Clinical Trial Experiences: Community Survey
The G1DF recently conducted a survey to get a better understanding of the clinical trial experiences in our community. We are engaged with researchers and companies who are working to develop better and easier treatments for Glut1 Deficiency patients. We are thankful they value and want to hear the patient voices and that we have these opportunities to share what is important and most meaningful to all of us.
As clinical trials are being designed, we want to share what has worked well in the past in terms of the burden for patients and families to be able to participate, and we also want to be able to offer suggestions for what needs to be done differently in the future.
We want to share some of the insights learned below, and we sincerely thank all who took time to share!
- Having them at the conferences
- Communication
- Explanation of goals of the trial, scheduling, and care from the team
- Having support and an actual person to ask when we had questions. It seemed like they really cared
- That travel, food, and accommodation was arranged for us and funded by the study facilitator. Without this we wouldn’t have been able to participate
- Having the flight and hotel covered financially
- The trials we have been able to be a part of, have been at the conferences, which have been very helpful for us, as we have not incurred any additional expenses. We are not afraid to travel for a clinical trial; however, we have not heard of any to participate in, except the ones at the conferences
- It was very encouraging to be able to take part in the research study, to feel we were making a difference
- They set up hotel rooms for us and accommodated my other kids to come along as we had to drive 6 hours to the hospital each trip. The coordinator was helpful when we had issues or questions
- Their making it as comfortable as possible
- We’ve been a part of many trials and those that leave us with a sense of purpose are the trials where all steps and purposes and our uses of the trial are thoroughly explained
- Everything was smooth and the team helped tremendously by explaining all the various steps. We were only a few months into post diagnosis and still learning a lot. Our anxiety was very high and the team helped keep us calm by walking us through every stage
- The process was easy and we felt very supported
- The enrollment, travel, the whole process was pretty straightforward and painless. Everyone was super helpful with everything
- Not knowing or understanding the results
- did not hear back about results, child had adverse effects
- No complaints/ except the trial did not help our daughter
- We needed to travel and pay travel costs for one of the studies
- Because we were not local we had to take time off work and take the children out of school
- Trial was great Flights were late luggage lost many times
- Nothing
- We would of appreciated follow up information regarding how our involvement in the trial was used and progressed the research
- Took off a lot time from work for travel
- The c7 oil did not work for my daughter (who is not on a MAD or keto diet) and caused her to stop eating food due to feeling full. We did not see a benefit in continuing the trial so we dropped out.
- Cooperation of the patient
- The C7 trial delivery of the oil was a disaster. We lost a good quantity of it due to its having broken during shipment which resulted in fed ex refusing to deliver it from their warehouse requiring us to travel a good distance and figure out how to travel home with a damage 5 gallon container of oil
- I can’t think of anything
- My daughter had an adverse reaction during the trial
- We did the C7 trial and there were many trials and tribulations to get our kiddo to take down the oil. It wasn’t anything negative from the trial itself, just some hardships that it put on us to be a part of it.
- Explanation of the results
- more transparency, follow up
- Nothing
- It was super helpful to have virtual visits when the in-person visit didn’t include an exam of any type
- More feedback from clinicians during trial
- We feel the two/three trials we’ve participated in, have gone really well for the participant
- Information to be shared between researcher and family of the progress
- The trial was set up wonderfully. It would be nice to have an option to participate in a trial without the upfront travel expenses though
- No opinion
- It would be good to always be sent the published findings
- Understanding that as parents we are not doctors or research experts. One of the staff helping while we were in the hospital assumed we knew something and reacted in a way that made us feel like bad parents. Being new to Glut1 and new to understanding petite seizures it was tough to swallow. Nothing terrible, but years later it has still stuck with me
- N/a
- I am not sure if there was a lot that could have been done differently. The one thing I would like to know is the result of the trial. We never had follow up once we were finished.
- People conducting the trials are nice
- probably would not put them through another trial depending on type
- No
- Could we have more research studies?
- No17 days ago
- We are appreciative and continue to be interested in participating .I wish the locations were more accessible
- Keep the information flowing about new clinical trials and how/when patients can participate
- We took part many years ago, protocols may well be different now
- Would like to participate in more trials but most are very far from home
- No. I’d love for my daughter to participate in any clinic trial that’s appropriate for her
- She’s been in two and both have been great!
- We’ve had wonderful experiences participating in Glut1 trials and we’re thankful for their existence.
- We really appreciate the team leading the way for those of us out here making it day to day.
- Lack of time on our part
- I didnt know about it
- Not one available
- None have been available to us
- Nothing that seemed of interest to us
- None available for us
- lack of time
- Our diagnosis is relatively new – only about four years into this journey. I am not aware of any clinical trials that we would have been eligible for in that timeframe
- Distance, time, availability, match for trial. I have participated in studies but no trials
- Not sure, I think because we thought that they couldn’t already be in the ketogenic diet
- We did not qualify for inclusion criteria/timing of the study (they had already recruited their subjects)
- We were not offered or made aware of the opportunity
- Nothing offered
- There has not been many clinical trial that we have known about and the ones we did know about we didn’t think was best for our child.
- We haven’t been asked to
- He is 40yo and hasn’t fit criteria that I know of
- we didn’t meet criteria
- Didn’t know about it
- First opportunity required going off keto for an extended time, and then risked the chance of receiving placebo treatment. Our Glut1 kiddo was recently diagnosed, not yet two years old, and obtained extended seizure control. The risk wasn’t worth it at the time. Since then, we have only heard of one trial opportunity but it was too difficult to have continued communication via email
- They didn’t seem to fit our symptoms
- Not aware of any
- Newly diagnosed
- Never been offered one
- I didn’t have this opportunity
- Not sure
- There are no clinical trials where I live
- Hasn’t been one that fit her
- We have not been offered any, I think the triheptanoin trial was being talked about but because my daughter hadn’t had seizures often enough she wasn’t eligible
- It was never offered to us
- Not had the opertunity
- We had no possibility
- Was not aware of a trial near us.
- Nothing seems to work
- There was a $1,000 stipend for travel and our flights alone would have been $950 so we couldn’t financially support a research trip
- Haven’t had the opportunity
- Not aware
- We never knew there was an option for a clinical trial of any kind for Glut1
- Haven’t heard of one
- She’s only 14 months and we are busy with pt ot speech and Dr appointments
- Not offered
- Besides a speech research study, we haven’t seen/pursued an opportunity to be involved because of an already overwhelming list of responsibilities. We also don’t know many other families in the glut1 community or been to any glut 1 events so haven’t felt plugged in enough to be aware of opportunities
- She did not meet the trial criteria
- None available in Canada
- Firstly, there have been very few clinical trials to our knowledge. We have given blood samples at various times for various purposes but those are not clinical trials. The only trials we’ve been aware of we haven’t been eligible for due to being on the ketogenic diet. One other trial involved an hours long unsedated MRI test that would not have worked for our child in addition to the fact that he has an orthodontic appliance permanently installed that would have had to be removed in order to participate
- I have no idea about them. Have not come across any
- We didn’t find any. Don’t know how to find the information
- Time and awareness
- Only recently diagnosed
- We live far from where the trials are being done
- No invitation
- Travel expenses
- We don’t have the opportunity in the country we live in
- Don’t know how to find them or enroll in one
- We haven’t been asked yet because our son has the diagnosis new
- Didn’t have a chance for one
- Haven’t needed to
- Concerned with potential side effects of the treatment arm
