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

NCT ID: NCT03819647

Last Updated: 2021-03-26

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-05-21
• Study Completion Date: 2021-03-08

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

stiripentol (Diacomit)

Group Type: EXPERIMENTAL

stiripentol (Diacomit)

Intervention Type: DRUG

Administration of stiripentol per os

Interventions

stiripentol (Diacomit)

Administration of stiripentol per os

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• Introduction, discontinuation or dose modification of vitamin B6 or potassium citrate treatment within 4 weeks prior to the inclusion visit
• 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
• Patient concurrently participating in another clinical trial or exclusion period following a previous trial

• Minimum Eligible Age: 6 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Biocodex

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Hôpital Necker

Paris, , France

Hôpital Robert Debré

Paris, , France

Hôpital Tenon

Paris, , France

Countries

France

Other Identifiers

HYPOP (STP194)

Identifier Type: -

Identifier Source: org_study_id