Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase De… (NCT07097311) | Clinical Trial Compass
RecruitingPhase 2
Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
United States24 participantsStarted 2026-07
Plain-language summary
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.
Who can participate
Age range4 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. A diagnosis of MCADD with molecular confirmation
✓. ≥4 years of age
✓. Able to perform and comply with study activities placement of a continuous glucose monitor, IV catheter, and all blood draws.
✓. Negative pregnancy test for all female subjects of childbearing age.
✓. Signed informed consent by the subject or parent/guardian of minors.
✓. All females of childbearing age and all sexually active males must agree to use an acceptable method of contraception throughout the study. Appropriate contraceptive methods include hormonal contraceptives (oral, injected, implanted, or transdermal), tubal ligation, intrauterine device, hysterectomy, vasectomy, or double barrier methods. Abstinence is an acceptable form of birth control, though appropriate contraception must be used if the subject becomes sexually active.
✓. Willing and able to adhere to requirements for maintaining continuous glucose monitoring.
Exclusion criteria
✕. Use of any investigational drug within 30 days of Screening (Visit 1).
✕. Active infection (viral or bacterial) or any other intercurrent condition as reported by the subject or noted on physical exam at screening.
✕. Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may put the subject at increased risk by participating in this study.
What they're measuring
1
Number of participants with treatment related adverse events as assessed by CTCAE v5.0
✕. Subjects with type 1 or type 2 diabetes, or who take medications as part of their routine care that can cause hypoglycemia
✕. Subjects who are taking medications in the antimetabolite drug class (e.g., hydroxyurea, 5-fluorouracil (5-FU), methotrexate) will be excluded; these medications can interfere with the DEXCOM sensor and cause inaccurate glucose readings