Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults Wit… (NCT03375047) | Clinical Trial Compass
CompletedPhase 1/2
Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis
United States42 participantsStarted 2018-05-10
Plain-language summary
This Phase 1/2, first-in-human study evaluated the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization to the respiratory tract of adult subjects with cystic fibrosis (CF).
Who can participate
Age range18 Years
SexALL
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Inclusion Criteria:
* Confirmed diagnosis of CF as defined by both of the following:
* Two CF disease-causing cystic fibrosis transmembrane conductance regulator (CFTR) mutations in Class I or II (genotype confirmed at the screening visit).
* Chronic sinopulmonary disease and/or gastrointestinal/nutritional abnormalities consistent with CF disease.
* Clinically stable CF disease, as judged by the investigator.
* Forced expiratory volume in 1 second (FEV1) ≥50% and ≤90% of the predicted normal for age, gender, and height at screening.
* Resting oxygen saturation ≥92% on room air (pulse oximetry).
Exclusion Criteria:
* An acute upper or lower respiratory infection, pulmonary exacerbation, or clinically significant episode of hemoptysis or change in chronic respiratory medications (including antibiotics) for CF lung disease within 28 days prior to dosing with investigational product on Day 1.
* Participants were receiving treatment with ivacaftor monotherapy (KALYDECO).
* Parts A and B only: Were receiving treatment with triple combination therapy (TRIKAFTA).
* Participants with a Class III, IV, or V CFTR gene mutation in at least 1 allele.
* Infection with highly virulent bacteria associated with accelerated decline in pulmonary function and/or decreased survival (e.g., Burkholderia cenocepacia, Burkholderia dolosa, Mycobacterium abscessus).
Treatment with ORKAMBI or SYMDEKO was not an exclusion for this study.
What they're measuring
1
Parts A, B and D: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events
Timeframe: From the first dose of study treatment administration (Day 1) up to 48 weeks (end of the follow-up period) after the last dose of study treatment administration (Part A: Day 337, Part B: Day 365 and Part D: Day 341)