followed by a 6-month open-label treatment period with blind maintained on results (period 2). Patients who benefit from the treatment after the first 12 months of study treatment will be proposed to enter the open-label extension (OLE) part of the study to continue to assess the long-term efficacy and safety of stiripentol.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants are randomly assigned to oral stiripentol (target 50 mg/kg/day, maximum 3,000 mg/day) or matching placebo for a 6‑month double‑blind period, then enter a 6‑month open‑label period during which placebo recipients switch to stiripentol. Visits include repeated 24‑hour urine collections (may be done at home when appropriate), blood draws (including pregnancy testing, genetic confirmation, plasma oxalate, vitamin B6, and stiripentol pharmacokinetics), compulsory renal ultrasound imaging, and quality-of-life questionnaires. Those judged to benefit after 12 months may be offered entry into a longer open‑label extension to continue assessing long‑term safety and efficacy; assessments are planned through month 60. No invasive procedures such as biopsies or spinal taps are described in the record.
How the study is structured. Randomized, parallel groups, quadruple‑masked (participants, care providers, investigators, outcomes assessors) phase 3 drug trial. Two periods: a 6-month double-blind period (stiripentol or placebo) followed by a 6-month open-label period in which participants receive stiripentol; a longer open-label extension may follow. Study procedures include repeated 24‑hour urine collections, blood tests, kidney ultrasound imaging, and quality-of-life questionnaires.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- No sites are listed on the registry yet. Where will the study take place?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on primary hyperoxaluria
Important dates
- Study start
- August 2024 (estimated)
- Primary completion
- August 2030 (estimated)
- Study completion
- August 2030 (estimated)
- Target enrollment
- 42 participants (estimated)
- Last updated on registry
- Jun 18, 2024
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Biocodex
- Collaborators
- Exystat
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Pauline DECIMA 📞 03.44.86.82.28 ext. +33 ✉ p.decima@biocodex.fr
- Carine FRANCOIS 📞 03.44.86.82.28 ext. +33 ✉ c.francois@biocodex.fr
Participating sites
Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.
Terms used on this page · explanations by MedicalUpdates.ai
- estimated Glomerular Filtration Rate
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- written informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- legal guardian
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- assent
- A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
- standard of care
- Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
- contraindications
- A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
- eligibility criteria
- The rules the study team uses to decide who may take part. They are usually split into inclusion criteria and exclusion criteria.
- childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- screening period
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- randomization
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- double-blind
- Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
- placebo-controlled
- A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
- placebo
- A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- open-label extension
- A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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