Phase IIa trial of fingolimod for amyotrophic lateral sclerosis demonstrates acceptable acute safety and tolerability
| Autor: | Berry, James D, Paganoni, Sabrina, Atassi, Nazem, Macklin, Eric A, Goyal, Namita, Rivner, Michael, Simpson, Ericka, Appel, Stanley, Grasso, Daniela L, Mejia, Nicte I, Mateen, Farrah, Gill, Alan, Vieira, Fernando, Tassinari, Valerie, Perrin, Steven |
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| Jazyk: | angličtina |
| Rok vydání: | 2017 |
| Předmět: |
Adult
Male circulating lymphocytes FOXP3 RNA profiling neuroinflammation diagnosis drug therapy [Amyotrophic Lateral Sclerosis] Clinical Research Medicine and Health Sciences Bradycardia Humans Single-Blind Method adverse effects therapeutic use [Fingolimod Hydrochloride] Fatigue Aged chemically induced [Bradycardia] therapeutic use [Immunosuppressive Agents] target engagement Fingolimod Hydrochloride Amyotrophic Lateral Sclerosis clinical trial Middle Aged chemically induced [Fatigue] Female Immunosuppressive Agents |
| Zdroj: | Muscle & Nerve Berry, James D; Paganoni, Sabrina; Atassi, Nazem; Macklin, Eric A; Goyal, Namita; Rivner, Michael; et al.(2017). Phase IIa trial of fingolimod for amyotrophic lateral sclerosis demonstrates acceptable acute safety and tolerability.. Muscle & nerve, 56(6), 1077-1084. UC Irvine: Institute for Clinical and Translational Science. Retrieved from: http://www.escholarship.org/uc/item/5513221p |
| ISSN: | 1097-4598 0148-639X |
| Popis: | Introduction Immune activation has been implicated in progression of amytrophic lateral sclerosis (ALS). Oral fingolimod reduces circulating lymphocytes. The objective of this phase IIa, randomized, controlled trial was to test the short‐term safety, tolerability, and target engagement of fingolimod in ALS. Methods Randomization was 2:1 (fingolimod:placebo). Treatment duration was 4 weeks. Primary outcomes were safety and tolerability. Secondary outcomes included circulating lymphocytes and whole‐blood gene expression. Results Thirty participants were randomized; 28 were administered a drug (fingolimod 18, placebo 10). No serious adverse events occurred. Adverse events were similar by treatment arm, as was study discontinuation (2 fingolimod vs. 0 placebo, with no statistical difference). Forced expiratory volume in 1 second (FEV1) and FEV1/slow vital capacity changes were similar in the fingolimod and placebo arms. Circulating lymphocytes decreased significantly in the fingolimod arm (P |
| Databáze: | OpenAIRE |
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