Renowned children's healthcare centre Great Ormond Street has gained recognition from the National Institute for Health Research for recruiting the first global patients to a number of industry multi-centre contract studies, focused on conditions including Duchenne Muscular Dystrophy, haemophilia, and the skin disorder Epidermolysis Bullosa

Prof Francesco Muntoni leads a trial entitled SMT C1100 – A Phase 1, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients with Duchenne Muscular Dystrophy, which is sponsored by Summit PLC.

The treatment is a compound capable of up-regulating the production of utrophin, a protein that can compensate for the lack of dystrophin, a protein that cannot be produced in boys with DMD – an approach that could slow or even stop the progression of this devastating condition. The recruitment of the first global patients to the study continues the success of Prof Muntoni, who has been awarded an NIHR’s award for ‘consistently delivering to time and target, and first global or European patient’.

Prof Muntoni says: “We are in a very privileged position for trials in DMD. Our group was the first to establish a UK consortium for investigator-driven trials in this condition more than a decade ago, in this way introducing for the first time a model of close collaboration between clinicians from UK, scientists, and advocacy groups.

As part of this consortium, I have led, as Chief Investigator, several DMD trials in the last decade, and have developed unique understanding of trial design, outcome measures, and practicalities of taking part in clinical trials. Secondly, in collaboration with Dr Adnan Manzur (also at GOSH), I coordinate the North Star UK network that collects longitudinal information on all DMD boys in the country. Children from this network can be referred to GOSH for clinical trials. Thirdly, the GOSH clinics are the largest DMD clinic in the country. Last but not least, the Clinical Research Facility at GOSH is a fantastic structure, which in true partnership with the clinical and therapy teams, allows efficient, safe and pleasant participation of DMD boys and their families in clinical trials.”

Dr Ri Liesner recruited the first global patient in a haemophilia study designed to evaluate the safety and efficacy of a recombinant fusion protein (Coagulation Factor VIII Fc Fusion Protein), sponsored by Biogen. Dr Liesner was identified by NIHR Clinical Research Network as a Leading Commercial Principal Investigator in 2015; this accolade is regarded by the NIHR, Department of Health and industry as the highest achievement in clinical research terms.

Dr Anna Martinez recruited the first European patient into a Phase 3 multi-centre, randomised, double-blind, placebo controlled trial investigating the efficacy and safety of SD-101 cream in patients with Epidermolysis Bullosa. This study is sponsored by Scioderm Inc.

Dame Sally Davies, Chief Medical Officer and Chief Scientific Advisor at the Department of Health, said: “These global firsts are a significant achievement and show the dedication of the research and research support teams at GOSH. It also demonstrates the impact of the measures we are putting in place to ensure that the UK stays internationally competitive as a place to undertake high quality life sciences research.”