Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis
Purpose
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.
Condition
- Cystic Fibrosis
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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 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) - 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.
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Low Dose |
8 mg MRT5005 |
|
|
Experimental Low/Mid Dose |
12 mg MRT5005 |
|
|
Experimental Mid Dose |
16 mg MRT5005 |
|
|
Experimental Mid/High Dose |
20 mg MRT5005 |
|
|
Experimental High Dose |
24 mg MRT5005 |
|
|
Placebo Comparator Placebo Comparator |
Normal Saline 0.9% USP |
|
|
Experimental Daily Dose |
20 mg MRT5005 delivered in 5 consecutive daily doses of 4mg |
|
Recruiting Locations
University of Alabama at Birmingham
Birmingham, Alabama 35233
Birmingham, Alabama 35233
More Details
- Status
- Unknown status
- Sponsor
- Translate Bio, Inc.