Expanded Access to Triheptanoin
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|ClinicalTrials.gov Identifier: NCT03773770|
Expanded Access Status : Available
First Posted : December 12, 2018
Last Update Posted : August 27, 2020
|Condition or disease||Intervention/treatment|
|Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)||Drug: Triheptanoin|
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.
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 Type :||Expanded Access|
|Expanded Access Type :||Individual Patients, Intermediate-size Population|
|See clinical trials of the intervention/treatment in this expanded access record.|
|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)|
- Drug: Triheptanoin
Liquid for oral (PO) or enteral tube administrationOther Name: UX007
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03773770
|Contact: Early Access||1-415-483-8800||EarlyAccess@ultragenyx.com|
|Study Director:||Medical Director||Ultragenyx Pharmaceutical Inc|