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Nucleic Acid-based Therapy

High Dose for Cystic Fibrosis (RESTORE-CF Trial)

Phase 1 & 2
Waitlist Available
Research Sponsored by Translate Bio, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 4 weeks after last dose
Awards & highlights

RESTORE-CF Trial Summary

This trial will study a new CF drug to see if it is safe and tolerated when given to adults with CF.

Eligible Conditions
  • Cystic Fibrosis

RESTORE-CF Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~4 weeks after last dose
This trial's timeline: 3 weeks for screening, Varies for treatment, and 4 weeks after last dose for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Types, frequency and severity of treatment-emergent Adverse Events
Secondary outcome measures
Biological activity of nebulized MRT5005

RESTORE-CF Trial Design

7Treatment groups
Experimental Treatment
Placebo Group
Group I: Mid/High DoseExperimental Treatment1 Intervention
20 mg MRT5005
Group II: Mid DoseExperimental Treatment1 Intervention
16 mg MRT5005
Group III: Low/Mid DoseExperimental Treatment1 Intervention
12 mg MRT5005
Group IV: Low DoseExperimental Treatment1 Intervention
8 mg MRT5005
Group V: High DoseExperimental Treatment1 Intervention
24 mg MRT5005
Group VI: Daily DoseExperimental Treatment1 Intervention
20 mg MRT5005 delivered in 5 consecutive daily doses of 4mg
Group VII: Placebo ComparatorPlacebo Group1 Intervention
Normal Saline 0.9% USP

Find a Location

Who is running the clinical trial?

Translate Bio, Inc.Lead Sponsor
1 Previous Clinical Trials

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis
2018. "Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03375047.
All > Fibrosis > Cystic Fibrosis
~6 spots leftby Apr 2025
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