Workpackage 01

Workpackage lead and partners

WP lead: Dr. Ronit Pressler (UCL)

WP partners: UCC, O4CP, INSERM, HUS, UNIVLEEDS, UU, UMCU, AP-HP, KI, GOSHFCNT, EMC

Workpackage objectives

It is the objective of this work package to develop an effective management framework to facilitate the clinical trials of NEMO. This includes managerial and administrative tasks at a central level as well as coordination and facilitation at individual centres including compliance with GCP (good clinical practice) as well as safety, legal and ethical issues. This will ensure the correct progress of the project towards its planned objectives.

Workpackage description

Two clinical trials are planned:

NEMO 1 -  phase I/IIa: dose finding, feasibility and pharmacokinetic study of bumetanide for the treatment of neonatal seizures not responding to phenobarbitone with the aim to estimate the optimal dose of bumetanide as an antiepileptic drug in neonatal seizures. 

NEMO 2 - phase IIb/III, randomized, placebo-controlled, double-blind, clinical trial to evaluate the efficacy and safety of the optimal dose of bumetanide for treatment of neonatal seizures not responding to phenobarbitone.

Both trials will be conducted by highly specialised centres from five different European countries requiring a detailed framework which will be set up during the first 6-9 months. This is the main task of WP 01 in close relationship with the sponsors of the trial (GOSH-FCNT and O4CP), other WP partners (UCC, INSERM, HUS, AP-HP) and the principal investigators of the clinical trial.

WP 01 will set up a trial management group and appoint clinical trial experts. Furthermore, setting up of the relevant independent advisory groups will be facilitated in accordance with the project milestones such as ethics advisory group (WP 05), clinical development management unit (WP 11) and the data and safety monitoring committee (WP 05).

Tasks of WP 01:

• The initial most important tasks will be to finalising the clinical trial protocol for the clinical trials with input form all principal investigators and scientific leads. We will ensure that the protocol complies with GCP (good clinical practice) and that all necessary quality standards will be observed for documentation, data collection, validation, evaluation, archiving and reporting of adverse events.
• Together with WP 5 we will write the patient information sheet and consent form for NEMO1.
• In collaboration with other work packages trial procedures will be reviewed, Standard Operating Procedures (SOPs) be written and distributed.
• Training in GCP is essential for all investigators and clinical trial staff. GOSH-FCNT will offer training to all clinical partners of NEMO.
• Together with WP 11 we will confirm that all sites are suitable for the trial and pre-trial monitoring report will be produced.
• We will organise a clinical investigator meeting prior to commencing the trial together with WP 02.
• We will oversee and facilitate the writing, collection, finalisation and distribution of essential study documents which will be kept in the Trial Master File. The overall responsibility of the Trial Master File will lay with the sponsor.

Several tasks in the setup are the responsibility of the local principal investigators and scientific leads. We will support local trial centres in these activities:

• Development of parent information sheets and consent forms compliant with GCP.
• Obtaining approval from their local Ethics Committees.
• Approval from local Research and Development centres.
• Authorisation of the Competent Authorities in each country.

GCP training of all clinical trial staff involved in the trial.