Could a fatty acid drug stop dangerous sugar crashes in rare disease?
NCT ID NCT07097311
First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 1 time
Summary
This study tests a drug called triheptanoin, already approved for similar conditions, to see if it can prevent dangerously low blood sugar in people with MCADD, a rare inherited disorder. About 24 participants aged 4 and older will take the medication and be monitored for safety and how long their blood sugar stays stable. The goal is to find a new way to manage this condition and reduce the risk of hypoglycemia.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Expected to start
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Jul 2026
An estimate. Start dates often move.
- Expected to finish
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Apr 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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4 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. A diagnosis of MCADD with molecular confirmation 2. ≥4 years of age 3. Able to perform and comply with study activities placement of a continuous glucose monitor, IV catheter, and all blood draws. 4. Negative pregnancy test for all female subjects of childbearing age. 5. Signed informed consent by the subject or parent/guardian of minors. 6. 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. 7. Willing and able to adhere to requirements for maintaining continuous glucose monitoring. Exclusion Criteria: 1. Use of any investigational drug within 30 days of Screening (Visit 1). 2. Active infection (viral or bacterial) or any other intercurrent condition as reported by the subject or noted on physical exam at screening. 3. 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. 4. Known hypersensitivity to triheptanoin 5. Breastfeeding or lactating females. 6. Subjects with type 1 or type 2 diabetes, or who take medications as part of their routine care that can cause hypoglycemia 7. 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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Small study tests exercise safety in kids with MCADD
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