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Triheptanoin for MCADD

Phase 2
Recruiting
Led By Gerard Vockley, MD, PhD
Research Sponsored by Jerry Vockley, MD, PhD
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Able to perform and comply with study activities including overnight admission to the research unit at UPMC Children's Hospital Pittsburgh, placement of an IV catheter, and all blood draws
Negative pregnancy test for all female subjects of childbearing age
Must not have
Active infection (viral or bacterial) or any other intercurrent condition as reported by the subject or noted on physical exam at screening
Diagnosis of pancreatic insufficiency or concomitant use of a pancreatic lipase inhibitor (e.g. Orlistat) which can interfere with absorption of triheptanoin
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 10 weeks
Awards & highlights
No Placebo-Only Group

Summary

This trial will study the safety & efficacy of triheptanoin as a treatment for MCADD in adult patients. Previous research suggests it may be effective.

Who is the study for?
Adults over 16 with MCADD, able to stay overnight for study activities at UPMC Children's Hospital Pittsburgh. Participants must not be pregnant or breastfeeding, agree to use contraception, and have no liver disease or diabetes. They can't have used investigational drugs recently or take meds that affect blood sugar levels.
What is being tested?
The trial is testing Triheptanoin in adults with MCADD to see if it's safe and effective. Triheptanoin is already approved for other metabolic disorders but might help prevent low blood sugar in MCADD too.
What are the potential side effects?
Potential side effects of Triheptanoin are not detailed here, but generally could include digestive issues due to its nature as a dietary supplement designed to provide an alternative energy source.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can stay overnight at the hospital, have an IV placed, and undergo blood tests.
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I am not pregnant.
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I have been diagnosed with MCAD deficiency confirmed by genetic testing.
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I am 16 years old or older.
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I have given my consent, or if under 18, I and my parents have consented.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I do not have any active infections or other ongoing health issues.
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I have pancreatic insufficiency or I'm taking medication that affects fat absorption.
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I have diabetes or take medication that can lower my blood sugar.
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My liver tests are higher than normal.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~10 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 10 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Normalization of biochemical markers of disease (plasma acylcarnitine)
Normalization of biochemical markers of disease (urine acylglycine)

Side effects data

From 2019 Phase 2 trial • 20 Patients • NCT02036853
83%
Diarrhea
33%
Headache
33%
Fever
33%
Stomach Virus
17%
Rash-Right Leg
17%
Decreased Appetite
17%
Hand Pain
17%
Convulsive Seizures
17%
Lethargy
17%
Skin Scale Hands
17%
Tongue Pain
17%
Gastric Reflux
17%
Urinary Incontinence
17%
Amenorrhea
17%
Otalgia
17%
Hirsutism
17%
Abdominal Discomfort
17%
Constipation
17%
Emesis
17%
Nausea
17%
Nasal Fracture
17%
Right Arm Fracture
17%
Decreased Hematocrit
17%
Weight Gain
17%
Seizure
17%
Insomnia
17%
Moodiness
17%
Upper Respiratory Infection
17%
Impetigo
17%
Onychomycosis
17%
Rhinovirus
100%
80%
60%
40%
20%
0%
Study treatment Arm
Schedule A
Schedule B

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: TriheptanoinExperimental Treatment1 Intervention
Open label study
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Triheptanoin
2020
Completed Phase 2
~200

Find a Location

Who is running the clinical trial?

Ultragenyx Pharmaceutical IncIndustry Sponsor
93 Previous Clinical Trials
104,328 Total Patients Enrolled
Jerry Vockley, MD, PhDLead Sponsor
4 Previous Clinical Trials
424 Total Patients Enrolled
Gerard Vockley, MD, PhDPrincipal InvestigatorUPMC Children's Hospital of Pittsburgh
3 Previous Clinical Trials
404 Total Patients Enrolled
~5 spots leftby Mar 2026
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