Last reviewed · How we verify
A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Participants With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
Phase 3 trial testing IVA in Cystic Fibrosis in 86 participants. Completed in 2 October 2023.
| Lead sponsor | Vertex Pharmaceuticals Incorporated |
|---|---|
| Phase | Phase 3 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | parallel |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 86 |
| Start date | 16 August 2017 |
| Primary completion | 2 October 2023 |
| Estimated completion | 2 October 2023 |
| Sites | 29 locations across Ireland, United Kingdom, Germany, Canada, Australia, United States |
Vertex Pharmaceuticals Incorporated — full company profile →
Adults 0 Months to 24 Months, any sex, with Cystic Fibrosis. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
| Group | Value | 95% CI |
|---|---|---|
| Ivacaftor Treatment | 85 |
| Group | Value | 95% CI |
|---|---|---|
| Ivacaftor Treatment | 21 |
Sweat samples were collected using an approved collection device.
| Group | Value | 95% CI |
|---|---|---|
| Ivacaftor Treatment | -55.3 | ± 25.0 |
Time frame: Day 1 up to Week 120. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Ivacaftor Arm |
|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | — |
| Constipation | Gastrointestinal disorders | — |
| Pseudomonas test positive | Investigations | — |
| Adrenocortical insufficiency acute | Endocrine disorders | — |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | — |
| Pyrexia | General disorders | — |
| Bronchiolitis | Infections and infestations | — |
| Bronchitis | Infections and infestations | — |
| Gastroenteritis | Infections and infestations | — |
| Gastroenteritis viral | Infections and infestations | — |
| Lower respiratory tract infection viral | Infections and infestations | — |
| Parainfluenzae virus infection | Infections and infestations | — |
| Periorbital cellulitis | Infections and infestations | — |
| Respiratory syncytial virus infection | Infections and infestations | — |
| Rhinovirus infection | Infections and infestations | — |
| Viral infection | Infections and infestations | — |
| Viral rash | Infections and infestations | — |
| Viral upper respiratory tract infection | Infections and infestations | — |
| Electrocardiogram QT shortened | Investigations | — |
| Electroencephalogram abnormal | Investigations | — |
| Dehydration | Metabolism and nutrition disorders | — |
| Malnutrition | Metabolism and nutrition disorders | — |
| Device dislocation | Product Issues | — |
| Reaction | System | Ivacaftor Arm |
|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | — |
| Pyrexia | General disorders | — |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | — |
| Upper respiratory tract infection | Infections and infestations | — |
| Vomiting | Gastrointestinal disorders | — |
| Rash | Skin and subcutaneous tissue disorders | — |
| Ear infection | Infections and infestations | — |
| Diarrhoea | Gastrointestinal disorders | — |
| Constipation | Gastrointestinal disorders | — |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | — |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | — |
| Rhinitis | Infections and infestations | — |
| Nasopharyngitis | Infections and infestations | — |
| Otitis media | Infections and infestations | — |
| Conjunctivitis | Infections and infestations | — |
| Gastroenteritis | Infections and infestations | — |
| Pseudomonas test positive | Investigations | — |
| Influenza | Infections and infestations | — |
| Teething | Gastrointestinal disorders | — |
| Alanine aminotransferase increased | Investigations | — |
| Seasonal allergy | Immune system disorders | — |
| Sinusitis | Infections and infestations | — |
| Tonsillitis | Infections and infestations | — |
| Viral upper respiratory tract infection | Infections and infestations | — |
| Gamma-glutamyltransferase increased | Investigations | — |
| Haemophilus test positive | Investigations | — |
| Respiratory tract infection viral | Infections and infestations | — |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | — |
Most-reported serious reactions: Infective pulmonary exacerbation of cystic fibrosis, Constipation, Pseudomonas test positive, Adrenocortical insufficiency acute, Distal intestinal obstruction syndrome, Pyrexia, Bronchiolitis, Bronchitis.
Data from ClinicalTrials.gov NCT03277196 adverse events section.
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in participants with cystic fibrosis (CF) who are \<24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation.
3 peer-reviewed publications reference this trial (live from Europe PMC):
Verify or expand the search:
Trials testing the same drug.
Currently open trials in the same condition.
Trials by the same sponsor.
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/NCT03277196.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing