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Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis (RESTORE-CF)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03375047
Recruitment Status : Unknown
Verified November 2020 by Translate Bio, Inc..
Recruitment status was:  Recruiting
First Posted : December 15, 2017
Last Update Posted : November 16, 2020
Sponsor:
Information provided by (Responsible Party):
Translate Bio, Inc.

Study Description
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Brief 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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: MRT5005 Drug: Normal saline Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : May 10, 2018
Estimated Primary Completion Date : October 2021
Estimated Study Completion Date : December 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arms and Interventions
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Arm Intervention/treatment
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


Outcome Measures
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Primary Outcome Measures :
  1. Types, frequency and severity of treatment-emergent Adverse Events [ Time Frame: 12 months after last dose ]
    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


Secondary Outcome Measures :
  1. Biological activity of nebulized MRT5005 [ Time Frame: 4 weeks after last dose ]
    Changes from baseline in ppFEV1


Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03375047


Contacts
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Contact: Elizabeth Brown 781-386-7261 ebrown@translate.bio
Contact: Caroline O'Hara 8572092450 cohara@translate.bio

Locations
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Sponsors and Collaborators
Translate Bio, Inc.
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Translate Bio, Inc.
ClinicalTrials.gov Identifier: NCT03375047    
Other Study ID Numbers: MRT5005-101
First Posted: December 15, 2017    Key Record Dates
Last Update Posted: November 16, 2020
Last Verified: November 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Translate Bio, Inc.:
Cystic Fibrosis
CF
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
 Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases