A support group for parents, friends, and family of children with Cystic Fibrosis at the Alberta Children's Hospital.
Wednesday 24 October 2012
Saturday 13 October 2012
Updated Support Group Dates
CONNECTING
FAMILIES
“Sometimes, reaching out and taking someone’s
hand is the beginning of a journey. At
other times, it is allowing another to take yours”
-Vera Nazarian
Support group and information
sharing for caregivers of children living with Cystic Fibrosis
Drop-in Sessions
October 10th
January 9, 2013
March 13th
May 8th
July 10th
7pm-8pm
Location: Alberta Children’s
Hospital – on day located in CF Clinic for room details
Facilitated by CF Clinic, Alberta
Children’s Hospital
Telehealth Video Conferences
available upon request
PLEASE EMAIL JILL REDFERNWITH QUESTIONS OR
FEEDBACK: Jillian.Redfern@albertahealthservices.ca
Wednesday 3 October 2012
The Kids
I thought it would be a neat idea for everyone to get to know the kids and their stories. So here is our story
My husband and I have twin girls Faith and Kaylee who are 4 months old. My daughter Faith was diagnosed with Cystic Fibrosis when she was 1 month old through the Newborn Screening. Her sister is a carrier, but does not have CF.At the time we did not know what CF was or what it meant for our daughter. However, through research and the Clinic at ACH we have learned a ton of information. When she was first diagnosed we were really sad, but quickly moved passed that. We have decided to have a positive approach and provide the best life for our daughter possible. We are getting involved as much as we can in hopes that with others we will find a cure.
My husband and I have twin girls Faith and Kaylee who are 4 months old. My daughter Faith was diagnosed with Cystic Fibrosis when she was 1 month old through the Newborn Screening. Her sister is a carrier, but does not have CF.At the time we did not know what CF was or what it meant for our daughter. However, through research and the Clinic at ACH we have learned a ton of information. When she was first diagnosed we were really sad, but quickly moved passed that. We have decided to have a positive approach and provide the best life for our daughter possible. We are getting involved as much as we can in hopes that with others we will find a cure.
 |
| Faith |
 |
| My girls and I |
Wednesday 26 September 2012
New Clinical Trial for Cystic Fibrosis
FAQs About Combined Kalydeco™ & VX-809 Clinical Trial
Vertex Pharmaceuticals Inc. is studying its cystic fibrosis drug Kalydeco™ in combination with another potential therapy, VX-809, in people with the most common mutation of CF, Delta F508.
Vertex Pharmaceuticals Inc. is studying its cystic fibrosis drug Kalydeco™ in combination with another potential therapy, VX-809, in people with the most common mutation of CF, Delta F508.
Both drugs are designed to treat the underlying cause of cystic fibrosis — a faulty gene and its protein product, CFTR — and are taken in pill form.
Results from a Phase 2 clinical trial of Kalydeco and VX-809 in multiple combinations showed significant improvements in lung function in people with two copies of the Delta F508 mutation.
The Delta F508 mutation creates a defective protein that does not move to its proper place at the cell surface. VX-809 is designed to move CFTR to the cell surface; Kalydeco is designed to improve the protein’s function once it has reached the cell surface.
Vertex plans to begin a pivotal trial of Kalydeco and VX-809 in people with two copies of the Delta F508 mutation in early 2013, pending discussion with regulatory agencies.
Vertex plans to begin a pivotal trial of Kalydeco and VX-809 in people with two copies of the Delta F508 mutation in early 2013, pending discussion with regulatory agencies.
The U.S. Food and Drug Administration (FDA) approved Kalydeco in January 2012 when taken alone for people with the G551D mutation of CF ages 6 and older.
Kalydeco was formerly called VX-770 and is also known by its generic name ivacaftor.
Vertex developed Kalydeco and VX-809 with significant scientific, clinical and financial support from the Cystic Fibrosis Foundation, including a $75 million investment.
Vertex developed Kalydeco and VX-809 with significant scientific, clinical and financial support from the Cystic Fibrosis Foundation, including a $75 million investment.
- What were the results of the Kalydeco™ and VX-809 Phase 2 clinical trial?
- What is the difference between Kalydeco and VX-809?
- What are the next steps for the Kalydeco and VX-809 combination trial?
- Will the Kalydeco and VX-809 combination therapy help people who have one copy of the Delta F508 mutation and a copy of another mutation?
- How many people with CF have the Delta F508 mutation?
- How can I find out which CF mutations I or my child have?
- How safe are CF clinical trials?
- How is my safety protected broadly if I participate in a clinical trial?
- Where can I find more information about clinical trials in general?
In June 2012, Vertex announced results from the second part of a Phase 2 clinical trial of Kalydeco and VX-809 in combination. The trial enrolled 109 people ages 18 and older with at least one copy of the Delta F508 mutation of CF.
People who received the combination treatment showed improvements in lung function, compared with those who received a placebo.
Those with two copies of the Delta F508 who received the highest dose of VX-809 had the greatest improvement. More than half of this group had an improvement in lung function of 5 percentage points, compared with those on placebo, and a quarter of this group had an improvement of 10 percentage points or more.
Participants with one copy of the Delta F508 mutation also showed improvements in lung function, however, the improvements were smaller than those seen in people with two copies of Delta F508.
The study also evaluated the safety and tolerability of the combination therapy. There were no serious adverse events reported during the trial.
back to topPeople who received the combination treatment showed improvements in lung function, compared with those who received a placebo.
Those with two copies of the Delta F508 who received the highest dose of VX-809 had the greatest improvement. More than half of this group had an improvement in lung function of 5 percentage points, compared with those on placebo, and a quarter of this group had an improvement of 10 percentage points or more.
Participants with one copy of the Delta F508 mutation also showed improvements in lung function, however, the improvements were smaller than those seen in people with two copies of Delta F508.
The study also evaluated the safety and tolerability of the combination therapy. There were no serious adverse events reported during the trial.
Kalydeco and VX-809 are two individual drugs that have been studied in separate clinical trials and are currently being studied in combination in people with the Delta F508 mutation of CF.
Kalydeco was approved by the FDA in January 2012 for people with the G551D mutation of CF ages 6 and older.
In people with the G551D mutation, Kalydeco helps improve the function of the defective CFTR protein at the surface of the cell. Kalydeco allows CFTR that is at the cell surface to function better, allowing a proper flow of salt and fluids on the surface of the lungs. This helps to thin the thick, sticky mucus caused by CF that builds up in the lungs.
In people with the G551D mutation, Kalydeco helps improve the function of the defective CFTR protein at the surface of the cell. Kalydeco allows CFTR that is at the cell surface to function better, allowing a proper flow of salt and fluids on the surface of the lungs. This helps to thin the thick, sticky mucus caused by CF that builds up in the lungs.
VX-809 is designed to move the defective CFTR protein to its proper place at the cell surface. The Delta F508 mutation leads to a CFTR protein that does not fold correctly. Because the defective CFTR is not the right shape, it does not make it to the surface of the cell.
back to top
What are the next steps for the Kalydeco and VX-809 combination trial?
Vertex plans to begin a pivotal trial of the combination treatment in people with two copies of the Delta F508 mutation of CF in early 2013, pending discussion with regulatory agencies. Pivotal trials are usually Phase 3 trials, designed to collect data that the U.S. Food and Drug Administration can use to decide whether to approve a potential drug.
Vertex has also said it will conduct additional studies of Kalydeco and VX-809 in combination in people with one copy of the Delta F508 mutation.
back to top
Vertex plans to begin a pivotal trial of the combination treatment in people with two copies of the Delta F508 mutation of CF in early 2013, pending discussion with regulatory agencies. Pivotal trials are usually Phase 3 trials, designed to collect data that the U.S. Food and Drug Administration can use to decide whether to approve a potential drug.
Vertex has also said it will conduct additional studies of Kalydeco and VX-809 in combination in people with one copy of the Delta F508 mutation.
back to top
Will the Kalydeco and VX-809 combination therapy help people who have one copy of the Delta F508 mutation and a copy of another mutation?
In the Phase 2 trial of Kalydeco and VX-809 in combination, participants with one copy of the Delta F508 mutation showed improvements in lung function, compared with those on placebo. However, these improvements were smaller than those seen in people with two copies of Delta F508.
Vertex has said it will conduct additional studies of Kalydeco and VX-809 in combination in people with one copy of the Delta F508 mutation.
back to top
In the Phase 2 trial of Kalydeco and VX-809 in combination, participants with one copy of the Delta F508 mutation showed improvements in lung function, compared with those on placebo. However, these improvements were smaller than those seen in people with two copies of Delta F508.
Vertex has said it will conduct additional studies of Kalydeco and VX-809 in combination in people with one copy of the Delta F508 mutation.
back to top
How many people with CF have the Delta F508 mutation?
About 50 percent of people with CF in the United States have two copies of the Delta F508 gene mutation. About 40 percent of people with CF in the United States have at least one copy of the Delta F508 mutation.
back to top
How can I find out which CF mutations I or my child have?
If you do not know what your or your child's two CF mutations are, contact your CF doctor or care center.
How can I find out which CF mutations I or my child have?
If you do not know what your or your child's two CF mutations are, contact your CF doctor or care center.
The Cystic Fibrosis Foundation’s Mutation Analysis Program (MAP) offers free and confidential genetic testing to patients with a confirmed diagnosis of cystic fibrosis. The MAP provides genotyping for cystic fibrosis patients who have not yet been tested, or who have been tested previously but still have one or more unknown mutations.
Nothing is more important than safety in developing new CF treatments. There are four layers of protection in every CF clinical trial. Each trial must be determined as safe and appropriate for patients by the:
- CF Foundation;
- FDA;
- Participating hospital or university’s Institutional Review Board (IRB); and
- Data Safety Monitoring Board (DSMB). The DSMB is an independent committee of experts in CF care that checks information on ongoing trials, watching for possible problems or unwanted side effects.
The CF Foundation is the only voluntary health organization to organize a DSMB whose members are experts in CF and completely independent and not involved in any way with the trial or its participants. In this way, the CF Foundation does its best to keep participants safe throughout the clinical trial.
back to top
How is my safety protected broadly if I participate in a clinical trial?
The U.S. government has strict guidelines and safeguards to help protect people who choose to participate in clinical research. Every clinical trial in the United States must be approved and monitored by an institutional review board (IRB). This is to keep risks as low as possible and ensure that the risks are worth any potential benefits.
The IRB is usually made up of doctors and the general public. They look at the trial’s protocol (a clear and detailed plan of the experiment) to make sure that participants’ rights are protected and the trial does not cause them unnecessary risk.
In addition, an independent committee of experts in CF care, the Data Safety Monitoring Board (DSMB), examines data from clinical trials and determines if there are safety issues that need to be brought to the attention of the IRB or the sponsors of a clinical trial.
The FDA also must approve all clinical trial protocols and make sure all of their procedures are being followed as the trial goes on.
In the United States, anyone participating in a clinical trial must sign an Informed Consent Form. This form explains the trial in full, including the risks, and a research team member will explain the trial and the consent form to individuals before they sign it.
back to top
How is my safety protected broadly if I participate in a clinical trial?
The U.S. government has strict guidelines and safeguards to help protect people who choose to participate in clinical research. Every clinical trial in the United States must be approved and monitored by an institutional review board (IRB). This is to keep risks as low as possible and ensure that the risks are worth any potential benefits.
The IRB is usually made up of doctors and the general public. They look at the trial’s protocol (a clear and detailed plan of the experiment) to make sure that participants’ rights are protected and the trial does not cause them unnecessary risk.
In addition, an independent committee of experts in CF care, the Data Safety Monitoring Board (DSMB), examines data from clinical trials and determines if there are safety issues that need to be brought to the attention of the IRB or the sponsors of a clinical trial.
The FDA also must approve all clinical trial protocols and make sure all of their procedures are being followed as the trial goes on.
In the United States, anyone participating in a clinical trial must sign an Informed Consent Form. This form explains the trial in full, including the risks, and a research team member will explain the trial and the consent form to individuals before they sign it.
Monday 24 September 2012
Welcome
Welcome to the group. Please feel free to make any posts you would like. I am excited about this support group and hope together we can get answers to questions, share stories, and so much more.
Once you are a member perhaps you could make a post and let me know about your CF story and a picture of your little one :) I will post mine soon
Once you are a member perhaps you could make a post and let me know about your CF story and a picture of your little one :) I will post mine soon
Saturday 15 September 2012
Welcome!!
Hello... My name is Rachel Newell and I am the proud mom of twin girls Faith and Kaylee. My one daughter Faith was diagnosed with Cystic Fibrosis when she was just over a month old. June 27, 2012 is a day my husband and I will never forgot. We had no idea we were carriers let alone what Cystic Fibrosis was or even meant for our daughter. Now the shock has worn off and we spend our time learning as much as we can and fighting to find a cure. Since starting at the CF Clinic at the Alberta Children's Hospital we have felt blessed to be surrounded by an amazing team of doctors, nurses, etc. My husband and I decided to start this blog as a support group for one another. We may not have all the answers, but what we do know is the more support you have the better and what better way for support than to surround yourself with others in a similar situation. It is our hope people will use this blog to ask questions, share experiences and challenges, tips you have found helpful, information and articles you have found, fundraisers, and anything that might be on your mind. Together we will find a cure and beat Cystic Fibrosis!!
Subscribe to:
Posts (Atom)