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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
Sponsor:
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

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
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.

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.

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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)



Intervention Details:
  • Drug: Triheptanoin
    Liquid for oral (PO) or enteral tube administration
    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
Criteria

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.

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 ClinicalTrials.gov identifier (NCT number): NCT03773770


Contacts
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Contact: Early Access 1-415-483-8800 EarlyAccess@ultragenyx.com

Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Investigators
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Study Director: Medical Director Ultragenyx Pharmaceutical Inc
Additional Information:
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Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT03773770    
Other Study ID Numbers: UX007-EAP
UX007G-EAP102 ( Other Identifier: Ultragenyx Pharmaceutical Inc )
First Posted: December 12, 2018    Key Record Dates
Last Update Posted: August 27, 2020
Last Verified: August 2020
Keywords provided by Ultragenyx Pharmaceutical Inc:
Expanded Access
Compassionate Use
Carnitine Palmitoyltransferase Deficiency
CPT I
CPT II
Very Long Chain acyl-CoA Dehydrogenase Deficiency
VLCAD
Long Chain 3-hydroxy-acyl-CoA Dehydrogenase Deficiency
LHCAD
Trifunctional Protein Deficiency
TFP
Carnitine-acylcarnitine Translocase Deficiency
CACT
Long Chain Fatty Acid Oxidation Disorders
LC-FAOD
Additional relevant MeSH terms:
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Carbohydrate Metabolism, Inborn Errors
Syndrome
Disease
Pathologic Processes
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases