Expanded Access to Triheptanoin

Sponsor

Ultragenyx Pharmaceutical Inc (Industry)

Overall Status

Available

CT.gov ID

NCT03773770

Collaborator

(none)

Study Details

Study Description

Brief Summary

Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.

Condition or Disease	Intervention/Treatment	Phase
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)	Drug: Triheptanoin

Detailed Description

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS): Available through Intermediate-Size Population Expanded Access.

The intermediate-size expanded access treatment protocol is intended to provide rapid access to triheptanoin for the treatment of seriously ill patients with Glut1 DS.

Consideration for access is for patients with previous exposure to triheptanoin.

Patients will be treated under this protocol for the duration of one year, with consideration on a yearly basis for extension of treatment based on the risk-benefit ratio assessed in the Treating Physician's quarterly progress reports. Patients may continue to receive triheptanoin under this intermediate-size treatment protocol until commercial availability of triheptanoin, should the drug receive regulatory approval for the specific disease indication.

Long Chain Fatty Acid Oxidation Disorders (LC-FAOD) and Non-FAOD conditions: Available outside of the United States through individual named patient compassionate access requests.

Expanded access may provide access for treatment prior to approval by the local regulatory agency.

For full details, please visit the link provided.

Study Design

Study Type:

Expanded Access

Official Title:

An Open-label Intermediate-size Treatment Protocol for the Urgent Treatment of Seriously Ill Patients With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) With Triheptanoin (UX007)

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

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.