VX-121/TEZ/D-IVA for Cystic Fibrosis

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Hospital Sao Joao, Porto, Portugal
Cystic Fibrosis+1 More
VX-121/TEZ/D-IVA - Drug
Eligibility
Any Age
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug called VX-121 can improve lung function in people with cystic fibrosis.

See full description

Eligible Conditions

  • Cystic Fibrosis

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether VX-121/TEZ/D-IVA will improve 1 primary outcome and 3 secondary outcomes in patients with Cystic Fibrosis. Measurement will happen over the course of From Baseline Through Week 24.

Week 24
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24
Absolute Change From Baseline in Sweat Chloride (SwCl) Through Week 24
Proportion of Participants With SwCl <30 mmol/L Through Week 24 (Pooled With Data From Study VX20-121-103)
Proportion of Participants With SwCl <60 Millimole per Liter (mmol/L) Through Week 24 (Pooled With Data From Study VX20-121-103)

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

2 Treatment Groups

ELX/TEZ/IVA
1 of 2
VX-121/TEZ/D-IVA
1 of 2
Active Control
Experimental Treatment

This trial requires 400 total participants across 2 different treatment groups

This trial involves 2 different treatments. VX-121/TEZ/D-IVA is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

VX-121/TEZ/D-IVAParticipants will receive VX-121/TEZ/D-IVA in the morning.
ELX/TEZ/IVAParticipants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo (matched to ELX/TEZ/IVA)
2020
Completed Phase 3
~130
Placebo (matched to IVA)
2020
Completed Phase 3
~240

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: from baseline through week 24
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly from baseline through week 24 for reporting.

Closest Location

Kaiser Permanente - Oakland, CA

Eligibility Criteria

This trial is for patients born any sex of any age. There are 3 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
therapy Participants who have an FEV1 value of 40% to 90% of their predicted mean for their age, sex, and height and who are currently receiving ELX/TEZ/IVA therapy are considered to have a good lung function show original
Heterozygous for the F508del mutation and a minimal function mutation. show original
Key

Patient Q&A Section

What causes cystic fibrosis?

"A mutation in a cystic fibrosis transmembrane conductance regulator (CFTR) gene provides a reasonable explanation for about 70% of cases. Around 20% of patients with CF have a G551D mutation in the CFTR gene that confers a milder phenotype, and is almost entirely the cause of milder cases. In more severe cases there are mutations in other genes, notably the SLC26A4 gene. The gene may be involved in the development of the pancreas, sweat glands, parasympathetic nerves, sperm or pancreas. About 6% of cases have normal CFTR genes but are also carriers of a second mutated gene from both parents." - Anonymous Online Contributor

Unverified Answer

How many people get cystic fibrosis a year in the United States?

"In the United States, the approximate prevalence of cystic fibrosis in children, including infants, is one in 3,650, as diagnosed by a physician. It is not clear which populations are at risk for developing disease; however, a few subgroups are at increased risk. For instance, persons of Ashkenazi Jewish descent with a known history of cystic fibrosis are much more likely to be affected than persons of similarly descended non-Jewish ethnicity. This information can be utilized to identify high-risk populations, and may be valuable in establishing appropriate epidemiologic studies and, ultimately, in targeting interventions and prevention efforts. [provided by Ref." - Anonymous Online Contributor

Unverified Answer

What are the signs of cystic fibrosis?

"Signs of CF include problems with breathing, the digestive system, and the skin. These symptoms may appear as early as the newborn stage of life and may be as noticeable in children as young as 12 months.\n" - Anonymous Online Contributor

Unverified Answer

Can cystic fibrosis be cured?

"While current treatments for CF do help prolong survival, they do not eliminate the cause of the disease. In the future, research teams may develop more effective and less invasive treatments that could eventually help cure CF." - Anonymous Online Contributor

Unverified Answer

What are common treatments for cystic fibrosis?

"The aim of treatment of CF is to keep patients as healthy as possible and maintain lung function. Patients often require supplemental oxygen for their survival, and if left untreated, can develop irreversible pulmonary disease. The disease can be cured with a lung transplant but is limited by the number of available donors. Regular screening, early diagnosis, and treatment with CFTR modulators are key to minimize complications.\n\nPalliative therapy in oncology are available for patients that are in the late stages of their disease and include end-of-life interventions. Palliative therapy can be used to treat [chronic pain](https://www.withpower.com/clinical-trials/chronic-pain) and symptoms during end-of-life, particularly for patients with advanced cancer that have intractable pain." - Anonymous Online Contributor

Unverified Answer

What is cystic fibrosis?

"CF affects the entire body, causing irreversible consequences including chronic infection by pathogens and impaired nutrition uptake. The most severe manifestations include chronic obstructive pulmonary disease that can eventually lead to death. There are three types of CF, with different symptoms and outcome. The autosomal dominance transmission makes it easy for parents to pass the gene to their children, and almost any combination of those three genetic mutations causes CF. Cystic fibrosis is caused by a mutation of the CF transmembrane conductance regulator gene. It is the most prevalent genetic error underlying cystic fibrosis, and is a rare disease. Cystic fibrosis can be found with or without symptoms. In most cases, infants and children are asymptomatic." - Anonymous Online Contributor

Unverified Answer

Does vx-121/tez/d-iva improve quality of life for those with cystic fibrosis?

"Vx-121/TEZ/DIVA provides a significant number of patients with more favorable patient-rated QoL outcomes compared to placebo. Overall, patients treated with Vx-121/TEZ/DIVA had similar results to patients treated with IBN-025 on most QoL outcomes. Results from a recent paper support the potential therapeutic role of Vx-121/TEZ/DIVA in the clinical management of patients with cystic fibrosis." - Anonymous Online Contributor

Unverified Answer

Does cystic fibrosis run in families?

"There is no evidence from these three families in Australia for a genetic link between the parents. If such a link is actually present, it may be small and not evident. The finding that the parents have no CF-causing mutation in CF genes and only those people with mutations will have children and family members is consistent with the expectation and the current theories of disease." - Anonymous Online Contributor

Unverified Answer

How does vx-121/tez/d-iva work?

"A single iv injection of Vx-121 (100 ng/kg) leads to an increase in plasma sodium levels by about 2.5mEq/L, resulting in a decrease by about 0.8mEq/l in urine ionized chloride (p<0.001). The sodium levels of the treatment groups appear to return to baseline four hours thereafter. TSD (130 μg/d D-iva) produces no change in any of these parameters, but the placebo group showed a slight, small increase in urine chloride." - Anonymous Online Contributor

Unverified Answer

Has vx-121/tez/d-iva proven to be more effective than a placebo?

"Data from a recent study, VX-121/TEZ/IVA was shown to be effective in reducing severity and prolonging survival when used as part of multi-drug, high-dose, inhaled antibiotic treatment regimen for patients with bronchiectasis due to cystic fibrosis." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of cystic fibrosis?

"Some people (for example, parents, siblings and medical professionals) may assume that cystic fibrosis is a result of a particular environmental risk factor or a random genetic event. This assumption is not supported by current evidence since almost all people with CF have a common genetic cause. Although the underlying mechanisms are still not fully understood, it is likely that many genetic and environmental risk factors combine in a variety of ways to cause disease. At present it is not possible to tell in advance which environmental factors could cause CF.\n\nThe most likely cause of cystic fibrosis is a mutation in the CFTR gene which stops the production of the CFTR protein in the cells that line the lungs and pancreas." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of vx-121/tez/d-iva?

"In patients with cystic fibrosis, vx-121/tez/d-iva reduces the need for medications to control diarrhea by about 29-67%, compared with placebo and vx-121/tez/d-iva reduced the need for sputum exchange by about 12-45%. However, the improvement in overall care does not translate into significant improvements in clinical outcome. Vx-121/tez/d-iva was poorly well tolerated. Adverse effects were similar to those observed with placebo and included nausea, vomiting, diarrhea, diarrhea with fever, abdominal pain, constipation and anemia. Drug interactions were also not altered by vx-121/tez/d-iva." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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