1,628
Views
14
CrossRef citations to date
0
Altmetric
Study Design Article

Setting up a clinical trial for a novel disease: a case study of the Doxycycline for the Treatment of Nodding Syndrome Trial – challenges, enablers and lessons learned

ORCID Icon, , , , , , , , , & show all
Article: 1431362 | Received 23 Oct 2017, Accepted 08 Jan 2018, Published online: 31 Jan 2018

References

  • Ho PM, Peterson PN, Masoudi FA. Evaluating the evidence is there a rigid hierarchy? Circulation. 2008;118:1675–9.
  • Arenz D, Hero B, Eichhorst BF, et al. Estimating site costs prior to conducting clinical trials. Clin Investig (Lond). 2014;4:227–234.
  • Sertkaya A, Wong -H-H, Jessup A, et al. Key cost drivers of pharmaceutical clinical trials in the USA. Clin Trials. 2016;13:117–126.
  • Byrom B. Using IVRS in clinical trial management. Appl Clin Trials. 2002;11:36–43.
  • Heneghan C, Blacklock C, Perera R, et al. Evidence for non-communicable diseases: analysis of Cochrane reviews and randomised trials by World Bank classification. BMJ Open. 2013;3:e003298.
  • Joseph PD, Caldwell PH, Barnes EH, et al. Disease burden-research match? Registered trials in child health from low- and middle-income and high-income countries. J Paediatr Child Health. 2017;53:667–674.
  • Chaudhry SH, Brehaut JC, Grimshaw JM, et al. Challenges in the research ethics review of cluster randomized trials: international survey of investigators. Clin Trials. 2013;10:257–268.
  • Hackshaw A, Farrant H, Bulley S, et al. Setting up non-commercial clinical trials takes too long in the UK: findings from a prospective study. J R Soc Med. 2008;101:299–304.
  • Berthon-Jones N, Courtney-Vega K, Donaldson A, et al. Assessing site performance in the Altair study, a multinational clinical trial. Trials. 2015;16:1.
  • Ahmed SM, Palermo A-GS. Community engagement in research: frameworks for education and peer review. Am J Public Health. 2010;100:1380–1387.
  • MacQueen KM, Bhan A, Frohlich J, et al. Evaluating community engagement in global health research: the need for metrics. BMC Med Ethics. 2015;16:44.
  • Idro R, Opar B, Wamala J, et al. Is nodding syndrome an Onchocerca volvulus-induced neuroinflammatory disorder? Uganda’s story of research in understanding the disease. Int J Infect Dis. 2016;45:112–117.
  • Idro R, Musubire KA, Byamah MB, et al. Proposed guidelines for the management of nodding syndrome. Afr Health Sci. 2013;13:219–225.
  • Idro R, Opoka RO, Aanyu HT, et al. Nodding syndrome in Ugandan children—clinical features, brain imaging and complications: a case series. BMJ Open. 2013;3:e002540.
  • Idro R, Namusoke H, Abbo C, et al. Patients with nodding syndrome in Uganda improve with symptomatic treatment: a cross-sectional study. BMJ Open. 2014;4:e006476.
  • Johnson T, Tyagi R, Lee PR, et al. Detection of auto-antibodies to leiomodin-1 in patients with nodding syndrome. J Neuroimmunol. 2014;275:103.
  • Johnson TP, Tyagi R, Lee PR, et al. Nodding syndrome may be an autoimmune reaction to the parasitic worm Onchocerca volvulus. Sci Transl Med. 2017;9:eaaf6953.
  • Marsh R Doxycycline for the treatment of nodding syndrome: U.S. National Institutes of Health; 2016. [ cited 2016 Sept 27th]. Available from: https://clinicaltrials.gov/ct2/show/NCT02850913.
  • UBOS. Statistical abstract. Vol. 2016, Kampala, Uganda: Uganda Bureau of Statistics (UBOS); 2016. p. 1–349.
  • Iyengar PJ, Wamala J, Ratto J, et al. Prevalence of nodding syndrome—uganda, 2012–2013. MMWR Morb Mortal Wkly Rep. 2014;63:603–606.
  • ICH. International council for harmonisation guidelines: International Council for Harmonisation (ICH); 2015. Available from: http://www.ich.org/products/guidelines.html.
  • Schluger N, Karunakara U, Lienhardt C, et al. Building clinical trials capacity for tuberculosis drugs in high-burden countries. PLoS Med. 2007;4:e302.
  • Trials AC The clinitiative: a new, community-centered approach to clinical trial promotion 2017. Available from: https://australianclinicaltrials.com/the-clinitiative-a-new-community-centered-approach-to-clinical-trial-promotion/.
  • Atim P, Ochola E, Ssendagire S, et al. Health seeking behaviours among caretakers of children with nodding syndrome in pader district-Northern Uganda: a mixed methods study. PloS One. 2016;11:e0159549.
  • Nakigudde J, Mutamba BB, Bazeyo W, et al. An exploration of caregiver burden for children with nodding syndrome (lucluc) in Northern Uganda. BMC Psychiatry. 2016;16:255.
  • Bonevski B, Randell M, Paul C, et al. Reaching the hard-to-reach: a systematic review of strategies for improving health and medical research with socially disadvantaged groups. BMC Med Res Methodol. 2014;14:42.
  • Webb DA, Coyne JC, Goldenberg RL, et al. Recruitment and retention of women in a large randomized control trial to reduce repeat preterm births: the philadelphia collaborative preterm prevention project. BMC Med Res Methodol. 2010;10:88.
  • Hahn EA, Cella D. Health outcomes assessment in vulnerable populations: measurement challenges and recommendations. Arch Phys Med Rehabil. 2003;84:S35–S42.
  • Brown DR, Fouad MN, Basen-Engquist K, et al. Recruitment and retention of minority women in cancer screening, prevention, and treatment trials. Ann Epidemiol. 2000;10:S13–S21.
  • Loftin WA, Barnett SK, Bunn PS, et al. Recruitment and retention of rural African Americans in diabetes research. Diabetes Educ. 2005;31:251–259.
  • Marsh V, Kamuya D, Parker M, et al. Working with concepts: the role of community in international collaborative biomedical research. Public Health Ethics. 2011;4:26–39.
  • Edwards SJ, Braunholtz DA, Lilford RJ, et al. Ethical issues in the design and conduct of cluster randomised controlled trials. Bmj. 1999;318:1407–1409.
  • George AS, Mehra V, Scott K, et al. Community participation in health systems research: a systematic review assessing the state of research, the nature of interventions involved and the features of engagement with communities. PLoS One. 2015;10:e0141091.
  • Angwenyi V, Kamuya D, Mwachiro D, et al. Complex realities: community engagement for a paediatric randomized controlled malaria vaccine trial in Kilifi, Kenya. Trials. 2014;15:1.
  • Marsh V, Kamuya D, Rowa Y, et al. Beginning community engagement at a busy biomedical research programme: experiences from the KEMRI CGMRC-wellcome trust research programme, Kilifi, Kenya. Soc Sci Med. 2008;67:721–733.
  • McCarthy MM, Woolley CS, Arnold AP. Incorporating sex as a biological variable in neuroscience: what do we gain?. Nat Rev Neurosci. 2017;18:707.
  • Joel D, McCarthy MM. Incorporating sex as a biological variable in neuropsychiatric research: where are we now and where should we be? Neuropsychopharmaco. 2017;42:379–385.
  • Shansky RM, Woolley CS. Considering sex as a biological variable will be valuable for neuroscience research. J Neurosci. 2016;36:11817–11822.
  • Robinson JM, Trochim WM. An examination of community members’, researchers’ and health professionals’ perceptions of barriers to minority participation in medical research: an application of concept mapping. Ethn Health. 2007;12:521–539.
  • Sheard L, Tompkins C, Wright N, et al. Non-commercial clinical trials of a medicinal product: can they survive the current process of research approvals in the UK? J Med Ethics. 2006;32:430–434.
  • Burgess L, Sulzer N. Examining the clinical trial feasibility process and its implications for a trial site. Open Access J Clin Trials. 2011;3:51–54.
  • CUREC. Studies approved by oxTREC university of Oxford: central University Research Ethics Committee; 2016 [ cited 2016 Sept 14th]. Available from: https://www.admin.ox.ac.uk/curec/about/oxtrec/studies/.
  • UNCST. National guidelines for research involving humans as research participants. Kampala, Uganda: Uganda National Council for Science and Technology; 2014.
  • NDA. Guidelines for the Conduct of Clinical Trials - Uganda National Drug Authority. Kampala, Uganda: Ministry of Health; 2008. Available from: http://apps.who.int/medicinedocs/documents/s19724en/s19724en.pdf
  • Dilts DM, Sandler AB, Cheng SK, et al. Steps and time to process clinical trials at the cancer therapy evaluation program. J Clin Oncol. 2009;27:1761–1766.