P.149The MYODA operational seamless clinical trial design phase I to III: a new approach for rare diseases to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of BIO101 (MAS activator) in paediatric patients with a genetically confirmed diagnosis of Duchenne muscular dystrophy

2019 ◽  
Vol 29 ◽  
pp. S92
Author(s):  
M. Chabane ◽  
W. Dioh ◽  
P. Dilda ◽  
R. Lafont ◽  
S. Veillet ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Phase I ◽  
Rare Diseases ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Design Phase ◽  
Pharmacokinetics And Pharmacodynamics ◽  
New Approach

scholarly journals Corticosteroid Treatment Impact on Spinal Deformity in Duchenne Muscular Dystrophy

2014 ◽  
Vol 2014 ◽  
pp. 1-9 ◽  
Author(s):  
Ilaria Sanzarello ◽  
Luciano Merlini ◽  
Francesco Traina ◽  
Michele Attilio Rosa ◽  
Cesare Faldini
Keyword(s):  
Clinical Trial ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Spinal Deformity ◽  
Trial Design ◽  
Progressive Disease ◽  
Clinical Trial Design ◽  
Corticosteroid Treatment ◽  
Surgical Interventions ◽  
History Of

Duchenne muscular dystrophy is a progressive disease with loss of ambulation at around 9-10 years of age, followed, if untreated, by development of scoliosis, respiratory insufficiency, and death in the second decade of life. This review highlights the natural history of the disease, in particular, with regard to the development of the spinal deformity and how this complication has been modified by surgical interventions and overall by corticosteroid treatment. The beneficial effect of corticosteroids may have also an impact on the clinical trial design of the new emerging causative therapies.

A novel, hybrid, single- and multi-site clinical trial design for CLN3 disease, an ultra-rare lysosomal storage disorder

2019 ◽  
Vol 16 (5) ◽  
pp. 555-560 ◽  
Author(s):  
Heather R Adams ◽  
Sara Defendorf ◽  
Amy Vierhile ◽  
Jonathan W Mink ◽  
Frederick J Marshall ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Rare Diseases ◽  
Trial Design ◽  
Neurodegenerative Disorder ◽  
Clinical Trial Design ◽  
Trial Participation ◽  
Local Participation ◽  
Cln3 Disease ◽  
Study Participants

Background Travel burden often substantially limits the ability of individuals to participate in clinical trials. Wide geographic dispersion of individuals with rare diseases poses an additional key challenge in the conduct of clinical trials for rare diseases. Novel technologies and methods can improve access to research by connecting participants in their homes and local communities to a distant research site. For clinical trials, however, understanding of factors important for transition from traditional multi-center trial models to local participation models is limited. We sought to test a novel, hybrid, single- and multi-site clinical trial design in the context of a trial for Juvenile Neuronal Ceroid Lipofuscinosis (CLN3 disease), a very rare pediatric neurodegenerative disorder. Methods We created a “hub and spoke” model for implementing a 22-week crossover clinical trial of mycophenolate compared with placebo, with two 8-week study arms. A single central site, the “hub,” conducted screening, consent, drug dispensing, and tolerability and efficacy assessments. Each participant identified a clinician to serve as a collaborating “spoke” site to perform local safety monitoring. Study participants traveled to the hub at the beginning and end of each study arm, and to their individual spoke site in the intervening weeks. Results A total of 18 spoke sites were established for 19 enrolled study participants. One potential participant was unable to identify a collaborating local site and was thus unable to participate. Study start-up required a median 6.7 months (interquartile range = 4.6–9.2 months). Only 33.3% (n = 6 of 18) of spoke site investigators had prior clinical trial experience, thus close collaboration with respect to study startup, training, and oversight was an important requirement. All but one participant completed all study visits; no study visits were missed due to travel requirements. Conclusions This study represents a step toward local trial participation for patients with rare diseases. Even in the context of close oversight, local participation models may be best suited for studies of compounds with well-understood side-effect profiles, for those with straightforward modes of administration, or for studies requiring extended follow-up periods.

scholarly journals Early 3+3 Trial Dose-Escalation Phase I Clinical Trial Design and Suitability for Immune Checkpoint Inhibitors

2020 ◽  
Author(s):  
Osama E. Rahma ◽  
Joshua E. Reuss ◽  
Anita Giobbie-Hurder ◽  
Ghazaleh Shoja E Razavi ◽  
Osama Abu-Shawer ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Phase I ◽  
Dose Escalation ◽  
Immune Checkpoint ◽  
Trial Design ◽  
Immune Checkpoint Inhibitors ◽  
Checkpoint Inhibitors ◽  
Clinical Trial Design ◽  
Phase I Clinical Trial

Evidence-Based Medicine for Rare Diseases: Implications for Data Interpretation and Clinical Trial Design

2007 ◽  
Vol 14 (2) ◽  
pp. 160-166 ◽  
Author(s):  
Madhusmita Behera ◽  
Ambuj Kumar ◽  
Heloisa P. Soares ◽  
Lubomir Sokol ◽  
Benjamin Djulbegovic
Keyword(s):  
Clinical Trial ◽  
Rare Diseases ◽  
Evidence Based Medicine ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Data Interpretation ◽  
Evidence Based ◽  
Based Medicine
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