To the editor: Response to ?use of nasal potential differences and sweat chloride as outcome measures in multicenter clinical trials in subjects with cystic fibrosis?

2002 ◽  
Vol 34 (2) ◽  
pp. 155-155
Author(s):  
Vicky A. LeGrys
Keyword(s):  
Cystic Fibrosis ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Sweat Chloride ◽  
Multicenter Clinical Trials

Use of nasal potential difference and sweat chloride as outcome measures in multicenter clinical trials in subjects with cystic fibrosis

2002 ◽  
Vol 33 (2) ◽  
pp. 142-150 ◽  
Author(s):  
Richard C. Ahrens ◽  
Thomas A. Standaert ◽  
Janice Launspach ◽  
Seung-Ho Han ◽  
Mary E. Teresi ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Potential Difference ◽  
Sweat Chloride ◽  
Nasal Potential Difference ◽  
Multicenter Clinical Trials

scholarly journals Elexacaftor-Tezacaftor-Ivacaftor: The First Triple-Combination Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapy

2020 ◽  
Vol 25 (3) ◽  
pp. 192-197 ◽  
Author(s):  
Kaden Ridley ◽  
Michelle Condren
Keyword(s):  
Cystic Fibrosis ◽  
Clinical Trials ◽  
Genetic Mutations ◽  
Transmembrane Conductance Regulator ◽  
Triple Combination ◽  
Transmembrane Conductance ◽  
Sweat Chloride ◽  
Cystic Fibrosis Transmembrane

Elexacaftor-tezacaftor-ivacaftor is a newly approved triple-combination cystic fibrosis transmembrane conductance regulator (CFTR) modulating therapy that contains 2 correctors and a potentiator of the CFTR channel. Its labeled indication for use is for persons 12 years of age and older with at least 1 F508del mutation for the CFTR gene. This drug combination provides potential therapy to many patients who had previously been excluded from CFTR modulation therapy due to the nature of their genetic mutations. The efficacy demonstrated in clinical trials surpasses the currently available therapies related to lung function, quality of life, sweat chloride reduction, and reducing exacerbations. The most common adverse events seen in clinical trials included rash and headache, and laboratory monitoring is recommended to evaluate liver function. Continued evaluation of patient data is needed to confirm its long-term safety and efficacy. Elexacaftor-tezacaftor-ivacaftor is a monumental and encouraging therapy for cystic fibrosis; however, approximately 10% of the CF population are not candidates for this or any other CFTR modulation therapy.

Change in Sweat Chloride as a Clinical End Point in Cystic Fibrosis Clinical Trials

CHEST Journal ◽  
2013 ◽  
Vol 143 (1) ◽  
pp. 14-18 ◽  
Author(s):  
Anthony G. Durmowicz ◽  
Kimberly A. Witzmann ◽  
Curtis J. Rosebraugh ◽  
Badrul A. Chowdhury
Keyword(s):  
Cystic Fibrosis ◽  
Clinical Trials ◽  
End Point ◽  
Sweat Chloride

The Use of the OMERACT Ultrasound Tenosynovitis Scoring System in Multicenter Clinical Trials

2017 ◽  
Vol 45 (2) ◽  
pp. 165-169 ◽  
Author(s):  
Mads Ammitzbøll-Danielsen ◽  
Mikkel Østergaard ◽  
Esperanza Naredo ◽  
Annamaria Iagnocco ◽  
Ingrid Möller ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Scoring System ◽  
Sensitivity To Change ◽  
Treatment Intensification ◽  
Standardized Response Mean ◽  
Multicenter Trials ◽  
High Responsiveness ◽  
Multicenter Clinical Trials

Objective.To test the sensitivity to change of the Outcome Measures in Rheumatology Clinical Trials (OMERACT) ultrasound (US) scoring system for tenosynovitis when applied in a multicenter design.Methods.RA patients with US-verified tenosynovitis were recruited when scheduled for treatment intensification. Tenosynovitis was assessed at baseline, and 3 and 6 months followup, using the semiquantitative OMERACT scoring system.Results.Expressed in median (25th; 75th percentiles), the overall greyscale and Doppler score decreased significantly from baseline at 4 (2; 7) and 3 (2; 6), to 6 months at 2 (0; 3) and 0 (0; 1, p < 0.01), respectively, and showed high responsiveness (standardized response mean ≥ 0.8).Conclusion.The OMERACT US scoring system for tenosynovitis showed high responsiveness, supporting its use for diagnosing and monitoring tenosynovitis in multicenter trials.

scholarly journals Novel outcome measures for clinical trials in cystic fibrosis

2014 ◽  
Vol 50 (3) ◽  
pp. 302-315 ◽  
Author(s):  
Harm A.W.M. Tiddens ◽  
Michael Puderbach ◽  
Jose G. Venegas ◽  
Felix Ratjen ◽  
Scott H. Donaldson ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Clinical Trials ◽  
Outcome Measures

scholarly journals The OMERACT Core Set of Outcome Measures for Use in Clinical Trials of ANCA-associated Vasculitis

2011 ◽  
Vol 38 (7) ◽  
pp. 1480-1486 ◽  
Author(s):  
PETER A. MERKEL ◽  
SIBEL Z. AYDIN ◽  
MAARTEN BOERS ◽  
HANER DIRESKENELI ◽  
KAREN HERLYN ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Outcome Measurement ◽  
Measurement Instrument ◽  
The United States ◽  
Anca Associated Vasculitis ◽  
Core Set ◽  
Patient Reported ◽  
Development Evaluation ◽  
Multicenter Clinical Trials

There has been a marked increase in the past 15 years in the number and quality of clinical trials in the idiopathic inflammatory vasculitides, especially the small-vessel vasculitides known as antineutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis [AAV; granulomatosis, with polyangiitis (Wegener’s)]. These trials have been conducted by multicenter, international groups in Europe and the United States with financial support provided by government agencies and biopharmaceutical companies. This increased clinical trial activity in vasculitis has been accompanied by the development and validation of new outcome measures — a challenging process for these complex, multiorgan system diseases. The international OMERACT Vasculitis Working Group has developed and implemented an iterative research agenda that has utilized accumulated experience and datasets from several multicenter clinical trials and large cohort studies. This work has led to the development, evaluation, validation, and endorsement, through the OMERACT consensus and validation processes, of a “core set” of outcome measurements for use in clinical trials of AAV. The core set includes domains of disease activity, damage assessment, patient-reported outcomes, and mortality; there is at least one validated outcome measurement instrument available for each domain. This report reviews the domains of illness in AAV included in the OMERACT core set, describes the instruments validated to measure these domains, and presents the approved core set.

scholarly journals Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy

2006 ◽  
Vol 35 (1) ◽  
pp. 36-42 ◽  
Author(s):  
Jill E. Mayhew ◽  
Julaine M. Florence ◽  
Thomas P. Mayhew ◽  
Erik K. Henricson ◽  
Robert T. Leshner ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Outcome Measures ◽  
Surrogate Outcome ◽  
Multicenter Clinical Trials
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