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Study on the Safety and Effects of SPL84 for Patients with Cystic Fibrosis

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying cystic fibrosis, a genetic condition that affects the lungs and digestive system. The trial will test a new treatment called SPL84, which is a solution for inhalation. This treatment is designed to help people with a specific genetic mutation known as the 3849 +10 Kb C->T mutation in the CFTR gene. The purpose of the study is to evaluate the safety and tolerability of SPL84 when given in increasing doses through inhalation.

Participants in the study will use a device called the Aerogen InnoSpire Go nebulizer to inhale the medication. This device helps deliver the treatment deep into the lungs. The study will compare the effects of SPL84 with a placebo solution, which looks like the treatment but does not contain the active substance. The trial will monitor participants for any side effects and changes in their health, including lung function and overall well-being.

The study will take place over several months, during which participants will receive multiple doses of SPL84. Researchers will collect information on how the body processes the medication and its impact on symptoms of cystic fibrosis. The goal is to determine the most suitable dose that is both safe and effective for patients with this genetic mutation. Participants will be closely monitored throughout the study to ensure their safety and to gather valuable data on the treatment's potential benefits.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on specific criteria such as age, diagnosis of cystic fibrosis, and lung function stability.

    Participants must be non-smokers or non-vapers for at least 180 days prior to the start of the study.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to establish baseline health metrics. This includes measuring vital signs, conducting a physical examination, and performing lung function tests such as spirometry.

    Blood and urine samples are collected to evaluate baseline clinical laboratory values.

  3. Step 3

    Treatment phase

    Participants receive multiple ascending doses of the investigational drug SPL84 administered by inhalation.

    A placebo solution for inhalation is used as a control in this phase.

    The treatment aims to evaluate the safety and tolerability of SPL84.

  4. Step 4

    Monitoring and follow-up

    Participants are monitored for any adverse events, changes in vital signs, and other health indicators.

    Regular follow-up visits are scheduled to assess lung function, body weight, and other health parameters.

    The concentration of SPL84 in blood and urine is measured to understand how the body processes the drug.

  5. Step 5

    Completion of study

    Upon completion of the study, a final assessment is conducted to evaluate the overall health and any changes from the baseline.

    Participants may be asked to complete a questionnaire regarding respiratory symptoms to assess the impact of the treatment.

Who can join the trial?

6 criteria

  • Participants must be **adults aged 18 and above** at the time they agree to join the study.
  • Participants must have a **diagnosis of cystic fibrosis (CF)**, which is a genetic condition affecting the lungs and other organs. They must have two specific genetic mutations causing CF, with one being the **3849+10 Kb C->T mutation** in the **CFTR gene**. Proof from a certified genetic lab is needed.
  • Participants must have a **body mass index (BMI)** of **17 kg/m² or higher**. BMI is a measure of body fat based on height and weight.
  • Participants must have a **FEV1** (Forced Expiratory Volume in 1 second) between **40% and 90%** of what is predicted for their age and size during the screening. FEV1 is a test that measures how much air you can forcefully exhale in one second.
  • Participants must have **stable lung function**, meaning their FEV1 at screening should not be more than 20% lower than their highest FEV1 recorded in the last 120 days. If past records are not available, the treating doctor can confirm stable lung function if the FEV1 at the start of the study is within 20% of the FEV1 at screening.
  • Participants must be **non-smokers or non-vapers** for at least **180 days (6 months)** before the screening, according to their own report.

Who cannot join the trial?

9 criteria

  • Participants who do not have cystic fibrosis cannot join the study. Cystic fibrosis is a genetic condition that affects the lungs and digestive system.
  • Participants who are not within the specified age range cannot join the study. The study is for certain age groups only.
  • Participants who are not able to follow the study procedures or take the study medication as required cannot join the study.
  • Participants who have other medical conditions that might interfere with the study cannot join.
  • Participants who are pregnant or breastfeeding cannot join the study.
  • Participants who are taking certain medications that might interfere with the study cannot join.
  • Participants who have had a recent infection or illness that might affect the study cannot join.
  • Participants who have a history of drug or alcohol abuse cannot join the study.
  • Participants who have participated in another clinical trial recently cannot join.
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Investigated drugs

SPL84 is a medication being tested in this clinical trial. It is administered by inhalation to patients with cystic fibrosis who have a specific genetic mutation known as 3849 +10 Kb C->T. The trial aims to assess the safety and tolerability of SPL84 when given in increasing doses. Researchers are also looking to determine the maximum tolerated dose (MTD) of this medication.

What is already known about the treatment

SPL84 – This medication is administered by inhalation and is currently being studied in clinical trials for its potential use in treating cystic fibrosis, specifically in patients with the 3849 +10 Kb C->T mutation. The main therapeutic indication for SPL84 is to improve lung function and manage symptoms associated with cystic fibrosis. At the molecular level, SPL84 is designed to target and modulate specific pathways involved in the disease process, although the exact mechanism of action is still under investigation. It is classified pharmacologically as a potential cystic fibrosis transmembrane conductance regulator (CFTR) modulator.

Investigated diseases

Cystic Fibrosis – Cystic fibrosis is a genetic disorder that affects the respiratory and digestive systems. It is caused by mutations in the CFTR gene, leading to the production of thick and sticky mucus. This mucus can clog the airways, causing breathing difficulties and frequent lung infections. Over time, the buildup of mucus can also affect the pancreas, leading to digestive problems and poor nutrient absorption. The disease is progressive, meaning symptoms typically worsen over time. It is most commonly diagnosed in childhood, but symptoms can vary widely among individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-511184-28-00Protocol codeSPL84-002Estimated enrolment60 patientsSponsorSplisense Ltd.

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