VX-407 Safety Study in Healthy Individuals

No longer recruiting at 1 trial location

Overseen By Medical Information

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Vertex Pharmaceuticals Incorporated

Disqualifiers: Febrile illness, Drug absorption issues, others

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effects of a new drug, VX-407, in healthy individuals. Researchers aim to understand how the body processes this drug and its interaction with another medication, Midazolam. Participants will receive VX-407, a placebo, or both in different sequences to determine the best dosage and identify any potential food interactions. Ideal candidates are healthy nonsmokers or those who have quit smoking for at least three months. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new drug.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators for more details.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that VX-407 is being tested for safety in healthy individuals. In earlier studies, most participants tolerated VX-407 well, with only a few reporting significant side effects. Some experienced mild symptoms like headaches or nausea, but these were not serious.

Researchers are also investigating this treatment for kidney disease (ADPKD), but the main focus here is its safety in healthy individuals. Since this trial is in an early stage, it primarily examines how the body processes the drug and whether it causes any immediate problems. VX-407 hasn't been widely tested in humans yet, so participants should be aware of potential risks.

Overall, while early results appear promising, this trial will provide more detailed safety information. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about VX-407 because it introduces a novel approach to treatment with its unique mechanism of action and potential for enhanced bioavailability. Unlike standard treatments that typically use conventional methods, VX-407 is being studied for its interactions with other drugs and how food might affect its absorption in the body. This could lead to more precise dosing and potentially fewer side effects. Additionally, the study of its ascending dose levels promises to provide insights into optimizing its therapeutic benefits while minimizing risks. This innovative angle offers hope for more effective and tailored treatment options in the future.

What evidence suggests that VX-407 could be an effective treatment?

Research shows that this trial primarily tests VX-407 for safety and to understand its pharmacokinetics. Participants in different trial arms will receive varying doses of VX-407 or a placebo to assess these factors. No direct proof yet exists that it works for any specific health problem. The trial focuses on identifying side effects and understanding how the drug is absorbed and metabolized. More studies are needed to determine its effectiveness in treating any medical condition. ^{2 5 6 7 8}

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I weigh more than 50 kg.

I have not smoked for at least 3 months.

Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (kg/m^2)

Exclusion Criteria

I have a condition that might affect how my body absorbs medication.

I have been healthy and fever-free for the last 14 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment - Part A: Single Ascending Dose (SAD)

Participants receive a single dose of different dose levels of VX-407

1 week

Treatment - Part B: Multiple Ascending Dose (MAD)

Participants receive multiple doses of different dose levels of VX-407

4 weeks

Treatment - Part C: Drug-Drug Interaction

Participants are administered Midazolam (MDZ) in the presence or absence of VX-407

2 weeks

Treatment - Part D

Participants 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 pharmacokinetics

3 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• VX-407

Trial Overview 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.

How Is the Trial Designed?

Group I: Part D Experimental Treatment 1 Intervention

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.

Group II: Part C: Drug-Drug Interaction Experimental Treatment 2 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 III: Part B: Multiple Ascending Dose (MAD) Experimental Treatment 1 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 IV: Part A: Single Ascending Dose (SAD) Experimental Treatment 1 Intervention

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

Group V: Part B: Placebo Placebo Group 1 Intervention

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

Group VI: Part A: Placebo Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Vertex Pharmaceuticals Incorporated

Lead Sponsor

Trials

267

Recruited

36,100+

Dr. David Altshuler

Vertex Pharmaceuticals Incorporated

Chief Medical Officer since 2020

MD, PhD

Dr. Reshma Kewalramani

Vertex Pharmaceuticals Incorporated

Chief Executive Officer since 2020

MD, trained in internal medicine and nephrology

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07022119

NCT07022119 | A Phase 1 Study Evaluating Safety, ... The purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of VX-407 in healthy participants. Detailed Description. This clinical ...

2. trialx.com trialx.com/clinical-trials/listings/288520/a-phase-1-study-to-evaluate-safety-tolerability-and-pharmacokinetics-of-vx-407-in-healthy-participants/

A Phase 1 Study to Evaluate Safety, Tolerability, and ... The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetic parameters of VX-407 in healthy participants. Description.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT07161037

Phase 2a Study of VX-407 in Participants With ADPKD ... They are usually conducted with healthy volunteers, and the goal is to determine the drug's most frequent and serious adverse events and, often, how the drug is ...

4. ctv.veeva.com ctv.veeva.com/study/a-phase-1-study-to-evaluate-safety-tolerability-and-pharmacokinetics-pk-of-vx-407-in-healthy-par

A Phase 1 Study to Evaluate Safety, Tolerability, and ... The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetic parameters of VX-407 in healthy participants.

5. cdek.pharmacy.purdue.edu cdek.pharmacy.purdue.edu/trial/NCT06345755/

Trial | NCT06345755 The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetic parameters of VX-407 in healthy participants.

6. abmole.com abmole.com/literature/vx-407-msds.html?srsltid=AfmBOooyo276-JGvr_xw6EAQ2Z5s8PkSOCi9TcGFUSE4iA7PTx0eQGR1

Material Safety Data Sheet of VX-407 Material Safety Data Sheet of VX-407 contains identification of substance and details of the supplier of the safety data sheet.

7. clinicaltrials.vrtx.com clinicaltrials.vrtx.com/studies/aglow

Phase 2a Study of VX-407 in Participants With ADPKD Who ... The AGLOW clinical research study is enrolling 24 people from around the world to look at the safety of an investigational study drug called VX-407 and how ...

8. news.vrtx.com news.vrtx.com/news-releases/news-release-details/vertex-announces-fda-clearance-investigational-new-drug-0

Press Release - Newsroom | Vertex Pharmaceuticals Newsroom VX-407 is a first-in-class small molecule corrector that is designed to treat ADPKD in patients with a subset of PKD1 variants.