Cystic Fibrosis
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2020-08-07Clinical Trial Update: A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation [Phase 3]
A Phase 3 rollover safety study evaluating lumacaftor/ivacaftor in cystic fibrosis patients aged 2 years and older with the F508del-CFTR mut…
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CLINICAL_TRIALS trial_result · 2020-07-31Clinical Trial Update: A Single Arm 48-Week Follow-on Safety Study to a Core Study Comparing the Efficacy and Tolerability of Tobrineb®/Actitob®/Bramitob® Versus TOBI® [Phase 3]
A single-arm 48-week safety follow-up study assessing Tobrineb/Actitob/Bramitob against TOBI in cystic fibrosis patients; phase 3 completed.
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FDA approval · 2020-07-13FDA Approved: TOBI PODHALER — Supplemental Application (Viatris Specialty LLC)
FDA approves a supplemental application for TOBI PODHALER (tobramycin) from Viatris Specialty LLC. The approval updates the product’s labeli…
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CLINICAL_TRIALS trial_result · 2020-07-13Clinical Trial Update: Efficacy and Safety of Tigerase® vs. Pulmozyme® in Patients With Cystic Fibrosis [Phase 3]
Phase 3 trial comparing Tigerase® to Pulmozyme® in cystic fibrosis patients completed.
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CLINICAL_TRIALS trial_result · 2020-07-07Clinical Trial Update: Polyethylene Glycol and Intestinal Inflammation in Cystic Fibrosis [Phase 2]
Phase 2 clinical trial update on polyethylene glycol for intestinal inflammation in cystic fibrosis.
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CLINICAL_TRIALS trial_result · 2020-06-30Clinical Trial Update: Human Factors Study to Validate the User Interface of TOBI Podhaler Using Placebo Capsules [Phase 4]
Phase 4 human factors study assessing the user interface of TOBI Podhaler with placebo capsules; completed with results posted in 2020.
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EMA approval · 2020-06-26New medicine for cystic fibrosis patients
EMA announces a new medicine for cystic fibrosis patients, signaling an important approval or landmark development for CF treatment in Europ…
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CLINICAL_TRIALS trial_result · 2020-06-16Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR Mutation [Phase 3]
Phase 3 trial evaluating long-term safety and efficacy of VX-661 (Tezacafor? actually VX-661 is a CFTR corrector) with Ivacaftor in CF patie…
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CLINICAL_TRIALS trial_result · 2020-06-11Clinical Trial Update: Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis [Phase 2]
Phase 2 clinical trial update on ataluren (PTC124) for cystic fibrosis evaluating safety and efficacy.
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CLINICAL_TRIALS trial_result · 2020-06-02Clinical Trial Update: The Role of Vitamin D3 in Pediatric Bronchiectasis Severity [Phase 2, Phase 3]
Phase 2/3 clinical trial evaluating the role of Vitamin D3 in pediatric bronchiectasis severity, including cystic fibrosis and non-CF bronch…
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CLINICAL_TRIALS trial_result · 2020-05-20Clinical Trial Update: A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT)
A completed multicenter longitudinal study in cystic fibrosis evaluating CFTR-dependent disease profiling; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-05-19Clinical Trial Update: A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) [Phase 3]
Phase 3 study of VX-445 combination therapy in cystic fibrosis patients with specific mutations has completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-05-14Clinical Trial Update: Study of Ataluren (PTC124™) in Cystic Fibrosis [Phase 3]
Phase 3 trial update for ataluren (PTC124) in cystic fibrosis completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-05-14Clinical Trial Update: Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF) [Phase 3]
Phase 3 trial update for ataluren in nonsense mutation cystic fibrosis has completed with results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-04-24Clinical Trial Update: Blood Flow and Vascular Function in Cystic Fibrosis [Phase 2]
Phase 2 clinical trial assessing blood flow and vascular function in cystic fibrosis has completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-04-22Clinical Trial Update: Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis [Phase 1, Phase 2]
Clinical trial update for PTI-808 focusing on safety, tolerability, and pharmacokinetics in healthy volunteers and adults with cystic fibros…
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CLINICAL_TRIALS trial_result · 2020-04-15Clinical Trial Update: Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis [Phase 3]
Extension study of ataluren in nonsense mutation cystic fibrosis participants has terminated; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-04-08Clinical Trial Update: Functional Respiratory Imaging and Orkambi in CF [Phase 4]
Phase 4 trial update investigating Functional Respiratory Imaging and Orkambi in cystic fibrosis has been terminated.
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CLINICAL_TRIALS trial_result · 2020-04-06Clinical Trial Update: Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic Fibrosis [Phase 1, Phase 2]
Phase 1/2 study assessing safety, tolerability, and pharmacokinetics of PTI-808, PTI-801, and PTI-428 combination in cystic fibrosis patient…
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CLINICAL_TRIALS trial_result · 2020-03-27Clinical Trial Update: Pharmacokinetics of Linezolid in Children With Cystic Fibrosis [Phase 2]
Phase 2 trial update on the pharmacokinetics of linezolid in children with cystic fibrosis.
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CLINICAL_TRIALS trial_result · 2020-03-26Clinical Trial Update: PTC Study to Evaluate Ataluren in Combination With Ivacaftor [Phase 4]
A Phase 4 clinical trial evaluating ataluren in combination with ivacaftor for cystic fibrosis has been completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-03-25Clinical Trial Update: Study of Ataluren (PTC124) in Cystic Fibrosis [Phase 3]
Phase 3 study of ataluren (PTC124) in cystic fibrosis was terminated; results posted in 2020.
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FDA approval · 2020-03-20FDA Approved: ZENPEP — Supplemental Application (Aimmune Therapeutics, Inc.)
The FDA approved a supplemental application for ZENPEP (pancrelipase) submitted by Aimmune Therapeutics, updating the labeling related to it…
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FDA approval · 2020-03-20FDA Approved: VIOKACE — Supplemental Application (Aimmune Therapeutics, Inc.)
FDA approved a supplemental application for Viokace (pancrelipase) by Aimmune Therapeutics, expanding its labeling for exocrine pancreatic i…
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FDA approval · 2020-03-20FDA Approved: PERTZYE — Supplemental Application (Digestive Care, Inc.)
The FDA approved a supplemental application for PERTZYE (pancrelipase) from Digestive Care, Inc., updating its labeling under the supplement…