NACFC | Plenary 2: Novel CF Therapeutics & Clinical Trial Strategy to Accelerate Our Mission
3.6 هزار بار بازدید -
2 سال پیش
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During plenary 2 of our
During plenary 2 of our North American Cystic Fibrosis Conference, Deepika Polineni, MD, MPH, and Nicole Mayer-Hamblett, PhD, discuss clinical trial advances in the CF therapeutics pipeline and highlight the diverse strategies being implemented to address the needs of all people living with the disease.
0:00 | Novel CF Therapeutics & Clinical Trial Strategy to Accelerate Our Mission
0:01 | JP Clancy, MD, senior vice president of clinical research of the CF Foundation, presents the Richard C. Talamo Clinical Achievement Award to Bonnie Ramsey, MD, endowed professor and vice chair for research, department of pediatrics, at University of Washington School of Medicine.
11:14 | Dr. Clancy introduces the topic and speakers for Plenary 2.
14:54 | Deepika Polineni, MD, MPH, associate professor and CF center director, department of pediatrics, at University of Washington School of Medicine in St. Louis, reflects on the 2019 approval of Trikafta® and other therapies that have improved the lives of people with CF.
17:23 | New data from three studies on Trikafta
21:33 | Expanding CFTR modulator access through theratyping
23:43 | CF affects a diverse population, and for people who cannot take modulators, our work is not done.
26:05 | Updates on genetic therapy trials in CF
29:23 | Paul Trey Newman shares his experience living with CF.
33:07 | CF therapeutics development pipeline: anti-inflammatory treatments
34:22 | CF therapeutics development pipeline: anti-infective treatments
36:09 | CF therapeutics development pipeline: mucociliary clearance treatments
37:53 | CF-related diabetes studies
39:27 | The pipeline over time
40:42 | Each person with CF experiences the disease differently.
42:26 | Closing remarks and introduction of Nicole Mayer Hamblett, co-executive director of the CF Therapeutics Development Network Coordinating Center at Seattle Children’s Hospital and professor in the department of pediatrics and biostatistics at University of Washington.
43:20 | Dr. Mayer Hamblett shares her experience in clinical trial design and declares there is nothing textbook about designing trials for cystic fibrosis.
44:48 | CF clinical trials are no longer being designed one trial at a time.
46:18 | Trials for people who not benefiting from modulators
48:10 | Reducing barriers for equitable opportunity in accessing clinical trials
50:13 | Designing trials for a smaller population
52:44 | Safety is a priority.
53: 47 | Kaylee Alvarado shares her experience living with CF.
57:46 | Trial considerations will need to be different for people who are on modulators and people who are not.
1:00:28 | Expanding our target for clinical efficacy as people taking Trikafta face a different course of their disease
1:03:40 | Getting treatments from trials to approval globally
1:05:55 | We need to listen to and understand the priorities of the CF community.
1:06:39 | New data showing the impact of reducing inhaled treatments after taking Trikafta
1:11:36 | Closing remarks
Originally recorded November 4, 2022.
0:00 | Novel CF Therapeutics & Clinical Trial Strategy to Accelerate Our Mission
0:01 | JP Clancy, MD, senior vice president of clinical research of the CF Foundation, presents the Richard C. Talamo Clinical Achievement Award to Bonnie Ramsey, MD, endowed professor and vice chair for research, department of pediatrics, at University of Washington School of Medicine.
11:14 | Dr. Clancy introduces the topic and speakers for Plenary 2.
14:54 | Deepika Polineni, MD, MPH, associate professor and CF center director, department of pediatrics, at University of Washington School of Medicine in St. Louis, reflects on the 2019 approval of Trikafta® and other therapies that have improved the lives of people with CF.
17:23 | New data from three studies on Trikafta
21:33 | Expanding CFTR modulator access through theratyping
23:43 | CF affects a diverse population, and for people who cannot take modulators, our work is not done.
26:05 | Updates on genetic therapy trials in CF
29:23 | Paul Trey Newman shares his experience living with CF.
33:07 | CF therapeutics development pipeline: anti-inflammatory treatments
34:22 | CF therapeutics development pipeline: anti-infective treatments
36:09 | CF therapeutics development pipeline: mucociliary clearance treatments
37:53 | CF-related diabetes studies
39:27 | The pipeline over time
40:42 | Each person with CF experiences the disease differently.
42:26 | Closing remarks and introduction of Nicole Mayer Hamblett, co-executive director of the CF Therapeutics Development Network Coordinating Center at Seattle Children’s Hospital and professor in the department of pediatrics and biostatistics at University of Washington.
43:20 | Dr. Mayer Hamblett shares her experience in clinical trial design and declares there is nothing textbook about designing trials for cystic fibrosis.
44:48 | CF clinical trials are no longer being designed one trial at a time.
46:18 | Trials for people who not benefiting from modulators
48:10 | Reducing barriers for equitable opportunity in accessing clinical trials
50:13 | Designing trials for a smaller population
52:44 | Safety is a priority.
53: 47 | Kaylee Alvarado shares her experience living with CF.
57:46 | Trial considerations will need to be different for people who are on modulators and people who are not.
1:00:28 | Expanding our target for clinical efficacy as people taking Trikafta face a different course of their disease
1:03:40 | Getting treatments from trials to approval globally
1:05:55 | We need to listen to and understand the priorities of the CF community.
1:06:39 | New data showing the impact of reducing inhaled treatments after taking Trikafta
1:11:36 | Closing remarks
Originally recorded November 4, 2022.
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