Autosomal Dominant Polycystic Kidney Disease (ADPKD)Phase 1NCT06345755
A Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants
A Phase 1 study testing VX-407 and Midazolam in people with Autosomal Dominant Polycystic Kidney Disease (ADPKD), run by Vertex Pharmaceuticals Incorporated at 2 sites in 2 countries.
Trial summary
The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetic parameters of VX-407 in healthy participants. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
- What is being tested?
- VX-407 and Midazolam
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 159 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 17 Apr 2024to 13 Jun 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (kg/m^2)
- A total body weight of greater than (>) 50 kg
- Nonsmoker or ex-smoker for at least 3 months before screening
Who cannot join
- History of febrile illness or other acute illness that has not fully resolved within 14 days before the first dose of study drug
- Any condition possibly affecting drug absorption
- Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: A Phase 1, Randomized, Double-blind, Placebo-controlled, First-in-human Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Single and Multiple Ascending Doses of VX-407 in Healthy Subjects
The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetic parameters of VX-407 in healthy participants.
Read the detailed description
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.).
- Being tested
- VX-407, Placebo, Midazolam
- Medical subject headings
- Polycystic Kidney, Autosomal Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-407Drug
Solution or Suspension for oral administration.
PlaceboDrug
Solution or Suspension for oral administration.
MidazolamDrug
Syrup for oral administration.
VX-407Drug
Suspension or Tablets for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| Part A: Single Ascending Dose (SAD) | Experimental | Participants will be randomized to receive a single dose of different dose levels of VX-407. Receives: Drug: VX-407 |
| Part A: Placebo | Placebo comparator | Participants will be randomized to receive placebo matched to VX-407. Receives: Drug: Placebo |
| 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. Receives: Drug: VX-407 |
| Part B: Placebo | Placebo comparator | Participants will be randomized to receive multiple doses of placebo matched to VX-407. Receives: Drug: Placebo |
| 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. Receives: Drug: VX-407; Drug: Midazolam |
| 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. Receives: Drug: VX-407 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: 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)
Measured: From Enrollment up to Day 23
Secondary outcomes (12)
Part A: Maximum Observed Plasma Concentration (Cmax) of VX-407
Measured: From Day 1 up to Day 6
Part B: Maximum Observed Plasma Concentration (Cmax) of VX-407
Measured: Days 1, 7, and 14
Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-407
Measured: From Day 1 up to Day 6
Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-407
Measured: Days 1, 7, and 14
Part C: Area Under the Concentration Versus Time Curve (AUC) of MDZ in Absence and Presence of VX-407
Measured: On Day 1, and Day 15
Part C: Maximum Observed Plasma Concentration (Cmax) of MDZ in Absence and Presence of VX-407
Measured: 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)
Measured: 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
Measured: 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
Measured: Days 1, 7, and 13
Part D: Maximum Observed Plasma Concentration (Cmax) of VX-407 Tablet Formulation Under Fed versus Fasted State
Measured: 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
Measured: 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)
Measured: From Enrollment up to Day 22
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2024
- Study start17 Apr 2024
- Primary completion27 Jan 2025
- Study completion13 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Autosomal Dominant Polycystic Kidney Disease (ADPKD)Studies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 159 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06345755
- Sponsor's protocol ID
- VX23-407-001
- Status last verified
- Jul 2025
- First submitted
- 28 Mar 2024
- Last updated
- 14 Jul 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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
Publications
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.
PubMed 41348481
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Double-blind, Placebo-controlled, First-in-human Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Single and Multiple Ascending Doses of VX-407 in Healthy Subjects
- ClinicalTrials.gov ID
- NCT06345755
- Acronym / sponsor ID
- VX23-407-001
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 159 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 17 Apr 2024 (actual)
- Primary completion
- 27 Jan 2025 (actual)
- Study completion
- 13 Jun 2025 (actual)
- First posted
- 3 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetic parameters of VX-407 in healthy participants.
Detailed description
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.).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Part A: Single Ascending Dose (SAD) | Experimental | Drug: VX-407Participants will be randomized to receive a single dose of different dose levels of VX-407. |
| Part A: Placebo | Placebo comparator | Drug: PlaceboParticipants will be randomized to receive placebo matched to VX-407. |
| Part B: Multiple Ascending Dose (MAD) | Experimental | Drug: VX-407Participants 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 | Drug: PlaceboParticipants will be randomized to receive multiple doses of placebo matched to VX-407. |
| Part C: Drug-Drug Interaction | Experimental | Drug: VX-407, Drug: MidazolamParticipants 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. |
| Part D | Experimental | Drug: VX-407Participants 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. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (kg/m^2)
- A total body weight of greater than (>) 50 kg
- Nonsmoker or ex-smoker for at least 3 months before screening
Key exclusion criteria (3 listed)
- History of febrile illness or other acute illness that has not fully resolved within 14 days before the first dose of study drug
- Any condition possibly affecting drug absorption
- Other protocol defined Inclusion/Exclusion criteria may apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VX-407Solution or Suspension for oral administration. | Drug | Part A: Single Ascending Dose (SAD), Part B: Multiple Ascending Dose (MAD), Part C: Drug-Drug Interaction |
| PlaceboSolution or Suspension for oral administration. | Drug | Part A: Placebo, Part B: Placebo |
| MidazolamSyrup for oral administration. | Drug | Part C: Drug-Drug Interaction |
| VX-407Suspension or Tablets for oral administration. | Drug | Part D |
Primary outcome
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
12 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2025.
- Derived from this study: PubMed 41348481Cortinovis 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.
- Individual participant dataSharing planned: 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VX-407 and Midazolam in people with Autosomal Dominant Polycystic Kidney Disease (ADPKD), run by Vertex Pharmaceuticals Incorporated at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06345755, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jul 2025.