Lancovutide (Moli1901) Inhalation Solution Study in Adolescents and Adults With Cystic Fibrosis

Study Overview
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Brief Summary
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This is a dose-finding study for the investigational product Lancovutide (Moli1901) in the exploratory phase IIb to establish minimum effective dose, optimal dose, and maximum safe dose. Additionally, the tolerability of Moli1901 shall be investigated.

Detailed Description
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Study Moli1901-010B is a multi center, parallel group, placebo controlled, double-blind efficacy and safety evaluation of three different dosage schedules of aerosolized Moli1901 in adolescents (12 years of age or older), and adults. Study will start with a screening period (visit 1) followed by an 8 weeks double blind comparative treatment period (visit 2-7). Thereafter, subjects will be observed for additional 4 weeks without treatment (follow-up period and visit 8). Spirometry, pulse oximetry and other safety measurements will be performed up to 120 min after study medication inhalation. The study will be conducted in 30 sites in 9 European countries.

Official Title
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A Randomized, Double-Blind, Placebo-Controlled, Dose-finding Study to Evaluate the Efficacy and Safety of Aerosolized Moli1901 in Adolescents (12 Years of Age or Older) and Adults With Cystic Fibrosis

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: Moli1901
* Drug: Moli1901 placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Body mass index Confirmed diagnosis of cystic fibrosis FEV1 between 50% and 85% of predicted Oxygen saturation level measured by pulse oximetry (SpO2) >90 % on room air Exclusion Criteria: Bronchial hyperresponsiveness Unstable lung function Pulmonary disease such as pneumonia, tuberculosis, or lung cancer Acute upper respiratory tract infection within the last 2 weeks Acute lower respiratory tract infection (requiring antibiotics or hospitalization) within the last 4 weeks Pulmonary exacerbation within the last 4 weeks Changes from routine maintenance therapy within the last 4 weeks Scheduled changes to inhaled antibiotics regimen during the course of the study Receive or are planned to receive any treatment via on-off regimen (e.g. Tobramycin - TOBI®); last dose of any on-off treatment within the last 6 weeks Any clinically significant liver, renal, cardiac, neurological, or hematological disease ABPA or colonization with Burkholderia cepacia Poorly controlled diabetes mellitus

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>daily inhalation | Drug: Moli1901<br>* 2,5 ml inhalation solution, 8 weeks treatment period<br>* Other names: Lancovutide, 2622U90, duramycin;|
| Experimental: 2<br>inhalation every other day | Drug: Moli1901<br>* 2,5 ml inhalation solution, 8 weeks treatment period<br>* Other names: Lancovutide, 2622U90, duramycin;|
| Experimental: 3<br>inhalation twice a week | Drug: Moli1901<br>* 2,5 ml inhalation solution, 8 weeks treatment period<br>* Other names: Lancovutide, 2622U90, duramycin;|
| Placebo Comparator: 4<br>daily inhalation | Drug: Moli1901 placebo<br>* 2,5 ml placebo solution, 8 weeks treatment period<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The change in the percentage of the predicted FEV1 (forced expiratory volume in first second) value |  | at every study visit | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The ensemble of two subject reported clinical scores, which are physical dimensions of the self-report, disease-specific quality of life test, the cystic fibrosis questionnaire in the revised version |  | every 2 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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cystic fibrosis, Moli1901, Lancovutide, 2622U90, duramycin, lung