Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis
NCT ID: NCT03375047
Last Updated: 2020-11-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE1/PHASE2
40 participants
INTERVENTIONAL
2018-05-10
2021-12-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Low Dose
8 mg MRT5005
MRT5005
Nebulization of MRT5005
Low/Mid Dose
12 mg MRT5005
MRT5005
Nebulization of MRT5005
Mid Dose
16 mg MRT5005
MRT5005
Nebulization of MRT5005
Mid/High Dose
20 mg MRT5005
MRT5005
Nebulization of MRT5005
High Dose
24 mg MRT5005
MRT5005
Nebulization of MRT5005
Placebo Comparator
Normal Saline 0.9% USP
Normal saline
Normal Saline for Inhalation
Daily Dose
20 mg MRT5005 delivered in 5 consecutive daily doses of 4mg
MRT5005
Nebulization of MRT5005
Interventions
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MRT5005
Nebulization of MRT5005
Normal saline
Normal Saline for Inhalation
Eligibility Criteria
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Inclusion Criteria
* 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 Criteria
* 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.
18 Years
ALL
No
Sponsors
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Translate Bio, Inc.
INDUSTRY
Responsible Party
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Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
National Jewish Health
Denver, Colorado, United States
University of Florida
Gainesville, Florida, United States
Central Florida Pulmonary Group
Orlando, Florida, United States
Northwestern University
Chicago, Illinois, United States
University of Indiana
Indianapolis, Indiana, United States
Maine Medical Center
Portland, Maine, United States
Johns Hopkins University
Baltimore, Maryland, United States
University of Michigan
Ann Arbor, Michigan, United States
University of Cincinnati
Cincinnati, Ohio, United States
University Hospitals
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Oregon Health and Sciences University
Portland, Oregon, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
New Orleans Center for Clinical Research
Knoxville, Tennessee, United States
University of Utah
Salt Lake City, Utah, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Countries
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Central Contacts
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References
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Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.
Other Identifiers
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MRT5005-101
Identifier Type: -
Identifier Source: org_study_id