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

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ClinicalTrials.gov Identifier: NCT03375047
Recruitment Status : Recruiting
First Posted : December 15, 2017
Last Update Posted : March 21, 2019
Sponsor:
Information provided by (Responsible Party):
Translate Bio, Inc.

Tracking Information
First Submitted Date  ICMJE September 7, 2017
First Posted Date  ICMJE December 15, 2017
Last Update Posted Date March 21, 2019
Actual Study Start Date  ICMJE May 10, 2018
Estimated Primary Completion Date January 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 11, 2017)
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
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03375047 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: December 11, 2017)
Delivery of mRNA [ Time Frame: 24 hours after 5th dose ]
Assessment of delivery of the mRNA to the bronchial epithelial cells by qPCR
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis
Official Title  ICMJE 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
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. 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Drug: MRT5005
    Nebulization of MRT5005
  • Drug: Normal saline
    Normal Saline for Inhalation
Study Arms  ICMJE
  • Experimental: Low Dose
    Low dose of MRT5005
    Intervention: Drug: MRT5005
  • Experimental: Mid Dose
    Mid dose of MRT5005
    Intervention: Drug: MRT5005
  • Experimental: High Dose
    High dose of MRT5005
    Intervention: Drug: MRT5005
  • Placebo Comparator: Placebo Comparator
    Normal Saline 0.9% USP
    Intervention: Drug: Normal saline
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 11, 2017)
32
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE February 2021
Estimated Primary Completion Date January 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Caroline O'Hara 8572092450 cohara@translate.bio
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03375047
Other Study ID Numbers  ICMJE MRT5005-101
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Translate Bio, Inc.
Study Sponsor  ICMJE Translate Bio, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Translate Bio, Inc.
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP