Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03375047 | Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis | PHASE1 | COMPLETED | 42 | — | — | May 10, 2018 | Mar 15, 2022 | Feb 9, 2026 | 16 | United States |
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily had a causal relationship with the study treatment. A serious adverse event (SAE) was any untoward medical occurrence (whether considered to be related to study treatment or not) that at any dose: resulted in death; or was life-threatening; or required inpatient hospitalization or prolongation of existing hospitalization; or resulted in persistent or significant disability/incapacity; or was a congenital abnormality/birth defect; or was an important medical event. The TEAEs were defined as events that were newly reported or reported to worsen in severity after the start of study treatment up to 48 weeks (end of follow-up period) after the last dose of study treatment administration.
| Arm | Type | Description |
|---|---|---|
| Part A - SAD Group 1: MRT5005 8 mg | EXPERIMENTAL | Participants received single dose of MRT5005 8 milligrams (mg) by nebulization on Day 1. |
| Part A - SAD Group 2: MRT5005 16 mg | EXPERIMENTAL | Participants received single dose of MRT5005 16 mg by nebulization on Day 1. |
| Part A - SAD Group 3: MRT5005 20 mg | EXPERIMENTAL | Participants received single dose of MRT5005 20 mg by nebulization on Day 1. |
| Part A - SAD Group 4: MRT5005 24 mg | EXPERIMENTAL | Participants received single dose of MRT5005 24 mg by nebulization on Day 1. |
| Part A - SAD Groups: Pooled Placebo | PLACEBO_COMPARATOR | Participants received single dose of placebo (normal saline) by nebulization on Day 1. Data were analyzed as a pooled population for all participants who received placebo in Part A SAD groups and reported in this arm. |
| Part B - MAD Group 1: MRT5005 8 mg | EXPERIMENTAL | Participants received MRT5005 8 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). |
| Part B - MAD Group 2: MRT5005 12 mg | EXPERIMENTAL | Participants received MRT5005 12 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). |
| Part B - MAD Group 3: MRT5005 16 mg | EXPERIMENTAL | Participants received MRT5005 16 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). |
| Part B - MAD Group 4: MRT5005 20 mg | EXPERIMENTAL | Participants received MRT5005 20 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). |
| Part B - MAD Groups: Pooled Placebo | PLACEBO_COMPARATOR | Participants received placebo (normal saline) once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). Data were analyzed as a pooled population for all participants who received placebo in Part B MAD groups and reported in this arm. |
| Part D: MRT5005 4 mg | EXPERIMENTAL | Participants received MRT5005 4 mg once daily by nebulization from Day 1 to Day 5. |
| Part D: Placebo | PLACEBO_COMPARATOR | Participants received placebo (normal saline) once daily by nebulization from Day 1 to Day 5. |
| Name | Type | Description |
|---|---|---|
| MRT5005 | DRUG | Nebulization of MRT5005 |
| Normal saline | DRUG | Normal Saline for Inhalation |
Inclusion Criteria: * Confirmed diagnosis of CF as defined by both of the following: * Two CF disease-causing cystic fibrosis transmembrane conductance regulator (CFTR) mutations in Class I or II (genotype confirmed at the screening visit). * Chronic sinopulmonary disease and/or gastrointestin...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 9 | PHASE3 | VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, VNZ/TEZ/D-IVA, VX-522 mRNA therapy |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719, SION-451, SION-2222, SION-109 |
| BiomX Ltd | PHGE | 1 | PHASE2 | BX004 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Illumina, Inc. | ILMN | 1 | — | Undisclosed |