One of our partners in the DevelopAKUre consortium is an organisation called PSR Orphan Experts. PSR Orphan Experts are located in Hoofddorp, the Netherlands, and are currently working on the SONIA 2 clinical trial. Their team consists of one Project Manager, Christa van Kan, one Data Manager, Dinny Laan, and two Clinical Research Associates (CRA), Jolanda Overweel and Dennis Omtzigt, and one Clinical Trial Assistant (CTA), Jeannette Bhola.
In this week’s blog they tell us about their role. We all work according to the principles of ICH GCP (International Conference on Harmonisation of Good Clinical Practice) which is “an international ethical and scientific quality standard for designing, conducting, recording and reporting trials that involve the participation of human subjects”. Christa is mainly involved in all the regulatory paperwork. She also shares important safety information with both the drug manufacturer, and independent physicians involved in the safety review, and she provides updates to the consortium about the progress of the study.
Our CTA Jeannette is doing the administrative tasks. She takes care that all documentation that is generated throughout the study is filed for future reference. Our Data Manager is involved in the quality control of the data collected throughout the study and entered in a database. Both CRAs are keeping good and regular contact with the local investigational site staff in the various countries to ensure that the trial is conducted in compliance with the protocol and good clinical practice.
They regularly visit the sites during so called monitoring visits to check data from the patient’s medical charts and study assessment reports is correctly entered into the database. But most important is that they check if the rights, safety, and well-being of the trial patients are protected. One important condition must be met prior to clinical trial participation, and any trial related activities: the patient must have freely given informed consent.
The patient is always informed upfront by the hospital staff about the study, its progress, and the requirements of participation before performing any study related assessment. An official document, the Informed Consent Form, is signed by the patient and a member of the site staff. The CRA will check this form before review of any patient’s medical records.
The patient is always free to withdraw consent at any time, and leave the trial. As patients participating in the SONIA2 study already know, there are many assessments to be performed at each hospital visit. All these assessments result in data to be entered in the database by the site staff and to be verified by the CRA and Data Manager.
Any discrepancies between the data as entered, and the data found in source documents are discussed in order to ensure that the correct data are in the database. In the end our Data Manager will provide an extract of the “clean” data to the statisticians who will evaluate the outcome of the study. This is the beginning of obtaining a possible approval of the study medication from the regulatory authorities and to make it available for all patients looking for AKU treatment.
As our ultimate goal is to make a positive impact on the lives of people with a rare disease, we are happy to support to this important project.
DevelopAKUre is a series of major international clinical trials, run by a consortium of 12 European partners. It aims to study a potential new drug, called nitisinone, and assess its potential effectiveness in treating the rare disease, alkaptonuria (AKU).
DevelopAKUre is co-funded by a grant from the European Commission. This website is run by a UK patient group, the AKU Society. Learn more about AKU on the AKU Society's What is AKU page.
In today’s blog Chief investigator Prof. Ranganath reinforces the need for SONIA 2 pat.. ...more