Cystic Fibrosis study: SPL84
Official title Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis
ClinicalTrials.gov ID: NCT06429176
What this study is testing
What is SPL84?
SPL84 is an investigational nebulized solution being studied for safety and effectiveness in adults with cystic fibrosis.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study aims to see if SPL84 is safe and if it may help treat cystic fibrosis in adults with the 3849+10kb C>T mutation. It compares the drug to a placebo.
- Who it's for
- Adults with cystic fibrosis who have the 3849+10kb C>T mutation and are stable on current therapy (for cohort 4).
- What's involved
- Participants will inhale either SPL84 or a placebo once a week. They will have clinic visits about once a week for check‑ups and tests.
- Time commitment
- The study lasts 9 weeks for cohorts 1‑3 and 12 weeks for cohort 4, with weekly clinic visits.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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...
- 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.
You likely can't join if
- 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...
- Use of CF medications, e.g. inhaled antibiotics, dornase alfa (Pulmozyme), hypertonic saline and physiotherapy should be on stable regimen for the...
- Any acute infection including acute upper respiratory or lower respiratory infections, pulmonary exacerbation, changes in therapy for pulmonary...
- 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...
See the full eligibility 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).
- 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.
- 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
- 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...
- 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.
- 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).
- 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.
- 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
- 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...
- 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.
The study team makes the final eligibility decision.
Where it is enrolling
- Long Beach, California, United States
- Los Angeles, California, United States
- San Francisco, California, United States
- Denver, Colorado, United States
- Boston, Massachusetts, United States
- New York, New York, United States
- Seattle, Washington, United States
- Montpellier, France
- Berlin, Germany
- Potsdam, Germany
- Jerusalem, Israel
- Petah Tikva, Israel
- Milan, Italy
- Barcelona, Spain
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Long Beach, California, United States; Los Angeles, California, United States; San Francisco, California, United States; Denver, Colorado, United States; Boston, Massachusetts, United States; New York, New York, United States and 8 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.