VX-828 Safety Study

Recruiting at 9 trial locations

Overseen By Medical Information

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Vertex Pharmaceuticals Incorporated

Disqualifiers: Transplantation, Cirrhosis, Lung infection, others

Compensation: Varies

Number of Visits: Unknown

Duration: 8-12 weeks

255 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial evaluates the safety and effects of a new medication, VX-828, for people with cystic fibrosis (CF). Participants will receive VX-828 alone or with other drugs to assess how the body processes it and identify any side effects. The trial includes multiple groups, such as healthy participants and those with CF, to test different drug combinations. People with CF who have a confirmed diagnosis, experience breathing issues, and do not respond to certain existing treatments might be suitable candidates. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this new medication.

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 or your doctor.

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

Research has shown that VX-828 is currently being tested for safety in humans. As this is an early study, limited information is available. In these initial studies, researchers focus on assessing the treatment's tolerability for participants, closely monitoring for any side effects or problems.

During early testing, researchers continue to learn about the treatment's safety. They proceed cautiously, closely observing participants to quickly identify and address any issues. Although VX-828 is new, the team remains committed to understanding its effects on the body. Prospective participants should know that their safety is the top priority. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about VX-828 for cystic fibrosis because it offers a new approach by combining with other treatments like D-IVA and TEZ. Unlike most existing treatments that focus on symptom management, VX-828 targets the root cause at a cellular level, potentially improving lung function more effectively. This multi-drug combination might enhance the efficacy and safety profile by maximizing therapeutic benefits while minimizing side effects, making it a promising option for patients.

What evidence suggests that this trial's treatments could be effective for cystic fibrosis?

Research has shown that VX-828 is under investigation as a potential treatment for cystic fibrosis (CF). The trial includes various treatment arms where participants may receive VX-828 alone or with other drugs like D-IVA and TEZ. This treatment aims to correct the genetic defect causing CF, potentially improving lung function and overall health. Early results suggest that combining VX-828 with these drugs might enhance the body's ability to manage the disease. Initial studies have examined the drug's absorption and safety. Although further research is needed to confirm its effectiveness for CF patients, using a combination of drugs appears promising for addressing the disease's root causes. ^{2 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for healthy adults with a BMI of 18.0 to 32.0 kg/m^2 and weighing over 50 kg. Participants should be nonsmokers or ex-smokers who quit at least 3 months prior, confirmed by tests.

Inclusion Criteria

I weigh more than 50 kg.

You have not smoked for at least 3 months before the screening, and a test will be done to confirm that you are currently not smoking.

Your body mass index (BMI) is between 18.0 and 32.0.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive VX-828 in various combinations and dosages, including single ascending dose, multiple ascending dose, and drug-drug interaction studies

8-12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term in an open-label cohort

What Are the Treatments Tested in This Trial?

Interventions

• VX-828

Trial Overview The study is testing VX-828's safety, how well it's tolerated, and its pharmacokinetics (how the drug moves through the body). It involves comparing VX-828 with Midazolam (a control drug), Itraconazole (to check interactions), and a placebo.

How Is the Trial Designed?

Group I: Part E: VX-828 in Combination with D-IVA with or without TEZ in CF Experimental Treatment 3 Interventions

Participants with cystic fibrosis will receive VX-828 in combination with D-IVA with or without TEZ.

Group II: Part D: VX-828 in combination with TEZ/VX-118 ,TEZ/D-IVA or D-IVA Experimental Treatment 4 Interventions

Participants will be randomized to receive VX-828 in combination with TEZ/VX-118, TEZ/D-IVA, or D-IVA.

Group III: Part C: Drug Drug Interaction Experimental Treatment 6 Interventions

Participants will receive a single dose of VX-828, followed by a washout period, Itraconazole administration, and concomitant administration of itraconazole and VX-828; or participants will receive Midazolam followed by VX-828/TEZ/D-IVA administration, and concomitant administration of VX-828/TEZ/D-IVA and Midazolam. Part C will be an open-label optional cohort.

Group IV: Part B: Multiple Ascending Dose (MAD) Experimental Treatment 1 Intervention

Participants will be randomized to receive multiple doses of different dose levels of VX-828. The dose levels will be determined based on the data from Part A.

Group V: 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-828.

Group VI: Part D: Placebo Placebo Group 3 Interventions

Participants will be randomized to receive placebo matched to VX-828/TEZ/VX-118 or placebo matched to VX-828 in combination with TEZ/D-IVA or D-IVA

Group VII: Part A: Placebo Placebo Group 1 Intervention

Participants will be randomized to receive placebo matched to VX-828.

Group VIII: Part B: Placebo Placebo Group 1 Intervention

Participants will be randomized to receive placebo matched to VX-828.

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/NCT06154447

Evaluation of VX-828 in Healthy Participants and in ... The purpose of the study is to evaluate safety, tolerability, and pharmacokinetics of VX-828 and VX-828 in triple combination (TC) with Tezacaftor (TEZ)/ VX- ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06861413

A Phase 1 Study to Evaluate the Relative Bioavailability of ... The purpose of this study is to evaluate the relative bioavailability of a VX-828 tablet formulation compared to a suspension and evaluate its safety and ...

3. trial.medpath.com trial.medpath.com/drug/report/9376ba420d2c0850

VX-828 | Advanced Drug Monograph - MedPath Comprehensive analysis about VX-828, including its mechanisms, clinical uses, and therapeutic considerations.

4. theglobeandmail.com theglobeandmail.com/investing/markets/stocks/VRTX/pressreleases/35744807/vertex-pharmaceuticals-vx-828-study-a-new-hope-for-cystic-fibrosis-treatment/

Vertex Pharmaceuticals' VX-828 Study: A New Hope for ... The goal is to assess its effectiveness and safety in treating cystic fibrosis. The study design is interventional, with a randomized ...

5. cysticfibrosisjournal.com cysticfibrosisjournal.com/article/S1569-1993(25)01467-5/fulltext

LONGITUDE: An observational study of the long-term ... LONGITUDE is an observational, registry-based cohort study using data from the UK CF Registry to evaluate outcomes of ELX/TEZ/IVA in people aged ≥6 years.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06154447?cond=%22Cystic%20Fibrosis%22&intr=%22psychotropic%20drugs%22&viewType=Table&rank=2

Study Details | Evaluation of VX-828 in Healthy Participants ... Also called a data safety and monitoring board, or DSMB. Early Phase 1 (formerly listed as Phase 0). A phase of research used to describe exploratory trials ...

7. withpower.com withpower.com/trial/phase-1-cystic-fibrosis-11-2023-e9f58

VX-828 Safety Study This trial is for healthy adults with a BMI of 18.0 to 32.0 kg/m^2 and weighing over 50 kg. Participants should be nonsmokers or ex-smokers who quit at least 3 ...

8. trialx.com trialx.com/clinical-trials/listings/270885/a-phase-1-first-in-human-study-of-vx-828/

A Phase 1, First-in-human Study of VX-828 Overview. The purpose of the study is to evaluate safety, tolerability, and pharmacokinetics of VX-828 in healthy participants.

9. connect.careboxhealth.com connect.careboxhealth.com/en-US/trial/listing/472755

Evaluation of VX-828 in Healthy Participants and in ... The purpose of the study is to evaluate safety, tolerability, and pharmacokinetics of VX-828 and VX-828 in triple combination (TC) with Tezacaftor (TEZ)/ ...