A Phase 1/2 Study of VX-121 in Subjects With Cystic Fibrosis and in Subjects without Cystic Fibrosis

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Study details

Phase 1/2 safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of VX-121

Phase The different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use: Phase 2
Recruitment target: 132
CF sponsor: Vertex Pharmaceuticals Incorporated
CF sponsor type: Commercial

Age range: 18 years and older
Including people: One copy of F508del mutation
Body weight ≥35 kg
Must be able to produce a sweat sample at screening
Excluding people: An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for sinopulmonary disease
History of cirrhosis (liver injury)
Risk factors for Torsade de Pointes (abnormal heart rhythm) and other ventricular arrhythmias (abnormal heartbeats)

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CF centres running this trial

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CTAP centre

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CTAP centre