Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients with Cystic Fibrosis

SpliSense

Status and phase

Enrolling

Phase 2

Conditions

Cystic Fibrosis

Treatments

Other: Placebo

Drug: SPL84

Study type

Interventional

Funder types

Industry

Identifiers

NCT06429176

SPL84-002

2024-511184-28 (EudraCT Number)

Details and patient eligibility

About

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

Enrollment

24 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.