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KIEKIDS Report Summary

Project ID: 282559
Funded under: FP7-HEALTH
Country: France

Periodic Report Summary 3 - KIEKIDS (Development of an innovative paediatric formulation of an antiepileptic agent for the treatment of absence epilepsy in children.)

Project Context and Objectives:
The KIEKIDS project is dedicated to the development of an innovative paediatric formulation of an antiepileptic agent for a safe alternative treatment of absence epilepsy in children. Development of such an age-appropriate formulation, using a drug with identified and documented efficacy in the target population, is regarded by the European Medicines Agency (EMA) to be amongst the highest priorities for the treatment of epilepsy.
The KIEKIDS project has gathered a very strong consortium of well trained and qualified partners from industry, academy, and hospital with extensive experience in drug development in clinical, pharmacological, data analysis and regulatory areas.
The proposed strategy for KIEKIDS implies to develop a dedicated paediatric formulation and to conduct a clinical programme, which will be endorsed by approval of the Paediatric Investigation Plan (PIP).
The consortium is coordinated by the Unit 1129 (ex 663) of INSERM (INSERM U1129), a French public organisation of research devoted to the paediatric epilepsies and brain plasticity. This consortium rallies all the competencies required to manage a paediatric drug development from the designing of an adapted formulation up to market.
With its solid track record of clinical success in pharmaceutical and biotech companies, and its huge expertise in absence epilepsy, this consortium will be able to avoid the pitfalls of pharmaceutical drug development and is in a strong position to guarantee the success of the programme.
The ultimate goal of KIEKIDS is to submit an application for a Paediatric-Use Marketing Authorisation, a PUMA, the tool identified by the EMA to bridge the gap for off-patent drugs, concerning this novel age-adapted paediatric formulation.

Project Results:
In work package 1, an age-adaptable formulation consisting of lipid granules was selected and optimised. The main selection criteria were (i) an acceptable palatability, (ii) an adequate kinetic profile and (iii) industrial feasibility. Twelve month stability data have been gathered with a first clinical batch and further stability studies are on-going. The first clinical batch was produced for phase I study part A and two optimised clinical batches were produced for the next parts of phase I study (part AR and part AS). The scale-up aimed at adapting the process for production of the clinical phase II batch (batch size of 5-10 kg), which will take place in June 2016.
In Work package 2 the phase I study was organised in the facilities of the Department of Clinical Pharmacology of CHUV and conducted the study. The pharmacokinetic parameters from part A of the study, allowed comparing two different formulations with the reference product. During parts AR and AS of the study, pharmacokinetic parameters have been confirmed (including bioequivalence with the reference formulation). Exploratory work was performed for saliva during the two first parts of the Phase I study. Part AS has allowed confirming the good palatability of the optimised formulation, which is now ready to be evaluated in paediatric patients.
As part of Work package 4, the whole investigation plan was discussed with the CHMP and the PDCO and validated.
The Phase I study protocol was amended twice and additional parts AR and AS were included and submitted for approval and the corresponding authorisations were obtained. The next step will be the submission of the CTA for the Phase II study in June 2016.
As part of Work package 3 phase II study protocol has been written and the investigators have been organised and are ready to start in September 2016, once the clinical batch will be available.
As part of the work package 5, an efficient organisation and decision making structure has been built, with a very active consortium that meets regularly and is characterised by open communication leading to fruitful discussions. The web site published in work package 6 allows sharing documents between partners.

Potential Impact:
The direct impact of the KIEKIDS project will be the availability of an adequate paediatric formulation for the treatment of absence and myoclonic epilepsy, identified as a high priority by EMA, with dedicated pharmacokinetic and safety data in children.
Absence and myoclonic seizures (5 to 10% each of the total paediatric epileptic population) represent 100 000 to 200 000 children in Europe. In most instances, epilepsy requires a prolonged treatment during childhood and has a significant impact on medical and social aspects of patient’s life:

• Seizures are not predictable: they can occur at any time. Apart from the fact that seizures are inconvenient and embarrassing for patients and family, they can represent a real danger to the patient depending on his environment during the crisis.

• Seizures also induce a negative impact on patient’s social behaviour, triggering isolation and loss of self-esteem.

• Childhood epilepsy is connected to learning disabilities and memory problems:

o Symptoms are overrepresented among epileptic children.

o Attention deficit disorders are emphasized during absence seizures.

o Adverse side-effects such as drowsiness and short attention impact education achievement.

o These symptoms are exacerbated by polytherapy!

Proposing an efficient treatment of childhood absence or myoclonic epilepsy would result in significant improvement of social conditions for these patients.

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