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


Study First Submitted Date 2019-08-13
Study First Posted Date 2019-08-15
Last Update Posted Date 2019-11-21
Verification Month Year November 2019
Verification Date 2019-11-30
Last Update Posted Date 2019-11-21

Browse Interventions

Sequence: 96149479 Sequence: 96149480 Sequence: 96149481 Sequence: 96149482 Sequence: 96149483
Mesh Term Ivacaftor Mesh Term Elexacaftor Mesh Term Chloride Channel Agonists Mesh Term Membrane Transport Modulators Mesh Term Molecular Mechanisms of Pharmacological Action
Downcase Mesh Term ivacaftor Downcase Mesh Term elexacaftor Downcase Mesh Term chloride channel agonists Downcase Mesh Term membrane transport modulators Downcase Mesh Term molecular mechanisms of pharmacological action
Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor


Sequence: 52232209
Name Cystic Fibrosis
Downcase Name cystic fibrosis

Id Information

Sequence: 40203254
Id Source org_study_id
Id Value VX18-445-901


Sequence: 52545965 Sequence: 52545966
Intervention Type Drug Intervention Type Drug
Description Fixed-dose combination tablet for oral administration. Description 150-mg tablet for oral administration.

Browse Conditions

Sequence: 193719311 Sequence: 193719312 Sequence: 193719313 Sequence: 193719314 Sequence: 193719315 Sequence: 193719316 Sequence: 193719317 Sequence: 193719318 Sequence: 193719319
Mesh Term Cystic Fibrosis Mesh Term Fibrosis Mesh Term Pathologic Processes Mesh Term Pancreatic Diseases Mesh Term Digestive System Diseases Mesh Term Lung Diseases Mesh Term Respiratory Tract Diseases Mesh Term Genetic Diseases, Inborn Mesh Term Infant, Newborn, Diseases
Downcase Mesh Term cystic fibrosis Downcase Mesh Term fibrosis Downcase Mesh Term pathologic processes Downcase Mesh Term pancreatic diseases Downcase Mesh Term digestive system diseases Downcase Mesh Term lung diseases Downcase Mesh Term respiratory tract diseases Downcase Mesh Term genetic diseases, inborn Downcase Mesh Term infant, newborn, diseases
Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor


Sequence: 48376668
Agency Class INDUSTRY
Lead Or Collaborator lead
Name Vertex Pharmaceuticals Incorporated


Sequence: 30801102
Gender All
Minimum Age 12 Years
Maximum Age N/A
Criteria 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:

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

Other protocol defined Inclusion/Exclusion criteria may apply

Adult True
Child True
Older Adult True

Calculated Values

Sequence: 254016015
Registered In Calendar Year 2019
Were Results Reported False
Has Single Facility False
Minimum Age Num 12
Minimum Age Unit Years

Intervention Other Names

Sequence: 26700946 Sequence: 26700947 Sequence: 26700948 Sequence: 26700949
Intervention Id 52545965 Intervention Id 52545965 Intervention Id 52545966 Intervention Id 52545966
Name elexacaftor/tezacaftor/ivacaftor Name VX-445/VX-661/VX-770 Name ivacaftor Name VX-770

Responsible Parties

Sequence: 28913458
Responsible Party Type Sponsor