Infantile SpasmsSoluWestNCT02220114
Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy
A study testing Vigabatrin: Vigabatrin new ST formulation then Sabril® in people with Infantile Spasms, run by Orphelia Pharma at 12 sites in France.
Trial summary
The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution… Read more about this study
- Who is it for?
- Ages 1 month to 6 years
- Conditions the study is for
- Infantile Spasms
- What is being tested?
- Vigabatrin: Vigabatrin new ST formulation then Sabril®
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 12 sitesFrance
- Study dates
- May 2014to Dec 2016
- Phase
- Interventional
- 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 1 month to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients with diagnosed infantile spasms (IS) or pharmacoresistant partial onset seizures (POS).
- Infants > 1 month and < 6 months; infants > 6 months and < 2 years; and children > 2 years and < 6 years.
- Patients under Sabril® or naive patients.
- Patients under a twice-a-day posology of Sabril® or patients for whom vigabatrin will be given twice daily.
- Non inclusion Criteria:
- Use of more than 2 other antiepileptic drugs as concomitant treatment (including steroids). Ketogenic diet can be in addition to these 2 other antiepileptic drugs.
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- Subjects receiving vigabatrin through a gastric tube.
- Weight < 1.750 Kgs.
- Any planned major surgery within the duration of the trial.
- Participation in any other clinical trial within 3 months prior to V1.
Who cannot join
None listed.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
France12 sites
Service de neurologie pédiatrique - CHU
Amiens, 80054
Service de neurologie pédiatrique - CHU
Angers, 49033²
Service de neuropédiatrie - CHU Pellegrin Enfants
Bordeaux, 33076
Service de neurologie infantile - Hôpital Salengro
Lille, 59037
Service de nuerologie pédiatrique - Hôpital Femme Mère Enfant
Lyon, 69677
Service de neurologie pédiatrique - Hôpital de la Timone
Marseille, 13385
Service de neurologie pédiatrique - Hôpital Necker Enfants Malades
Paris, 75015
Service de neuropédiatrie - Hôpital Robert Debré
Paris, 75019
Service de neurologie pédiatrique - Hôpital Sud
Rennes, 35203
Centre référent des épilepsies rares pédiatrique associé - Hôpital de Hautepierre
Strasbourg, 67098
Service de neuropédiatrie - Hôpital Purpan
Toulouse, 331059
Service de neuropédiatrie - Hôpital de Clocheville
Tours, 37044
About this study
Official title: Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy. Observational, Descriptive, Open-label, Multi-centric, Non-randomized Study
The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). Sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. Manual splitting of the sachet or lengthy and error-prone dilutions are often required.
This study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. The primary objective is to describe the adherence to the new formulation. Secondary objectives include:
- evaluation of the palatability and user-friendliness of the new treatment,
- evaluation of the pharmacokinetic parameters of the new formulation,
- PK parameters,
- evaluation of the tolerance,
- measurement of taurine plasma levels. This study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in France.
- Conditions
- Infantile Spasms
- Being tested
- Vigabatrin: Vigabatrin new ST formulation then Sabril®
- Keywords
- Infantile spasms, vigabatrin, soluble tablets, acceptability, adherence, pharmacokinetics
- Medical subject headings
- Spasms, Infantile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vigabatrin: Vigabatrin new ST formulation then Sabril®Drug
- first "treatment" phase (V1/D1-V3/D84), in which patients already under Sabril® "granules for oral solution" and naive patients start the new ST formulation; patients already under Sabril® will start at the same dose and regimen as their usual Sabril®. Dose and regimen for naive patients will be chosen according to SPC.
- second "treatment" phase (V3/D84-V4/D98) in which the patient is switched to Sabril® "granules for oral solution" (supplied by sponsor) for 15 days at the same dose as under the new ST formulation.
Dose and treatment regimen should be maintained as in first treatment phase.
- At V4/D98, patients who received Sabril® "granules for oral solution" (supplied by sponsor) continue with marketed Sabril® treatment (or switches to another AED, according to the natural evolution of the patient's condition and upon investigator decision).
| Study group | Role | What they receive |
|---|---|---|
| Vigabatrin: Vigabatrin new ST formulation then Sabril® | Other | Sabril®: sachet for oral solution 500 mg, 50 to 100mg/Kg/day, twice a day, 14 days.
Vigabatrin new ST formulation: Soluble tablets 100 or 500 mg, 50 to 100mg/Kg/day, twice a day, 12 weeks. Receives: Drug: Vigabatrin: Vigabatrin new ST formulation then Sabril® |
What is being measured
The primary outcome is the main question the study is designed to answer.
Individual adherence to the new Soluble Tablets (ST) formulation of Vigabatrin (VGB) using Medication Event Monitoring System (MEMS)
Adherence will be assessed by measurement of the dosing history of patients using an electronic Medication Event Monitoring System (MEMS).The date and time of each opening will be recorded
Measured: from V1 (day 1) to V3 (day 84), continuous assessment.
Secondary outcomes (6)
Adherence to the new ST formulation and to Sabril® granules for oral solution, by treatment unit accountability
Accountability of used and unused treatment units, retrieved by the patient at V3 and V4
Measured: VGB-ST: V1 (day 1) to V3 (day 84). Sabril®:V3 (day 85) to V4 (day 98)
palatability of the new ST formulation and of Sabril® "granules for oral solution".
Palatability of the treatment will be evaluated using a two face visual hedonic scale filled in by the parents and/or by the child, if feasible, on a daily basis. Each "face" of the scale will be assigned a score (1 to 2), and the average score will be calculated for the group. Palatability will be considered good if the average score for the group is at least 1.5 (out of a maximum of 2; Motte et al. 2005).
Measured: during 7 consecutive days: from D90 to D96 under Sabril® and from D29 to D35 under the new ST formulation
Ease of use of the new ST formulation and of Sabril® "granules for oral solution".
Ease of use will be evaluated using diaries filled by the parents or care-givers during 7 consecutive days: . Time required for preparation of both new ST formulation and Sabril® administrations will be averaged and compared, together with the global use satisfaction.
Measured: during 7 consecutive days: from D90 to D96 under Sabril® and from D29 to D35 under the new ST formulation.
Safety and tolerance
VGB safety profile is well known. The new ST formulation is expected to be bioequivalent to Sabril®, and since the new formulation does not contain excipients known to have a recognized action or effect at the dose used, the safety of the new formulation should be similar to that of Sabril®. Hence no other measures specific to the new ST formulation are included in the clinical acceptability study.
Measured: Results of electroretinogram: when available from D1 to D126; Blood assessment: at D1 & D84; Vital signs at D1, D28, D84, D98 & D126; Adverse events, serious adverse events: evaluated for the duration of study participation (at D1, D28, D84, D98 & D126)
pharmacokinetic parameters for the new ST formulation (1 sample). Pharmacokinetic parameters for the new ST formulation (population PK) : Area under the curve (AUC), Tmax, Cmax, T½, Ka, V/F, Cl/F
The objective is to better characterize the developmental PK of vigabatrin during childhood.
Measured: PK D84: 1 sample before treatment.
Evaluation of the taurine plasma levels in children treated by vigabatrin. Taurine plasma concentration will be measured and a relationship between vigabatrin exposition and taurine plasma levels will be sought.
Measured: 1 sample will be drawn at V3 (day 84): just before treatment when patient is fasting.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2014 (estimated)
- Study startMay 2014
- Primary completionDec 2016
- Study completionDec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Infantile SpasmsStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- Orphelia Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Institut National de la Santé Et de la Recherche Médicale, France, Hospices Civils de Lyon, National Research Agency, France
- Responsible party
- Sponsor
- Investigators
- Rima NABBOUT, Principal investigator, Hôpital Necker Enfants Malades - APHP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02220114
- Sponsor's protocol ID
- TGO-VGB-III-01
- Acronym
- SoluWest
- Status last verified
- Apr 2018
- First submitted
- 14 Aug 2014
- Last updated
- 24 Apr 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy. Observational, Descriptive, Open-label, Multi-centric, Non-randomized Study
- ClinicalTrials.gov ID
- NCT02220114
- Acronym / sponsor ID
- SoluWest / TGO-VGB-III-01
- Conditions
- Infantile Spasms
- Keywords
- Infantile spasms, vigabatrin, soluble tablets, acceptability, adherence, pharmacokinetics
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- Orphelia PharmaIndustry (pharma, biotech, devices)
- Collaborators
- Institut National de la Santé Et de la Recherche Médicale, France, Hospices Civils de Lyon, National Research Agency, France
- Study start
- May 2014
- Primary completion
- Dec 2016 (actual)
- Study completion
- Dec 2016 (actual)
- First posted
- 19 Aug 2014 (estimated)
- 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.
The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). Sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. Manual splitting of the sachet or lengthy and error-prone dilutions are often required.
This study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. The primary objective is to describe the adherence to the new formulation. Secondary objectives include:
- evaluation of the palatability and user-friendliness of the new treatment,
- evaluation of the pharmacokinetic parameters of the new formulation,
- PK parameters,
- evaluation of the tolerance,
- measurement of taurine plasma levels. This study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in France.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vigabatrin: Vigabatrin new ST formulation then Sabril® | Other | Drug: Vigabatrin: Vigabatrin new ST formulation then Sabril®Sabril®: sachet for oral solution 500 mg, 50 to 100mg/Kg/day, twice a day, 14 days. Vigabatrin new ST formulation: Soluble tablets 100 or 500 mg, 50 to 100mg/Kg/day, twice a day, 12 weeks. |
Who may qualify
- Ages
- 1 month to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- Patients with diagnosed infantile spasms (IS) or pharmacoresistant partial onset seizures (POS).
- Infants > 1 month and < 6 months; infants > 6 months and < 2 years; and children > 2 years and < 6 years.
- Patients under Sabril® or naive patients.
- Patients under a twice-a-day posology of Sabril® or patients for whom vigabatrin will be given twice daily.
- Use of more than 2 other antiepileptic drugs as concomitant treatment (including steroids). Ketogenic diet can be in addition to these 2 other antiepileptic drugs.
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| Vigabatrin: Vigabatrin new ST formulation then Sabril®* first "treatment" phase (V1/D1-V3/D84), in which patients already under Sabril® "granules for oral solution" and naive patients start the new ST formulation; patients already under Sabril® will start at the same dose and regimen as their usual Sabril®. Dose and regimen for naive patients will be chosen according to SPC. * second "treatment" phase (V3/D84-V4/D98) in which the patient is switched to Sabril® "granules for oral solution" (supplied by sponsor) for 15 days at the same dose as under the new ST formulation. Dose and treatment regimen should be maintained as in first treatment phase. - At V4/D98, patients who received Sabril® "granules for oral solution" (supplied by sponsor) continue with marketed Sabril® treatment (or switches to another AED, according to the natural evolution of the patient's condition and upon investigator decision). | Drug | Vigabatrin: Vigabatrin new ST formulation then Sabril® |
Primary outcome
Individual adherence to the new Soluble Tablets (ST) formulation of Vigabatrin (VGB) using Medication Event Monitoring System (MEMS)
Time frame: from V1 (day 1) to V3 (day 84), continuous assessment.
Adherence will be assessed by measurement of the dosing history of patients using an electronic Medication Event Monitoring System (MEMS).The date and time of each opening will be recorded
6 secondary outcomes
Adherence to the new ST formulation and to Sabril® granules for oral solution, by treatment unit accountability
Time frame: VGB-ST: V1 (day 1) to V3 (day 84). Sabril®:V3 (day 85) to V4 (day 98)
palatability of the new ST formulation and of Sabril® "granules for oral solution".
Time frame: during 7 consecutive days: from D90 to D96 under Sabril® and from D29 to D35 under the new ST formulation
Ease of use of the new ST formulation and of Sabril® "granules for oral solution".
Time frame: during 7 consecutive days: from D90 to D96 under Sabril® and from D29 to D35 under the new ST formulation.
Safety and tolerance
Time frame: Results of electroretinogram: when available from D1 to D126; Blood assessment: at D1 & D84; Vital signs at D1, D28, D84, D98 & D126; Adverse events, serious adverse events: evaluated for the duration of study participation (at D1, D28, D84, D98 & D126)
pharmacokinetic parameters for the new ST formulation (1 sample). Pharmacokinetic parameters for the new ST formulation (population PK) : Area under the curve (AUC), Tmax, Cmax, T½, Ka, V/F, Cl/F
Time frame: PK D84: 1 sample before treatment.
Evaluation of the taurine plasma levels in children treated by vigabatrin. Taurine plasma concentration will be measured and a relationship between vigabatrin exposition and taurine plasma levels will be sought.
Time frame: 1 sample will be drawn at V3 (day 84): just before treatment when patient is fasting.
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 24 Apr 2018.
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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.
Rima NABBOUT principal investigator
Hôpital Necker Enfants Malades - APHP
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Vigabatrin: Vigabatrin new ST formulation then Sabril® in people with Infantile Spasms, run by Orphelia Pharma at 12 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 10 inclusion and 0 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 24 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 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.
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?
Individual adherence to the new Soluble Tablets (ST) formulation of Vigabatrin (VGB) using Medication Event Monitoring System (MEMS) (time frame: from V1 (day 1) to V3 (day 84), continuous assessment.). 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 24 Apr 2018.