Clinical Trials Logo
  1. Home
  2. Cystic Fibrosis
  3. NCT04058210
  4. Details
NCT04058210 Clinical Trial

VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)

Cystic Fibrosis Clinical Trial

Official title:
VX-445/TEZ/IVA Triple Combination Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis Who Are Heterozygous for F508del and a Minimal Function CFTR Mutation

Clinical Trial Details — Status: Approved for marketing

Administrative data
NCT number NCT04058210
Other study ID # VX18-445-901
Secondary ID
Status Approved for marketing
Phase
First received
Last updated

Study information
Verified date November 2019
Source Vertex Pharmaceuticals Incorporated
Contact n/a
Is FDA regulated No
Health authority
Study type Expanded Access

Clinical Trial Summary

The purpose of this program is to provide elexacaftor(ELX, VX-445)/tezacaftor(TEZ)/ivacaftor(IVA) combination therapy to CF patients in critical need who are 12 years of age and older, heterozygous for F508del and a minimal function (MF) mutation in response to unsolicited physician requests.

Recruitment information / eligibility
Status Approved for marketing
Enrollment 0
Est. completion date
Est. primary completion date
Accepts healthy volunteers
Gender All
Age group 12 Years and older
Eligibility Inclusion Criteria:

1. Patients who have F/MF genotypes AND who meet at least 1 of the following criteria:

- The percent predicted forced expiratory volume in 1 second (ppFEV1) is <40 for a minimum of 2 months before the date of the request, OR

- Documentation of being active on a lung transplant waiting list or documentation of being evaluated for lung transplantation, but deemed unsuitable because of contraindications

Exclusion Criteria:

1. Patients with severe hepatic impairment (Child-Pugh Class C)

2. History of any other comorbidity that, in the opinion of the treating physician, might pose undue risk in administering ELX/TEZ/IVA to the patient

3. Pregnancy

Other protocol defined Inclusion/Exclusion criteria may apply

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• ELX/TEZ/IVA
Fixed-dose combination tablet for oral administration.
• IVA
150-mg tablet for oral administration.

Locations
Country Name City State
n/a

Sponsors (1)
Lead Sponsor Collaborator
Vertex Pharmaceuticals Incorporated
See also
  Status Clinical Trial Phase
Completed NCT04696198 - Thoracic Mobility in Cystic Fibrosis Care N/A
Completed NCT00803205 - Study of Ataluren (PTC124™) in Cystic Fibrosis Phase 3
Terminated NCT04921332 - Bright Light Therapy for Depression Symptoms in Adults With Cystic Fibrosis (CF) and COPD N/A
Completed NCT03601637 - Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del Phase 3
Terminated NCT02769637 - Effect of Acid Blockade on Microbiota and Inflammation in Cystic Fibrosis (CF)
Recruiting NCT06030206 - Lung Transplant READY CF 2: A Multi-site RCT N/A
Recruiting NCT06012084 - The Development and Evaluation of iCF-PWR for Healthy Siblings of Individuals With Cystic Fibrosis N/A
Recruiting NCT06032273 - Lung Transplant READY CF 2: CARING CF Ancillary RCT N/A
Recruiting NCT06088485 - The Effect of Bone Mineral Density in Patients With Adult Cystic Fibrosis
Recruiting NCT05392855 - Symptom Based Performance of Airway Clearance After Starting Highly Effective Modulators for Cystic Fibrosis (SPACE-CF) N/A
Recruiting NCT04039087 - Sildenafil Exercise: Role of PDE5 Inhibition Phase 2/Phase 3
Recruiting NCT04056702 - Impact of Triple Combination CFTR Therapy on Sinus Disease.
Completed NCT04058548 - Clinical Utility of the 1-minute Sit to Stand Test as a Measure of Submaximal Exercise Tolerance in Patients With Cystic Fibrosis During Acute Pulmonary Exacerbation N/A
Completed NCT04038710 - Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.
Completed NCT03637504 - Feasibility of a Mobile Medication Plan Application in CF Patient Care N/A
Recruiting NCT03506061 - Trikafta in Cystic Fibrosis Patients Phase 2
Completed NCT03566550 - Gut Imaging for Function & Transit in Cystic Fibrosis Study 1
Recruiting NCT04828382 - Prospective Study of Pregnancy in Women With Cystic Fibrosis
Completed NCT04568980 - Assessment of Contraceptive Safety and Effectiveness in Cystic Fibrosis
Recruiting NCT04010253 - Impact of Bronchial Drainage Technique by the Medical Device Simeox® on Respiratory Function and Symptoms in Adult Patients With Cystic Fibrosis N/A