Orkambi® ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
299嚢胞性線維症3

299. 嚢胞性線維症


臨床試験数 : 1,696 薬物数 : 1,644 - (DrugBank : 272) / 標的遺伝子数 : 96 - 標的パスウェイ数 : 170
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
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PhaseCountries
1NCT03894657
(ClinicalTrials.gov)
December 18, 201912/2/2019Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®.Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. Pathway to Personalized Therapy in Cystic FibrosisCystic Fibrosis;Homozygous F508del MutationDiagnostic Test: Nasal brushing;Other: Sputum sample;Other: blood sample;Drug: OrkambiAssistance Publique - Hôpitaux de ParisNULLRecruiting5 YearsN/AAll104N/AFrance
2NCT03150719
(ClinicalTrials.gov)
May 24, 20173/5/2017A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)Phase 3b, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Efficacy, and Tolerability of Tezacaftor/Ivacaftor (TEZ/IVA) in an Orkambi-experienced Population Who Are Homozygous for the F508del CFTR MutationCystic FibrosisDrug: Tezacaftor/Ivacaftor;Drug: Ivacaftor;Drug: PlaceboVertex Pharmaceuticals IncorporatedNULLCompleted12 YearsN/AAll98Phase 3United States;France;Germany
3EUCTR2018-002624-16-FR
(EUCTR)
21/11/2018NA Validation of respiratory epithelial functional assessment to predict clinical efficacy of Orkambi®. Pathway to personalized therapy in Cystic Fibrosis PREDICT-CF - PREDICT-CF Homozygous F508del patient aged 12 years or older
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Orkambi®
Product Name: Orkambi®
ASSISTANC-PUBLIQUE-HOPITAUX DE PARIS (AP-HP)NULLNA Female: yes
Male: yes
104 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yesFrance