Challenges recruiting to a proof-of-concept pharmaceutical trial for a rare disease: The trigeminal neuralgia experience
| Autor: | Mark Obermann, Giulia Di Stefano, Lars Bendtsen, Valerie Morisset, Deb Steiner, Stine Maarbjerg, Joanne Palmer, Joanna M Zakrzewska, Giorgio Cruccu, Simon Tate, Dominik A Ettlin |
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| Přispěvatelé: | University of Zurich, Zakrzewska, Joanna M |
| Jazyk: | angličtina |
| Rok vydání: | 2018 |
| Předmět: |
Time Factors
Pregabalin Medizin Medicine (miscellaneous) law.invention South Africa 0302 clinical medicine Randomized controlled trial law Trigeminal neuralgia 2736 Pharmacology (medical) Pharmacology (medical) 030212 general & internal medicine Sodium channel blocker Pain Measurement lcsh:R5-920 Analgesics Underrepresentation Vixotrigine NAV1.7 Voltage-Gated Sodium Channel 10223 Clinic for Masticatory Disorders 2701 Medicine (miscellaneous) Middle Aged NAV1.7 Voltage-Gated Sodium Channel/drug effects Clinical trial Europe Treatment Outcome Analgesics/adverse effects Anxiety medicine.symptom lcsh:Medicine (General) Sodium Channel Blockers medicine.drug Adult medicine.medical_specialty Gabapentin Trigeminal Neuralgia/diagnosis 610 Medicine & health Placebo 03 medical and health sciences Young Adult Rare Diseases Double-Blind Method Internal medicine medicine Humans Trigeminal Nerve Aged business.industry Research Patient Selection Rare Diseases/diagnosis Trigeminal Neuralgia medicine.disease Patient recruitment Trigeminal Nerve/drug effects Sample Size business 030217 neurology & neurosurgery Sodium Channel Blockers/adverse effects |
| Zdroj: | Zakrzewska, J M, Palmer, J, Bendtsen, L, Di Stefano, G, Ettlin, D A, Maarbjerg, S, Obermann, M, Morisset, V, Steiner, D, Tate, S & Cruccu, G 2018, ' Challenges recruiting to a proof-of-concept pharmaceutical trial for a rare disease : the trigeminal neuralgia experience ', Trials, vol. 19, 704 . https://doi.org/10.1186/s13063-018-3045-1 Trials Trials, Vol 19, Iss 1, Pp 1-10 (2018) |
| DOI: | 10.1186/s13063-018-3045-1 |
| Popis: | BACKGROUND: This study aimed to describe recruitment challenges encountered during a phase IIa study of vixotrigine, a state and use-dependent Nav1.7 channel blocker, in individuals with trigeminal neuralgia.METHODS: This was an international, multicenter, placebo-controlled, randomized withdrawal study that included a 7-day run-in period, a 21-day open-label phase, and a 28-day double-blind phase in which patients (planned n = 30) were randomized to vixotrigine or placebo. Before recruitment, all antiepileptic drugs had to be stopped, except for gabapentin or pregabalin. After the trial, patients returned to their original medications. Patient recruitment was expanded beyond the original five planned (core) centers in order to meet target enrollment (total recruiting sites N = 25). Core sites contributed data related to patient identification for study participation (prescreening data). Data related to screening failures and study withdrawal were also analyzed using descriptive statistics.RESULTS: Approximately half (322/636; 50.6%) of the patients who were prescreened at core sites were considered eligible for the study and 56/322 (17.4%) were screened. Of those considered eligible, 26/322 (8.1%) enrolled in the study and 6/322 (1.9%) completed the study. In total, 125 patients were screened across all study sites and 67/125 (53.6%) were enrolled. At prescreening, reasons for noneligibility varied by site and were most commonly diagnosis change (78/314; 24.8%), age > 80 years (75/314; 23.9%), language/distance/mobility (61/314; 19.4%), and noncardiac medical problems (53/314; 16.9%). At screening, frequently cited reasons for noneligibility included failure based on electrocardiogram, insufficient pain, and diagnosis change.CONCLUSIONS: Factors contributing to recruitment challenges encountered in this study included diagnosis changes, anxiety over treatment changes, and issues relating to distance, language, and mobility. Wherever possible, future studies should be designed to address these challenges.TRIAL REGISTRATION: ClinicalTrials.gov, NCT01540630 . EudraCT, 2010-023963-16. 07 Aug 2015. |
| Databáze: | OpenAIRE |
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