Expanded Access to Triheptanoin

March 1, 2024 updated by: Ultragenyx Pharmaceutical Inc

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)

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

Study Overview

Status

Available

Conditions

Intervention / Treatment

Detailed Description

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

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: Expanded access may be available outside of the US in countries prior to approval by the local regulatory agencies.

For full details, please visit the links provided below.

Study Type

Expanded Access

Expanded Access Type

  • Individual Patients
  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ultragenyx Pharmaceutical Inc

Investigators

  • Study Director: Medical Director, Ultragenyx Pharmaceutical Inc

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

December 10, 2018

First Submitted That Met QC Criteria

December 10, 2018

First Posted (Actual)

December 12, 2018

Study Record Updates

Last Update Posted (Estimated)

March 5, 2024

Last Update Submitted That Met QC Criteria

March 1, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • UX007-EAP
  • UX007G-EAP102 (Other Identifier: Ultragenyx Pharmaceutical Inc)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Clinical Trials on Triheptanoin

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Kyrgyzstan

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe