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    DRUG CLASS:

    CFTR potentiator

    9d
    Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants (clinicaltrials.gov)
    P1, N=18, Completed, Vertex Pharmaceuticals Incorporated | Active, not recruiting --> Completed
    Trial completion
    1m
    Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants (clinicaltrials.gov)
    P1, N=18, Active, not recruiting, Vertex Pharmaceuticals Incorporated | Recruiting --> Active, not recruiting
    Enrollment closed
    1m
    Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older (clinicaltrials.gov)
    P3, N=25, Enrolling by invitation, Vertex Pharmaceuticals Incorporated | N=14 --> 25
    Enrollment change
    1m
    Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older (clinicaltrials.gov)
    P3, N=71, Completed, Vertex Pharmaceuticals Incorporated | Active, not recruiting --> Completed | Trial completion date: Apr 2026 --> Jan 2026 | Trial primary completion date: Apr 2026 --> Jan 2026
    Trial completion • Trial completion date • Trial primary completion date
    1m
    TIPS: Immunomodulatory Treatment of Interstitial Lung Disease Associated With Surfactant Related Gene Variants (clinicaltrials.gov)
    P2, N=30, Not yet recruiting, Assistance Publique - Hôpitaux de Paris
    New P2 trial
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    ABCA3 (ATP Binding Cassette Subfamily A Member 3) • SFTPA1 (Surfactant Protein A1)
    |
    prednisone • hydroxychloroquine
    1m
    Drugs Associated with Pediatric Cataracts: A Real-World Pharmacovigilance Study. (PubMed, Children (Basel))
    Difluprednate showed the highest pharmacovigilance signal (ROR: 963.67; 95% CI: 316.27-2936.31; n = 4). Notably, CFTR modulators exhibited striking signals: ivacaftor (ROR: 30.75; 95% CI: 18.06-52.37; n = 14), elexacaftor-ivacaftor-tezacaftor (ROR: 15.58; 95% CI: 9.86-24.63; n = 19), and ivacaftor-lumacaftor (ROR: 13.2; 95% CI: 7.9-22.07; n = 15). This study provides a comprehensive large-scale pharmacovigilance profile of drug-induced pediatric cataracts, identifying agents with high-risk pharmacovigilance signals and underscoring the need for proactive ocular monitoring. These findings can inform clinical decision making and prevention strategies and guide future mechanistic research.
    Journal • Adverse events • Real-world evidence
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    CFTR (CF Transmembrane Conductance Regulator)
    2ms
    Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators (clinicaltrials.gov)
    P=N/A, N=500, Recruiting, Charite University, Berlin, Germany | N=200 --> 500
    Enrollment change
    2ms
    IMPROVED: Population Pharmacokinetics of Elexacaftor-tezacaftor-ivacaftor in a Paediatric Population (clinicaltrials.gov)
    P=N/A, N=150, Recruiting, Hospices Civils de Lyon | Not yet recruiting --> Recruiting | Trial completion date: Feb 2027 --> Aug 2027 | Trial primary completion date: Dec 2026 --> Aug 2027
    Enrollment open • Trial completion date • Trial primary completion date
    2ms
    PKCAFTORs: A low-intervention prospective-retrospective study to evaluate the pharmacokinetics of elexacaftor/tezacaftor/ivacaftor combination in a Cystic Fibrosis population (2024-518785-28-00)
    P4, N=50, Not yet recruiting, IRCCS Istituto Giannina Gaslini
    New P4 trial
    2ms
    Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants (clinicaltrials.gov)
    P1, N=18, Recruiting, Vertex Pharmaceuticals Incorporated | Not yet recruiting --> Recruiting
    Enrollment open
    2ms
    Trikafta for Patients With Non-cystic Fibrosis Bronchiectasis (clinicaltrials.gov)
    P4, N=32, Completed, Emory University | Recruiting --> Completed
    Trial completion
    2ms
    Evaluation of the Need for Comprehensive Care for Patients with Cystic Fibrosis. (PubMed, J Am Board Fam Med)
    There is a gap in healthcare for pwCF, especially for those who do not routinely interact with a PCP. CF clinicians may not be up to date on primary care management and PCPs may not be comfortable with treating patients with a high acuity chronic condition. PCPs should be better integrated into the CF care team to ensure pwCF are receiving comprehensive care.
    Journal
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    CFTR (CF Transmembrane Conductance Regulator)