RECRUITING

Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Conditions

Medium-chain Acyl-CoA Dehydrogenase Deficiency

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

A Phase II, Open Label Study to Evaluate the Safety of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Quick Facts

Study Start:2025-11

Study Completion:2027-03

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT07097311

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:4 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	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

Inclusion Criteria

Exclusion 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.

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

Contacts and Locations

Study Contact

Elizabeth McCracken

CONTACT

412-692-5662

elizabeth.mccracken@chp.edu

Principal Investigator

Gerard Vockley

PRINCIPAL_INVESTIGATOR

UPMC Children's Hospital of Pittsburgh

Study Locations (Sites)

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224

United States

Collaborators and Investigators

Sponsor: Jerry Vockley, MD, PhD

Gerard Vockley, PRINCIPAL_INVESTIGATOR, UPMC Children's Hospital of Pittsburgh

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-11

Study Completion Date2027-03

Study Record Updates

Study Start Date2025-11

Study Completion Date2027-03

Terms related to this study

Keywords Provided by Researchers

Medium-chain Acyl-CoA Dehydrogenase Deficiency

Additional Relevant MeSH Terms

Medium-chain Acyl-CoA Dehydrogenase Deficiency