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Purpose

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. The purpose of this research study is to: - test the safety and effectiveness of multiple doses of the study drug, SPL84 - test how multiple doses of the drug are processed by the body 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. Participants will: Take drug SPL84 or a placebo by inhalation every week for 9 weeks months Visit the clinic approximately 14 times over 17.5 weeks for checkups and tests

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Active Comparator
SPL84
  • Drug: SPL84
    SPL84 solution for nebulization
Placebo Comparator
Placebo
  • Other: Placebo
    Placebo solution for nebulization

Recruiting Locations

University of Alabama at Birmingham
Birmingham, Alabama 35233
Contact:
Kathryn Monroe
205-638-5599
kathrynmonroe@uabmc.edu

More Details

Status
Recruiting
Sponsor
SpliSense Ltd.

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.