Phase 3
Completed N=1,163
A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis
Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Source: ClinicalTrials.gov NCT01931839 ↗
Enrolled (actual)
1,163
Serious AEs
43.9%
Results posted
May 2017
Primary outcomePrimary: Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) — 331; 177; 333; 176 participants
◆ Published Evidence
Highly cited
285citations · ~32 / year
Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study.
Summary
The purpose of this study is to evaluate the efficacy and safety of long-term treatment with lumacaftor in combination with ivacaftor in people 12 years and older with Cystic Fibrosis.
Linked Publications (3)
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Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study.
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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
331; 177; 333; 176; 156; 77 | — |
| PRIMARY Part B Treatment Cohort: Number of Participants With Treatment-Emergent AEs and SAEs |
52; 57; 18; 21 | — |
| SECONDARY Part A Treatment Cohort: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) At Day 15, Week 8, 16, 24, 36, 48, 60 and 72 |
3; 2.8; 2.8; 3; 3.1; 2.8 | — |
| SECONDARY Part B Treatment Cohort: Absolute Change From Baseline in Percent Predicted FEV1 at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 |
-2; -3.4; -3.9; -1.8; -3.1; -2.8 | — |
| SECONDARY Part A Treatment Cohort: Relative Change From Baseline in Percent Predicted FEV1 at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 |
5.3; 5.1; 5.3; 4.8; 5.6; 5.6 | — |
| SECONDARY Part B Treatment Cohort: Relative Change From Baseline in Percent Predicted FEV1 at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 |
-2.2; -5.8; -5.2; -3.1; -4.2; -4.9 | — |
| SECONDARY Part A Treatment Cohort: Absolute Change From Baseline in Body Mass Index (BMI) at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 |
0.56; 0.06; 0.5; 0.1; 0.58; 0.14 | — |
| SECONDARY Part B Treatment Cohort: Absolute Change From Baseline in BMI at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 |
-0.05; -0.03; -0.01; 0.14; 0.08; 0.2 | — |
| SECONDARY Part A Treatment Cohort: Number of Pulmonary Exacerbations Events Per Patient-Year |
0.38; 0.42; 0.32; 0.37 | — |
| SECONDARY Part A Treatment Cohort: Absolute Change From Baseline in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain Score at Day 15, Week 8, 16, 24, 48 and 72 |
5.9; 2.3; 6.2; 3.5; 5; 4.1 | — |
| SECONDARY Part B Treatment Cohort: Absolute Change From Baseline in CFQ-R Respiratory Domain Score at Day 15, Week 8, 16, 24, 48 and 72 |
3.9; -4.1; 4.8; 1; 6.2; -0.2 | — |
| SECONDARY Part A Treatment Cohort: Absolute Change From Baseline in BMI Z-score at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 |
0.16; -0.01; 0.14; -0.02; 0.15; 0.04 | — |
| SECONDARY Part A Treatment Cohort: Absolute Change From Baseline in Body Weight at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 |
1.7; -0.1; 1.3; 0.1; 1.8; 0.2 | — |
| SECONDARY Part B Treatment Cohort: Absolute Change From Baseline in Body Weight at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 |
-0.1; -0.1; 0; 0.4; 0.3; 0.6 | — |
| SECONDARY Part A Treatment Cohort: Time-to-First Pulmonary Exacerbation |
364; 505; 481; 466 | — |
| SECONDARY Part A Treatment Cohort: Percentage of Participants With at Least 1 Pulmonary Exacerbation |
64.7; 53.6; 59.9; 55.7 | — |
| SECONDARY Part A Treatment Cohort: Percentage of Participants With Response Based on Relative Change in Percent Predicted FEV1 From Baseline |
39.4; 42.5; 40.9; 42.6; 25; 26.8 | — |
| SECONDARY Part B Treatment Cohort: Percentage of Participants With Response Based on Relative Change in Percent Predicted FEV1 From Baseline |
21; 12.7; 11.3; 19; 19.4; 15.9 | — |
| SECONDARY Part A Observation Cohort: Number of Participants With Serious Adverse Events (SAEs) |
7 | — |
Eligibility Criteria
Inclusion Criteria
- Signed informed consent form (ICF), and where appropriate, signed assent form.
- Participants entering the Part A Treatment Cohort: Completed 24 weeks of study drug treatment in Study 103 or Study 104 and elect to enroll in Part A treatment cohort.
- Participants entering the Part B Treatment Cohort: Completed 56 days of study drug treatment in Cohort 4 of Study 102 and elect to enroll in Part B treatment cohort.
- Participants entering the Part A Observational Cohort: Completed 24 weeks of study drug treatment in Study 103 or Study 104, but do not elect to enroll in the Part A Treatment Cohort or do not qualify to enroll in Part A treatment cohort.
- Willing to remain on a stable CF medication regimen through the end of study (Part A and Part B Treatment Cohorts only).
Exclusion Criteria
- Any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the participant (e.g., cirrhosis with portal hypertension).
- Pregnant and nursing females. Females of childbearing potential must have a negative pregnancy test at the Day 1 Visit.
- History of drug intolerance in the prior study that would pose an additional risk to the participant in the opinion of investigator or Vertex.
- History of poor compliance with study drug and/or procedures in the previous study as deemed by the investigator.
- Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor, or studies requiring blood collections with or without administration of study drug)
Data sourced from ClinicalTrials.gov (NCT01931839) and the linked publication. 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. Informational only — not medical advice.