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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01684410 | Safety and Tolerability Trial of Inhaled Alpha1-Proteinase Inhibitor (Human), Hydrophobic Chromatography Process (Alpha-1 HC) in Subjects With Cystic Fibrosis | PHASE2 | COMPLETED | 30 | — | — | Aug 1, 2012 | Oct 1, 2013 | Feb 8, 2016 | 6 | United States |
adverse event frequency
| Arm | Type | Description |
|---|---|---|
| Alpha-1 HC 100 mg | EXPERIMENTAL | 100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks. |
| Alpha-1 HC 200 mg | EXPERIMENTAL | 200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks. |
| Placebo | PLACEBO_COMPARATOR | Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate). |
| Name | Type | Description |
|---|---|---|
| Alpha-1 HC 100 mg | BIOLOGICAL | Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma. Alpha-1 HC 100 mg inhaled once daily for 21 days for a total of 21 inhaled treatments. |
| Placebo | BIOLOGICAL | Phosphate Buffer Saline with Polysorbate (placebo) composed of the same elements listed for Alpha-1 HC, minus the 50 mg/mL of Alpha-1 HC. Placebo inhaled once daily for 21 days for a total of 21 inhaled treatments. |
| Alpha-1 HC 200 mg | BIOLOGICAL | Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma. Alpha-1 HC 200 mg inhaled once daily for 21 days for a total of 21 inhaled treatments. |
Inclusion Criteria: * Age 18 years or older. * Documentation of CF diagnosis. * Have a pre-bronchodilator FEV1 ≥ 40% of predicted at Visit 1 and have a Visit 2 pre-investigational product FEV1 that is ≥ 40% of predicted and within ± 15% of the Visit 1 result. * Deemed by the Investigator to be a su...
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|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 9 | PHASE3 | VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, VNZ/TEZ/D-IVA, VX-522 mRNA therapy |
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| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
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