Platform trial design for neurofibromatosis type 1, NF2-related schwannomatosis and non-NF2-related schwannomatosis: A potential model for rare diseases.
Britt A E DhaenensGünter HeimannAnnette BakkerMarco NievoRosalie E FernerD Gareth EvansPierre WolkensteinJonas LeubnerCornelia PotratzCharlotte CartonUchenna IloejeGeorge KirkJaishri O BlakeleyScott PlotkinMichael J FisherAe Rang KimPablo Hernáiz DrieverAmedeo A AziziBrigitte C WidemannAndrea M GrossTom ParkeEric LegiusRianne OostenbrinkPublished in: Neuro-oncology practice (2024)
These innovative platform-basket trials for NF may serve as a model for other rare diseases, as they will enhance the chance of identifying beneficial treatments through optimal learning from a small number of patients. The goal of these trials is to identify beneficial treatments for NF more rapidly and at a lower cost than traditional, single-agent clinical trials.
Keyphrases
- signaling pathway
- lps induced
- clinical trial
- pi k akt
- nuclear factor
- oxidative stress
- end stage renal disease
- inflammatory response
- newly diagnosed
- high throughput
- ejection fraction
- chronic kidney disease
- study protocol
- phase iii
- prognostic factors
- phase ii
- peritoneal dialysis
- randomized controlled trial
- risk assessment
- patient reported outcomes
- open label