A Phase 3 interventional study of Stiripentol Oral Capsule and Placebo Oral Capsule in Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2 and Primary Hyperoxaluria Type 3, sponsored by Biocodex. Not yet recruiting. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2024-06-18.
Sponsored by Biocodex · Phase 3, Interventional, and Treatment
Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3.
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.
22 studies on the registry are indexed under Primary hyperoxaluria type 1; 8 are open to participants now.
This study's planned enrollment of 42 is above the median of 28 across 16 interventional studies indexed under Primary hyperoxaluria type 1.
Browse Primary hyperoxaluria type 1 studies →Biocodex is the lead sponsor of 19 studies on the registry; 6 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Screening Criteria :
Inclusion Criteria:
Exclusion Criteria:
Presenting any of the following liver function tests abnormalities during the screening period:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients in this arms will receive 50 mg/kg/day oral stiripentol during the first 6 months double-blind placebo-controlled study period 1 (Day 1 through Month 6) then continue the same treatment for the following 6 months (period 2).
Drug: Stiripentol Oral Capsule · Biological: Urine samples collect · Biological: Blood samples collect · Other: Kidney imaging · Other: Quality of Life questionnaires
Patients in this arms will receive 50 mg/kg/day oral placebo during the first 6 months double-blind placebo-controlled study period 1 (Day 1 through Month 6) then patients receiving placebo will switch over the 6 to 12 month-period to stiripentol (period 2).
Drug: Placebo Oral Capsule · Biological: Urine samples collect · Biological: Blood samples collect · Other: Kidney imaging · Other: Quality of Life questionnaires
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 capsules will be administered for the first 6 months. Then patients will switch to stiripentol over the 6- to 12-month period.
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.
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 will be obtained. Renal ultrasounds will be compulsory for all patients.
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.
% 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
% 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.
% of patients with normalisation of 24-hour urinary oxalate level corrected for bode surface area 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.
Blood samples for assessment of change in estimated glomerular filtration rate (eGFR in mL/min/1.73m2) from baseline to Month 6
Blood samples for assessment of change in estimated glomerular filtration rate (eGFR). EGFR (mL/min/1.73m2) will be calculated from baseline to Month 6
Time frame: Change in estimated glomerular filtration rate (eGFR) between baseline value and month 6 value
Occurrence of and frequency of kidney stone events during the follow-up of the patient
Number of and frequency of kidney stone events reported during the follow-up of the patient
Time frame: From start of participation of the patient to end of the study (Month 60)
Change in urine oxalate/creatinine ratios as assessed in spot urine collections between baseline and Month 6
Urine oxalate/creatinine ratios will be calculated from the oxalate and creatinine levels measured in spot urine collected during patients' hospitalizations or at home. The concentrations will be determined using a validated assay.
Time frame: From start of participation of the patient to end of the study (Month 60)
Change in biological parameters : oxalate concentration measured in urinary spots collections between baseline and Month 6
Change in oxalate concentration, related to kidney stone formation, measured in urinary spots collections between baseline and Month 6
Time frame: From baseline to 6 months of treatment.
Change in biological parameters : creatinine concentration measured in urinary spots collections between baseline and Month 6
Change in creatinine concentration, related to kidney stone formation, measured in urinary spots collections between baseline and Month 6
Time frame: From baseline to 6 months of treatment.
Absolute change in quality of life measured by the Pediatric Quality of Life Inventory (PedsQL) questionnaire
Absolute change in the Pediatric Quality of Life Inventory (PedsQL \[the generic and kidney failure (KF) modules\]) for patients \< 18 years of age (at screening) which is a 23 items 5-point Likert scale from: 0 (Never) to 4 (Almost always). Scores are transformed on a scale from 0 to 100 so higher score means better health.
Time frame: At baseline and every 6 months until the end of the study (Month 60)
Absolute change in quality of life measured by the Kidney Disease Quality of Life Questionnaire (KDQOL)
Absolute changes in the Kidney Disease Quality of Life Questionnaire (KDQOL) for patients ≥ 18 years of age (at screening) which is a 36 items survey with five subscales. Scores are transformed on a scale from 0 to 100 so higher score means better health.
Time frame: At baseline and every 6 months until the end of the study (Month 60)
Change in quality of life measured by the Euro Quality of Life Health State Profile Questionnaire (EQ-5D)
Change in Euro Quality of Life Health State Profile Questionnaire (EQ-5D) is a standardized instrument consisting of a questionnaire. Scoring of the questionnaire is based on degrees of disability. Higher scores indicate better health status.
Time frame: At baseline and every 6 months until the end of the study (Month 60)
Change in quality of life measured by the Euro Quality of Life Health State Profile Visual Analog Scale (VAS) : EQ-5D
Change in EQ-5D Visual Analog Scale EQ-5D is a standardized instrument consisting of a visual analog scale pertaining to 5 dimensions. 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.
Time frame: At baseline and every 6 months until the end of the study (Month 60)
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)
Frequency and nature of adverse events
Collect of the frequency and nature of adverse events
Time frame: From baseline to the end of the study (Month 60)
Change in 24-hour urinary glyoxylate, glycine and glycolate
Change in 24-hour urinary glyoxylate, glycine and glycolate
Time frame: From start of participation of the patient to end of the study (Month 60)
Change from baseline (percent and absolute) in 24-hour urine oxalate excretion, percentage of time that 24-hour urinary oxalate ≤ 1.5 x ULN, 24-hour urine oxalate/creatinine ratios and eGFR
Change from baseline (percent and absolute) in 24-hour urine oxalate excretion, percentage of time that 24-hour urinary oxalate ≤ 1.5 x ULN, 24-hour urine oxalate/creatinine ratios and eGFR
Time frame: From start of participation of the patient to end of the study (Month 60)
PK parameters : volume of distribution of stiripentol
PK parameters : volume of distribution of stiripentol based on appropriate PK sampling scheme (on Month 0, Month 6 and Month 12), and further determined through a population PK (popPK) approach
Time frame: From start of participation of the patient to end of the study (Month 60)
PK parameters : clearance of stiripentol
PK parameters : clearance of stiripentol based on appropriate PK sampling scheme (on Month 0, Month 6 and Month 12), and further determined through a population PK (popPK) approach
Time frame: From start of participation of the patient to end of the study (Month 60)
Exposure parameters : Peak Plasma Concentration (Cmax) of stiripentol
Peak Plasma Concentration (Cmax) of stiripentol based on appropriate PK sampling scheme (on Month 0, Month 6 and Month 12), and further determined through a population PK (popPK) approach
Time frame: From start of participation of the patient to end of the study (Month 60)
Exposure parameters : Minimum Plasma Concentration (Cmin) of stiripentol
Minimum Plasma Concentration (Cmin) of stiripentol based on appropriate PK sampling scheme (on Month 0, Month 6 and Month 12), and further determined through a population PK (popPK) approach
Time frame: From start of participation of the patient to end of the study (Month 60)
Exposure parameters : Area under the plasma concentration versus time curve (AUC) of stiripentol
Area under the plasma concentration versus time curve (AUC) of stiripentol based on appropriate PK sampling scheme (on Month 0, Month 6 and Month 12), and further determined through a population PK (popPK) approach
Time frame: From start of participation of the patient to end of the study (Month 60)
Change in plasma oxalate concentrations
Change in plasma oxalate concentrations
Time frame: At baseline and every three Months until month 12
Change in plasma vitamin B6 concentrations
Change in plasma vitamin B6 concentrations
Time frame: At baseline and every three Months until month 12
Change in nephrocalcinosis as assessed by kidney imaging
Change in nephrocalcinosis as assessed by kidney imaging
Time frame: At baseline and every 6 months until the end of the study (Month 60)
Change in bone conditions during the follow-up
Change in bone conditions during the follow-up based on questions from the investigator to the patient collected in the CRF
Time frame: At Baseline and at Month 3,6 and 12
Prediction of urinary oxalate excretion at exposure of clinical interest for stiripentol from a PK/PD model
Prediction of urinary oxalate excretion at exposure of clinical interest for stiripentol from a PK/PD model
Time frame: At baseline and every 6 months until month 12
Change in patient, caregiver and investigator impact and experiences as evaluated by patient and investigator experience surveys
Change in patient, caregiver and investigator impact and experiences as evaluated by patient and investigator experience surveys
Time frame: At baseline and every 6 months until end of the study (Month 60)
No study locations are listed for this record.
Start is estimated for Aug 2024; primary completion for Aug 2030, 72 months later.
How these figures are worked out, and which studies are left out of them.
This study is not yet recruiting, as verified in Jun 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Stiripentol
Primary hyperoxaluria type 1 →
Biocodex