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NCT02722057
A Study in US Cystic Fibrosis Patients With the R117H-CFTR Mutation to Confirm the Long-term Safety and Effectiveness of Kalydeco, Including Patients <18 Years of Age, Combining Data Captured in the Cystic Fibrosis Foundation Registry From an Interventional Cohort and a Non-Interventional Cohort
trial in Cystic Fibrosis in 368 participants. Completed in 1 December 2019.
1 December 2019
Quick facts
| Lead sponsor | Vertex Pharmaceuticals Incorporated |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 368 |
| Start date | 1 December 2015 |
| Primary completion | 1 December 2019 |
| Estimated completion | 1 December 2019 |
Conditions studied
- Cystic Fibrosis — all drugs for Cystic Fibrosis →
Sponsor
Vertex Pharmaceuticals Incorporated — full company profile →
Who can join
Eligibility, any sex, with Cystic Fibrosis. Patients with the condition only — healthy volunteers not accepted.
What's being measured
Primary outcomes are the specific endpoints the trial is designed to prove or disprove.
-
Lung function measurements (percent predicted forced expiratory volume in 1 second [FEV1] and forced vital capacity [FVC])
Time frame: 36 Months
Spirometry will be performed according to the standard procedure at each site, and FEV1 values as recorded in the registry will be evaluated. All descriptive and summary data collected for FEV1 will be repeated for FVC -
Pulmonary exacerbations, use of IV antibiotics
Time frame: 36 Months
Pulmonary exacerbation data will be collected as recorded in the registry. -
Nutritional parameters (body mass index [BMI], BMI-for-age z-score, weight, and weight-for-age z-score)
Time frame: 36 Months
Height and weight measurements as recorded in the registry will be evaluated. BMI, BMI-for-age z-score, and weight-for-age z-score will be derived -
Death or transplantation
Time frame: 36 months
Death will be collected from the registry database. -
Hospitalizations
Time frame: 36 Months
Hospitalizations will be collected from the registry database. -
Selected Complications (Symptomatic sinus disease, Pulmonary complications, CF-related diabetes (CFRD) and distal intestinal obstruction syndrome (DIOS), Hepatobiliary complications, Pancreatitis)
Time frame: 36 Months
Information for the above shown CF-related complications as recorded in the registry will be evaluated
Sponsor's own description
The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation \<18 years of age and to describe the long-term safety and effectiveness of Kalydeco in CF patients with the R117H-CFTR mutation overall and in patients ≥18 years. The long-term safety and effectiveness of Kalydeco will be examined in totality through the evaluation of the primary outcome measures.
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
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Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis.
Skilton M, Krishan A, Patel S, Sinha IP, et al · · 2019 · cited 18× · PMID 30616300 · DOI 10.1002/14651858.cd009841.pub3
Verify or expand the search:
- PubMed search for NCT02722057
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Cystic Fibrosis
Currently open trials in the same condition.
- NCT07437105 — Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants · Phase 1 · recruiting
- NCT07283770 — Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy Participants · Phase 1 · recruiting
- NCT07274631 — A Cohort for Inflammatory Respiratory Diseases: From Phenotyping to Personalised Medicine · recruiting
- NCT06810167 — Assessing Tenapanor as a Treatment of CF-related Constipation. · Phase 3 · recruiting
- NCT06962852 — A Long-term Study to Monitor the Health Status of People With Cystic Fibrosis Who Took Part in a Previous Study With BI · Phase 1, PHASE2 · active not recruiting
Other Vertex Pharmaceuticals Incorporated trials
Trials by the same sponsor.
- NCT05951205 — Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Ge · Phase 3 · withdrawn
- NCT07501702 — A Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia Gravis · Phase 2 · not yet recruiting
- NCT07437105 — Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants · Phase 1 · recruiting
- NCT07378865 — Evaluation of the Excretion of Suzetrigine Into Breast Milk in Healthy Lactating Female Participants · Phase 1 · recruiting
- NCT07349394 — Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants · Phase 1 · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT02722057 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Vertex Pharmaceuticals Incorporated
- Last refreshed: 4 February 2020
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02722057.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing