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Clinical trial

A Phase 3, Open-label, and Rollover Study to Evaluate the Long-term Safety and Tolerability of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

ID
Name
VX19-809-124
Description
This is a Phase 3, multicenter, open-label and roll-over study in subjects who are 12 to \<24 months of age at initiation of Lumacaftor/Ivacaftor (LUM/IVA) treatment.
Trial arms
Trial start
2020-02-24
Estimated PCD
2023-08-22
Trial end
2023-08-22
Status
Completed
Phase
Early phase I
Treatment
LUM/IVA
LUM/IVA granules for oral administration
Arms:
LUM/IVA
Other names:
lumacaftor/ivacaftor, VX-809/VX-770
Size
52
Primary endpoint
Safety and tolerability as assessed by the number of adverse events (AEs) and serious adverse events (SAEs)
Up to 120 weeks
Eligibility criteria
Key Inclusion Criteria: * Subjects From Study VX16-809-122 Part B (Study 122) * Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B * Subjects Not From Study 122 * Subjects will be 1 to less than 2 years of age * Homozygous for the F508del mutation (F/F) Key Exclusion Criteria: * Any clinically significant laboratory abnormalities that would interfere with the study assessments or pose an undue risk for the subject * Solid organ or hematological transplantation Other protocol defined Inclusion/Exclusion criteria may apply.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 52, 'type': 'ACTUAL'}}
Updated at
2023-09-21

1 organization

1 product

1 indication

Organization
Vertex Pharmaceuticals
Product
LUM/IVA
Indication
Cystic Fibrosis