A Phase 1/2 Randomized Double-Blind Placebo-Controlled Combined Single and Multiple Ascending Dose Study Evaluating the Safety Tolerability and Biological Activity of MRT5005 (COhCFTR mRNA/ICE LNP) Administered by Nebulization to Adult Subjects with Cystic Fibrosis

Recruiting
99 years or below
All
Phase 1
4 participants needed
1 Location

Brief description of study

This research study is being done to study a new way to possibly treat cystic fibrosis lung disease. The investigational drug to be tested in the present study is known by its code name MRT5005. MRT5005 consists of very small particles containing messenger ribonucleic acid (mRNA), which has the genetic information for cells to make normal CFTR protein. These particles are delivered to the cells in the lungs by a standard nebulizer. Following uptake of MRT5005 by the cells, the mRNA leads to the production of normally functioning CFTR protein in the cells. MRT5005 is intended to restore CFTR channel function in the lungs of all cystic fibrosis patients, regardless of the type of CFTR gene defect the patient has.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: TBD
  • Age: 99 years or below
  • Gender: All
Updated on 19 Feb 2024. Study ID: 829530

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