What side effects are associated with this type of intervention?

Common treatments for cystic fibrosis include CFTR modulators like Trikafta (elexacaftor/tezacaftor/ivacaftor), inhaled antibiotics such as tobramycin and aztreonam lysine, and oral antibiotics like trimethoprim-sulfamethoxazole. Known side effects of CFTR modulators include liver enzyme elevations, cataracts, and respiratory events. Inhaled tobramycin can cause bronchospasm, voice alteration, and tinnitus, while aztreonam lysine may lead to cough, nasal congestion, and wheezing. Oral antibiotics like trimethoprim-sulfamethoxazole can cause gastrointestinal upset, rash, and increased liver enzymes. These side effects are important considerations when evaluating the safety and tolerability of new treatments like VX-828.

What prior FDA approvals exist?

Several CFTR modulators have received FDA approval for the treatment of Cystic Fibrosis. Ivacaftor, a CFTR potentiator, was one of the first approved and has shown efficacy in improving lung function and other clinical outcomes. Tezacaftor-ivacaftor and elexacaftor-tezacaftor-ivacaftor are combination therapies that have also been approved, offering benefits for patients with different CFTR mutations. These treatments work by improving the function of the defective CFTR protein, similar to the pharmacokinetic focus of VX-828.

What other types of research exist?

Promising alternatives to VX-828 for cystic fibrosis treatment include the triple-combination therapies VX-659-tezacaftor-ivacaftor and VX-445-tezacaftor-ivacaftor. These combinations have shown significant improvements in CFTR processing, trafficking, and function, as well as clinical improvements in lung function (FEV1) in patients with one or two F508del alleles. Both combinations have demonstrated acceptable safety and side-effect profiles in clinical trials.

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VX-828 Safety Study

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 more than (>) 50 kg

Key

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial is studying a new medicine called VX-828 in healthy people to see if it is safe and how the body handles it.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Possible side effects are not detailed but may include reactions typical of clinical trials such as headaches, nausea, dizziness or allergic reactions due to the investigational nature of VX-828.

Eligibility Criteria

Inclusion Criteria

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

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I weigh more than 50 kg.

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It seems like the criterion is incomplete. Could you please provide more details or context so I can assist you better?

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

5Treatment groups

Experimental Treatment

Placebo Group

Group I: Part C: Drug Drug InteractionExperimental Treatment3 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 administration, and concomitant administration of VX-828 and Midazolam. Part C will be an open-label optional cohort.

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-828. 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-828.

Group IV: Part A: PlaceboPlacebo Group1 Intervention

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

Group V: Part B: PlaceboPlacebo Group1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Midazolam

2018

Completed Phase 4

~1910

Itraconazole

2017

Completed Phase 2

~830

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Cystic Fibrosis (CF) include CFTR modulators, antibiotics, and airway clearance therapies. CFTR modulators, such as ivacaftor, tezacaftor, and elexacaftor, enhance the function of the defective CFTR protein, improving salt and water balance in cells and reducing mucus thickness. Antibiotics target chronic pulmonary infections by eliminating bacteria trapped in the thick mucus. Airway clearance therapies, like inhaled hypertonic saline and DNase, help thin and remove mucus from the lungs. These treatments are vital for CF patients as they address the underlying cause of the disease, improve lung function, and decrease infection rates, significantly enhancing quality of life and longevity.

Targeting the Root Cause of Cystic Fibrosis.Cystic fibrosis and the use of pharmacogenomics to determine surrogate endpoints for drug discovery.Cellular heterogeneity of CFTR expression and function in the lung: implications for gene therapy of cystic fibrosis.