Phase 3
N=69
A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)
Cystic Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT04599465 ↗Enrolled (actual)
69
Serious AEs
8.7%
Results posted
Aug 2023
Primary outcome: Primary: Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 48 — -35.0 milligrams per deciliter (mg/dl)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- ELX/TEZ/IVA (Drug); IVA (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Primary completion
- Jul 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 48 |
-35.0 | — |
| SECONDARY Percentage of Participants With Improvement in Dysglycemia Categorization at Week 48 |
37.7 | — |
| SECONDARY Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
67; 6 | — |
Summary
This study was evaluate the effect of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) on glucose tolerance in CF participants, 12 years of age and older who are heterozygous for the F508del mutation and a minimal function mutation (F/MF genotypes), with abnormal glucose metabolism.
Eligibility Criteria
Key Inclusion Criteria
- Heterozygous for F508del and an MF mutation (F/MF genotypes)
- Forced expiratory volume in 1 second (FEV1) value ≥ 30% of predicted mean for age, sex, and height
- Abnormal glucose tolerance determined by an OGTT as either:
- Impaired glucose tolerance (IGT) defined as 2 hour post OGTT blood glucose level ≥140 to <200 mg/dL (≥7.77 to <11.10 mmol/L) and fasting blood glucose level <126 mg/dL (<7.00 mmol/L)
- CF-related diabetes (CFRD) defined as either fasting hyperglycemia (blood glucose level ≥126 mg/dL [≥7.00 mmol/L] after an 8-hour fast) or 2-hour post OGTT blood glucose level ≥200 mg/dL (≥11.10 mmol/L)
Key Exclusion Criteria
- Clinically significant liver cirrhosis with or without portal hypertension
- Solid organ or hematological transplantation
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Type 1 or Type 2 diabetes
- Duration of CFRD ≥5 years
Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT04599465). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.