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

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Kinderspital Zuerich, Zürich, SwitzerlandCystic FibrosisVX-121/TEZ/D-IVA - Drug
Eligibility
1 - 11
All Sexes
What conditions do you have?
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Study Summary

This trial is to study VX-121/tezacaftor/deutivacaftor, a drug combo for treating cystic fibrosis, in patients with a certain mutation.

Eligible Conditions
  • Cystic Fibrosis

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Similar Trials

1
VX-121/TEZ/D-IVA
1
VX-522
1
Group B

Study Objectives

3 Primary · 19 Secondary · Reporting Duration: From Day 1 up to Day 50

Week 24
Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale
Week 24
Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
Part B: Absolute Change in Sweat Chloride (SwCl)
Part B: Number of CF-Related Hospitalizations
Part B: Number of Pulmonary Exacerbation (PEx)
Part B: Proportion of Participants With SwCl <30 mmol/L
Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L)
Week 24
Part B: Absolute Change in BMI-for-age Z-score
Part B: Absolute Change in Body Mass Index (BMI)
Part B: Absolute Change in Height
Part B: Absolute Change in Height-for-age Z-score
Part B: Absolute Change in Length
Part B: Absolute Change in Length-for-age Z-score
Part B: Absolute Change in Weight
Part B: Absolute Change in Weight-for-age Z-score
Part B: Absolute Change in Weight-for-length
Part B: Absolute Change in Weight-for-length Z-score
Day 22
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites
Day 50
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Week 16
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites
Week 28
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Similar Trials

1
VX-121/TEZ/D-IVA
1
VX-522
2
Group B

Trial Design

2 Treatment Groups

Part A: VX-121/TEZ/D-IVA
1 of 2
Part B: VX-121/TEZ/D-IVA
1 of 2

Experimental Treatment

210 Total Participants · 2 Treatment Groups

Primary Treatment: VX-121/TEZ/D-IVA · No Placebo Group · Phase 3

Part A: VX-121/TEZ/D-IVA
Drug
Experimental Group · 1 Intervention: VX-121/TEZ/D-IVA · Intervention Types: Drug
Part B: VX-121/TEZ/D-IVA
Drug
Experimental Group · 1 Intervention: VX-121/TEZ/D-IVA · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
VX-121/TEZ/D-IVA
2022
Completed Phase 1
~20

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: from day 1 up to day 50

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor
223 Previous Clinical Trials
30,541 Total Patients Enrolled
118 Trials studying Cystic Fibrosis
16,887 Patients Enrolled for Cystic Fibrosis

Eligibility Criteria

Age 1 - 11 · All Participants · 1 Total Inclusion Criteria

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References

Frequently Asked Questions

Are investigators still looking for participants for this research project?

"Yes, an inspection of clinicaltrials.gov discloses that this trial is currently looking for participants. This specific study was posted on June 21st, 2022 and updated as recently as August 30th, 8/30/2022. 210 individuals are needed across 8 locations." - Anonymous Online Contributor

Unverified Answer

Does this research project consider applicants who are adults?

"The age bracket for patients that this study can accept begins at 1 year old and ends at 10 years old." - Anonymous Online Contributor

Unverified Answer

Could you give me an estimate of how many individuals are involved in this research project?

"In order to start this clinical trial, 210 eligible patients must enroll. These individuals can be recruited from several locations, such as Boston Children's Hospital in Boston, Massachusetts and UPMC Children's Hospital of Pittsburgh in Pittsburgh, Pennsylvania." - Anonymous Online Contributor

Unverified Answer

How many different hospitals are participating in this clinical trial?

"To make things more convenient for participants, this clinical trial is taking place at 8 locations. Those who live near Boston, Pittsburgh or Aurora will have a shorter distance to travel. However, there are also 8 other sites located in different cities." - Anonymous Online Contributor

Unverified Answer

Who does this study aim to target as potential participants?

"The ideal candidate for this study would be somebody with cystic fibrosis who is between 1 and 11 years old. They should also have at least one TCR mutation in the CFTR gene." - Anonymous Online Contributor

Unverified Answer

Are there any reported dangers of VX-121/TEZ/D-IVA to human beings?

"VX-121/TEZ/D-IVA is a phase 3 trial drug, which means that while there is some evidence of efficacy, mostly there is data backing up its safety. Power rates it as a 3 on their safety scale." - 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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