A Phase 1 interventional study of VX-407 and Placebo in Autosomal Dominant Polycystic Kidney Disease (ADPKD), sponsored by Vertex Pharmaceuticals Incorporated. Completed at 2 sites in 2 countries. Open to participants aged 18 Years to 55 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2025-07-14.
Sponsored by Vertex Pharmaceuticals Incorporated · Phase 1, Interventional, and Treatment
The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetic parameters of VX-407 in healthy participants.
This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).
173 studies on the registry are indexed under Polycystic Kidney, Autosomal Dominant; 37 are open to participants now.
This study's enrollment of 159 is above the median of 54 across 128 interventional studies indexed under Polycystic Kidney, Autosomal Dominant.
Browse Polycystic Kidney, Autosomal Dominant studies →Vertex Pharmaceuticals Incorporated is the lead sponsor of 243 studies on the registry; 19 are open to participants now.
Of its 102 completed or terminated interventional studies of FDA-regulated products, 49 (48%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
Participants will be randomized to receive a single dose of different dose levels of VX-407.
Drug: VX-407
Participants will be randomized to receive placebo matched to VX-407.
Drug: Placebo
Participants will be randomized to receive multiple doses of different dose levels of VX-407. The dose levels will be determined based on the data from Part A.
Drug: VX-407
Participants will be randomized to receive multiple doses of placebo matched to VX-407.
Drug: Placebo
Participants will be administered Midazolam (MDZ) in the presence or absence of VX-407. The dose levels will be determined based on the data from Part B.
Drug: VX-407 · Drug: Midazolam
Participants will be randomized to receive VX-407 in 1 of 3 treatment sequences with 3 dosing periods to assess the relative bioavailability of VX-407 formulations and the effect of food on the pharmacokinetics of VX-407.
Drug: VX-407
Solution or Suspension for oral administration.
Solution or Suspension for oral administration.
Syrup for oral administration.
Suspension or Tablets for oral administration.
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Enrollment up to Day 10
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Enrollment up to Day 23
Part A: Maximum Observed Plasma Concentration (Cmax) of VX-407
Time frame: From Day 1 up to Day 6
Part B: Maximum Observed Plasma Concentration (Cmax) of VX-407
Time frame: Days 1, 7, and 14
Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-407
Time frame: From Day 1 up to Day 6
Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-407
Time frame: Days 1, 7, and 14
Part C: Area Under the Concentration Versus Time Curve (AUC) of MDZ in Absence and Presence of VX-407
Time frame: On Day 1, and Day 15
Part C: Maximum Observed Plasma Concentration (Cmax) of MDZ in Absence and Presence of VX-407
Time frame: On Day 1, and Day 15
Part C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Enrollment up to Day 24
Part D: Maximum Observed Plasma Concentration (Cmax) of VX-407 Tablet Formulation (test) compared to a Suspension Formulation (reference) Under Fasted Conditions
Time frame: Days 1, 7, and 13
Part D: Area Under the Concentration Versus Time Curve From the Time of Dosing Extrapolated to Infinity (AUC0-inf) of VX-407 Under Fasted Conditions
Time frame: Days 1, 7, and 13
Part D: Maximum Observed Plasma Concentration (Cmax) of VX-407 Tablet Formulation Under Fed versus Fasted State
Time frame: Days 1, 7, and 13
Part D: Area Under the Concentration Versus Time Curve From the Time of Dosing Extrapolated to Infinity (AUC0-inf) of VX-407 Tablet Formulation Under Fed versus Fasted State
Time frame: Days 1, 7, and 13
Part D: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Enrollment up to Day 22
Plan to share: No — Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing
This study is completed, as verified in Jul 2025. You cannot join it, but the record below documents what was studied.
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Polycystic Kidney, Autosomal Dominant→
Vertex Pharmaceuticals Incorporated