Primary Hyperoxaluria Type 1Phase 3CRYSTALNCT06465472
Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3
A Phase 3 study testing Stiripentol Oral Capsule, Urine samples collect and Blood samples collect in people with Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2, run by Biocodex.
Trial summary
Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary Hyperoxaluria Type 3
- What is being tested?
- Stiripentol Oral Capsule, Urine samples collect and Blood samples collect
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 42 participants
- Where
- No sites listed yet
- Study dates
- Aug 2024to Aug 2030
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 1. Diagnosed with primary hyperoxaluria disease and subtype (type 1, 2 or 3) confirmed by genetic testing
- 2. 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
- 3. With mean 24-hour urinary oxalate excretion from 2 valid 24-hour urine collections ≥ 0.70 mmol/24h/1.73m²
- 4. With estimated Glomerular Filtration Rate ≥ 45 mL/min/1.73 m2 (Schwartz et al., 2009 in pediatric patients and CKD-EPI in adults)
- 5. 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
- 6. In France, patient affiliated with or who benefits from a social security scheme
Who cannot join
- 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
- 4. 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
Show all 19Show fewer
- 5. Recent (4 weeks before the screening visit) or planned change in eating habits
- 6. Intermittent fasting planned during the 6 first months of the study period (e.g., Ramadan)
- 7. Other medical conditions or comorbidities, treatment, which in the opinion of the investigator, would interfere with study compliance or data interpretation
- 8. Presenting any significant biological or clinical anomalies that are not compatible with participation in the study according to the investigator
- 9. History of severe allergy, asthma, skin rashes, intolerance to lactose or hypersensitivity to the study treatments
- 10. 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
- 11. 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
- 12. Contraindications to stiripentol as defined in the applicable Investigator's Brochure (i.e. patients presenting a hypersensitivity to the active substance or any excipients)
- 13. Patient at risk of pregnancy, pregnant or breastfeeding female
- 14. Patient under guardianship or curatorship
- 15. Patient under the protection of the Court or deprived of liberty
- 16. 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
- 17. Patient whose current state of health does not allow him/her to give consent
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3.
Read the detailed description
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.
- Being tested
- Stiripentol Oral Capsule, Placebo Oral Capsule, Urine samples collect, Blood samples collect, Kidney imaging
- Keywords
- Stiripentol, Primary Hyperoxaluria, Oxalate
- Medical subject headings
- Primary hyperoxaluria type 1, Primary hyperoxaluria type 2, Hyperoxaluria, Primary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stiripentol Oral CapsuleDrug
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 CapsuleDrug
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 collectBiological
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 collectBiological
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 imagingOther
Kidney imaging will be obtained. Renal ultrasounds will be compulsory for all patients.
Quality of Life questionnairesOther
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.
| Study group | Role | What they receive |
|---|---|---|
| Stiripentol | Experimental | 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). Receives: Drug: Stiripentol Oral Capsule; Biological: Urine samples collect; Biological: Blood samples collect; Other: Kidney imaging; Other: Quality of Life questionnaires |
| Placebo | Placebo comparator | 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). Receives: Drug: Placebo Oral Capsule; Biological: Urine samples collect; Biological: Blood samples collect; Other: Kidney imaging; Other: Quality of Life questionnaires |
What is being measured
The primary outcome is the main question the study is designed to answer.
% 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
Measured: % change in 24-hour urinary oxalate excretion between baseline value and value at month 6
Secondary outcomes (14)
% 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
Measured: % 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
Measured: 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
Measured: 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)
Measured: 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)
Measured: 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
Measured: 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
Measured: 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.
Measured: 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
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: At baseline and every 6 months until the end of the study (Month 60)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2024
- Study startAug 2024 (estimated)
- Primary completionAug 2030 (estimated)
- Study completionAug 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Hyperoxaluria Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- The study will be conducted in 2 periods: a 6-month, placebo-controlled, double-blind treatment period on which the primary endpoint will be evaluated (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 4 years open-label extension (OLE) part of the study to continue to assess the long-term efficacy and safety of stiripentol.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Biocodex
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Exystat
- Responsible party
- Sponsor
- Investigators
- Oana BERNARD, MD, Study director, Chief Scientific Officer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06465472
- Sponsor's protocol ID
- CRYSTAL - STP226
- Other IDs
- 2023-508062-15-00, 2023
- Acronym
- CRYSTAL
- Status last verified
- Jun 2024
- First submitted
- 3 Jun 2024
- Last updated
- 18 Jun 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3
- ClinicalTrials.gov ID
- NCT06465472
- Acronym / sponsor ID
- CRYSTAL / CRYSTAL - STP226
- Keywords
- Stiripentol, Primary Hyperoxaluria, Oxalate
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- BiocodexIndustry (pharma, biotech, devices)
- Collaborators
- Exystat
- Study start
- Aug 2024 (estimated)
- Primary completion
- Aug 2030 (estimated)
- Study completion
- Aug 2030 (estimated)
- First posted
- 18 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- The study will be conducted in 2 periods: a 6-month, placebo-controlled, double-blind treatment period on which the primary endpoint will be evaluated (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 4 years open-label extension (OLE) part of the study to continue to assess the long-term efficacy and safety of stiripentol.
| Arm | Type | Receives |
|---|---|---|
| Stiripentol | Experimental | Drug: Stiripentol Oral Capsule, Biological: Urine samples collect, Biological: Blood samples collect, Other: Kidney imaging, Other: Quality of Life questionnairesPatients 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). |
| Placebo | Placebo comparator | Drug: Placebo Oral Capsule, Biological: Urine samples collect, Biological: Blood samples collect, Other: Kidney imaging, Other: Quality of Life questionnairesPatients 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). |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 1. Diagnosed with primary hyperoxaluria disease and subtype (type 1, 2 or 3) confirmed by genetic testing
- 2. 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
- 3. With mean 24-hour urinary oxalate excretion from 2 valid 24-hour urine collections ≥ 0.70 mmol/24h/1.73m²
- 4. With estimated Glomerular Filtration Rate ≥ 45 mL/min/1.73 m2 (Schwartz et al., 2009 in pediatric patients and CKD-EPI in adults)
- 5. 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
Key exclusion criteria (19 listed)
- 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
- 4. Presenting any of the following liver function tests abnormalities during the screening period:
- 5. Recent (4 weeks before the screening visit) or planned change in eating habits
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Stiripentol Oral CapsuleThe 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 | Stiripentol |
| Placebo Oral CapsulePlacebo capsules will be administered for the first 6 months. Then patients will switch to stiripentol over the 6- to 12-month period. | Drug | Placebo |
| Urine samples collectCollections 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 | Placebo, Stiripentol |
| Blood samples collectSeries of blood samples will be taken (serum pregnancy test, Primary Hyperoxaluria genetic characterization, clinical laboratory assessments, vitamin B6 dosage, plasma oxalate dosage, stiripentol pharmacokinetics) | Biological | Placebo, Stiripentol |
| Kidney imagingKidney imaging will be obtained. Renal ultrasounds will be compulsory for all patients. | Other | Placebo, Stiripentol |
| Quality of Life questionnairesKidney 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. | Other | Placebo, Stiripentol |
Primary outcome
% change in 24-hour urinary oxalate excretion corrected for body surface area (BSA) 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 in mg/kg corrected for body surface area (BSA) between baseline and Month 6 and determined from 24-hour urine sample collections
14 secondary outcomes
% change in 24-hour urinary oxalate excretion corrected for body surface area (BSA) 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
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
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
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
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
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
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
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
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
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
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)
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)
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
Time frame: At baseline and every 6 months until the end of the study (Month 60)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Oana BERNARD, MD study director
Chief Scientific Officer
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Stiripentol Oral Capsule, Urine samples collect and Blood samples collect in people with Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2, run by Biocodex.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 18 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Pauline DECIMA (03.44.86.82.28 ext. +33, p.decima@biocodex.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
% change in 24-hour urinary oxalate excretion corrected for body surface area (BSA) determined from 24-hour urine sample collections (time frame: % change in 24-hour urinary oxalate excretion between baseline value and value at month 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jun 2024.