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Individual Patient Compassionate Use of Triheptanoin

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03773770
Expanded Access Status : Available
First Posted : December 12, 2018
Last Update Posted : September 4, 2019
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Brief Summary:
Individual patient expanded access requests may be considered for patients who have no other treatment options

Condition or disease Intervention/treatment
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Drug: Triheptanoin

Detailed Description:
Expanded access may provide access for treatment prior to approval by the local regulatory agency. For full details, please visit the link provided.

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Study Type : Expanded Access
Expanded Access Type : Individual Patients
  See clinical trials of the intervention/treatment in this expanded access record.

Resource links provided by the National Library of Medicine

Intervention Details:
  • Drug: Triheptanoin
    Other Name: UX007

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All

Inclusion Criteria:


Exclusion Criteria:


Information from the National Library of Medicine

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 identifier (NCT number): NCT03773770

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Contact: Early Access 1-415-483-8800

Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc

Additional Information:
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Responsible Party: Ultragenyx Pharmaceutical Inc Identifier: NCT03773770     History of Changes
Other Study ID Numbers: UX007-CU
First Posted: December 12, 2018    Key Record Dates
Last Update Posted: September 4, 2019
Last Verified: September 2019
Keywords provided by Ultragenyx Pharmaceutical Inc:
Expanded Access
Compassionate Use