| Eligibility |
Inclusion Criteria:
Criteria per Intermediate-Size Population Protocol for Glut1 DS
- Confirmed diagnosis of Glut1 DS by documented SLC2A1 mutation or documented
improvement on other forms of tripheptanoin administered based on a clinical
presentation consistent with Glut1 DS diagnosis, including cerebrospinal fluid glucose
levels.
- Patients of any age who are seriously ill and, in the Treating Physician's opinion,
experiencing clinical manifestations of Glut1 DS despite other management.
- Willing and able to comply with all aspects of the treatment, including visits and
tests specified by the Treating Physician, documentation of symptoms and diet, and
administration of triheptanoin. If a minor, have a caregiver(s) willing and able to
assist in all applicable treatment requirements.
- Provide written informed consent (patients aged = 18 years), or provide written assent
(where appropriate) and have a legally authorized representative willing and able to
provide written informed consent, after the nature of the treatment program has been
explained and prior to any treatment-related procedures.
Exclusion Criteria:
Criteria per Intermediate-Size Population Protocol for Glut1 DS
- Patient qualifies for any other clinical trial designed to progressively evaluate the
safety and efficacy of tripheptanoin in Glut1 DS.
- Any known hypersensitivity to triheptanoin that, in the judgement of the Treating
Physician, places the patient at an increased risk for adverse events.
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