Medium-chain Acyl-CoA Dehydrogenase DeficiencyPhase 2NCT07097311

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

A Phase 2 study testing Triheptanoin in people with Medium-chain Acyl-CoA Dehydrogenase Deficiency, run by Jerry Vockley, MD, PhD at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record

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Trial summary

Who is it for?: Ages 4 years and older
Conditions the study is for: Medium-chain Acyl-CoA Dehydrogenase Deficiency
What is being tested?: Triheptanoin
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 0 participants
Where: 1 siteUnited States
Study dates: Jul 2026to Apr 2028
Phase: Phase 2
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 4 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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.

Who cannot join

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.
Known hypersensitivity to triheptanoin
Breastfeeding or lactating females.
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

Trial locations and contacts

1 site in 1 country.

United States1 site

Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224

Other trials in United States

About this study

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)

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.

Read the detailed description

Participation in the study will require four outpatient visits at the Clinical and Translational Research Center at the UPMC Children's Hospital of Pittsburgh (also called the PCTRC). The total length of the study participation is approximately 8 weeks.

Participants will come in for a screening visit (Visit 1) to where they will undergo some lab work testing to confirm they are eligible for the study. If it's determined that they are eligible, they will undergo training on the use of a continuous glucose monitor (CGM). They will be sent home and asked to wear the CGM for the next 10 days. This 10-day period is considered the run-in period.

Following the run-in period, participants will return to the site (Visit 2) in a fasting state. Fasting labs will be drawn, then the participant will be provided with a meal and they will then be dosed with triheptanoin. Following the dose of triheptanoin, the participant will be sent home with instructions on how to titrate (increase) the dose of triheptanoin over the next 28 days.

Following the 28 day dose titration period, the participant will return to the site (Visit 3) in a fasting state. Fasting labs will be drawn, then the participant will be provided with a meal and then will then be dosed with triheptanoin Following the dose of ACER-001, the participant will be sent home with continuous glucose monitoring and will be asked to log their triheptanoin doses as well as overnight fasting times.

Triheptanoin doses will be weight-based with the maximum dose being 1.0 gm/kg daily divided into 3 or 4 doses.

Participants will be asked to return approximately 2 weeks later for Visit 4 to complete the same procedures outlined for Visit 3.

Study staff will contact the participant by phone approximately 1 week after Visit 4 to follow up on any adverse events.

All study procedures will be done at no cost to the participants.

Conditions: Medium-chain Acyl-CoA Dehydrogenase Deficiency
Being tested: Triheptanoin
Keywords: Medium-chain Acyl-CoA Dehydrogenase Deficiency
Medical subject headings: Medium chain acyl CoA dehydrogenase deficiency

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

TriheptanoinDrug
Also called Dojolvi
Open-label design with doses of triheptanoin up to 1.0 gm/kg/day. The dose will be titrated from Day 1-24.

Study groups in NCT07097311
Study group	Role	What they receive
1.0 gm/kg/day triheptanoin	Experimental	Up to 24 subjects (8: 4-9 years of age, 8: 10-15 years of age; 8: 16 years of age or older) will take 1.0 gm/kg/day divided into 3 or 4 doses Receives: Drug: Triheptanoin

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of participants with treatment related adverse events as assessed by CTCAE v5.0
Measured: 8 weeks

Secondary outcomes (2)

Length of time before glucose falls below 60 mg/dL
Comparison of the length of time each participant's glucose level stays above 60 mg/dL, comparing their values from the run-in period to the results obtained between Visits 3 and 4
Measured: 8 weeks
Length of time before glucose falls below 70 mg/dL
Comparison of the length of time each participant's glucose level stays above 70 mg/dL, comparing their values from the run-in period to the results obtained between Visits 3 and 4
Measured: 8 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jul 2026Apr 2028

First posted31 Jul 2025
Study startJul 2026 (estimated)
Primary completionDec 2027 (estimated)
Study completionApr 2028 (estimated)

Why it stopped: funding was pulled

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Medium-chain Acyl-CoA Dehydrogenase DeficiencyStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: The study will include 3 cohorts with up to 8 participants enrolled in each cohort. Cohort 1: ≥ 16 years of age; Cohort 2: 10-15 years of age; Cohort 3: 4-9 years of age. At least 2 participants will be enrolled in Cohort 1 to assess for any safety signals. If no safety issues are identified, enrollment in Cohort 2 will begin. At least 2 participants will be enrolled in Cohort 2 to assess for any safety signals before opening enrollment in Cohort 3.
Masking: None (open label)
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT07097311
Sponsor's protocol ID: STUDY25050173
Status last verified: Jun 2026
First submitted: 24 Jul 2025
Last updated: 2 Jul 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2
Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Active, not recruitingPhase 3
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
RecruitingObservational study
Natural History of MADD
Multiple Acyl-CoA Dehydrogenase Deficiency
Recruiting
Metabolic Effects of Medium-Chain Fatty Acids in Patients With Medium-Chain Acyl-CoA Dehydrogenase Deficiency and Healthy Individuals
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome

All Medium-chain Acyl-CoA Dehydrogenase Deficiency trials

Study at a glance

From the registry record, last updated by the sponsor on 2 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

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)
ClinicalTrials.gov ID: NCT07097311
Acronym / sponsor ID: STUDY25050173
Conditions: Medium-chain Acyl-CoA Dehydrogenase Deficiency
Keywords: Medium-chain Acyl-CoA Dehydrogenase Deficiency
Phase: Phase 2
Study type: Interventional
Recruitment status: WithdrawnReason stopped: funding was pulled
Enrolment: 0 participants (actual)
Lead sponsor: Jerry Vockley, MD, PhDUniversity, hospital or other
Collaborators: Ultragenyx Pharmaceutical Inc
Study start: Jul 2026 (estimated)
Primary completion: Dec 2027 (estimated)
Study completion: Apr 2028 (estimated)
First posted: 31 Jul 2025 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Triheptanoin doses will be weight-based with the maximum dose being 1.0 gm/kg daily divided into 3 or 4 doses.

Participants will be asked to return approximately 2 weeks later for Visit 4 to complete the same procedures outlined for Visit 3.

Study staff will contact the participant by phone approximately 1 week after Visit 4 to follow up on any adverse events.

All study procedures will be done at no cost to the participants.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: The study will include 3 cohorts with up to 8 participants enrolled in each cohort. Cohort 1: ≥ 16 years of age; Cohort 2: 10-15 years of age; Cohort 3: 4-9 years of age. At least 2 participants will be enrolled in Cohort 1 to assess for any safety signals. If no safety issues are identified, enrollment in Cohort 2 will begin. At least 2 participants will be enrolled in Cohort 2 to assess for any safety signals before opening enrollment in Cohort 3.

Arm	Type	Receives
1.0 gm/kg/day triheptanoin	Experimental	Drug: TriheptanoinUp to 24 subjects (8: 4-9 years of age, 8: 10-15 years of age; 8: 16 years of age or older) will take 1.0 gm/kg/day divided into 3 or 4 doses

Who may qualify

Ages: 4 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (7 listed)

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.

Key exclusion criteria (7 listed)

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.
Known hypersensitivity to triheptanoin
Breastfeeding or lactating females.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
TriheptanoinAlso: DojolviOpen-label design with doses of triheptanoin up to 1.0 gm/kg/day. The dose will be titrated from Day 1-24.	Drug	1.0 gm/kg/day triheptanoin

Primary outcome

Number of participants with treatment related adverse events as assessed by CTCAE v5.0
Time frame: 8 weeks

2 secondary outcomes

Length of time before glucose falls below 60 mg/dL
Time frame: 8 weeks
Length of time before glucose falls below 70 mg/dL
Time frame: 8 weeks

Results and safety

The study was withdrawn before enrolling participants, so there are no results.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2026.
Individual participant dataSharing planned: No

Related trials for Medium-chain Acyl-CoA Dehydrogenase Deficiency

RecruitingPhase 2
Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Active, not recruitingPhase 3
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
RecruitingObservational study
Natural History of MADD
Multiple Acyl-CoA Dehydrogenase Deficiency
Recruiting
Metabolic Effects of Medium-Chain Fatty Acids in Patients With Medium-Chain Acyl-CoA Dehydrogenase Deficiency and Healthy Individuals
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Gerard Vockley principal investigator

UPMC Children's Hospital of Pittsburgh

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Triheptanoin in people with Medium-chain Acyl-CoA Dehydrogenase Deficiency, run by Jerry Vockley, MD, PhD at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 2 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with treatment related adverse events as assessed by CTCAE v5.0 (time frame: 8 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07097311.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jul 2026.