Primary HyperoxaluriaPhase 2NCT03819647
Evaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary Hyperoxaluria
A Phase 2 study testing stiripentol (Diacomit) in people with Primary Hyperoxaluria, run by Biocodex at 3 sites in France.
Trial summary
Evaluation of the efficacy of stiripentol (Diacomit) as monotherapy for the treatment of primary hyperoxaluria. Pilot clinical study, open, prospective and multicenter. Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Primary Hyperoxaluria
- What is being tested?
- stiripentol (Diacomit)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 3 sitesFrance
- Study dates
- 21 May 2019to 8 Mar 2021
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
3 sites in 1 country.
France3 sites
Hôpital Necker
Paris
Hôpital Robert Debré
Paris
Hôpital Tenon
Paris
About this study
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
- Being tested
- stiripentol (Diacomit)
- Medical subject headings
- Hyperoxaluria, Primary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
stiripentol (Diacomit)Drug
Administration of stiripentol per os
| Study group | Role | What they receive |
|---|---|---|
| stiripentol (Diacomit) | Experimental | Not described Receives: Drug: stiripentol (Diacomit) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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]
Response to treatment defined by a decrease> 20% of the molar ratio [oxaluria / creatinuria]
Measured: 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
Measured: 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
Relative variation (%) in overall crystalline volume measured by crystalluria on fresh urine between the start and the end of treatment period
Measured: 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]
Measured: 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]
Measured: 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
Measured: 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
Measured: 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
Measured: From start of participation of the patient to end of the treatment period (up to 8 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2019
- Study start21 May 2019
- Primary completion18 Dec 2020
- Study completion8 Mar 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary HyperoxaluriaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biocodex
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03819647
- Sponsor's protocol ID
- HYPOP (STP194)
- Status last verified
- Nov 2020
- First submitted
- 18 Jan 2019
- Last updated
- 26 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary Hyperoxaluria
- ClinicalTrials.gov ID
- NCT03819647
- Acronym / sponsor ID
- HYPOP (STP194)
- Conditions
- Primary Hyperoxaluria
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- BiocodexIndustry (pharma, biotech, devices)
- Study start
- 21 May 2019 (actual)
- Primary completion
- 18 Dec 2020 (actual)
- Study completion
- 8 Mar 2021 (actual)
- First posted
- 28 Jan 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Evaluation of the efficacy of stiripentol (Diacomit) as monotherapy for the treatment of primary hyperoxaluria.
Pilot clinical study, open, prospective and multicenter.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| stiripentol (Diacomit) | Experimental | Drug: stiripentol (Diacomit) |
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| stiripentol (Diacomit)Administration of stiripentol per os | Drug | stiripentol (Diacomit) |
Primary outcome
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 two 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.
Relative variation (%) of the molar ratio [oxaluria / creatinuria] between baseline and after three weeks of treatment.
8 secondary outcomes
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
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 (%) 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]
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]
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
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
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
Time frame: From start of participation of the patient to end of the treatment period (up to 8 weeks)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Mar 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing stiripentol (Diacomit) in people with Primary Hyperoxaluria, run by Biocodex at 3 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Mar 2021.