Clinical Trial Alerts – September 2019

Cystic Fibrosis Foundation issued the following clinical trial alerts in the month of September. September 17, 2019 Study of QBW276 in adults with CF Status: Completed with results Description: This study evaluated the safety and tolerability of QBW276, an inhaled drug designed to improve mucus clearance. This study also evaluated how the body processes the drug. Age: 18 Years and Older Mutation: No Mutation Requirement Fev1% Predicted: 40 to 100% Number of Visits: 6 Length of Participation: 14 days link: Study of adherence to lumacaftor/ivacaftor using a smart pill bottle device Status: Completed with results Description: This study evaluated the impact of a smart pill bottle device on adherence to lumacaftor/ivacaftor (Orkambi®).This study was for people with CF who were not currently being treated with lumacaftor/ivacaftor (Orkambi®) prior to enrolling in the study. Age: 16 Years and Older Mutation: Two Copies F508del Fev1% Predicted: 40% or greater Number of Visits: 7 Length of Participation: 48 weeks link: Study of VX-661 plus ivacaftor in people with CF who have two copies of the F508del CFTR mutation Status: Completed with results Description: This study evaluated the safety and effectiveness of the CFTR modulator drug VX-661 in combination with ivacaftor (Symdeko®). This study was for people with CF who have two copies of the F508del CFTR mutation. Age: 18 Years and Older Mutation: Two Copies F508del Fev1% Predicted: 40 to 90% Number of Visits: 6 Length of Participation: 60 days link:


CFFC Facebook Posts – September 2019 – Part 1

09/01/2019 - Clinical Trial Alerts - August 2019 – Cystic Fibrosis Foundation released several clinical trial updates in August, most of which provide reports of competed trials. 09/02/2019 - MAC Lung Infections Best Treated When Specific Bacterial Species Known, Study Finds – This study demonstrates two things. One, the advances in genetics allow for more precise diagnosis. Two, more precise diagnosis leads to more effective treatment. 09/04/2019 - Resources for Supporting Families Coping with Chronic Illness – A collection of links to a lot of good information has been compiled by Online MSW Programs. 09/05/2019 - Long-term use of Kalydeco reduces lung infections according to Registry analysis – An interesting finding drawn from the data captured in the UK CF registry. 09/06/2019 - The Kind of Friend We Deserve – Your personal support system is a critical part of overcoming the challenges presented by CF. 09/07/2019 – Living with CF Golf Tournament - One week to go until CFFC's golf tournament. Our sponsors are lined up. Southern Meadows Golf Club is in great condition. The long range weather forecast is look marvelous. All we need is you! Send an email to to secure a slot in the tournament. It's not too late to sign up. 09/08/2019 - Things I Learned After Gallbladder Surgery – We know that CF does not give you a pass on the rest of the health issues that affect the rest of the population. This blog shares some observations on minor surgery. 09/09/2019 - Amikacin Liposome Therapy Has Similar Efficacy as Tobramycin for P. aeruginosa, Study Shows – Another weapon in the arsenal to combat Pseudomonas. 09/10/2019 - Cystic fibrosis carriers: What to know – This article provides information about the ...


Living with CF Golf Tournament – 2019 Results

After last year's break in the action, CFFC's Living with CF Golf Tournament returned in 2019 with good spirits, good weather and, as a result, good golf. Eighteen golfers gathered on September 14th for nine holes of golf. The clouds that lingered at tee time (see above picture) dissolved and we finished in a pleasantly warm sunshine. Thanks to our generous sponsors, each player was able to return home with a prize. Therefore, the scores just determined the order for prize selection. Here are the results: With a score of 32 (par), first place went to Cindy Garcia, Jean Lazeroff, Helena Spring and Mary Moss (in spirit). Second place was secured by Dan Sullivan, Jamie Romeo and Kristen Shaw with a score of 34. Shooting 36 put Jackie Goldberger, Jeff Goldberger, Sue Smith and Greg Smith in third place. Linda Maloney, Julie Maxon, Bruce Oliver and Tom Wickerham shot a 38 to place fourth. The field was rounded out by Caroline Burtner, Linda Beach, Peggy Rickman and Stella Burtner with a 41. Gift baskets also went to Greg Smith and Tom Wickerham for placing their tee shots closest to the pin on holes 2 and 9, respectively. Of course, the real winner was our local CF community. The net proceeds from the event will go toward funding our Hospital Help Packet program. For that, we are grateful to all of our supporters. Our returning prize sponsors, Marketview Liqour and Wegmans, were joined by Wright Beverage Distributing. Allan Mason provided some last minute additions to the prize table. We also inherited some unclaimed prizes from the Lillian Invitational held the preceding night at Southern Meadows Golf Club. Additional funding was provided by our hole sponsors: Fitz Fitzpatrick and Linda Stackman, Plantation Party House, Jackie and Jeff Goldberger, Walt and Mary McCanna, Ken and Sue Graham, and the Dwyer Family. These sponsors were supplemented with contributions from: Dan and Jane Sullivan, Bob Parise, Kevin Donavan, Allan Mason and Joan Updaw. Last but not least, we appreciate the ongoing assistance from Southern Meadows Golf Club. In addition to acting as host, golf pro Mike Clawson served as our official scorer and menu coordinator. The golfers also directly benefited from Rick ...


CFFC Facebook Posts – August 2019 – Part 2

08/16/2019 - Comment Period for Palliative Care Guidelines – Draft guidelines from the Cystic Fibrosis Foundation call for the incorporation of palliative care in the CF treatment plan from diagnosis throughout the life cycle. We encourage you to review the guidelines and provide your feedback before the close of the comment period on August 30th. 08/17/2019 - Lungs Grown in Yale Lab Have Potential for Patients with CF, Other Chronic Lung Diseases – Researchers are working on a solution to the shortage of donor lungs for transplantation. 08/18/2019 - Ask a Case Manager: What Should I Consider When Going to College? - Some timely advice for planning the move (or return) to college. 08/19/2019 - Losing Control: A CF Patient’s Journey Through Mental Health – The main thrust of this blog is to dispel the stigma of seeking mental health care. The story also touches on the topics of coordination among care teams, seeking multiple opinions and the impact of insurance. 08/20/2019 - Can a person catch cystic fibrosis from someone else? - Have you ever been asked if CF is contagious? (Bet you have!) This article doesn't present any new information, but it can be a good reference for acquainting family and friends with key facts about CF. 08/21/2019 - Adapting the Routine – Here's another look at adjusting routines for the return to (or start of) school. 08/22/2019 - When Will I Be ‘Sick Enough’ for a Transplant? - While the title of this blog may suggest a discussion of medical criteria, it presents the mental exercise of one person weighing the pros and cons of ...


Clinical Trial Alerts – August 2019

Cystic Fibrosis Foundation issued the following clinical trial alerts in the month of August. August 7, 2019 Study to evaluate the effects of the triple-combination modulator, elexacaftor/tezacaftor/ivacaftor (PROMISE) Status: Enrolling Description: This observational study will measure the effects of the triple-combination therapy, elexacaftor/tezacaftor/ivacaftor, in people with CF. These drugs are intended to help CFTR protein function closer to normal. This study will look at how treatment with the triple-combination therapy affects people with CF across many different aspects of the disease. Age: 12 Years and Older Mutation: Two Copies F508del or One Copy F508del Fev1% Predicted: No FEV1 Limit Number of Visits: 6 Length of Participation: 2 years link: August 21, 2019 Study of BI 1265162, a mucus clearance drug, in teens and adults with CF Status: Enrolling Description: This study will look at the safety and effectiveness of BI 1265162, a drug intended to improve the clearance of mucus from the lungs. This study is for people with CF ages 12 and older. Age: 12 Years and Older Mutation: No Mutation Requirement Fev1% Predicted: 40 to 90% Number of Visits: 5 Length of Participation: 7 weeks link: Phase 2a study of Galapagos GLPG2222 in adults with CF Status: Completed with results Description: This study evaluated the safety and effectiveness of the CFTR modulator GLPG2222. This study was for people with CF who have two copies of the F508del mutation. Age: 18 Years and Older Mutation: Two Copies F508del Fev1% Predicted: 40% or greater Number of Visits: 5 Length of Participation: 10 weeks link: Phase 3 study of tezacaftor (VX-661) combination drug in children with cystic fibrosis Status: Completed with results


CFFC Facebook Posts – August 2019 – Part 1

08/01/2019 - Data Safety Monitoring Board – This article describes the safety oversight that the CF Foundation provides for clinical trials in addition to the procedures employed by government agencies and research institutions. 08/02/2019 - Andrew W. Eve Memorial Scholarship – At this mid-point of summer, you may be making preparations for college. The Andrew W. Eve Memorial Scholarship may help you with your financial planning. 08/03/2019 - Using Nebulizer While Lying on Alternating Sides Can Better Deposit Therapy Across Lungs, Trial Finds – This article notes that researches not only look into the components of CF treatment, they also look at how the treatments are delivered. 08/04/2019 - Cystic Fibrosis Newborn Screening – In this video, staff from Cincinnati Children's Hospital explain their protocol for newborn screening. 08/05/2019 – Hospital Help Packets – Hospital Help Packets have been the cornerstone of support offered by CFFC since its inception. Learn about, or refresh your memory on, the program at this link. 08/06/2019 - Translate Bio’s MRT5005 May Improve Lung Function With a Single Dose, Interim Trial Data Shows – The preliminary results of this trial show the potential for a CFTR correction that is independent of the mutation type. 08/07/2019 - Opening Up About My Struggle Getting Pregnant – With the advances in treatments, the majority of people living with CF are adults. The benefits of adulthood come with new challenges. This blog presents the struggles that may be encountered when pursuing parenthood. 08/08/2019 - Local cystic fibrosis patient gets help from ‘The Bachelor’ star Colton Underwood – In case you missed the broadcast ...


Comment Period for Palliative Care Guidelines

A team sponsored by Cystic Fibrosis Foundation (CFF) has produced draft recommendations for models of best practices for palliative care in CF. Members of the CF community has the opportunity to provide feedback on the draft through August 30, 2019. As pointed out in the draft, the concept of palliative care is commonly misunderstood. Rather than an end of life option, the draft guidelines establish a role for palliative care in CF treatment throughout life, starting with diagnosis. The scope of palliative care includes family and caregivers as well as those living with CF. Here is the announcement from the CFF Community Voice team: The CF Foundation would like your input on the draft of the Models of palliative care delivery for individuals with cystic fibrosis: Cystic Fibrosis Foundation evidence-informed guidelines. [NOTE: This link provides the option to read or download a PDF version of the draft guidelines.] A committee of CF health care providers, two adults with CF, and a parent of an individual with CF researched and developed the consensus recommendation statements presented in the draft. It is our hope that you will add your input and feedback to ensure that our community issues actionable practice recommendations for palliative care delivery. If you are interested in this opportunity, please review the Models of palliative care delivery for individuals with cystic fibrosis: Cystic Fibrosis Foundation evidence-informed guidelines. Please read the instructions that are listed on the first page of the survey and submit your comments using the button below by Friday, August 30, 2019 at 5:00 p.m., ET. You will be able to provide feedback on each recommendation and have the opportunity to provide input on other sections as well. Submit Comments Thank you in advance for taking time to review and provide us with feedback on this important aspect of CF care. Feel free to share this opportunity with others in your network. If you have any questions, please contact Sarah Hempstead at Sincerely,Albert Faro and Sarah Hempstead Albert Faro, MDSenior Director of Clinical AffairsCystic Fibrosis Foundation Sarah Hempstead, MSPractice Guidelines ManagerCystic Fibrosis Foundation Once these guidelines are adopted, they will influence the course of CF care. Therefore, we encourage you to review and comment ...


CF Roundtable – Summer 2019

The Summer 2019 edition of the CF Roundtable quarterly newsletter was just released by United States Adult Cystic Fibrosis Association, Inc (USACFA). The primary focus of this issue is planning for education and Careers. To download a PDF version of the current issues click here. To access an online version and review back issues, go here: The contents of the current issue are described in the Editor's Comments presented below. We are happy to tell you of two new directors of USACFA. They are Mark Anthony Tremblay and Tré LaRosa. You may read more about them on pages 26 and 27. We also have some sad news of a former director, Richard “Rich” DeNagel. Rich, who had been both a director and a columnist, died in May. Please see his obituary on page 39. Once again we have an issue that is full of good articles by a variety of authors. I hope that you have read the front cover where Amy Sylvis clues us in on the cross-infection protection guidelines from the CFF. This leads us right into the Focus topic of this issue, which is ENT Problems and Sinus Disease. Jeanie Hanley writes of vestibular problems. Andrea Eisenman tells of her six sinus surgeries. Katie Lockwood discusses her lack of a sense of smell. Sydna Marshall and Brittany Wager tell of their differing results from obliteration of the frontal sinuses. I bring up my sinus pain and surgery in “Speeding Past 50.” In “Ask The Attorney,” Beth Sufian answers questions from readers about household deductions for SSI and overpayments of Social Security. Isabel Stenzel Byrnes talks about being special in “Spirit Medicine.” Be sure to check out the review by Rob De La Noval of the book — Salt in My Soul: An Unfinished Life — by Mallory Smith. Also, take a look at “Information From The Internet” that is so ably compiled by Laura Tillman. We find Rebecca Mueller “In The Spotlight.” Three people have contributed to “Voices From The Roundtable.” They are Alexis Schuller, who writes of the monkey wrenches that CF can throw into our plans for our lives and how to work with that. Mark Anthony Tremblay writes of being sober for 32 years and working to help others. Emily Trout tells of her journey toward transplant. I am using the final lines ...


CFFC Facebook Posts – July 2019 – Part 2

07/16/2019 - Different CFTR Potentiators Bind to Same Flexible Spot on Protein, Study Finds –This study looked at how drugs like Kalydeco actually work which could lead to the development of other, more effective treatments. 07/17/2019 - Focusing on the Now Helped Us Survive Losing Our Daughter to CF – This blog addresses an issue that is always in the back of our mind. 07/18/2019 - CFFC Annual Financial Report for 2018-19 – We have published the financial report for CFFC's fiscal year that ended June 30, 2019. 07/19/2019 - Building Trust In the Relationship with Your CF Clinic Team – In this blog, Canadians living with CF share their perspectives on building a trust relationship with their CF care teams. 07/20/2019 - Pancreas-on-a-chip Expected to Shed Light, Help Find New Treatments for CF-related Diabetes – Using this new approach, scientists have confirmed that a faulty CFTR gene is directly related to CF-related diabetes. That knowledge can be the basis for developing effective treatments. 07/21/2019 - My Colonoscopy Wasn’t so Crappy! - Gunnar Esiason's blog is a little crass, but he addresses an important topic. 07/22/2019 - Specialized Care Helping Cystic Fibrosis Patient Breathe Easier – This page is basically an ad for the adult CF center in Denver; however, since it features Kori Tolbert Doherty, CFFC's past president, it's worth sharing here. 07/23/2019 - ‘Everything’ Will Never Be Perfect, and That’s OK – This blogger reflects on the peril of pursuing some sort of “ideal.” 07/24/2019 -


CF Foundation 2019 CF Health Insurance Survey

The Cystic Fibrosis Foundation is conducting a follow up to last year's survey on health insurance concerns in the CF community. Here is the text of the announcement. Last year more than 2,500 members of the cystic fibrosis community responded to our landmark CF Health Insurance Survey. This survey, along with in-depth interviews with people with cystic fibrosis and parents of children with CF, has shaped Compass programming and helped us advocate with decision-makers across the country. The 2019 CF Health Insurance Survey is now open and available at for people with CF, parents, and caretakers. Take the Survey This year’s survey, designed with researchers at George Washington University, will help the CF Foundation to better understand how people with cystic fibrosis navigate health insurance, access to care, and cost. The survey should take about 15 minutes to complete. Your voice is critical in helping to inform Foundation policy efforts and programming. We greatly appreciate your continued support!