Actively Recruiting
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis
Led by SpliSense Ltd. · Updated on 2026-05-14
64
Participants Needed
3
Research Sites
183 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of cystic fibrosis with two CF causing mutations including 3849+10 Kb C->T mutation on one allele confirmed by certified genetic testing
- Body mass index (BMI) of 17 kg/m2 or higher
- FEV1 between 40% and 90% predicted at screening for cohorts 1-3
- FEV1 between 40% and 80% predicted at screening for cohort 4
- Non-smokers or non-vapers for at least 180 days prior to screening
- For cohort 4 only: stable use of Trikafta/Kaftio or Alyftrek for at least 3 months, or Alyftrek for 1 month after switching from Trikafta/Kaftio
You will not qualify if you...
- Use of Kalydeco, Orkambi, Symdeko/Symkevi, or Trikafta/Kaftrio within 30 days before first dose (cohorts 1-3)
- Use of any investigational drug or device other than SPL84 within 30 days before first dose
- Use of systemic steroids over 3 consecutive months in last 6 months or any systemic steroids in last month prior to screening
- Use of inhaled steroids above 1 mg
- Unstable use of CF medications such as inhaled antibiotics, dornase alfa, hypertonic saline, or physiotherapy within 28 days prior to screening; inhaled antibiotics for prophylaxis must be stable for 90 days prior
- Any acute infection or pulmonary exacerbation requiring new therapy within 14 days prior to first dose
- Hemoptysis greater than 30 mL within 90 days prior to Day 1 or hospitalization for hemoptysis within 6 months prior
- Liver disease with significant cirrhosis or portal hypertension
- History of any organ transplantation
- Documented COVID-19 infection within 4 weeks prior to dosing
AI-Screening
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Trial Site Locations
Total: 3 locations
1
University of Southern California
Los Angeles, California, United States, 90033
Actively Recruiting
2
National Jewish Health
Denver, Colorado, United States, 80206
Actively Recruiting
3
Boston Children'S Hospital
Boston, Massachusetts, United States, 02115
Not Yet Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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