Evaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary Hyperoxaluria

NCT03819647 | Completed Phase 2

• 1 other identifier: HYPOP (STP194)
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 3

Brief Summary

Evaluation of the efficacy of stiripentol (Diacomit) as monotherapy for the treatment of primary hyperoxaluria. Pilot clinical study, open, prospective and multicenter.

Conditions

Primary Hyperoxaluria

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  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.

Secondary Outcomes (8)

• Response to treatment defined by a decrease> 20% of the molar ratio [oxaluria / creatinuria]

  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

  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 (%) in overall crystalline volume measured by crystalluria on fresh urine between the start and the end of treatment period

  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]

  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]

  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)

Study Arms (1)

stiripentol (Diacomit)

EXPERIMENTAL

Drug: stiripentol (Diacomit)

Interventions

stiripentol (Diacomit) DRUG

Administration of stiripentol per os

stiripentol (Diacomit)

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient with primary hyperoxaluria type 1, 2 or 3, diagnosed according to standard methods
• Having at least one molar ratio \[oxaluria / creatinuria\] greater than 0.08 since diagnosis
• Having Glomerular Filtration Rate ≥ 45 mL / min / 1.73m2
• Age ≥ 6 months
• Having read, or whose parents have read, the information note and signed the consent form. For children, if their level of understanding allows, their assent will also be sought
• Proficient enough, or whose parents or legal representatives have sufficient mastery, the French language to read, understand and complete study documents
• Affiliate or beneficiary of a social security scheme
• Ability to respect the protocol, including treatment, and can be followed regularly in the study
• For pubertal patients, contraception deemed effective by the investigator or abstinence

You may not qualify if:

• Consumption of jelly candies and / or dark chocolate in the week preceding the study
• Patient having a kidney and / or liver transplant
• Presence of a clinically significant acute or chronic pathology, other than primary hyperoxaluria, that may interfere with the evaluation of the study results according to the investigator
• During biological or physical examinations, presence of significant anomaly (s) inconsistent with participation in the study according to the investigator
• History of severe allergy, asthma, skin rash or hypersensitivity to a drug
• Treatment affecting hepatic metabolism (cimetidine, ketoconazole, fluconazole, itraconazole, phenytoin, rifampicin, rifabutin) in progress or taken during the month preceding the start of the study
• Treatment affecting the renal tubule (probenecid, β-lactams, ...) in progress or taken during the last two weeks preceding the start of the study
• Presence of a pathology or treatment that, according to the investigator, renders the subject unfit
• Contraindications to stiripentol as defined in the current SmPC (hypersensitivity to the active substance or to any of the excipients listed in section 6.1 of the SmPC, history of psychosis in the form of delusional episodes)
• Pregnant or lactating woman
• Patient under guardianship

Sponsors & Collaborators

• Biocodex: lead

Study Sites (3)

Hôpital Necker

Paris, France

Location

Hôpital Robert Debré

Paris, France

Location

Hôpital Tenon

Paris, France

Location

MeSH Terms

Conditions

Hyperoxaluria, Primary

Interventions

stiripentol

Condition Hierarchy (Ancestors)

Hyperoxaluria; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 18, 2019
• First Posted: January 28, 2019
• Study Start: May 21, 2019
• Primary Completion: December 18, 2020
• Study Completion: March 8, 2021
• Last Updated: March 26, 2021

Record last verified: 2020-11

Data Sharing

• IPD Sharing: Will not share

Locations

FR (3)