Some data from the clinical trials of KRN23/burosumab were shared with clinicians and researchers during the recent conference of the American Society for Bone and Mineral Research. Dr. Karl Insogna presented about the treatment of adults, and Dr. Michael Whyte presented for pediatric issues.
The data were summarized in an article by MedPage Today, which you can read here: https://www.medpagetoday.com/meetingcoverage/asbmr/67919
Of particular interest is the lack of significant adverse effects reported in the adult study, as reported by MedPage Today:
"The safety profile of burosumab was similar to what was seen for placebo, with no serious drug-related adverse events and no events leading to study discontinuation.
"We conclude that burosumab represents an exciting new therapy for XLH," Insogna said."
And for the pediatric study, as reported by MedPage Today, the improvement in the rickets score (lower is better), even though at least some of the patients had previously been treated with the current therapy (phos and calcitriol): "the most substantial improvements [in rickets scores] being seen for children who had more severe rickets (RSS of 1.5 or higher at baseline). In that subgroup, RSS decreased from a baseline score of 2.46 to 1.01 .... "
Also, side effects in the pediatric study were considered either insignificant or unrelated to the new treatment: "Renal function remained normal and there was no evidence of ectopic mineralization of the myocardium. The most common adverse events were transient injection site reactions, and only one child had a serious adverse event, which was considered unrelated to the therapy. "The favorable benefit-risk profile of burosumab holds promise for long-term outcomes for these children," Whyte concluded."
Showing posts with label clinical trial. Show all posts
Showing posts with label clinical trial. Show all posts
Wednesday, September 20, 2017
Thursday, October 27, 2016
KRN23 pediatric trial is enrolling patients
Ultragenyx Pharmaceutical, Inc. just announced that the first patient has been enrolled in the most recent KRN23 clinical trial. As explained by the CEO and President of Ultragenyx, Emil D. Kakkis, "This study will allow us to directly assess whether KRN23's potential impact on rickets ... is superior to that of conventional oral phosphate and vitamin D therapy for children with XLH."
You can read the press release here: http://ir.ultragenyx.com/releasedetail.cfm?ReleaseID=995543
You can read more about this Phase 3 pediatric clinical trial, which is recruiting pediatric patients (currently recruiting at two locations in California, plus one location in each of Indiana, Missouri and Tennessee, with additional sites worldwide not yet recruiting) here:
https://clinicaltrials.gov/ct2/show/NCT02915705?term=xlh&rank=6
The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
If you're considering participation in a trial, please remember that discussion of the details of others' experiences (especially with respect to symptoms and side effects) may have some adverse consequences for the validity of the study. On the other hand, discussions of things like the time commitments, disruptions to daily life and the quality of the staff are safer to discuss with other participants in the trial.
You can read the press release here: http://ir.ultragenyx.com/releasedetail.cfm?ReleaseID=995543
You can read more about this Phase 3 pediatric clinical trial, which is recruiting pediatric patients (currently recruiting at two locations in California, plus one location in each of Indiana, Missouri and Tennessee, with additional sites worldwide not yet recruiting) here:
https://clinicaltrials.gov/ct2/show/NCT02915705?term=xlh&rank=6
The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
If you're considering participation in a trial, please remember that discussion of the details of others' experiences (especially with respect to symptoms and side effects) may have some adverse consequences for the validity of the study. On the other hand, discussions of things like the time commitments, disruptions to daily life and the quality of the staff are safer to discuss with other participants in the trial.
Thursday, September 29, 2016
Pediatric clinical trial of KRN23
The Phase 3 clinical trial of KRN23 for children from 1 to 12 years old has been announced. This is a head-to-head comparison study of treatment with KRN23 to the current treatment with phosphate/calcitriol, happening throughout the world.
You can find the location information and read more about the inclusion/exclusion criteria here: https://clinicaltrials.gov/ct2/show/NCT02915705?term=xlh&rank=6
The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
You can find the location information and read more about the inclusion/exclusion criteria here: https://clinicaltrials.gov/ct2/show/NCT02915705?term=xlh&rank=6
The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
Thursday, May 12, 2016
On participation in clinical trials
If you don't already know about the clinical trials of KRN23, there are two Phase 3 trials going on now for adults, and we expect that there will be a Phase 3 pediatric trial beginning sometime this year. Check back for updates here, or in our discussion platform, forum.xlhnetwork.org or at our official facebook page: facebook.com/xlhnetwork.
You can read more about the current adult trials here:
https://clinicaltrials.gov/ct2/show/NCT02526160?term=XLH&rank=1
and here:
https://clinicaltrials.gov/ct2/show/NCT02537431?term=XLH&rank=2
Scroll down to the link to "show study locations" for contact information.
The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
If you're wondering about the experience of a clinicial trial generally, however, we have some insight to share from an XLHer who has participated in clinical trials in the past. He is a male college professor and in his fifties now.
In response to a question about why he enrolled in a clinical trial, he stated he had several reasons, including intellectual curiosity. He added, "As a middle-aged XLHer, any treatment is probably too late for me, but perhaps I can make some type of contribution to 'pay it forward' for future generations that include my two sweet daughters."
He also found the experience educational. He reported, "During the intake process I had the opportunity to meet another XLHer who was being discharged. This individual, a male who was about 30 years younger than me, was unable to walk. We chatted, somewhat awkwardly because of the variance in the extent of our shared illness. It was evident to both of us that his health was visibly and significantly more profoundly affected by XLH than mine. I almost started feeling like an imposter despite the fact that I have had 8 osteotomies. The fact remained: I was standing upright and he was bedridden when not in a wheelchair. It was then that I truly understood the range in the severity of XLH."
This particular member was also in a unique position to be able to use his experience as a teaching moment to educate others: "The best part of my participation in the research trial involved my teaching an online summer graduate research methods class. The students and I 'met' online via conferencing technology. When we began the chapter on Experimental Research, which happened to fall during the time when I was in the hospital for the clinical trial, I had an idea for providing the most authentic learning experience. During our online class discussion, I turned on the web camera and allowed the students to see me, the lab rat, sitting in a hospital bed. I wish I could have seen all 25 of the reactions to this stark reality and authenticity resulting from the true visualization of Experimental Research in action. Several of the students did use the chat window to type things like, 'Wow!' 'Incredible,' and 'You are very dedicated to what you teach!' Two months later I received the highest course evaluations I had ever received in the 5 years I have been teaching research methods."
The only challenges this member experienced were those involved with travel planning, but he reports that the staff who made the arrangements were very helpful.
You can read more about the current adult trials here:
https://clinicaltrials.gov/ct2/show/NCT02526160?term=XLH&rank=1
and here:
https://clinicaltrials.gov/ct2/show/NCT02537431?term=XLH&rank=2
Scroll down to the link to "show study locations" for contact information.
The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
If you're wondering about the experience of a clinicial trial generally, however, we have some insight to share from an XLHer who has participated in clinical trials in the past. He is a male college professor and in his fifties now.
In response to a question about why he enrolled in a clinical trial, he stated he had several reasons, including intellectual curiosity. He added, "As a middle-aged XLHer, any treatment is probably too late for me, but perhaps I can make some type of contribution to 'pay it forward' for future generations that include my two sweet daughters."
He also found the experience educational. He reported, "During the intake process I had the opportunity to meet another XLHer who was being discharged. This individual, a male who was about 30 years younger than me, was unable to walk. We chatted, somewhat awkwardly because of the variance in the extent of our shared illness. It was evident to both of us that his health was visibly and significantly more profoundly affected by XLH than mine. I almost started feeling like an imposter despite the fact that I have had 8 osteotomies. The fact remained: I was standing upright and he was bedridden when not in a wheelchair. It was then that I truly understood the range in the severity of XLH."
This particular member was also in a unique position to be able to use his experience as a teaching moment to educate others: "The best part of my participation in the research trial involved my teaching an online summer graduate research methods class. The students and I 'met' online via conferencing technology. When we began the chapter on Experimental Research, which happened to fall during the time when I was in the hospital for the clinical trial, I had an idea for providing the most authentic learning experience. During our online class discussion, I turned on the web camera and allowed the students to see me, the lab rat, sitting in a hospital bed. I wish I could have seen all 25 of the reactions to this stark reality and authenticity resulting from the true visualization of Experimental Research in action. Several of the students did use the chat window to type things like, 'Wow!' 'Incredible,' and 'You are very dedicated to what you teach!' Two months later I received the highest course evaluations I had ever received in the 5 years I have been teaching research methods."
The only challenges this member experienced were those involved with travel planning, but he reports that the staff who made the arrangements were very helpful.
Thursday, May 5, 2016
Social media and clinical research
Social media has been a boon for many patients with rare disorders, enabling them to share their experiences with people who truly understand what they're going through.
It's not without its down sides, though. Misinformation can be passed around, sensitive subjects can be distressing, and the negative news can feel overwhelming sometimes. Those things are true of any social activity, whether in person or in the virtual world, but now there's a risk that is particularly associated with the virtual world: the possibility that patients in clinical trials will undermine the trials by sharing their experiences with other patients.
The concern is that patients will "unblind" the study (figure out which patients are on placebo and which are not). Once the study is unblinded, according to a Wall Street Journal article, "Drug makers and researchers ... worry that patients may drop out if they suspect they aren't getting the drug being tested, or may report symptoms inaccurately because of the influence or suggestions of others in the trial."
While there may not be contractual or regulatory provisions against publicly sharing detailed information about clinical trial participation, it's important to keep in mind the risks of tainting the data. The last thing anyone wants is for the equivalent of a "mistrial" to be called and the research suspended or discarded, because the study was unblinded or otherwise affected by outside factors, and therefore the reported outcomes and adverse effects can't be relied upon. You can read more of the Wall Street Journal article here:
http://www.wsj.com/articles/researchers-fret-as-social-media-lift-veil-on-drug-trials-1406687404
It's not without its down sides, though. Misinformation can be passed around, sensitive subjects can be distressing, and the negative news can feel overwhelming sometimes. Those things are true of any social activity, whether in person or in the virtual world, but now there's a risk that is particularly associated with the virtual world: the possibility that patients in clinical trials will undermine the trials by sharing their experiences with other patients.
The concern is that patients will "unblind" the study (figure out which patients are on placebo and which are not). Once the study is unblinded, according to a Wall Street Journal article, "Drug makers and researchers ... worry that patients may drop out if they suspect they aren't getting the drug being tested, or may report symptoms inaccurately because of the influence or suggestions of others in the trial."
While there may not be contractual or regulatory provisions against publicly sharing detailed information about clinical trial participation, it's important to keep in mind the risks of tainting the data. The last thing anyone wants is for the equivalent of a "mistrial" to be called and the research suspended or discarded, because the study was unblinded or otherwise affected by outside factors, and therefore the reported outcomes and adverse effects can't be relied upon. You can read more of the Wall Street Journal article here:
http://www.wsj.com/articles/researchers-fret-as-social-media-lift-veil-on-drug-trials-1406687404
Monday, December 21, 2015
KRN23 adult trials go global
There are a total of 29 locations for the KRN23 adult clinical trials across the globe now (some not yet recruiting, while they wait for local approvals, but you can contact them now to let them know you're interested when the location is fully set up), including eight in the U.S., two in Canada, one in Denmark, three in France, five in Japan, two in Korea, six in the UK, and one each in Denmark, Ireland, and Italy.
You can get all the details here (age 18-65, regardless of past treatment): https://clinicaltrials.gov/ct2/show/NCT02526160?term=xlh&rank=1
or here (age 25-65, no treatment in past two years): https://clinicaltrials.gov/ct2/show/NCT02537431?term=xlh&rank=2
As always, the XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
You can get all the details here (age 18-65, regardless of past treatment): https://clinicaltrials.gov/ct2/show/NCT02526160?term=xlh&rank=1
or here (age 25-65, no treatment in past two years): https://clinicaltrials.gov/ct2/show/NCT02537431?term=xlh&rank=2
As always, the XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
Friday, December 4, 2015
Adult Clinical trials are recruiting
We just learned that the Phase 3 clinical trial of KRN23 for adults is recruiting now. You can get the details and contact information here: https://clinicaltrials.gov/ct2/show/NCT02526160?term=xlh&rank=1
U.S. locations are: California, Connecticut, Indiana, North Carolina and Texas.
The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
U.S. locations are: California, Connecticut, Indiana, North Carolina and Texas.
The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
Wednesday, September 16, 2015
Additional KRN23 adult trial
In addition to the Phase 3 clinical trials of KRN23 that we mentioned previously (starting in October, not yet recruiting), there's another, smaller study that will begin in November (also not yet recruiting). You can read about it here: https://clinicaltrials.gov/ct2/show/NCT02537431?term=XLH&rank=2
This study is for XLHers who have NOT been on the standard treatment regimen at any time during the past two years. This study will consist of approximately ten patients, and will involve bone biopsies rather than blood and urine samples. The intent is to study the effects on the bone directly, rather than indirectly (using blood tests as a proxy for what is happening to the bones).
If you're interested in participating, be sure to read the information at the link above, and watch for an announcement (here, on the listserv and at the official Facebook page) that recruiting has begun.
The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
This study is for XLHers who have NOT been on the standard treatment regimen at any time during the past two years. This study will consist of approximately ten patients, and will involve bone biopsies rather than blood and urine samples. The intent is to study the effects on the bone directly, rather than indirectly (using blood tests as a proxy for what is happening to the bones).
If you're interested in participating, be sure to read the information at the link above, and watch for an announcement (here, on the listserv and at the official Facebook page) that recruiting has begun.
The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
Wednesday, September 2, 2015
The patient's perspective on clinical trials
In light of the upcoming Phase 3 trials for KRN23, we've been talking to some XLHers who have participated in a variety of clinical trials in the past, so we can share a bit about the experience generally. These comments are not specific to the KRN23 trials, but are just meant to shed some light on an experience that most people are unfamiliar with.
Today's insights, the first in a series on this topic, are from a male in his fifties, who lives in the southeast.
1. How and why did you decide to participate in the trial?
I decided to participate in the trials due to my health concerns and those of my children. I wasn't sure if I could get any relief for my self but wanted to participate with the hope that it would benefit those who are younger and still have a lot of living to do. If I could help them relieve some of their issues thru the trials then I was all for it.
2. Was there anyone in particular who helped you decide whether to participate or to cope with any challenges?
Family and a fellow XLHer.
3. What do you know now that you wish you'd known before participating in your first trial?
The trials I have participated in have been well explained and there really wasn't any hidden info or circumstances. So really nothing.
4. Anything else you want to share with the Network's members?
I know people have different varying conditions of hypophosphatemia and everyone will feel different on trial medications, but my thought on the whole trial study is if you or I could help the next generations with this disease then why shouldn't we? It may not help us now but our children, their children, and generations down the line may benefit from something we help accomplish.
Disclaimer: The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
Today's insights, the first in a series on this topic, are from a male in his fifties, who lives in the southeast.
1. How and why did you decide to participate in the trial?
I decided to participate in the trials due to my health concerns and those of my children. I wasn't sure if I could get any relief for my self but wanted to participate with the hope that it would benefit those who are younger and still have a lot of living to do. If I could help them relieve some of their issues thru the trials then I was all for it.
2. Was there anyone in particular who helped you decide whether to participate or to cope with any challenges?
Family and a fellow XLHer.
3. What do you know now that you wish you'd known before participating in your first trial?
The trials I have participated in have been well explained and there really wasn't any hidden info or circumstances. So really nothing.
4. Anything else you want to share with the Network's members?
I know people have different varying conditions of hypophosphatemia and everyone will feel different on trial medications, but my thought on the whole trial study is if you or I could help the next generations with this disease then why shouldn't we? It may not help us now but our children, their children, and generations down the line may benefit from something we help accomplish.
Disclaimer: The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
Tuesday, August 25, 2015
KRN23 adult clinical trials
The Phase 3 KRN23 clinical trials for adults with XLH are set to begin in October. The announcement indicates that they're not yet recruiting, but we'll update when we find out that recruiting has begun.
In the meantime, if you're considering participating in the trials, you can review the inclusion/exclusion criteria (available here: https://clinicaltrials.gov/ct2/show/NCT02526160?term=XLH&rank=1 ) and discuss your participation with trusted advisers.
You might also find the following sites useful for background information on participating in clinical trials and questions to ask:
http://www.nih.gov/health/clinicaltrials/basics.htm
Board member Kathy Buchanan, who has extensive experience as a clinical research nurse and is a certified clinical research coordinator, suggests that anyone considering participation in a trial should read all of the documents carefully, especially the consent form. Some questions that ought to be addressed by the paperwork, but that you may wish to ask about are: 1) how much time your involvement will require, 2) how often you'll be required to travel, 3) how often bloodwork and other lab work will be done, 4) what side effects may occur and how/when to report them. Finally, if payment is offered for participation, make sure you understand when the monies will be paid (e.g., at the end of the study or at shorter intervals).
In the meantime, if you're considering participating in the trials, you can review the inclusion/exclusion criteria (available here: https://clinicaltrials.gov/ct2/show/NCT02526160?term=XLH&rank=1 ) and discuss your participation with trusted advisers.
You might also find the following sites useful for background information on participating in clinical trials and questions to ask:
http://www.nih.gov/health/clinicaltrials/basics.htm
https://www.ciscrp.org/education-center/
Board member Kathy Buchanan, who has extensive experience as a clinical research nurse and is a certified clinical research coordinator, suggests that anyone considering participation in a trial should read all of the documents carefully, especially the consent form. Some questions that ought to be addressed by the paperwork, but that you may wish to ask about are: 1) how much time your involvement will require, 2) how often you'll be required to travel, 3) how often bloodwork and other lab work will be done, 4) what side effects may occur and how/when to report them. Finally, if payment is offered for participation, make sure you understand when the monies will be paid (e.g., at the end of the study or at shorter intervals).
Note that, as always, the XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.
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