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Metformin for treatment of polycystic ovary syndrome in children

Objective

PCOS, a common disorder affecting 5-10% of women of reproductive age is characterized by cystic ovarian morphology, androgen excess, and/or irregular periods and emerges during or shortly after puberty. The common off-label use of metformin for treating PCOS in children appears to have no greater risk than that in adults; drug tolerance is good with limited adverse effects. However, clinical trials are needed and requested by EMA to validate the safety and efficacy of this agent in PCOS.
In January 2012, the EMA released a revised priority list of PUMA generic medicines and Metformin was identified as a requested drug for treatment of PCOS in children. The call requests data on pharmacokinetics, efficacy and safety in this indication. A PIP was submitted to EMA in August 2012. In its final opinion of 17 May 2013 the PDCO recommend to agree to the PIP. The PDCO and the consortium have agreed that by restricting exposure to no more than 2000 mg metformin / day, no additional pharmacokinetic investigations are required or even requested by EMA.
METFIZZ will conduct the required product development and clinical studies to establish efficacy and safety of administering age-appropriate innovative effervescent soluble formulations of metformin. The studies will focus attention on ethics in this psychologically sensitive condition, guided by the current legislation and by engaging patients and caregivers along with an independent ethics expert.
Currently, there are no age-appropriate dosing forms of metformin for treatment of PCOS in paediatric populations. This can result in over or under dosing, lack of medication compliance, dangers of iatrogenic harms. METFIZZ embraces clinical development, manufacturing scale up, and registration aspects of this pharmaceutical development programme.
Coordinator SME EffRx together with 4 SME-CROs and at least 20-30 related study centres will carry out the clinical study in 4 EU member states. The statistical analysis plan requires approximately 150 patients to be randomized into the PCOS study. EffRx will lead the METFIZZ product commercialisation to benefit children in Europe.

Call for proposal

FP7-HEALTH-2013-INNOVATION-1
See other projects for this call

Funding Scheme

CP-FP - Small or medium-scale focused research project

Coordinator

EFFRX PHARMACEUTICALS SA
Address
Wolleraustrasse 41 B
8807 Freienbach
Switzerland
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
EU contribution
€ 1 839 700
Administrative Contact
Ana Johansson (Ms.)

Participants (6)

KLIFO AS
Denmark
EU contribution
€ 1 681 637
Address
Fruebjergvej 3
2100 Kobenhavn
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Alejandra Moerk (Ms.)
CAMPBELL CHARLES ASSOCIATES (2000)LIMITED
United Kingdom
EU contribution
€ 1 486 921
Address
Station Road 48
KT11 3BN Stoke D Abernon
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Ignazio Di Giovanna (Dr.)
SERMES PLANIFICACION
Spain
EU contribution
€ 366 229
Address
Calle Rufino Gonzalez 14
28037 Madrid
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Antonio Berlanga (Mr.)
RCTS - RANDOMIZED CLINICAL TRIALS
France
EU contribution
€ 398 875
Address
Rue Du Plat 38
69002 Lyon
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Yves Alamercery (Mr.)
SCIPROM SARL
Switzerland
EU contribution
€ 177 000
Address
Rue Du Centre 70
1025 Saint Sulpice
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Kirsten Leufgen (Dr.)
THE UNIVERSITY OF LIVERPOOL
United Kingdom
EU contribution
€ 49 600
Address
Brownlow Hill 765 Foundation Building
L69 7ZX Liverpool
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Jenny Lawson (Ms.)