A phase 2a study with SPL84: A novel RNA-targeted approach to restore CFTR function in people with CF carrying the 3849+10 kb C→T mutation.
In a small trial, inhaled SPL84 showed promise and good tolerability in cystic fibrosis patients with a specific genetic mutation, signaling potential new treatment options.
This randomized placebo-controlled phase 2a trial evaluated The 3849+10 kb C→T mutation in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene leads to aberrant splicing and the inclusion of a cryptic exon harboring a premature stop codon, resulting in the production of non-functional CFTR protein. The authors report that in a 17-participant randomized phase 2a trial, inhaled SPL84 was well tolerated and produced encouraging lung-function signals in cystic fibrosis with the 3849+10 kb C-to-T mutation.
What the study was
- Study design
- Randomized placebo-controlled phase 2a trial
- Category
- Treatment Innovation
- Maturity
- Exploratory
- Journal
- The European respiratory journal
Why it surfaced
9/10: Randomized placebo-controlled phase 2a trial with direct relevance to Rare diseases with high unmet need; scoring remains conservative because triage used the PubMed abstract without full-text review.
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