Tests inhaled SPL84 in adults with cystic fibrosis and a specific gene mutation
Tests whether inhaled SPL84 is safe and may help adults with cystic fibrosis.
Plain-language title generated from the registry record
Registry title: Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis
Key facts
- Where
- Berlin and Potsdam, GermanySee the 2 sites
- Phase
- Phase 2
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with cystic fibrosis who have the 3849+10 Kb C->T mutation in the CFTR gene. They inhale SPL84 or a placebo once a week.
- Age
- 18 years or older
- Sex
- All sexes can join
- Disease stage
- Cystic fibrosis with FEV1 40-90% predicted (cohorts 1-3) or 40-80% predicted (cohort 4), and a body mass index of at least 17 kg/m2.
- Earlier treatment
- Cohorts 1-3: no Kalydeco, Orkambi, Symdeko/Symkevi or Trikafta/Kaftrio in the 30 days before the first dose. Cohort 4: stable use of Trikafta/Kaftio or Alyftrek for at least 3 months, or Alyftrek for 1 month after switching from Trikafta/Kaftio.
- Biomarker or test result
- Two cystic fibrosis causing CFTR mutations, with the 3849+10 Kb C->T mutation on one allele. A certified genetic laboratory must document it.
Main reasons for not taking part
- Smoking or vaping in the last 180 days
- Any organ transplant in the past
- Liver disease with clinically significant cirrhosis or portal hypertension
- Acute infection or lung flare-up within 14 days before the first dose
- Another investigational drug or device within 30 days before the first dose
Read by machine on 11 November 2026.
Where in Germany
- Charité Universitätsmedizin BerlinBerlin, GermanyTaking people
- Klinikum Westbrandenburg CF Zentrum PotsdamPotsdam, GermanyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- SpliSense Ltd.
- Trial code
- NCT06429176
- Record updated
- 10 November 2026
- Original 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 (3849 +10kb C-->T).Read full original descriptionShow less
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.
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.
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 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; 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; Documented coronavirus disease (COVID-19) infection within 4 weeks prior to dosing; Liver disease characterized by clinically significant cirrhosis and/or documented portal hypertension; History of any organ transplantation; Liver disease characterized by clinically significant cirrhosis and/or documented portal hypertension; History of any organ transplantation; Use of Kalydeco, Orkambi, Symdeko/Symkevi or Trikafta/Kaftrio within 30 days of first dose with study intervention.
Cohort 1-3:.