Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3 - CRYSTA

Phase 1

• Conditions: Primary Hyperoxaluria (PH) of all subtypes (PH1, PH2 and PH3); MedDRA version: 23.0Level: PTClassification code: 10084111Term: Primary hyperoxaluria Class: 100000004850; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: CTIS2023-508062-15-00
• Lead Sponsor: Biocodex

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-01-19
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 42

Inclusion Criteria

Male or female subjects aged =6 years at the time of consent signature, 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), 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 2009 in pediatric patients and CKD-EPI in adults), Pubescent patients and adult female subjects 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 subject to be eligible., Able to understand and willing to comply with study requirements and to provide written informed consent. In the case of patient under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent per local and national requirements

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, Treatment affecting hepatic metabolism (i.e., cimetidine, ketoconazole, fluconazole, itraconazole, phenytoine, rifampicine, rifabutine) that is ongoing or has been taken in the month prior to the selection visit, Treatment affecting the renal tubule (probénécide, ß-lactames, 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 Brochure, Patient at risk of pregnancy, pregnant or breastfeeding female patient, 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, 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 if such 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: a) Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2 × upper limit of normal (ULN) b) 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 or hypersensitivity to the study treatments

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified