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DRUG CLASS:

CFTR potentiator

3d
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
12d
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
14d
Trikafta for Patients With Non-cystic Fibrosis Bronchiectasis (clinicaltrials.gov)
P4, N=32, Completed, Emory University | Recruiting --> Completed
Trial completion
24d
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)
1m
New P1 trial
2ms
Repurposing of ivacaftor shows potential to treat ROR1 expressing high-grade serous ovarian cancer. (PubMed, Ther Adv Med Oncol)
Flow cytometry was used to assess apoptosis, DNA damage and cell proliferation following treatment with either 15 µM ivacaftor or 30 µM carboplatin at 24, 48 and 72 h. ROR1 signalling associated oncogenic pathways including the BMI-1 and the PI3K/AKT pathways were modulated following ivacaftor treatment. In summary, ivacaftor demonstrated significant anti-tumour potential in preclinical HGSOC models, supporting its further investigation as a repurposed therapy for ovarian cancer.
Journal
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ROR1 (Receptor Tyrosine Kinase Like Orphan Receptor 1) • BMI1 (BMI1 proto-oncogene, polycomb ring finger) • ANXA5 (Annexin A5)
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carboplatin
2ms
New trial
3ms
ACTIVMUCO: Physical Activity Profile and Sedentary Behaviour in Adults With Cystic Fibrosis (clinicaltrials.gov)
P=N/A, N=270, Recruiting, Hospices Civils de Lyon | Trial completion date: Mar 2026 --> Mar 2027 | Trial primary completion date: Sep 2025 --> Sep 2026
Trial completion date • Trial primary completion date
5ms
Trial completion
5ms
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older (clinicaltrials.gov)
P3, N=50, Active, not recruiting, Vertex Pharmaceuticals Incorporated | Enrolling by invitation --> Active, not recruiting
Enrollment closed