Publishing protocols for trials of complex interventions before trial completion – potential pitfalls, solutions and the need for public debate

Background - Open Science is ‘the movement to make scientific research, data and dissemination accessible to all levels of an inquiring society’. In the spirit of the Open Science movement, advance publication of protocols for clinical trials is now being advocated by BioMed Central, BMJ Open and others. Simultaneously, participants are becoming increasingly active in their pursuit and sharing of trial- and health- related information. Whilst access to protocols alongside published trial findings has clear benefits, advance publication of trial protocols is potentially problematic for trials of complex behavioural interventions. In this article we explain, with examples, how this could lead to unblinding, ‘contamination’ between intervention and control groups and deliberate biasing of assessment outcomes by participants. We discuss potential solutions and demonstrate the need for public debate about how this issue is best managed. Conclusion - Triallists may still be underestimating participants’ interest in information. This needs to change: joint and open discussions with the public are needed to inform how we should proceed

Publishing protocols for trials of complex interventions before trial completion – potential pitfalls, solutions and the need for public debate

Background - Open Science is ‘the movement to make scientific research, data and dissemination accessible to all levels of an inquiring society’. In the spirit of the Open Science movement, advance publication of protocols for clinical trials is now being advocated by BioMed Central, BMJ Open and others. Simultaneously, participants are becoming increasingly active in their pursuit and sharing of trial- and health- related information. Whilst access to protocols alongside published trial findings has clear benefits, advance publication of trial protocols is potentially problematic for trials of complex behavioural interventions. In this article we explain, with examples, how this could lead to unblinding, ‘contamination’ between intervention and control groups and deliberate biasing of assessment outcomes by participants. We discuss potential solutions and demonstrate the need for public debate about how this issue is best managed. Conclusion - Triallists may still be underestimating participants’ interest in information. This needs to change: joint and open discussions with the public are needed to inform how we should proceed

Assessment of musculoskeletal abnormalities in children with mucopolysaccharidoses using pGALS

Background - Children with mucopolysaccharidoses (MPS) often have musculoskeletal (MSK) abnormalities. Paediatric Gait, Arms, Legs, and Spine (pGALS), is a simple MSK assessment validated in school-age children to detect abnormal joints. We aimed to identify MSK abnormalities in children with MPS performing pGALS. Methods - Videos of children with a spectrum of MPS performing pGALS were analysed. A piloted proforma to record abnormalities for each pGALS manoeuvre observed in the videos (scored as normal/abnormal/not assessable) was used by three observers blinded to MPS subtype. Videos were scored independently and rescored for intra- and inter-observer consistency. Data were pooled and analysed. Results - Eighteen videos of children [12 boys, 6 girls, median age 11 years (4–19)] with MPS (13 type I [5 Hurler, 8 attenuated type I]; 4 type II; 1 mannosidosis) were assessed. The most common abnormalities detected using pGALS were restrictions of the shoulder, elbow, wrist, jaw (>75% cases), and fingers (2/3 cases). Mean intra-observer Κ 0.74 (range 0.65–0.88) and inter-observer Κ 0.62 (range 0.51–0.77). Hip manoeuvres were not clearly demonstrated in the videos. Conclusions - In this observational study, pGALS identifies MSK abnormalities in children with MPS. Restricted joint movement (especially upper limb) was a consistent finding. Future work includes pGALS assessment of the hip and testing pGALS in further children with attenuated MPS type I. The use of pGALS and awareness of patterns of joint involvement may be a useful adjunct to facilitate earlier recognition of these rare conditions and ultimately access to specialist care