Evaluation of the efficacy of stiripentol (Diacomit) as monotherapy for the treatment of primary hyperoxaluria. Pilot clinical study, open, prospective and multicenter.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
Administration of stiripentol per os
Hôpital Necker
Paris, France
Hôpital Robert Debré
Paris, France
Hôpital Tenon
Paris, France
Relative variation (%) of the molar ratio [oxaluria / creatinuria] between baseline and after two weeks of treatment.
Relative variation (%) of the molar ratio \[oxaluria / creatinuria\] between baseline and after two weeks of treatment.
Time frame: Change (%) of the molar ratio [oxaluria / creatinuria] between the baseline value (average of 3 measures done during pre-treatment period) and the value (average of 2 measures) after 2 weeks of treatment.
Relative variation (%) of the molar ratio [oxaluria / creatinuria] between baseline and after three weeks of treatment.
Relative variation (%) of the molar ratio \[oxaluria / creatinuria\] between baseline and after three weeks of treatment.
Time frame: Change (%) of the molar ratio [oxaluria / creatinuria] between the baseline value (average of 3 measures done during pre-treatment period) and the value (average of 2 measures) after 3 weeks of treatment.
Response to treatment defined by a decrease> 20% of the molar ratio [oxaluria / creatinuria]
Response to treatment defined by a decrease\> 20% of the molar ratio \[oxaluria / creatinuria\]
Time frame: 3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)
Relative variation (%) of supersaturation of urine with calcium oxalate between the start and the end of treatment period
Relative variation (%) of supersaturation of urine with calcium oxalate between the start and the end of treatment period
Time frame: 3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)
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Relative variation (%) in overall crystalline volume measured by crystalluria on fresh urine between the start and the end of treatment period
Relative variation (%) in overall crystalline volume measured by crystalluria on fresh urine between the start and the end of treatment period
Time frame: 3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)
Effect of stiripentol dose increase on absolute decrease of the molar ratio [oxaluria / creatinuria]
Effect of stiripentol dose increase on absolute decrease of the molar ratio \[oxaluria / creatinuria\]
Time frame: 3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)
Effect of stiripentol dose increase on relative decrease (%) of the molar ratio [oxaluria / creatinuria]
Effect of stiripentol dose increase on relative decrease (%) of the molar ratio \[oxaluria / creatinuria\]
Time frame: 3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)
Blood test results (hepatic assessment) at the start and at the end of the study
Blood test results (hepatic assessment) at the start and at the end of the study
Time frame: From start of participation of the patient to end of the treatment period (up to 8 weeks)
Blood test results (blood cells count) at the start and at the end of the study
Blood test results (blood cells count) at the start and at the end of the study
Time frame: From start of participation of the patient to end of the treatment period (up to 8 weeks)
Frequency and nature of the adverse events throughout the study
Frequency and nature of the adverse events throughout the study
Time frame: From start of participation of the patient to end of the treatment period (up to 8 weeks)