ClinicalTrials.gg
CompletedNCT03819647Updated Mar 26, 2021

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

A Phase 2 interventional study of stiripentol (Diacomit) in Primary Hyperoxaluria, sponsored by Biocodex. Completed at 3 sites in France. Open to participants aged 6 Months and older. Per ClinicalTrials.gov, last updated 2021-03-26.

Sponsored by Biocodex · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
15
Allocation
Not applicable
Ages
6 Months and older
Sex
All
01

Study summary

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

Pilot clinical study, open, prospective and multicenter.

02

Conditions studied

  • Primary Hyperoxaluria

Browse trials for

03

In context

Hyperoxaluria, Primary

42 studies on the registry are indexed under Hyperoxaluria, Primary; 11 are open to participants now.

This study's enrollment of 15 is below the median of 24 across 26 interventional studies indexed under Hyperoxaluria, Primary.

Browse Hyperoxaluria, Primary studies →

Lead sponsor

Biocodex is the lead sponsor of 19 studies on the registry; 6 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Months and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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 ≥ 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, deemed effective by the investigator or abstinence

Exclusion criteria

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 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 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
  • to stiripentol as defined in the current SmPC (hypersensitivity to the active substance or to any of the 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
Terms in these criteria, in plain language(6)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

Contraindication

A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.

Excipients

The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
15 participants (actual)

Study arms

  • Experimental
    stiripentol (Diacomit)

    Drug: stiripentol (Diacomit)

Interventions

06

What researchers measure

Primary outcomes

  1. 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.

  2. 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.

Secondary outcomes

  1. 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)

  2. 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)

  3. 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)

  4. 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)

  5. 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)

  6. 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)

  7. 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)

  8. 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)

07

Study locations

3 sites
  • Hôpital Necker
    Paris, France
  • Hôpital Robert Debré
    Paris, France
  • Hôpital Tenon
    Paris, France
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from May 2019 to primary completion in Dec 2020, 18 months.

How these figures are worked out, and which studies are left out of them.

Registered
Jan 18, 2019
First posted
Jan 28, 2019
StartActual
May 21, 2019
Primary completionActual
Dec 18, 2020
CompletionActual
Mar 8, 2021
Last update
Mar 26, 2021
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Mar 26, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT03819647
Lead sponsor
Biocodex
Responsible party
Sponsor
First posted
Jan 28, 2019
Start date
May 21, 2019
Primary completion
Dec 18, 2020
Completion
Mar 8, 2021
Last update
Mar 26, 2021

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Nov 2020. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Studies of the drug tested here: Stiripentol

Start the discussion