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Tests treatment safety and results for Cystic Fibrosis
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 medicine, given as an once-weekly inhaled treatment, being studied as a potential treatment for 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 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).
- 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
- Cohort 1-3:
- 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
- Cohort 1-3:
- 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...
- 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; those participants taking inhaled...
- 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. 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...
- 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...
- 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's taking place
- Los Angeles, California, United States
- Denver, Colorado, United States
- Boston, Massachusetts, United States
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 - 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?
Study sites include Los Angeles, California, United States; Denver, Colorado, United States; Boston, Massachusetts, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.