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SPL84 for Cystic Fibrosis with a Specific Mutation

This study is testing a drug called SPL84 for adults with cystic fibrosis (CF) who have a specific genetic mutation (3849+10kb C->T). The main goal is to see if SPL84 is safe and well-tolerated. Researchers will also look at how the body handles SPL84 and if it helps treat CF. You would receive either SPL84 or a placebo (a substance with no drug). Some participants will take SPL84 alone, while others will take it along with their current CF medication. The study aims to enroll 64 participants.

Study design
This study compares SPL84 to a placebo and plans to enroll 64 participants. It is an interventional study, but the phase is not specified.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for safety and tolerability for up to 87 or 108 days, depending on the group.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06429176

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

Recruiting
PHASE2Ages 18+InterventionalTreatment
SpliSense Ltd.
~64 participants
Updated 2026-05-14 on ClinicalTrials.gov
What's tested:SPL84Placebo

At a glance

Recruiting sites
2 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
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)
Measured over Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
+12 more outcomes measured

Conditions

Cystic Fibrosis

Explore related trials

Cystic FibrosisCFTR Gene

Where it's being run

3 sites across 3 states
California1
Colorado1
Massachusetts1

Who to contact

Study Coordinator
[email protected]
+1 323 409 5383
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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.
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

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.
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.

What this trial 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)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 rateDay 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 rateDay 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 pressureDay 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
  • Safety and Tolerability of SPL84 as assessed by number of participants with abnormal oximetryDay 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
  • Safety and Tolerability of SPL84 as assessed by number of participants with abnormal temperatureDay 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 resultsDay 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 resultsDay 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 resultsDay 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) parametersDay 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 findingsDay 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 resultsDay 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 resultsDay 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)

    assessment of anti-SPL84 antibodies will be performed both in serum and sputum