Clinical Trials Logo
  1. Home
  2. Cystic Fibrosis
  3. NCT04702360
  4. Details
NCT04702360 Clinical Trial

ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation

Cystic Fibrosis Clinical Trial

Official title:
ELX/TEZ/IVA Expanded Access Program for Patients 6 Through 11 Years of Age With Cystic Fibrosis Who Have At Least One F508del Mutation

Clinical Trial Details — Status: Approved for marketing

Administrative data
NCT number NCT04702360
Other study ID # VX20-445-905
Secondary ID
Status Approved for marketing
Phase
First received
Last updated

Study information
Verified date July 2021
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/tezacaftor/ivacaftor (ELX/TEZ/IVA) to CF patients in critical need who are 6 to 11 years of age with at least one F508del 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 6 Years to 11 Years
Eligibility Key Inclusion Criteria: - Patients with confirmed diagnosis of CF who have an F/any genotype - Prior treatment for at least 6 months with an approved alternative CFTR modulator (if the patient is eligible for such treatment) - Patients with percent predicted forced expiratory volume in 1 second (ppFEV1) <40 for a minimum of 60 consecutive days before the date of completion of the request form Key Exclusion Criteria: - Patients with severe hepatic impairment 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
IVA monotablet 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 NCT06032273 - Lung Transplant READY CF 2: CARING CF Ancillary RCT N/A
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 NCT05392855 - Symptom Based Performance of Airway Clearance After Starting Highly Effective Modulators for Cystic Fibrosis (SPACE-CF) N/A
Recruiting NCT06088485 - The Effect of Bone Mineral Density in Patients With Adult Cystic Fibrosis
Recruiting NCT04056702 - Impact of Triple Combination CFTR Therapy on Sinus Disease.
Recruiting NCT04039087 - Sildenafil Exercise: Role of PDE5 Inhibition Phase 2/Phase 3
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