Actively Recruiting
Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Led by Jerry Vockley, MD, PhD · Updated on 2026-03-05
24
Participants Needed
1
Research Sites
91 weeks
Total Duration
On this page
Sponsors
J
Jerry Vockley, MD, PhD
Lead Sponsor
U
Ultragenyx Pharmaceutical Inc
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a medical research study to test a medication in patients 4 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD). The medication is triheptanoin, which is currently FDA approved for the treatment of long chain fatty acid oxidation disorders (FAODs). Previous research suggests that triheptanoin may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of triheptanoin patients with MCADD.
CONDITIONS
Official Title
Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of MCADD with molecular confirmation
- 4 years of age or older
- Able to perform and comply with study activities including placement of a continuous glucose monitor, IV catheter, and blood draws
- Negative pregnancy test for females of childbearing age
- Signed informed consent by subject or parent/guardian for minors
- Agree to use acceptable contraception throughout the study if sexually active (acceptable methods include hormonal contraceptives, tubal ligation, intrauterine device, hysterectomy, vasectomy, double barrier methods, or abstinence)
- Willing and able to maintain continuous glucose monitoring requirements
You will not qualify if you...
- Use of any investigational drug within 30 days before screening
- Active viral or bacterial infection or other condition at screening
- Any medical condition or lab abnormality that may increase risk in the investigator's judgment
- Known allergy to triheptanoin
- Breastfeeding or lactating females
- Diagnosis of type 1 or type 2 diabetes or use of medications that cause hypoglycemia
- Taking antimetabolite drugs such as hydroxyurea, 5-fluorouracil, or methotrexate that interfere with glucose monitoring
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
Research Team
E
Elizabeth McCracken
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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