Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis



Status:Recruiting
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:18 - Any
Updated:3/23/2019
Start Date:May 10, 2018
End Date:February 2021
Contact:Caroline O'Hara
Email:cohara@translate.bio
Phone:8572092450

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A Phase 1/2, Randomized, Double-Blinded, Placebo-Controlled, Combined Single and Multiple Ascending Dose Study Evaluating the Safety, Tolerability, and Biological Activity of MRT5005 Administered by Nebulization to Adult Subjects With Cystic Fibrosis

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 subjects with CF. This study will also assess the delivery of the drug to bronchial
epithelial cells following multiple doses of MRT5005, and will characterize its biological
activity by measuring changes in CFTR protein levels and CFTR chloride channel activity
post-treatment.


Inclusion Criteria:

- Confirmed diagnosis of CF as defined by all of the following:

- A sweat chloride value of ≥60 mmol/L by quantitative pilocarpine iontophoresis
(documented in the subject's medical record).

- 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:

- 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).

- 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 is not an exclusion for this study.
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326 North Mills Avenue
Orlando, Florida 32803
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3400 N Charles St
Baltimore, Maryland 21205
410-516-8000
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1720 2nd Ave S
Birmingham, Alabama 35233
(205) 934-4011 
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
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Gainesville, Florida 32610
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22 Bramhall St
Portland, Maine 04102
(207) 662-0111
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801 North 29th Street
Billings, Montana 59107
406-238-2500
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303 East Superior Street
Chicago, Illinois 60611
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Cleveland, Ohio 44106
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1400 Jackson St
Denver, Colorado 80206
(303) 388-4461
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Indianapolis, Indiana
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Knoxville, Tennessee 37920
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3451 Walnut St
Philadelphia, Pennsylvania 19104
1 (215) 898-5000
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Portland, Oregon 97227
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Richmond, Virginia 23298
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