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A Study to Determine the Effect of Triheptanoin Compared with Even-chain MCT on MCEs in Pediatric Patients with LC-FAOD
EU CTIS ID: 2023-509809-76-00
What this study is testing
Evaluate the effect of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of major clinical events (MCE)
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Confirmed diagnosis of LC-FAOD: carnitine palmitoyl transferase (CPT) I deficiency, CPT II deficiency, carnitine/acylcarnitine translocase (CACT) deficiency, very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency, long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency, or mitochondrial trifunctional protein (TFP) deficiency. Diagnosis must be confirmed by results of acylcarnitine profiles, fatty acid oxidation probe studies in cultured fibroblasts, or mutation analysis obtained from medical records
- 2. Males and females, from 0 (including newborns) to < 18 years of age at time of randomination
- 3. Have a caregiver(s) willing and able to assist in all applicable study requirements
- 4. 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, and the study subject to be able to provide age-appropriate written assent
- 5. Have ANY ONE of the following significant clinical manifestations of LC-FAOD: - At least 2 in the prior year, or 3 in the prior 2 years, of severe major episodes of metabolic decompensation (eg, hypoglycemia, rhabdomyolysis, or exacerbation of cardiomyopathy, requiring ER/urgent care unit visits or hospitalizations) - Recurrent symptomatic hypoglycemia (clinical symptoms of hypoglycemia) requiring intervention - Susceptibility to hypoglycemia after short periods of fasting (less than 4 to 12 hours, depending on age) - Evidence of functional cardiomyopathy requiring ongoing medical management or clinical manifestation of heart failure - Sibling(s) with the same pathogenic variant who presented with MCEs - Subject with pathogenic variants that are known or suspected to be associated with absent or severely reduced enzyme activity or with severe disease manifestations.
- 6. From the time of informed consent to 5 days after the last dose of study drug in this study, females of childbearing potential and fertile males must consent to use highly effective methods of contraception as described in Appendix 2. If female, agree not to become pregnant. If male, agree not to father a child or donate sperm. Inclusion Criteria for Liver Sub-study: 1. Enrollment in the Main Study of Study UX007-CL302 2. Age > 2 years 3. Liver fat content ≥ 2% and < 20% PDFF as assessed by 1H-MRS 4. Body mass index < 95th percentile 5. Able to comply with instructions (remaining still during scan) and requirements (eg, constraints on recent meals, no metallic items or implanted devices in the body, no recent contrast agents) for liver HMRS scan
You likely can't join if
- 1. Enrolled in a clinical study involving concurrent use of an investigational drug product within 30 days before Screening 2. Use of a prohibited medication (eg, valproate products or pancreatic lipase inhibitors) within 30 days before Screening, or unwilling to avoid a prohibited medication or other substance that may confound study objectives 3. Treatment with triheptanoin within 60 days of Screening 4. History of known hypersensitivity to triheptanoin or MCT or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects 5. Caregiver unwilling or unable to sign informed consent, or release of medical records, or follow study procedures 6. Have any comorbid conditions, including unstable major organsystem 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 interpretation of results. History of metabolic decompensation(s) with metabolic acidosis, hyperammonemia, and/or liver enzyme elevations does not constitute an exclusion criterion unless 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 interpretation of results. 7. Have a diagnosis of pancreatic insufficiency 8. Pregnant, breastfeeding, or planning to become pregnant (self or partner) at any time during the study. Exclusion Criteria for Liver Sub-study 1. Acute or chronic liver disease other than LC-FAOD that presents with increased risk of liver fat (eg, hepatic cirrhosis, viral toxic or drug hepatitis, diabetes mellitus) and/or metabolic syndrome 2. Need for anesthesia/sedation to perform liver H-MRS
The study team makes the final eligibility decision.
Where it's taking place
- Saudi Arabia
- Turkey
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Saudi Arabia; Turkey. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.