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Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients with Cystic Fibrosis
EU CTIS ID: 2024-511184-28-00
What this study is testing
To evaluate the safety and tolerability of multiple ascending doses of SPL84 administered by inhalation and identify the MTD in participants with CF.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male and female adults aged 18 and above at the time of consent.
- Diagnosis of CF and two CF causing mutations; 3849+10 Kb C->T mutation on one allele in the 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.
- Stable lung functions as defined at screening FEV1 not more than 20% lower than the highest absolute FEV1 (in liters) within 120 days prior to Day 1. If adequate documentation of prior spirometry is not available, determination of stable lung function per the treating physician is acceptable, provided the FEV1 at Day 1 is within 20% of the FEV1 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 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.
- Unstable adherence to standard use of CF medications, e.g. inhaled antibiotics, dornase alfa (Pulmozyme), hypertonic saline and physiotherapy in the period of 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 30 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.
See the full eligibility criteria
- Male and female adults aged 18 and above at the time of consent.
- Diagnosis of CF and two CF causing mutations; 3849+10 Kb C->T mutation on one allele in the 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.
- Stable lung functions as defined at screening FEV1 not more than 20% lower than the highest absolute FEV1 (in liters) within 120 days prior to Day 1. If adequate documentation of prior spirometry is not available, determination of stable lung function per the treating physician is acceptable, provided the FEV1 at Day 1 is within 20% of the FEV1 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 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.
- Unstable adherence to standard use of CF medications, e.g. inhaled antibiotics, dornase alfa (Pulmozyme), hypertonic saline and physiotherapy in the period of 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 30 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 COVID-19 infection within 4 weeks prior to dosing.
The study team makes the final eligibility decision.
Where it's taking place
- Israel
- United States
- Canada
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, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Israel; United States; Canada. 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 EU CTIS; the study team decides eligibility.