Special Statement on Clinical Trial Recruitment
The Glut1 Deficiency Foundation is a non-profit 501(c)3 patient and family organization dedicated to educating others about Glut1 Deficiency by creating a forum for sharing support, experiences, resources, and information between patients, families, and healthcare professionals; increasing awareness of and advocacy for Glut1 Deficiency; and supporting and funding researchers as they work for a cure. The Foundation is honored to be served by world-renowned Glut1 Deficiency and ketogenic diet experts through its Medical Advisory Board. The Foundation strives to fulfill its mission in the best interests of the global Glut1 Deficiency community.
The Foundation has recently been made aware of some activities, both in the United States and abroad, surrounding patient recruitment for an active clinical trials program for pharmaceutical (drug) grade triheptanoin. The protocol for these trials requires that patients wishing to enroll not be on a ketogenic diet, document a 6 week seizure baseline period, and then begin a double blind placebo treatment plan if certain seizure type and frequency benchmarks are met.
The Foundation recognizes that some patients are not compliant on a ketogenic diet or their symptoms are not well controlled. However, several instances have been reported to us where patient families have been contacted by their own or a different treatment center and offered the opportunity to take their child off a ketogenic diet in order to participate in the trials, even when the diet is effective and established in the patient. Patients would be required to stop the diet, go without treatment for 6 weeks, document any seizure frequency and type, then enroll in the treatment plan with possibility of a placebo.
It is the view of the Glut1 Deficiency Foundation Board of Directors and Medical Advisory Board that no patient should go without treatment or discontinue an effective one. We recommend consulting with a member of our Medical Advisory Board or another medical professional with significant expertise in Glut1 Deficiency before proceeding with changes in established treatment methods.
The ketogenic diet has been the standard of care for Glut1 Deficiency since the condition was first described over 20 years ago, and it remains the standard of care today with years of clinical and family experience to support its efficacy in treating seizures and many other symptoms associated with Glut1 Deficiency. Of particular importance, the diet provides nourishment to the brain during the critical childhood periods of brain growth and development. The Foundation recognizes that the ketogenic diet is not a perfect treatment and supports research leading to better treatments and ultimately a cure. However, the conduct of clinical trial investigations should not put prospective participants in a position to terminate or avoid an established effective treatment.
The Foundation is dedicated to promoting the wellbeing of each and every Glut1 Deficiency patient. We respectfully recognize that patients and families are in the best position to make decisions regarding the treatment of their loved ones. Not all patients are alike, and not all situations are the same. However, we urge families to consider carefully any changes in treatment or participation in studies and trials and to seek the expert guidance of medical professionals well versed in Glut1 Deficiency and in their child’s medical needs.
Glut1 Deficiency Foundation Board of Directors
Glut1 Deficiency Foundation Medical Advisory Board
The Foundation has recently been made aware of some activities, both in the United States and abroad, surrounding patient recruitment for an active clinical trials program for pharmaceutical (drug) grade triheptanoin. The protocol for these trials requires that patients wishing to enroll not be on a ketogenic diet, document a 6 week seizure baseline period, and then begin a double blind placebo treatment plan if certain seizure type and frequency benchmarks are met.
The Foundation recognizes that some patients are not compliant on a ketogenic diet or their symptoms are not well controlled. However, several instances have been reported to us where patient families have been contacted by their own or a different treatment center and offered the opportunity to take their child off a ketogenic diet in order to participate in the trials, even when the diet is effective and established in the patient. Patients would be required to stop the diet, go without treatment for 6 weeks, document any seizure frequency and type, then enroll in the treatment plan with possibility of a placebo.
It is the view of the Glut1 Deficiency Foundation Board of Directors and Medical Advisory Board that no patient should go without treatment or discontinue an effective one. We recommend consulting with a member of our Medical Advisory Board or another medical professional with significant expertise in Glut1 Deficiency before proceeding with changes in established treatment methods.
The ketogenic diet has been the standard of care for Glut1 Deficiency since the condition was first described over 20 years ago, and it remains the standard of care today with years of clinical and family experience to support its efficacy in treating seizures and many other symptoms associated with Glut1 Deficiency. Of particular importance, the diet provides nourishment to the brain during the critical childhood periods of brain growth and development. The Foundation recognizes that the ketogenic diet is not a perfect treatment and supports research leading to better treatments and ultimately a cure. However, the conduct of clinical trial investigations should not put prospective participants in a position to terminate or avoid an established effective treatment.
The Foundation is dedicated to promoting the wellbeing of each and every Glut1 Deficiency patient. We respectfully recognize that patients and families are in the best position to make decisions regarding the treatment of their loved ones. Not all patients are alike, and not all situations are the same. However, we urge families to consider carefully any changes in treatment or participation in studies and trials and to seek the expert guidance of medical professionals well versed in Glut1 Deficiency and in their child’s medical needs.
Glut1 Deficiency Foundation Board of Directors
Glut1 Deficiency Foundation Medical Advisory Board
