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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.).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part A: Single Ascending Dose (SAD) | Experimental | Participants will be randomized to receive a single dose of different dose levels of VX-407. |
|
| Part A: Placebo | Placebo Comparator | Participants will be randomized to receive placebo matched to VX-407. |
|
| Part B: Multiple Ascending Dose (MAD) | Experimental | 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. |
|
| Part B: Placebo | Placebo Comparator | Participants will be randomized to receive multiple doses of placebo matched to VX-407. |
|
| Part C: Drug-Drug Interaction | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-407 | Drug | Solution or Suspension for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | 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) | From Enrollment up to Day 23 |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Maximum Observed Plasma Concentration (Cmax) of VX-407 | From Day 1 up to Day 6 | |
| Part B: Maximum Observed Plasma Concentration (Cmax) of VX-407 | Days 1, 7, and 14 | |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| ICON Lenexa | Lenexa | Kansas | 66219 | United States | ||
| Altasciences Montreal |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41348481 | Derived | Cortinovis M, Perico N, Remuzzi G. The Need for Novel Therapeutic Directions in Autosomal Dominant Polycystic Kidney Disease Patient Care. Clin J Am Soc Nephrol. 2025 Dec 5. doi: 10.2215/CJN.0000000975. Online ahead of print. |
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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
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| ID | Term |
|---|---|
| D016891 | Polycystic Kidney, Autosomal Dominant |
| ID | Term |
|---|---|
| D007690 | Polycystic Kidney Diseases |
| D052177 | Kidney Diseases, Cystic |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
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| ID | Term |
|---|---|
| D008874 | Midazolam |
| ID | Term |
|---|---|
| D001569 | Benzodiazepines |
| D001552 | Benzazepines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
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| Part D |
| Experimental |
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. |
|
| Placebo | Drug | Solution or Suspension for oral administration. |
|
| Midazolam | Drug | Syrup for oral administration. |
|
| VX-407 | Drug | Suspension or Tablets for oral administration. |
|
| Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-407 |
| From Day 1 up to Day 6 |
| Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-407 | Days 1, 7, and 14 |
| Part C: Area Under the Concentration Versus Time Curve (AUC) of MDZ in Absence and Presence of VX-407 | On Day 1, and Day 15 |
| Part C: Maximum Observed Plasma Concentration (Cmax) of MDZ in Absence and Presence of VX-407 | 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) | 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 | 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 | Days 1, 7, and 13 |
| Part D: Maximum Observed Plasma Concentration (Cmax) of VX-407 Tablet Formulation Under Fed versus Fasted State | 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 | 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) | From Enrollment up to Day 22 |
| Montreal |
| Canada |
| D052776 |
| Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000072661 | Ciliopathies |
| D030342 | Genetic Diseases, Inborn |
| D006571 | Heterocyclic Compounds |