Trial reportPediatric diabetes2013
Insulin secretion improves in cystic fibrosis following ivacaftor correction of CFTR: a small pilot study.
Trial report in Pediatric diabetes, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 93 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Study of the Effect of Combination Lumacaftor and Ivacaftor on Glucose Tolerance in Persons With Cystic Fibrosis Who Are Homozygous for the Phe508del Cystic Fibrosis Transmembrane Conductance Regulator Mutation.
A Study of the Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia in Persons With Cystic Fibrosis
Who cites it
93 citing papers in PubMed, 1 synthesis or guideline pooled it, 189 citations in OpenAlex.
- Effect of CFTR modulators on glucose homeostasis in children and young adults with cystic fibrosis-related diabetes: a systematic review.Frontiers in endocrinology · 2025Pooled it
- Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor.Digestive diseases and sciences · 2016Trial
- Impact of Genetics on Endocrine Manifestations of Cystic Fibrosis.Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists · 2026Review
- The evolving understanding of cystic fibrosis-related diabetes in the highly effective modulator therapy era: a scoping review.Diabetologia · 2026Article
- From Pathophysiology to Treatment: Contemporary Approaches to CFRD in the Pediatric and Adolescent Population.Pediatric diabetes · 2026Review
- A genetics-guided integrative framework for drug repurposing: identifying antihypertensive drugs for type 2 diabetes.NPJ drug discovery · 2026Article
- Beta cell function and insulin sensitivity during elexacaftor/tezacaftor/ivacaftor therapy in people with cystic fibrosis.Frontiers in endocrinology · 2026Article
- Hitting the Sweet Spot: The Impact of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance in Cystic Fibrosis.American journal of respiratory and critical care medicine · 2025Article
- Glycemia and Insulin Secretion in Cystic Fibrosis 2 Years After Elexacaftor/Tezacaftor/Ivacaftor: PROMISE-ENDO.The Journal of clinical endocrinology and metabolism · 2025Observational
- Cystic fibrosis-related diabetes in the era of modern treatment using CFTR modulators in pediatric patients-a systematic review.Frontiers in pediatrics · 2025Review
- Preservation of β-cell Function in Pancreatic Insufficient Cystic Fibrosis With Highly Effective CFTR Modulator Therapy.The Journal of clinical endocrinology and metabolism · 2023Article
- An Update in Cystic Fibrosis-Related Diabetes in Children and Adolescents.Children (Basel, Switzerland) · 2023Review
- Article
- A Gastroenterologist's Guide to Care Transitions in Cystic Fibrosis from Pediatrics to Adult Care.International journal of molecular sciences · 2023Review
- Delivering a New Future for People With Cystic Fibrosis.Pediatrics · 2023Article
- Combined CFTR modulator therapies are linked with anabolic benefits and insulin-sparing in cystic fibrosis-related diabetes.Journal of clinical & translational endocrinology · 2023Article
- Hypoglycemia in children and young adults with cystic fibrosis during oral glucose tolerance testing vs. continuous glucose monitoring.Pediatric pulmonology · 2023Article
- Cystic Fibrosis-Related Diabetes Workshop: Research Priorities Spanning Disease Pathophysiology, Diagnosis, and Outcomes.Diabetes care · 2023Review
- Cystic Fibrosis-Related Diabetes Workshop: Research Priorities Spanning Disease Pathophysiology, Diagnosis, and Outcomes.Diabetes · 2023Review
- Nutritional Care in Children with Cystic Fibrosis.Nutrients · 2023Review
33 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 1 institution in 1 country.
Funding
Abstract
objectiveTo determine whether the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) is involved in human insulin secretion by assessing the metabolic impact of the new CFTR corrector-ivacaftor.
methodsThis open-label pilot study was conducted in CF patients with the G551D mutation given new prescriptions for ivacaftor. At baseline and 4 wk after daily ivacaftor therapy, intravenous glucose tolerance tests (IVGTT) and oral glucose tolerance tests (OGTT) were performed.
resultsFive patients aged 6-52 were studied. After 1 month on ivacaftor, the insulin response to oral glucose improved by 66-178% in all subjects except one with long-standing diabetes. OGTT glucose levels were not lower in the two individuals with diabetes or the two with normal glucose tolerance (NGT), but the glucose tolerance category in the subject with impaired glucose tolerance (IGT) improved to NGT after treatment. In response to intravenous glucose, the only patient whose acute insulin secretion did not improve had newly diagnosed, untreated CFRD. The others improved by 51-346%. Acute insulin secretion was partially restored in two subjects with no measurable acute insulin response at baseline, including the one with IGT and the one with long-standing diabetes.
conclusionsThis small pilot study suggests there is a direct role of CFTR in human insulin secretion. Larger, long-term longitudinal studies are necessary to determine whether early initiation of CFTR correction, particularly in young children with CF who have not yet lost considerable β-cell mass, will delay or prevent development of diabetes in this high-risk population.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.