Recruiting
Phase 2

SPL84

Sponsor:

SpliSense Ltd.

Code:

NCT06429176

Conditions

Cystic Fibrosis

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

SPL84

Placebo

Study Details

Brief 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).

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

Cystic Fibrosis

Study ID

NCT06429176

Start date

Jun 24, 2024

Status verified date

May, 2026

Completion date

Dec 31, 2027

Anticipated

Primary completion date

Oct 1, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Cohort 1-3:

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 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.

Cohort 4:

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 40-80% 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.

Exclusion Criteria:

  • 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.

Study Design

Enrollment

64 participants

Anticipated

Allocation

Randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

active comparator: SPL84

placebo comparator: Placebo

Interventions

SPL84

SPL84 solution for nebulization

Placebo

Placebo solution for nebulization

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) ]
  • 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) ]
  • 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) ]
  • 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) ]
  • 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) ]

Central Contacts and Locations

Locations

University of Southern California

Recruiting

Los Angeles, California, United States, 90033

Contacts

Study Coordinator

+1 323 409 5383lynn.fukushima@med.usc.edu

National Jewish Health

Recruiting

Denver, Colorado, United States, 80206

Contacts

Site Manager

303-270-2517wilsona@njhealth.org

More Information

Sponsor

SpliSense Ltd.

Last update posted

May 14, 2026

Last verified

May, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by SpliSense Ltd. on 2026-05-14.