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

Biocodex

Status and phase

Completed

Phase 2

Conditions

Primary Hyperoxaluria

Treatments

Drug: stiripentol (Diacomit)

Study type

Interventional

Funder types

Industry

Identifiers

NCT03819647

HYPOP (STP194)

Details and patient eligibility

About

Evaluation of the efficacy of stiripentol (Diacomit) as monotherapy for the treatment of primary hyperoxaluria.

Pilot clinical study, open, prospective and multicenter.

Enrollment

15 patients

Sex

All

Ages

6+ months old

Volunteers

No Healthy Volunteers

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