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Part A: Single Ascending Dose (SAD) for Polycystic Kidney Disease

Phase 1

Recruiting

Research Sponsored by Vertex Pharmaceuticals Incorporated

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

A total body weight of greater than 50 kg

Be between 18 and 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up days 1, 7, and 14

Awards & highlights

Study Summary

"This trial aims to test how safe and well-tolerated VX-407 is in healthy individuals, as well as how the body processes this drug."

Who is the study for?

This trial is for healthy individuals over 50 kg and with a BMI of 18.0 to 32.0 kg/m^2. Participants should be nonsmokers or have quit smoking at least three months prior to screening.Check my eligibility

What is being tested?

The study tests VX-407's safety, how well it's tolerated, and how the body processes it (pharmacokinetics). It involves comparing VX-407 against a placebo and Midazolam in healthy participants.See study design

What are the potential side effects?

Specific side effects are not listed but generally may include reactions at the site of administration, potential allergic responses, general discomforts like headaches or nausea, and any unexpected changes in health.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I weigh more than 50 kg.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ days 1, 7, and 14

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~days 1, 7, and 14

This trial's timeline: 3 weeks for screening, Varies for treatment, and days 1, 7, and 14 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Part C: Area Under the Concentration Versus Time Curve (AUC) of MDZ in Absence and Presence of VX-407

+1 more

Secondary outcome measures

Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-407

Part A: Maximum Observed Plasma Concentration (Cmax) of VX-407

Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-407

+2 more

Trial Design

5Treatment groups

Experimental Treatment

Placebo Group

Group I: Part C (Optional): Drug-Drug InteractionExperimental Treatment2 Interventions

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.

Group II: Part B: Multiple Ascending Dose (MAD)Experimental Treatment1 Intervention

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.

Group III: Part A: Single Ascending Dose (SAD)Experimental Treatment1 Intervention

Participants will be randomized to receive a single dose of different dose levels of VX-407.

Group IV: Part B: PlaceboPlacebo Group1 Intervention

Participants will be randomized to receive multiple doses of placebo matched to VX-407.

Group V: Part A: PlaceboPlacebo Group1 Intervention

Participants will be randomized to receive placebo matched to VX-407.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Midazolam

2018

Completed Phase 4

~1910

0 / 1Eligibility criteria met

Find a Location

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor

245 Previous Clinical Trials

32,445 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What level of risk does Part A: Single Ascending Dose (SAD) pose to individuals?

"Based on our assessment at Power, the safety rating for Part A: Single Ascending Dose (SAD) is 1. This score reflects that it is a Phase 1 trial with limited evidence supporting both safety and efficacy."

Answered by AI

What are the eligibility criteria for individuals to participate in this clinical study?

"Individuals eligible for participation must have a diagnosis of polycystic kidney disease and fall within the age range of 18 to 55 years. The study aims to recruit approximately 95 suitable candidates."

Answered by AI

Is there an age limit under 35 for individuals to participate in this medical study?

"Applicants aged between 18 and 55 years old are welcomed to join this study. It is noted that there are 49 trials tailored for individuals below the age of adulthood, while a substantial number of 497 studies cater to those over the age of 65."

Answered by AI

Are potential participants currently able to enroll in this ongoing investigation?

"As per clinicaltrials.gov, patient enrollment is currently ongoing for this investigation. The trial was initially published on April 1st, 2024 and most recently revised on April 25th, 2024."

Answered by AI

What is the upper limit for the number of participants enrolled in this medical research study?

"Affirmative. Information available on clinicaltrials.gov affirms that this medical investigation is presently in the process of seeking suitable participants. The study was initially made public on April 1st, 2024, and most recently revised on April 25th, 2024. For this research endeavor, a total of 95 individuals are sought from a single designated site."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics (PK) of VX-407 in Healthy Participants

2024. "A Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics (PK) of VX-407 in Healthy Participants". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06345755.

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