Cystic FibrosisPhase 2NCT06429176
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis
A Phase 2 study testing SPL84 in people with Cystic Fibrosis, run by SpliSense Ltd. at 3 sites in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- SPL84
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 64 participants
- Where
- 3 sitesUnited States
- Study dates
- 24 Jun 2024to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- 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.
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- FEV1 40-80% predicted at screening.
- Non-smokers or vapers for at least 180 days (6 months) prior to screening, per participant report.
- Stable adherence to standard use of Trikafta/Kaftio or Alyftrek for at least 3 months, or Alyftrek for 1 month after switching from Trikafta/Kaftio, according to prescribing information.
Who cannot join
- 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 saline and physiotherapy should be on stable regimen for the period 28 days prior to screening; those participants taking inhaled antibiotics for prophylaxis must be on a stable regimen of these drugs for at least 90 days prior to first dose with study intervention.
- Any acute infection including acute upper respiratory or lower respiratory infections, pulmonary exacerbation, changes in therapy for pulmonary disease, or any non CF-related illness which results in the initiation of any new therapy within 14 days prior to first dose with study intervention.
- Hemoptysis of greater than 30 mL within 90 days prior to Day 1, or hospitalization for hemoptysis within 6 months of first dose with study intervention.
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- Liver disease characterized by clinically significant cirrhosis and/or documented portal hypertension.
- History of any organ transplantation.
- Documented coronavirus disease (COVID-19) infection within 4 weeks prior to dosing.
- Cohort 4:
- Previous participation in active arm of SPL84-002 study (Cohort 1-3)
- 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 saline and physiotherapy should be on stable regimen for the period 28 days prior to screening; those participants taking inhaled antibiotics for prophylaxis must be on a stable regimen of these drugs for at least 90 days prior to first dose with study intervention.
- Any acute infection including acute upper respiratory or lower respiratory infections, pulmonary exacerbation, changes in therapy for pulmonary disease, or any non CF-related illness which results in the initiation of any new therapy within 14 days prior to first dose with study intervention.
- Hemoptysis of greater than 30 mL within 90 days prior to Day 1, or hospitalization for hemoptysis within 6 months of first dose with study intervention.
- Liver disease characterized by clinically significant cirrhosis and/or documented portal hypertension.
- History of any organ transplantation.
- Documented coronavirus disease (COVID-19) infection within 4 weeks prior to dosing.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Not yet recruiting
Boston Children'S Hospital
Boston, Massachusetts, 02115
Julia Giancola, 617-355-4171, Julia.Giancola@childrens.harvard.edu
- Recruiting
National Jewish Health
Denver, Colorado, 80206
Site Manager, 303-270-2517, wilsona@njhealth.org
- Recruiting
University of Southern California
Los Angeles, California, 90033
Study Coordinator, +1 323 409 5383, lynn.fukushima@med.usc.edu
About this study
Official title: A Phase 2a, Randomized, Placebo-Controlled, Double Blind Multiple Ascending Dose Study in Patients With Cystic Fibrosis Carrying the 3849 +10 Kb C->T Mutation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84
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.
Read more
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
- Cystic Fibrosis
- Being tested
- SPL84, Placebo
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SPL84Drug
SPL84 solution for nebulization
PlaceboOther
Placebo solution for nebulization
| Study group | Role | What they receive |
|---|---|---|
| SPL84 | Active comparator | Not described Receives: Drug: SPL84 |
| Placebo | Placebo comparator | Not described Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Incidence, nature, and severity of AEs and SAEs
Measured: 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 heart rate
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
using an ECG machine that automatically calculates heart rate and measure PR, QRS, QT, and QTc intervals
Measured: 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 physical examination findings
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.
Measured: 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 pulmonary function tests results
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
Measured: 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 immunogenicity results
assessment of anti-SPL84 antibodies will be performed both in serum and sputum
Measured: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Secondary outcomes (14)
Characterization of pharmacokinetics (PK) of SPL84: maximum serum concentration (Cmax)
Measured: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: Time to Cmax (Tmax)
Measured: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: terminal elimination half-life (t1/2)
Measured: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: Area under the curve to the final sample (AUC0-t)
Measured: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: Area under the curve to infinity (AUC0-∞)
Measured: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: Apparent clearance (CL/F)
Measured: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of excretion of SPL84: concentration of SPL84 in urine
Measured: Day 1 through Day 87 (Cohort 1-3) or Day 85 (Cohort 4)
Efficacy of SPL84 as assessed by change from baseline in percent predicted FEV1
Pulmonary function tests will be performed according to the ATS/ERS
Measured: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Efficacy of SPL84 as assessed by change from baseline in percent predicted FEF25-75
Measured: Day 1 to Day 87 (Cohort 1-3) or Day 108 (Cohort 4)
Efficacy of SPL84 as assessed by change from baseline in Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score
The score for is standardized on a 0- to 100-point scale on which higher scores represent a higher quality of life
Measured: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Efficacy of SPL84 as assessed by change from baseline in body weight
Measured: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Preliminary efficacy of SPL84 as assessed by change from baseline of antibiotic treatment (Cohort 1-3 only)
Measured: Day 1 through Day 87
Cohort 1-3: Safety and Tolerability of SPL84 as assessed by number of participants with abnormal sputum microbiology results Cohort 4: Exploratory efficacy of SPL84 as assessed by number of participants with change in abnormal sputum microbiology result
Sputum microbiology will be performed with a microbiology based assay; organism growth will be identified.
Measured: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Exploratory efficacy of SPL84 as assessed by number of participants with change in Lung Clearance Index at 2.5% of starting concentration (LCI2.5)
Measured using multiple breath washout (at select study sites only)
Measured: Day 1 to Day 85
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2024
- Study start24 Jun 2024
- Primary completion1 Oct 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 64 participants (estimated)
Sponsor and investigators
- Lead sponsor
- SpliSense Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06429176
- Sponsor's protocol ID
- SPL84-002
- Other IDs
- 2024-511184-28
- Status last verified
- May 2026
- First submitted
- 15 May 2024
- Last updated
- 14 May 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Randomized, Placebo-Controlled, Double Blind Multiple Ascending Dose Study in Patients With Cystic Fibrosis Carrying the 3849 +10 Kb C->T Mutation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84
- ClinicalTrials.gov ID
- NCT06429176
- Acronym / sponsor ID
- SPL84-002
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 64 participants (estimated)
- Lead sponsor
- SpliSense Ltd.Industry (pharma, biotech, devices)
- Study start
- 24 Jun 2024 (actual)
- Primary completion
- 1 Oct 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 24 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SPL84 | Active comparator | Drug: SPL84 |
| Placebo | Placebo comparator | Other: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
- 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.
Key exclusion criteria (19 listed)
- 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 saline and physiotherapy should be on stable regimen for the period 28 days prior to screening; those participants taking inhaled antibiotics for prophylaxis must be on a stable regimen of these drugs for at least 90 days prior to first dose with study intervention.
- Any acute infection including acute upper respiratory or lower respiratory infections, pulmonary exacerbation, changes in therapy for pulmonary disease, or any non CF-related illness which results in the initiation of any new therapy within 14 days prior to first dose with study intervention.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SPL84SPL84 solution for nebulization | Drug | SPL84 |
| PlaceboPlacebo solution for nebulization | Other | Placebo |
Primary outcome
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
14 secondary outcomes
Characterization of pharmacokinetics (PK) of SPL84: maximum serum concentration (Cmax)
Time frame: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: Time to Cmax (Tmax)
Time frame: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: terminal elimination half-life (t1/2)
Time frame: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: Area under the curve to the final sample (AUC0-t)
Time frame: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: Area under the curve to infinity (AUC0-∞)
Time frame: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: Apparent clearance (CL/F)
Time frame: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of excretion of SPL84: concentration of SPL84 in urine
Time frame: Day 1 through Day 87 (Cohort 1-3) or Day 85 (Cohort 4)
Efficacy of SPL84 as assessed by change from baseline in percent predicted FEV1
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Efficacy of SPL84 as assessed by change from baseline in percent predicted FEF25-75
Time frame: Day 1 to Day 87 (Cohort 1-3) or Day 108 (Cohort 4)
Efficacy of SPL84 as assessed by change from baseline in Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Efficacy of SPL84 as assessed by change from baseline in body weight
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Preliminary efficacy of SPL84 as assessed by change from baseline of antibiotic treatment (Cohort 1-3 only)
Time frame: Day 1 through Day 87
Cohort 1-3: Safety and Tolerability of SPL84 as assessed by number of participants with abnormal sputum microbiology results Cohort 4: Exploratory efficacy of SPL84 as assessed by number of participants with change in abnormal sputum microbiology result
Time frame: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Exploratory efficacy of SPL84 as assessed by number of participants with change in Lung Clearance Index at 2.5% of starting concentration (LCI2.5)
Time frame: Day 1 to Day 85
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
University of Southern California
Los Angeles, United States
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SPL84 in people with Cystic Fibrosis, run by SpliSense Ltd. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). The study lists 13 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 May 2026.