Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3

Summary

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

Conditions

• Primary Hyperoxaluria Type 1
• Primary Hyperoxaluria Type 2
• Primary Hyperoxaluria Type 3

Interventions

DRUG

Stiripentol Oral Capsule

The target dose of stiripentol will be 50 mg/kg/day with a maximum dose of 3,000 mg/day. Patients allocated to the Stiripentol group will receive this treatment during the first 6 months, and in continuation up to 12 months.

DRUG

Placebo Oral Capsule

Placebo capsules will be administered for the first 6 months. Then patients will switch to stiripentol over the 6- to 12-month period.

BIOLOGICAL

Urine samples collect

Collections of urine over 24 hours and the first urine in the morning (spot urines) will be carried out. Urine collections will be performed either during hospitalizations, or at home if appropriate conditions are met.

BIOLOGICAL

Blood samples collect

Series of blood samples will be taken (serum pregnancy test, Primary Hyperoxaluria genetic characterization, clinical laboratory assessments, vitamin B6 dosage, plasma oxalate dosage, stiripentol pharmacokinetics)

OTHER

Kidney imaging

Kidney imaging will be obtained. Renal ultrasounds will be compulsory for all patients.

OTHER

Quality of Life questionnaires

Kidney Disease Quality of Life Questionnaire : KDQOL-36 for patients ≥18 years of age at screening, and the Pediatric Quality of Life Inventory (PedsQL) including the generic and KF modules (parent and/or self-report versions) for patients \<18 years of age at screening. EQ-5D: a standardized instrument consisting of a questionnaire and a visual analog scale pertaining to 5 dimensions. Scoring of the questionnaire is based on degrees of disability. Scoring of the visual analog scale is based on a visual scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state). Higher scores indicate better health status. The EQ-5D-5L questionnaire (will be utilized in patients ≥18 years of age at screening, and the EQ-5D-Y questionnaire will be utilized in patients \<18 years of age at screening, where available.

Sponsors & Collaborators

• Exystat

  collaborator OTHER

• Biocodex

  lead INDUSTRY

Principal Investigators

• Oana BERNARD, MD · Chief Scientific Officer

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

6 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2024-08-31

Primary Completion

2030-08-31

Completion

2030-08-31