Wave FOCUS-C9 studie met C9orf72 ASO in ALS en/of FTD
A multicenter , randomized, double blind, placebo controlled , phase 1b/2a study of WVE-004 administered intrathecally to patients with C9orf72 associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD) .
This phase 1a/2b study is sponsored by the company Wave and aims to determine whether reducing certain C9ORF72 transcripts using antisense oligonucleotides (ASO) directed against repeat expansion in the C9ORF72 gene can be safely used in ALS patients with a C9ORF72 mutation.
Only patients with C9orf72-associated ALS or FTD or both can participate in the study. WVE-004 is an antisense oligonucleotide (ASO) administered intrathecally (via lumbar puncture) to inhibit the damaging effects of a C9ORF72 mutation.
The development of WVE-004 is still at an early stage. In the first part of the study, only one dose will be administered and the dose will be slowly increased in successive groups of patients. In the second part of the study, multiple doses will be administered, again with increasing doses. The purpose of the study is to determine the safety and tolerability of the study product. In the first part of the study, 1 in 5 patients will receive placebo. In the second part, 1 in 3 patients will be assigned to the placebo group. The study will enroll about 42 patients worldwide.
More info on this study can be found on the website https://www.clinicaltrials.gov/ct2/show/NCT04931862?cond=amyotrophic+C9orf72&draw=2&rank=10
Interested, or desire further information? Contact us at onderzoek@als.be
