Manchester University NHS Foundation Trust (MFT) was formed on 1st October 2017 following the merger of Central Manchester University Hospitals NHS Foundation Trust (CMFT) and University Hospital of South Manchester NHS Foundation Trust (UHSM).
Royal Manchester Children's Hospital provides specialist healthcare services for children and young people throughout the North West.
The hospital sees 220,000 patient visits each year across a range of specialities and has 371 beds.
RMCH has a dedicated National Institute for Health Research (NIHR) Manchester / Wellcome Trust Clinical Research Facility at Royal Manchester Children’s Hospital. This is a bespoke research unit, with state of the art equipment, dedicated to the testing of and development of new therapies for a wide range of childhood diseases. The CRF has the capacity for complex inpatient clinical trials including rare metabolic disease, cancer, autism and kidney failure. There are 2 clinical trial coordinators, 16 research nurses and practitioners and a data administrator. The unit has been open 8 years, is positioned in the heart of RMCH and has extensive experience of early phase trials including trials in DMD.
The Paediatric Neurology Department currently has eight Consultants two of whom have an interest in neuromuscular disease and see and care for approximately 400 patients with various neuromuscular conditions. Of these patients around 70 have Duchenne Muscular Dystrophy (DMD). The Manchester neuromuscular service is well-established with close links with relevant clinical services, including respiratory, with facility for polysomnography and ventilator support, cardiology, metabolic bone, orthopaedics (spinal surgeons, foot surgeon and upper limb surgeon), orthotics, histopathology, radiology, genetics and inherited metabolic disease. Joint clinics are held with respiratory and orthopaedic colleagues. There is a regular neuromuscular scoliosis MDT. There is a transition clinic
Since 2013 the Manchester team is or has already been involved in a number of clinical trials, these have included early phase and phase 3 trials and natural history studies.
RMCH participation (past & current) in neuromuscular clinical trials and studies:
SMT C1100 A Phase 1, Openlabel, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients with Duchenne Muscular Dystrophy
A Phase 3 Efficacy and Safety Study of Ataluren (ptc124) in Patients with Nonsense Mutation Dystrophinopathy
SMT C11003 - A Phase 1b placebo-controlled, multi-centre, randomized, double-blind 3-period dose escalation study to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a balanced diet
A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects with spinal muscular atrophy who are not eligible to participate in the clinical studies ISIS 396443-CS3B or ISIS 396443-CS4
A Phase I, 2-Part, Open-label, Multiple Oral Dose Study of the Safety, Tolerability and Pharmacokinetics of up to 2 Formulations of SMT C1100 in Healthy Adult Male Subjects and a Selected Formulation of SMT C1100 in Paediatric Subjects with Duchenne Muscular Dystrophy (DMD)
A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005)
Isolation of mesoangioblasts from patients affected by Duchenne Muscular Dystrophy with a skippable mutation of exon 51 for the development of a novel ATIMP (Advanced Therapy Investigational Medicinal Product): a non-randomized, open-label, phase I study
DMD05/04 Wellcome Trust sponsored trial currently pre-clinical stage
North Star Clinical Network
Royal Manchester Children’s Hospital
Manchester University Hospitals NHS Foundation Trust
|Study Name||Trial Status at Manchester||Overall Trial Status|
|Phase 3 PolarisDMD Trial||Recruiting||Recruiting|
|Patient Registry Translarna (Ataluren)||Recruiting||Recruiting|
|PTC Ataluren Phase 3||Trial complete/terminated||Trial complete|
|Summit- Ezutromid (PhaseOut DMD)||Trial complete/terminated||Trial terminated|
|FOR-DMD||Fully recruited||Fully recruited|
Having completed paediatric training in Belfast and Bath and Paediatric Neurology training in Southampton Dr Hughes has been a Consultant Paediatric Neurologist and Lead Clinician for the Neuromuscular Service at Royal Manchester children’s Hospital since 1996.
Janet Johnson trained as a Paediatric Nurse at Manchester University acquiring firstly a DPSN and then a BSC (Hons) 2;1 IN NURSING PRACTICES. Her background is mainly in the care of children with complex needs, life-limiting conditions, invasive and non-invasive ventilation. She has been a neuromuscular Specialist practitioner for the last 7 years and supports patients and families both in hospital and in the community. She helps to coordinate services, support education and help with housing and the social and emotional wellbeing of children with neuromuscular conditions and their families.
Dr McCullagh is a Consultant Paediatric Neurologist with expertise in paediatric neuromuscular disorders. Having initially trained in paediatrics in Belfast, he completed paediatric neurology training in Manchester. Followingthis he completed a fellowship in neuromuscular disorders at the Dubowitz Neuromuscular Centre, Great Ormond Street Hospital, London.
Sinead Warner completed her physiotherapy degree in Manchester and following this moved to New Zealand where she completed rotational posts including paediatric specialities. On returning to Manchester Sinead worked as a paediatric community physiotherapist where she developed an interest in neuromuscular conditions. Sinead now works as a highly specialised physiotherapist within the Neuromuscular team.