Harvey has been taking part in a clinical trial at Alder Hey to help improve the treatment of rare seizure disorders.
Clinical trials help scientists to find new treatments, modify use of existing treatments, help prevent diseases and improve care. By volunteering to take part in research and with the support of his mother, Harvey is playing a crucial part in testing how we manage treatment of patients with rare seizure disorders (such as Dravet Syndrome).
What is Dravet Syndrome?
Dravet Syndrome is a rare, life-limiting and devastating genetic neurological condition occurring in 1 in 15,000 people in the UK. The condition causes treatment-resistant epilepsy and intellectual disability alongside a spectrum of associated conditions including autism, ADHD, challenging behaviour, difficulties with speech, mobility, feeding and sleep. The range and severity of symptoms vary from one individual to another. Seizures typically begin during the first year of life. Over time other seizure types can occur and other symptoms emerge
Harvey’s clinical trial
Harvey is part of an international research trial led by a pharmaceutical company named Zogenix International Limited, that recruited participants from the UK and EU. The study is testing the effects and benefits of a treatment called fenfluramine hydrochloride, which has previously been used to treat other conditions. Harvey has been part of an open label extension of an earlier research study into the effects of this treatment for this condition, and it is hoped he will benefit from continuing to receive this treatment.
Harvey’s mother Alison has given her thoughts on their experience of participating in this research study at the NIHR Alder Hey Clinical Research Facility. Alison says she was “very happy and willing to take part”, “extremely grateful to be given the opportunity” and that “we feel Harvey mattered.” Alison wanted Harvey to continue with the treatment as “we had been well informed about the possible success of the study medication and were hopeful that Harvey might gain better seizure control with it (alongside his current medications).”
With regards to their experience of taking part in research at Alder Hey, Alison says it has been a “very positive and rewarding experience… from doctors, nurses, cardiology and pharmacy we have been treated extremely well… and given extremely dedicated support.” In relation to Harvey’s treatment, Alison advises that Harvey has had a positive experience “both seizure wise and overall behaviour and developmentally. His care during each visit was very good and he was treated with dignity.”
Finally, if Alison could share a message to other parents who are considering being part of a research study at Alder Hey, she would…
“fully encourage any parent to enrol on the research study. It is run sufficiently with much care and attention to details…and all the staff are caring and professional and always supportive!”
Alison, Harvey’s mum
Catherine McBurney, the lead research nurse for Harvey’s research study along with research nurses based at the CRF and Clinical Research Division, advises that, “It is lovely to hear from patients and their families about the positive impact this research has had. I took over this study from another nurse who lead on the partner study. When I introduced myself to the family, they explained the positive impact this study had had on Harvey and were delighted at his seizure reduction. It was lovely working with the family and being part of such an important life changing study.”
Dr Dan Hawcutt, Director of the NIHR Alder Hey Clinical Research Facility and lead investigator for the study at Alder Hey said:
“Without families and patients like Harvey, we would not be able to get medicine like Fenfluramine to patients. This is a success story both for them, and for giving people across the UK as the drug is now licensed and available.”
Dr. Dan Hawcutt