Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Jerry Vockley, MD, PhD
With
Ultragenyx Pharmaceutical Inc
This treatment plan is intended for the use of UX007 (triheptanoin) in the treatment of a single patient with Glucose Transporter 1 (GLUT1) Deficiency Syndrome. Patient has already tried the other acceptable treatments for GLUT1 with either side effects that caused treatment to be discontinued (Ketogenic Diet) or a current treatment that is not showing any efficacy and is difficult to sustain for a long duration given this patient's age (modified Atkins diet). Due to the patient's ongoing neuroregression and lack of other viable treatments, the use of UX007 (triheptanoin) is warranted. There is no comparable or satisfactory alternative therapy to treat this patient's severely debilitating condition and the potential benefits to the patient justify the potential risks of the treatment. The potential risks from UX007 (triheptanoin) are not unreasonable given the context of this patient's condition.
Age
3–any
Sex
FEMALE
Healthy volunteers
Not accepted
You may be eligible if
This treatment plan is intended for the use of UX007 (triheptanoin) in the treatment of a single patient with Glucose Transporter 1 (GLUT1) Deficiency Syndrome.
You may not be if
Not applicable as study only includes one patient.
Clareo Health | Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome
