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Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis

StatusRecruiting
PhasePhase 2
Started2024-06-24
View on ClinicalTrials.gov ↗
The goal of this clinical trial is to learn if drug SPL84 is safe for adult patients with cystic fibrosis (CF). It will also learn if the drug works to treat works to treat CF with a specific mutation (3849 +10kb C--\>T). The purpose of this research study is to test the safety and effectiveness of multiple doses of the study drug, SPL84. Researchers will compare drug SPL84 to a placebo (a look-alike substance that contains no drug) to see if drug SPL84 is safe and if it works to treat CF. In cohorts 1-3, SPL84 will be tested as a monotherapy, and in Cohort 4, SPL84 will be tested in participants who are already stable on CFTR modulator therapy. Participants will take drug SPL84 or a placebo by inhalation every week for 9 weeks (cohorts 1-3) or 12 weeks (cohort 4) and visit the clinic approximately weekly for checkups and tests.

Amendment record

Amended 2 times since 2024-05-20; most recent filed 2026-05-11. Every amendment is listed below.

Dates here are the day the sponsor filed the amendment, not the day ClinicalTrials.gov published it. Version history last read from ClinicalTrials.gov on 2026-08-30.

2026-05-11
Amendment Study Identification, Study Status, Oversight, Study Description, Study Design, Outcome Measures, Eligibility, Contacts/Locations v2
2024-12-09
Amendment Study Identification, Study Status, Oversight, Contacts/Locations v1
2024-05-20
Original filing
Trial Details
NCT Number NCT06429176
Lead Sponsor SpliSense Ltd.
Conditions Cystic Fibrosis
Enrollment 64 participants
Start Date 2024-06-24
Primary Completion 2027-10-01 (estimated)
Study Completion 2027-12-31 (estimated)
Updated on ClinicalTrials.gov 2026-05-14