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

Cystic Fibrosis Clinical Trial

— RESTORE-CF  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03375047
• Other study ID #: MRT5005-101
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: May 10, 2018
• Est. completion date: December 2021

Study information

• Verified date: November 2020
• Source: Translate Bio, Inc.
• Contact: Elizabeth Brown
• Phone: 781-386-7261
• Email: ebrown[@]translate.bio
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 subjects with CF.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: December 2021
• Est. primary completion date: October 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• MRT5005
Nebulization of MRT5005
• Normal saline
Normal Saline for Inhalation

Locations

Country	Name	City	State
United States	University of Michigan	Ann Arbor	Michigan
United States	Johns Hopkins University	Baltimore	Maryland
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Northwestern University	Chicago	Illinois
United States	University of Cincinnati	Cincinnati	Ohio
United States	University Hospitals	Cleveland	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	National Jewish Health	Denver	Colorado
United States	University of Florida	Gainesville	Florida
United States	University of Indiana	Indianapolis	Indiana
United States	New Orleans Center for Clinical Research	Knoxville	Tennessee
United States	Central Florida Pulmonary Group	Orlando	Florida
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Maine Medical Center	Portland	Maine
United States	Oregon Health and Sciences University	Portland	Oregon
United States	Virginia Commonwealth University	Richmond	Virginia
United States	University of Utah	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Translate Bio, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Types, frequency and severity of treatment-emergent Adverse Events	Safety and tolerability of nebulized MRT5005 will be assessed through the types, frequency and severity of treatment-emergent Adverse Events experienced by participants on the trial	12 months after last dose
Secondary	Biological activity of nebulized MRT5005	Changes from baseline in ppFEV1	4 weeks after last dose