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

ConditionsPrimary Hyperoxaluria Type 1 Primary Hyperoxaluria Type 2 Primary Hyperoxaluria Type 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

This trial does not accept healthy volunteers

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

Design model

Parallel

Treatments tested in this trial

Stiripentol Oral Capsule
Drug
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.
Placebo Oral Capsule
Drug
Placebo capsules will be administered for the first 6 months. Then patients will switch to stiripentol over the 6- to 12-month period.
Urine samples collect
Biological/Vaccine
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.
Blood samples collect
Biological/Vaccine
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)
Kidney imaging
Other intervention
Kidney imaging will be obtained. Renal ultrasounds will be compulsory for all patients.
Quality of Life questionnaires
Other intervention
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.

Treatment groups

42 Participants

are divided into 2 treatment groups

Group A: StiripentolExperimental treatment 5 interventions

Group B: PlaceboPlacebo comparator 5 interventions

Trial outcomes

Primary outcomes

% change in 24-hour urinary oxalate excretion corrected for body surface area (BSA) determined from 24-hour urine sample collections

% change in 24-hour urinary oxalate excretion in mg/kg corrected for body surface area (BSA) between baseline and Month 6 and determined from 24-hour urine sample collections

Time frame

% change in 24-hour urinary oxalate excretion between baseline value and value at month 6

Secondary outcomes

% change in 24-hour urinary oxalate excretion corrected for body surface area (BSA) determined from 24-hour urine sample collections

% change in 24-hour urinary oxalate excretion in mg/kg corrected for body surface area (BSA) from baseline to Month 3 and determined from 24-hour urine sample collections

Time frame

% change in 24-hour urinary oxalate excretion between baseline value and value at month 3

Absolute change in 24-hour urinary oxalate excretion in mg/kg corrected for body surface area (BSA) from baseline to Month 3 and Month 6

Absolute change in 24-hour urinary oxalate excretion in mg/kg corrected for BSA from baseline to Month 3 and Month 6

Time frame

Absolute change in 24-hour urinary oxalate excretion between baseline value to Month 3 and Month 6 values.

Change in 24-hour urine oxalate/creatinine ratio from baseline to Month 3 and Month 6

Concentration of 24-hour urine oxalate and 24-hour urine creatinine will be combined to report urine oxalate/creatinine ratio from baseline to Month 3 and Month 6. The concentrations will be determined using a validated assay

Time frame

Change in 24-hour urine oxalate/creatinine ratio between baseline value to month 3 and month 6 values

% of patients with urinary oxalate lower than 1.5 x upper limit of normal (ULN)) at Month 3 and Month 6

%of patients with near normalisation of 24-hour urinary oxalate level corrected for BSA (defined as urinary oxalate lower than 1.5 x upper limit of normal (ULN)) at Month 3 and Month 6)

Time frame

At 3 and 6 months of treatment.

Other outcomes

Height in meters

Changes in height in meters

Time frame

At baseline and every 3 months until the end of the study (Month 60)

Weight in kilograms

Changes in weight in kilograms

Time frame

At baseline and every 3 months until the end of the study (Month 60)

Changes in liver function tests (clinical laboratory parameters)

Changes in liver function tests (AST, ALT, GGT, ALP, Bilirubin (total and direct))

Time frame

From start of participation of the patient to the end of the study

Complete blood count with differential

Changes in complete blood count with differential ( blood test results)

Time frame

At baseline and every months until the end of the study (Month 60)

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Biocodex

Lead sponsor

Exystat

Collaborator

Source ClinicalTrials.gov

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