Study to Evaluate the Use of Triheptanoin in Patients Wit... | NCT07097311

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

NCT07097311

Sponsor

Jerry Vockley, MD, PhD

Status

Withdrawn

Last Update Posted

Jul 2, 2026Actual

Enrollment

0Actual

Phase

Phase 2

Conditions

Medium-chain Acyl-CoA Dehydrogenase Deficiency

Interventions

Triheptanoin

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jun 2026

Overall Recruitment Status or Expanded Access Status

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

funding was pulled

Expanded Access Info

Not provided

Start Date

Jul 2026Estimated

Primary Completion Date

Dec 2027Estimated

Completion Date

Apr 2028Estimated

First Submitted Date

Jul 24, 2025

First Submission Date that Met QC Criteria

Jul 24, 2025

First Posted Date

Jul 31, 2025Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jun 30, 2026

Last Update Posted Date

Jul 2, 2026Actual

Name	Class
Ultragenyx Pharmaceutical Inc	INDUSTRY

Description Module

Brief 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.

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

0Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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	Drug: Triheptanoin

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Triheptanoin	Drug	Open-label design with doses of triheptanoin up to 1.0 gm/kg/day. The dose will be titrated from Day 1-24.	1.0 gm/kg/day triheptanoin

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of participants with treatment related adverse events as assessed by CTCAE v5.0		8 weeks

Secondary Outcomes

Measure	Description	Time Frame
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	8 weeks
Length of time before glucose falls below 70 mg/dL

Other Outcomes

Measure	Description	Time Frame
Length of time before glucose falls below 80 mg/dL	Comparison of the length of time each participant's glucose level stays above 80 mg/dL, comparing their values from the run-in period to the results obtained between Visits 3 and 4	8 weeks

Eligibility Module

Eligibility Criteria

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.
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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

4 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Affiliation	Role
Gerard Vockley	UPMC Children's Hospital of Pittsburgh	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
Children's Hospital of Pittsburgh of UPMC		Pittsburgh	Pennsylvania	15224	United States

ID	Term
C536038	Medium chain acyl CoA dehydrogenase deficiency

ID	Term
C531010	triheptanoin

Intervention 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.