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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03375047
Recruitment Status : Recruiting
First Posted : December 15, 2017
Last Update Posted : March 21, 2019
Information provided by (Responsible Party):
Translate Bio, Inc.

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.

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 32 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 : January 2021
Estimated Study Completion Date : February 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Low Dose
Low dose of MRT5005
Drug: MRT5005
Nebulization of MRT5005

Experimental: Mid Dose
Mid dose of MRT5005
Drug: MRT5005
Nebulization of MRT5005

Experimental: High Dose
High dose of MRT5005
Drug: MRT5005
Nebulization of MRT5005

Placebo Comparator: Placebo Comparator
Normal Saline 0.9% USP
Drug: Normal saline
Normal Saline for Inhalation

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. Delivery of mRNA [ Time Frame: 24 hours after 5th dose ]
    Assessment of delivery of the mRNA to the bronchial epithelial cells by qPCR

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

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.

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 identifier (NCT number): NCT03375047

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Contact: Caroline O'Hara 8572092450

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United States, Alabama
University of Alabama at Birmingham Not yet recruiting
Birmingham, Alabama, United States, 35233
United States, Colorado
National Jewish Health Recruiting
Denver, Colorado, United States, 80206
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32608
Central Florida Pulmonary Group Recruiting
Orlando, Florida, United States, 32803
United States, Illinois
Northwestern University Recruiting
Chicago, Illinois, United States, 60611
United States, Indiana
University of Indiana Recruiting
Indianapolis, Indiana, United States, 46202
United States, Maine
Maine Medical Center Recruiting
Portland, Maine, United States, 04102
United States, Maryland
Johns Hopkins University Not yet recruiting
Baltimore, Maryland, United States, 21202
United States, Montana
Billings Clinic Not yet recruiting
Billings, Montana, United States, 59107
United States, Ohio
University Hospitals Recruiting
Cleveland, Ohio, United States, 44106
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
United States, Oregon
Oregon Health and Sciences University Recruiting
Portland, Oregon, United States, 97239
United States, Pennsylvania
University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
New Orleans Center for Clinical Research Recruiting
Knoxville, Tennessee, United States, 37920
United States, Virginia
Virginia Commonwealth University Recruiting
Richmond, Virginia, United States, 23219
Sponsors and Collaborators
Translate Bio, Inc.

Alton EWFW, Armstrong DK, Ashby D, Bayfield KJ, Bilton D, Bloomfield EV, Boyd AC, Brand J, Buchan R, Calcedo R, Carvelli P, Chan M, Cheng SH, Collie DDS, Cunningham S, Davidson HE, Davies G, Davies JC, Davies LA, Dewar MH, Doherty A, Donovan J, Dwyer NS, Elgmati HI, Featherstone RF, Gavino J, Gea-Sorli S, Geddes DM, Gibson JSR, Gill DR, Greening AP, Griesenbach U, Hansell DM, Harman K, Higgins TE, Hodges SL, Hyde SC, Hyndman L, Innes JA, Jacob J, Jones N, Keogh BF, Limberis MP, Lloyd-Evans P, Maclean AW, Manvell MC, McCormick D, McGovern M, McLachlan G, Meng C, Montero MA, Milligan H, Moyce LJ, Murray GD, Nicholson AG, Osadolor T, Parra-Leiton J, Porteous DJ, Pringle IA, Punch EK, Pytel KM, Quittner AL, Rivellini G, Saunders CJ, Scheule RK, Sheard S, Simmonds NJ, Smith K, Smith SN, Soussi N, Soussi S, Spearing EJ, Stevenson BJ, Sumner-Jones SG, Turkkila M, Ureta RP, Waller MD, Wasowicz MY, Wilson JM, Wolstenholme-Hogg P; UK Cystic Fibrosis Gene Therapy Consortium. Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial. Lancet Respir Med. 2015 Sep;3(9):684-691. doi: 10.1016/S2213-2600(15)00245-3. Epub 2015 Jul 3. Erratum in: Lancet Respir Med. 2015 Sep;3(9):e33.

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Responsible Party: Translate Bio, Inc. Identifier: NCT03375047    
Other Study ID Numbers: MRT5005-101
First Posted: December 15, 2017    Key Record Dates
Last Update Posted: March 21, 2019
Last Verified: March 2019
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
Additional relevant MeSH terms:
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Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases