Testing the safety and effectiveness of SPL84 for cystic fibrosis

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

This study is testing if the drug SPL84 is safe and effective for adults with cystic fibrosis who have certain genetic mutations.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: SpliSense Ltd. (Industry-sponsored)
• Locations: 2 sites (Boston, Massachusetts and 1 other locations)
• Trial ID: NCT06429176 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of the drug SPL84 in adult patients diagnosed with cystic fibrosis who have specific genetic mutations. Participants will receive either SPL84 or a placebo through inhalation weekly for nine weeks, with regular clinic visits for monitoring and assessments. The study will compare the effects of SPL84 against a placebo to determine its safety and potential effectiveness in treating cystic fibrosis.

Who should consider this trial

Good fit: Ideal candidates include adults with cystic fibrosis who have the 3849+10 Kb C->T mutation and meet specific health criteria.

Not a fit: Patients currently using certain CF medications or those with specific health conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for patients with cystic fibrosis, particularly those with specific mutations.

How similar studies have performed: While this approach is being tested, similar studies have shown promise in evaluating new treatments for cystic fibrosis.

Eligibility criteria

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 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.

Exclusion Criteria:

* 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.

Where this trial is running

Boston, Massachusetts and 1 other locations

• Boston Children'S Hospital — Boston, Massachusetts, United States (Recruiting)
• Nationwide Children'S Hospital — Columbus, Ohio, United States (Recruiting)

Conditions Cystic Fibrosis