NCT06429176 · SpliSense Ltd.
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients with Cystic Fibrosis
What this study 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.
View original scientific description
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.
Interventions
DRUG
SPL84
SPL84 solution for nebulization
OTHER
Placebo
Placebo solution for nebulization
Primary outcome measures
Safety and Tolerability of SPL84 as evaluated by number of subjects with at least one treatment-related adverse event (AE) or serious adverse event (SAEs)
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Incidence, nature, and severity of AEs and SAEs
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal heart rate
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal respiratory rate
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal systolic and diastolic blood pressure
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal oximetry
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal temperature
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal hematology lab test results
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal biochemistry lab test results
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal urinalysis lab test results
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal electrocardiogram (ECG) parameters
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
using an ECG machine that automatically calculates heart rate and measure PR, QRS, QT, and QTc intervals
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal physical examination findings
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Complete physical examinations include general appearance, head, ears, eyes, nose, throat, thyroid, chest (heart, lungs), abdomen, skin, neurological, extremities, back, neck, musculoskeletal, and lymph nodes.
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal pulmonary function tests results
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Pulmonary function tests will be performed according to the American Thoracic Society (ATS)/European Respiratory Society (ERS) and forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), and forced mid-expiratory flow (FEF25-75) will be measured
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal immunogenicity results
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
assessment of anti-SPL84 antibodies will be performed both in serum and sputum
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Diagnosis of CF and two CF causing mutations; 3849+10 Kb C-\>T mutation on one allele in the CF transmembrane conductance regulator (CFTR) gene (homozygote or compound heterozygote). Source documentation from a certified genetic laboratory is required.
- Body mass index (BMI) of ≥ 17 kg/m2.
- FEV1 40-90% predicted at screening.
- Non-smokers or vapers for at least 180 days (6 months) prior to screening, per participant report.
Exclusion criteria
- Use of Kalydeco, Orkambi, Symdeko/Symkevi or Trikafta/Kaftrio within 30 days of first dose with study intervention.
- Use of any investigational drug (other than SPL84) or device within 30 days of first dose with study intervention.
- Use of systemic steroids over 3 consecutive months in the last 6 months prior to screening, or use of systemic steroids in the last month prior to screening. Use of inhaled steroids above 1 mg.
- Use of CF medications, e.g. inhaled antibiotics, dornase alfa (Pulmozyme), hypertonic salin
Where
- Los Angeles, California
- Denver, Colorado
- Boston, Massachusetts
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced May 14, 2026 · Source of record for eligibility and locations