Acceptability study of a new paediatric form of vigabatrin in infants and children with infantile spasms or pharmaco-resistant partial epilepsy.

• Register: EUCTR
• Last refreshed on: 2 October 2017
• Main ID: EUCTR2014-000360-17-FR
• Date of registration: 17/06/2015
• Prospective Registration: Yes
• Primary sponsor: TARGEON
• Public title: Acceptability study of a new paediatric form of vigabatrin in infants and children with infantile spasms or pharmaco-resistant partial epilepsy.
• Scientific title: Acceptability study of a new paediatric form of vigabatrin in infants and children with infantile spasms or pharmaco-resistant partial epilepsy. Observational, descriptive, open-label, multi-centric, non-randomized study - SOLUWEST
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-000360-17
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

France

Contacts

• Name: Behrouz KASSAI KOUPAI
• Affiliation: Centre d'Investigation Clinique, HCL Lyon

• Name: Behrouz KASSAI KOUPAI
• Affiliation: Centre d'Investigation Clinique, HCL Lyon

Key inclusion & exclusion criteria

Inclusion criteria:
- Boys and girls.
- Patients with diagnosed infantile spasms (IS) or pharmaco-resistant partial onset seizures (POS).
- Infants > 1 month and < 6 months; infants > 6 months and < 2 years; and children > 2 years and < 6 years.
- Patients stabilized under Sabril® for at least 2 weeks prior to V1: patients with no spasms/POS or with a stable frequency of spasms/POS (i.e. with no more than 50% variation in the number of spasms/POS) within 2 weeks prior to V1.
- Patients under a twice-a-day posology of Sabril®.
- Subjects covered by or having the rights to social security.
- Signed informed consent and/or child assent.

Are the trial subjects under 18? yes
Number of subjects for this age range: 50
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- Change in anti-epileptic treatment and/or Sabril® dose within 7 days before V1.
- 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.
- Subjects receiving vigabatrin through a gastric tube.
- Weight < 4 Kgs.
- Any planned major surgery within the duration of the trial.
- Participation in any other clinical trial within 3 months prior to V1.
- Lack of ability or willingness to give informed consent.
- Anticipated non-availability for study visits / procedures.
- Lack of willingness or inability to co-operate adequately.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Infantile spasms and pharmaco-resistant partial epilepsy
MedDRA version: 18.0 Level: PT Classification code 10021750 Term: Infantile spasms System Organ Class: 10029205 - Nervous system disorders
MedDRA version: 18.0 Level: LLT Classification code 10065336 Term: Partial epilepsy System Organ Class: 10029205 - Nervous system disorders

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Product Name: vigabatrin
Product Code: VGB-ST
Pharmaceutical Form: Soluble tablet
INN or Proposed INN: VIGABATRIN
CAS Number: 60643-86-9
Current Sponsor code: VGB-ST
Other descriptive name: VGB-ST soluble tablets
Concentration unit: mg milligram(s)
Concentration type: range
Concentration number: 100-500

Trade Name: Sabril "granules for oral use"
Pharmaceutical Form: Granules for oral solution in sachet

Primary Outcome(s)

Primary end point(s): The primary endpoint is the proportion of adherent patients for the new VGB formulation. Adherence will be assessed by measurement of the dosing history of patients using an electronic Medication Event Monitoring System (MEMS).

Main Objective: To describe the adherence to a new vigabatrin formulation (soluble tablets).

Timepoint(s) of evaluation of this end point: Continuous endpoint between D15 and D98, 12 weeks under VGB-ST.

Secondary Objective: 1)Evaluate the palatability and the ease of use of the new vigabatrin formulation.
2)Describe the adherence to Sabril and the new formulation (by treatments accountability)
3)Evaluate the pharmacokinetic parameters of the new vigabatrin formulation.
4)Evaluate the treatment safety, including the visual safety (by electroretinogram).
5)Evaluate the taurine plasma concentrations in children treated with vigabatrin.

Secondary Outcome(s)

Secondary end point(s): - Adherence by accountability of Treatment units for the new ST formulation (number of tablets) and to Sabril® “granules for oral solution” (number of sachets).
- Palatability 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.
- Ease of use will be evaluated for Sabril® « granules for oral solution » and for the new ST formulation using two diaries filled by the parents or care-givers during 7 consecutive days. Time required for preparation of both VGB ST and Sabril® administrations will be averaged and compared, together with the global use satisfaction .
- Safety measures include:
o Results of electroretinogram (when available).
o General safety, including:
• blood cells count, blood electrolytes, blood ionogram, serum creatinine, liver function assessment
• vital signs (cardiac frequency and blood pressure)
• adverse events and serious adverse events.
- Pharmacokinetic parameters for the new ST formulation (population PK) : AUC, T max, Cmax, T½, Ka, V/F, Cl/F .
- Taurine plasma concentration.

Timepoint(s) of evaluation of this end point: - Accountability : D1 to D14 under Sabril ; D15 to D98 under VGB-ST.
- Palatability and ease of use, 7 consecutive days :D8 to D14 for Sabril® ; D22 to D28 under VGB-ST.
- Safety measures :
• blood assessment at V1 and V4,
• vital signs at V1, V2, V4 and V5.
• AE and SAE at each study visit.
- Pharmacokinetic:
• After V3, the same day, 1 sample just before treatment and 1 sample 1h after treatment.
• At least 1 week before V4, the same day, 1 sample 3 to 5h after treatment and 1 sample 6 to 9 h after treatment.
• At V4, 1 sample just before treatment and 1 sample 2h after treatment.
- Taurine concentration at V4 fasting.

Secondary ID(s)

TGO-VGB-III-01

Source(s) of Monetary Support

targeon

ANR BIOTECS