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Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy (SoluWest)

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Targeon SAS
Sponsor:
Collaborators:
Institut National de la Santé Et de la Recherche Médicale, France
Hospices Civils de Lyon
National Research Agency, France
Information provided by (Responsible Party):
Targeon SAS
ClinicalTrials.gov Identifier:
NCT02220114
First received: August 14, 2014
Last updated: August 18, 2014
Last verified: August 2014
  Purpose

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 study,
  • evaluation of the tolerance,
  • measurement of taurine plasma levels. This study will recruit 50 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 12 clinical sites in France.

Condition Intervention
Infantile Spasms
Drug: Vigabatrin: Sabril® then Vigabatrin new ST formulation.

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Targeon SAS:

Primary Outcome Measures:
  • Adherence to the new Soluble Tablets (ST) formulation of Vigabatrin (VGB) using Medication Event Monitoring System (MEMS) [ Time Frame: from V2 (day 14) to V4 (day 98), continuous assessment. ] [ Designated as safety issue: No ]
    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


Secondary Outcome Measures:
  • Adherence to the new ST formulation and to Sabril® granules for oral solution, by treatment unit accountability [ Time Frame: Sabril®:V1 (day 1) to V2 (day 14) and VGB-ST: V2 (day 14) to V4 (day 98). ] [ Designated as safety issue: No ]
    Accountability of used and unused treatment units, retrieved by the patient at V2 and V4

  • palatability of the new ST formulation and of Sabril® "granules for oral solution". [ Time Frame: during 7 consecutive days: from D8 to D14 under Sabril® and from D22 to D28 under the new ST formulation ] [ Designated as safety issue: No ]
    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).

  • Ease of use of the new ST formulation and of Sabril® "granules for oral solution". [ Time Frame: during 7 consecutive days: from D8 to D14 under Sabril® and from D22 to D28 under the new ST formulation. ] [ Designated as safety issue: No ]
    Ease of use will be evaluated using diaries filled by the parents or care-givers during 7 consecutive days: from D8 to D14 under Sabril® and from D22 to D28 under the new ST formulation. Time required for preparation of both new ST formulation and Sabril® administrations will be averaged and compared, together with the global use satisfaction.

  • Safety and tolerance [ Time Frame: Results of electroretinogram: when available from D1 to D126; Blood assessment: at D1 & D98; Vital signs at D1, D14, D98 & D126; Adverse events, serious adverse events: evaluated for the duration of study participation (at D1, D14, D42, D98 & D126) ] [ Designated as safety issue: Yes ]
    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.

  • pharmacokinetic parameters for the new ST formulation (population PK). Pharmacokinetic parameters for the new ST formulation (population PK) : Area under the curve (AUC), Tmax, Cmax, T½, Ka, V/F, Cl/F [ Time Frame: PKA (1 week after D42): 1 sample before and 1 sample 1h after treatment; PKB (1 week before D98 and 1 month post-PKA): 1 sample between 3-5h and 1 sample between 6-9h post-treatment; PK D98: 1 sample before and 1 sample 2h after treatment. ] [ Designated as safety issue: No ]
    The objective of the population PK analysis is to better characterize the developmental PK of vigabatrin during childhood.

  • 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 V4 (day 98): just before treatment when patient is fasting. ] [ Designated as safety issue: No ]

Estimated Enrollment: 50
Study Start Date: May 2014
Estimated Primary Completion Date: April 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Vigabatrin: Sabril® then Vigabatrin new ST formulation

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.

Drug: Vigabatrin: Sabril® then Vigabatrin new ST formulation.
  • a first "run-in" phase (D1-D14), in which the patient (already stabilized under Sabril® "granules for oral solution") continues treatment with Sabril®,
  • a second "treatment" phase (D15-D98) in which the patient is switched to the new vigabatrin ST form at the same dose and treatment regimen than Sabril®. At D99 the patient is switched back to its earlier Sabril® treatment (or another antiepileptic drug) at discretion of investigator. During the "treatment phase", the concomitant antiepileptic treatments are permitted to be changed (dose, type) if necessary, according to clinical practice, but no change in the dose of the new vigabatrin ST form is permitted,
  • a third 'follow-up" phase with no study treatment (D99-D126).

  Eligibility

Ages Eligible for Study:   1 Month to 6 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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 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®.

Non inclusion 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02220114

Contacts
Contact: Hugues BIENAYME +33142770818 h.bienayme@targeon-pharma.com
Contact: Segolene GAILLARD +33427857728 segolene.gaillard@chu-lyon.fr

Locations
France
Service de neurologie pédiatrique - CHU Recruiting
Amiens, France, 80054
Principal Investigator: Patrick BERQUIN         
Service de neurologie pédiatrique - CHU Recruiting
Angers, France, 49033²
Principal Investigator: Sylvie N'GUYEN         
Service de neuropédiatrie - CHU Pellegrin Enfants Completed
Bordeaux, France, 33076
Service de neurologie infantile - Hôpital Salengro Not yet recruiting
Lille, France, 59037
Principal Investigator: Louis VALLEE         
Service de nuerologie pédiatrique - Hôpital Femme Mère Enfant Recruiting
Lyon, France, 69677
Principal Investigator: Dorothée VILLE         
Service de neurologie pédiatrique - Hôpital de la Timone Recruiting
Marseille, France, 13385
Principal Investigator: Nathalie VILLENEUVE         
Service de neurologie pédiatrique - Hôpital Necker Enfants Malades Active, not recruiting
Paris, France, 75015
Service de neuropédiatrie - Hôpital Robert Debré Not yet recruiting
Paris, France, 75019
Principal Investigator: Stephane AUVIN         
Service de neurologie pédiatrique - Hôpital Sud Recruiting
Rennes, France, 35203
Principal Investigator: Sylvia NAPURI         
Centre référent des épilepsies rares pédiatrique associé - Hôpital de Hautepierre Recruiting
Strasbourg, France, 67098
Principal Investigator: Anne DE SAINT MARTIN         
Service de neuropédiatrie - Hôpital Purpan Recruiting
Toulouse, France, 331059
Principal Investigator: Claude CANCES         
Service de neuropédiatrie - Hôpital de Clocheville Recruiting
Tours, France, 37044
Principal Investigator: Marie-Anne BARTHEZ         
Sponsors and Collaborators
Targeon SAS
Institut National de la Santé Et de la Recherche Médicale, France
Hospices Civils de Lyon
National Research Agency, France
Investigators
Principal Investigator: Rima NABBOUT Hôpital Necker Enfants Malades - APHP
  More Information

No publications provided

Responsible Party: Targeon SAS
ClinicalTrials.gov Identifier: NCT02220114     History of Changes
Other Study ID Numbers: TGO-VGB-III-01
Study First Received: August 14, 2014
Last Updated: August 18, 2014
Health Authority: France: Agence Nationale de Sécurité du Médicament et des produits de santé

Keywords provided by Targeon SAS:
Infantile spasms
vigabatrin
soluble tablets
acceptability
adherence
pharmacokinetics

Additional relevant MeSH terms:
Vigabatrin
Epilepsy
Epilepsies, Partial
Spasm
Spasms, Infantile
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Signs and Symptoms
Epilepsy, Generalized
Anticonvulsants
Central Nervous System Agents
Therapeutic Uses
Pharmacologic Actions
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
GABA Agents
Neurotransmitter Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on August 28, 2014