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 Sex
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

ArmDescriptionAssigned Intervention
Experimental
Low Dose 		8 mg MRT5005
  • Drug: MRT5005
    Nebulization of MRT5005
Experimental
Low/Mid Dose 		12 mg MRT5005
  • Drug: MRT5005
    Nebulization of MRT5005
Experimental
Mid Dose 		16 mg MRT5005
  • Drug: MRT5005
    Nebulization of MRT5005
Experimental
Mid/High Dose 		20 mg MRT5005
  • Drug: MRT5005
    Nebulization of MRT5005
Experimental
High Dose 		24 mg MRT5005
  • Drug: MRT5005
    Nebulization of MRT5005
Placebo Comparator
Placebo Comparator 		Normal Saline 0.9% USP
  • Drug: Normal saline
    Normal Saline for Inhalation
Experimental
Daily Dose 		20 mg MRT5005 delivered in 5 consecutive daily doses of 4mg
  • Drug: MRT5005
    Nebulization of MRT5005

Recruiting Locations

University of Alabama at Birmingham
Birmingham 4049979, Alabama 4829764 35233

National Jewish Health
Denver 5419384, Colorado 5417618 80206

University of Florida
Gainesville 4156404, Florida 4155751 32608

Northwestern University
Chicago 4887398, Illinois 4896861 60611

University of Indiana
Indianapolis 4259418, Indiana 4921868 46202

Maine Medical Center
Portland 4975802, Maine 4971068 04102

Johns Hopkins University
Baltimore 4347778, Maryland 4361885 21202

University of Michigan
Ann Arbor 4984247, Michigan 5001836 48109

University of Cincinnati
Cincinnati 4508722, Ohio 5165418 45267

University Hospitals
Cleveland 5150529, Ohio 5165418 44106

Nationwide Children's Hospital
Columbus 4509177, Ohio 5165418 43205

Oregon Health and Sciences University
Portland 5746545, Oregon 5744337 97239

University of Pennsylvania
Philadelphia 4560349, Pennsylvania 6254927 19104

UPMC Children's Hospital of Pittsburgh
Pittsburgh 5206379, Pennsylvania 6254927 15224

University of Utah
Salt Lake City 5780993, Utah 5549030 84132

Virginia Commonwealth University
Richmond 4781708, Virginia 6254928 23219

More Details

Status
Recruiting
Sponsor
Translate Bio, Inc.

Study Contact

Elizabeth Brown
781-386-7261
ebrown@translate.bio