Status:
UNKNOWN
Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis
Lead Sponsor:
Translate Bio, Inc.
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
This Phase 1/2, first-in-human study will evaluate the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization to the respiratory tract of adult subject...
Eligibility Criteria
Inclusion
- Confirmed diagnosis of CF as defined by both of the following:
- Two CF disease-causing 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.
- 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
- An acute upper or lower respiratory infection, pulmonary exacerbation, 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.
- Receiving treatment with ivacaftor monotherapy (KALYDECO)
- For all groups except Daily dosing: Receiving treatment with triple combination therapy (TRIKAFTA).
- Subjects 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 is not an exclusion for this study.
Key Trial Info
Start Date :
May 10 2018
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2021
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT03375047
Start Date
May 10 2018
End Date
December 1 2021
Last Update
November 16 2020
Active Locations (18)
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1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
2
National Jewish Health
Denver, Colorado, United States, 80206
3
University of Florida
Gainesville, Florida, United States, 32608
4
Central Florida Pulmonary Group
Orlando, Florida, United States, 32803