Study design & results
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Trial intelligence / Profile preview
Phase 2a multiple ascending dose study of SPL84 in adults with cystic fibrosis carrying the 3849+10kb C>T CFTR mutation
2024-511184-28-00Phase 2Authorised
- Sponsor
- SpliSense
- Start
- Oct 2024
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 12
01
Overview
Phase 2, randomised, double-blind trial in cystic fibrosis (CF) comparing SPL84 multiple ascending dose with Placebo inhalation (2 arms). Primary endpoints: Incidence, nature, and severity of adverse events, Change from baseline in safety assessments and 2 more. Planned start Oct 2024; status: authorised.
02
Drugs
SPL84
03
Conditions
cystic fibrosis (CF)
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Study design, results and competitive context are available in the Gosset platform where reported.
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