Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies.



Status:Enrolling by invitation
Healthy:No
Age Range:Any
Updated:3/6/2019
Start Date:December 2014
End Date:September 2021

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An Open-label Long-Term Safety and Efficacy Extension Study in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007 or Triheptanoin Studies

The primary objective of this study is to evaluate the long-term safety and efficacy of UX007
in LC-FAOD subjects. The secondary objectives of this study are to evaluate the effect of
UX007 on energy metabolism in LC-FAOD and evaluate the impact of UX007 on clinical events
associated with LC-FAOD.


Inclusion Criteria:

1. Male or female, 6 months of age or older

2. Prior participation in a clinical study assessing UX007/triheptanoin treatment for LC
FAOD. Study Sponsors/Collaborators include: Oregon Health & Science University,
University of Pittsburgh, and Ultragenyx Pharmaceutical (ClinicalTrials.gov
Identifiers: NCT01379625, NCT01461304, and NCT01886378). Patients who received
UX007/triheptanoin treatment as part of other clinical studies; investigator sponsored
trials (IST); expanded access/compassionate use treatment programs; or patients who
are treatment naïve (i.e., naïve to both UX007 and food-grade triheptanoin), have
failed conventional therapy and, in the opinion of the investigator and sponsor, have
documented severe unmet need, may also be eligible at the discretion of the sponsor

3. Confirmed diagnosis of LC-FAOD including: carnitine palmitoyltransferase (CPT I or CPT
II) deficiency, very long chain acyl-CoA dehydrogenase (VLCAD) deficiency, long chain
3-hydroxy-acyl-CoA dehydrogenase (LCHAD) deficiency, trifunctional protein (TFP)
deficiency, or carnitine-acylcarnitine translocase (CACT) deficiency. Information on
diagnosis will be obtained from medical records and should include confirmed diagnosis
by results of acylcarnitine profiles, fatty acid oxidation probe studies in cultured
fibroblasts, and/or mutation analysis

4. Willing and able to complete all aspects of the study through the end of the study,
including visits and tests, documentation of symptoms and diet, and administration of
study medications. If a minor, have a caregiver(s) willing and able to assist in all
applicable study requirements

5. Provide written informed consent (subjects 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 study has been explained and
prior to any research-related procedures.

6. Females of child-bearing potential must have a negative urine pregnancy test at
Baseline and be willing to have additional pregnancy tests during the study. Females
considered not of child-bearing potential include those who have not experienced
menarche, are post-menopausal (defined as having no menses for at least 12 months
without an alternative medical cause), or are permanently sterile due to total
hysterectomy, bilateral salpingectomy, or bilateral oophorectomy

7. Participants of child‐bearing potential or fertile males with partners of
child-bearing potential who are sexually active must consent to use a highly effective
method of contraception as determined by the investigator from the period following
the signing of the informed consent through 30 days after last dose of study drug

Exclusion Criteria:

1. Diagnosis of medium-chain acyl coenzyme A dehydrogenase (MCAD) deficiency, short- or
medium-chain FAOD, ketone body metabolism defect, propionic acidemia or methylmalonic
acidemia

2. Patient qualifies for any other clinical trial designed to progressively evaluate the
safety and efficacy of triheptanoin in LC-FAOD

3. History of serious adverse reactions or known hypersensitivity to triheptanoin

4. Pregnant and/or breastfeeding an infant at Screening or planning to become pregnant
(self or partner) at any time during the study

5. Have any co-morbid conditions, including unstable major organ-system disease(s) that
in the opinion of the Investigator, places the subject at increased risk of
complications, interferes with study participation or compliance, or confounds study
objectives, or unwilling to discontinue prohibited medications
We found this trial at
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4202 E Fowler Ave
Tampa, Florida 33620
(813) 974-2011
University of South Florida The University of South Florida is a high-impact, global research university...
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1211 Medical Center Dr
Nashville, Tennessee 37232
(615) 322-5000
Vanderbilt Univ Med Ctr Vanderbilt University Medical Center (VUMC) is a comprehensive healthcare facility dedicated...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
University of Utah Research is a major component in the life of the U benefiting...
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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San Francisco, California 94143
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111 Michigan Ave NW
Washington, District of Columbia
(202) 476-5000
Childrens National Medical Center As the nation’s children’s hospital, the mission of Children’s National Medical...
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Washington,
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