Your invitation to take part in a new qualitative study on ‘Burden Of and Lessons Learnt from Trials’ by our patient community
Patient organisations and our communities have learnt important lessons from the participation and involvement in the first studies for Myotubular and Centronuclear Myopathies (MTM & CNM). We would like to share the experiences of individual trial participants and of the patient organisations as a whole with clinicians, researchers and companies involved in future trial design. The lessons learnt by individual patients and patient advocacy groups will help drug studies in the future.
How you can help with this Study
Since 2017, the first natural history studies and clinical trials (studies to test the safety, and effect, of new drugs) in MTM & CNM have been taking place:
• ASPIRO: Gene Transfer Clinical Study in X-Linked Myotubular Myopathy (formerly by Audentes, now Astellas)
• Unite-CNM: Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies (by Dynacure)
• Tamoxifen Therapy for Myotubular Myopathy (TAM4MTM), (by James Dowling, The Hospital for Sick Children, Toronto, Canada, and Giovanni Baranello, Great Ormond Street Hospital, London)
The first results of the ASPIRO trial have been discussed with the patient organisations and presented at international conferences. This includes information on the effects of the trial, and some serious adverse events. Reports have been submitted to and will be published in medical journals. The Dynacure ‘Unite-CNM’ trial was stopped in the summer of 2022. The tamoxifen trial is still ongoing, with the Great Ormond Street Hospital London site due to begin recruitment.
The German patient organisation, ZusammenStark, set out to investigate the burden of, and the lessons learnt, from the first studies from a patient perspective by a qualitative approach with two Focus Groups at their family and scientific conference in May 26th – 29th in Bad Nauheim, Germany. They have subsequently invited Myotubular Trust to involve the UK community in two virtual workshops in the UK to take place in September and October.
A Focus Group is a small group of six to ten people led through an open discussion by a skilled moderator. The group needs to be large enough to generate rich discussion but not so large that some participants are left out.
Dr Nicol Voermans will participate as the medical expert. She is a neurologist specialised in congenital myopathies in Nijmegen, the Netherlands and member of the scientific advisory board of ZusammenStark.
Professor Heinz Jungbluth will
participate as the medical expert in the UK focus groups. He is a pediatric neurologist specialised in congenital myopathies in London, UK, and active member of Myotubular Trust.
Your invitation to join our Focus Group
You are invited to participate in one of the Focus Groups.
The meeting will be recorded, and the data will be analysed. The researchers aim is to present the results of this next year (2023); in a manual for future trials for other patient organisations; and in a scientific publication for researchers. The data analysis will be performed anonymously: your contribution will not be recognisable. The study will focus on the burden of participation and on your experience that can help to improve future trial design. You will not be asked to share any details from the trial design you took part in, so there will be no conflict of interest.
Get in touch with us to take part
You can contact Nicol Voermans and Lizan Stinissen (research assistant) if you are willing to participate or in case of any questions on the study:
Please email: Lizan.Stinissen@radboudumc.nl
You will need to send us
Your name, date of birth, email address, which study you were involved with, and what your role is or was (study participant, carer, parent or interested member of Myotubular Trust). The aim is to have mixed groups.
You can also contact the Myotubular Trust with any questions you may have.
We look forward to hearing from you,
Nicol Voermans, Lizan Stinissen and Wija Oortwijn
Radboudumc, Nijmegen, the Netherlands
Heinz Jungbluth
Evelina’s Children Hospital, London, UK
Supported by:
