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 (CO-hCFTR mRNA/ICE LNP) Administered by Nebulization to Adult Subjects with Cystic Fibrosis
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- STATUS
- Not Recruiting
Summary
Study MRT5005-101 represents an important first step in the evaluation of MRT5005 as a novel mRNA-based therapeutic approach for treating CF lung disease. Adult CF subjects participating in this Phase 1/2 study will be the first subjects to receive MRT5005. As such, the primary objective of the study is to characterize the safety profile and tolerated dose range of MRT5005 in CF subjects. Study MRT5005-101 will investigate the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization to the respiratory tract. This study will also assess the delivery of CO-hCFTR mRNA (the active drug substance of MRT5005) to bronchial epithelial cells following multiple doses of 2 different dose levels MRT5005, and will characterize its biological activity by measuring changes in CFTR protein levels and CFTR chloride channel activity post-treatment. These results will be essential in establishing the initial safety profile of MRT5005 as well as guiding dose selection for subsequent clinical development.
Description
Study MRT5005-101 represents an important first step in the evaluation of MRT5005 as a novel mRNA-based therapeutic approach for treating CF lung disease. Adult CF subjects participating in this Phase 1/2 study will be the first subjects to receive MRT5005. As such, the primary objective of the study is to characterize the safety profile and tolerated dose range of MRT5005 in CF subjects. Study MRT5005-101 will investigate the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization to the respiratory tract. This study will also assess the delivery of CO-hCFTR mRNA (the active drug substance of MRT5005) to bronchial epithelial cells following multiple doses of 2 different dose levels MRT5005, and will characterize its biological activity by measuring changes in CFTR protein levels and CFTR chloride channel activity post-treatment. These results will be essential in establishing the initial safety profile of MRT5005 as well as guiding dose selection for subsequent clinical development.
Details
| Condition | cystic fibrosis |
|---|---|
| Age | 100years or below |
| Clinical Study Identifier | TX8748 |
| Last Modified on | 19 February 2024 |
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