{"type":"trials","record":{"id":56453,"source_id":"clinicaltrials-gov","external_id":"NCT01161537","title":"Study of the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis and the G551D Mutation","brief_summary":"Cystic Fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The encoded protein, CFTR, is an epithelial chloride ion channel responsible for aiding in the regulation of salt and water absorption and secretion in various tissues. Although the disease affects multiple organs, the leading cause of mortality is the progressive loss of lung function. Obstruction of airways with thick mucus, chronic bacterial infection of the airways, and inflammatory response are all thought to play a role in causing lung damage. Through its function as a chloride channel, CFTR is believed to be integral in epithelial ion and water transport and hence, maintaining the normal hydration of lung secretions. VX-770 (ivacaftor) is a potent and selective potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR. Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, VX-770 has been selected for clinical development as a possible treatment for patients with CF. Hyperpolarized noble gas magnetic resonance imaging (HG-MRI) is a promising new means of assessing lung function by direct imaging of certain non-radioactive isotopes of an inert noble gas, such as helium or xenon. Through this technique, high-resolution 3-dimensional images of lung ventilation can be obtained in both pediatric and adult patients during a single short breath-hold following inhalation of the gas. This is a 2-part study to evaluate the effect of VX-770 on hyperpolarized helium-3 magnetic resonance imaging (3He-MRI), and to evaluate the safety and efficacy of VX-770 in subjects aged 12 years and older with CF who have the G551D-CFTR mutation. Part A is a single-blind, placebo-controlled study that includes 4 weeks of VX-770 treatment and 4 weeks of placebo treatment. Part B is an open-label, 48 week study of long-term effect of VX 770 on hyperpolarized 3He-MRI.","overall_status":"Completed","phases":["PHASE2"],"study_type":"INTERVENTIONAL","sponsor":"Vertex Pharmaceuticals Incorporated","enrollment":13,"countries":["United States"],"start_date":"2010-10-01","completion_date":"2013-02-01","last_update_date":"2014-07-31","source_url":"https://clinicaltrials.gov/study/NCT01161537","editorial_summary":"A registered PHASE2 study indexed because it matched monitored longevity research terms. Registry status: Completed. Registration does not establish safety or effectiveness.","first_seen_at":"2026-09-25T02:16:44.048862+00:00","last_seen_at":"2026-09-25T06:15:20.281+00:00","metadata":{"sex":"ALL","acronym":null,"age_range":"12 Years","comparator":null,"organization":"Vertex Pharmaceuticals Incorporated","interventions":["VX-770","Placebo"],"registry_source":"ClinicalTrials.gov","outcome_measures":["Part A: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging…","Part B: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging…","Part A: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs — Part A: Day 1…","Part A: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Day 43 — Part A: Basel…","Part A: Absolute Change From Baseline in Sweat Chloride at Day 43 — Part A: Baseline (pre-dose Day 15), Day 43","Part A: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score At Day…","Part B: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs — Part B: Day 1…","Part B: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 48 — Part B: Base…","Part B: Absolute Change From Baseline in Sweat Chloride at Week 48 — Part B: Baseline (Day -1), Week 48","Part B: Absolute Change From Baseline in in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score At W…"],"design_description":"NON RANDOMIZED · SINGLE GROUP · TREATMENT · SINGLE","source_has_results":true},"controlled_terms":["Cystic Fibrosis","VX-770","Placebo"],"relevance_confidence":100,"source_quality_score":100,"freshness_score":35,"publication_state":"published","match_explanation":"Title contains controlled term: fibrosis.","quality_checked_at":"2026-09-25T06:15:30.843286+00:00","duplicate_cluster_key":"id:nct01161537","duplicate_of_id":null,"evidence_snapshot":{"status":"structured","version":1,"duration":"2010-10-01 to 2013-02-01","comparator":null,"confidence":"structured-source","population":"ALL · 12 Years","provenance":{"duration":"start_date and completion_date","comparator":"registry arm fields","population":"registry eligibility fields","intervention":"registry intervention fields","participants":"enrollment","study_design":"study_type and registry design fields","evidence_stage":"phases","reported_outcome":"not available","outcomes_measured":"registry outcome-measure fields"},"generated_at":"2026-09-25T06:15:20.411Z","intervention":["VX-770","Placebo"],"participants":13,"study_design":"NON RANDOMIZED · SINGLE GROUP · TREATMENT · SINGLE","subject_scope":"Human clinical study registration","evidence_stage":"PHASE2","safety_context":"Eligibility, adverse-event details, and clinical decisions must be checked in the official registry and with qualified clinicians.","source_support":"Structured registry protocol metadata; no finding-level conclusion is generated.","main_limitation":"This is a study registration. No reusable structured result is available here, so it cannot show whether the intervention worked or was safe.","reported_outcome":null,"outcomes_measured":["Part A: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging…","Part B: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging…","Part A: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs — Part A: Day 1…","Part A: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Day 43 — Part A: Basel…","Part A: Absolute Change From Baseline in Sweat Chloride at Day 43 — Part A: Baseline (pre-dose Day 15), Day 43","Part A: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score At Day…","Part B: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs — Part B: Day 1…","Part B: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 48 — Part B: Base…","Part B: Absolute Change From Baseline in Sweat Chloride at Week 48 — Part B: Baseline (Day -1), Week 48","Part B: Absolute Change From Baseline in in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score At W…"],"regulatory_context":"Trial registration is not regulatory approval and does not establish that an intervention is available."},"clinical_trial_topics":[{"topic_slug":"extracellular-matrix-aging","is_published":true,"match_reasons":["Title contains controlled term: fibrosis.","Abstract contains controlled term: fibrosis.","Source terminology contains: fibrosis.","Title supplies longevity context: aging.","Study type is explicitly identified as INTERVENTIONAL."],"matched_fields":["title","abstract","controlled terminology","title context","study type"],"relevance_score":100,"intelligence_topics":{"name":"Extracellular matrix ageing","slug":"extracellular-matrix-aging"}}],"content_sources":{"name":"ClinicalTrials.gov","homepage_url":"https://clinicaltrials.gov/"}},"canonical_url":"https://www.immortal.life/trials/56453","automation_disclosure":"Generated automatically from cited source metadata. No scientist, clinician, researcher, editor, or human reviewer evaluates this publication before release."}