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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 10 weeks

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 10 weeks

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of participants with treatment related adverse events as assessed by CTCAE v5.0

Secondary outcome measures

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%

Fever

33%

Headache

33%

Stomach Virus

17%

Convulsive Seizures

17%

Skin Scale Hands

17%

Rash-Right Leg

17%

Decreased Appetite

17%

Lethargy

17%

Hand Pain

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%

Study treatment Arm

Schedule A

Schedule B

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

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

Jerry Vockley, MD, PhDLead Sponsor

4 Previous Clinical Trials

424 Total Patients Enrolled

Ultragenyx Pharmaceutical IncIndustry Sponsor

89 Previous Clinical Trials

179,067 Total Patients Enrolled

Gerard Vockley, MD, PhDPrincipal InvestigatorUPMC Children's Hospital of Pittsburgh

3 Previous Clinical Trials

404 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is Triheptanoin reported to cause any adverse effects for individuals?

"After evaluating the safety profile of Triheptanoin, our team assigned it a rating of 2. This is due to limited efficacy data but some evidence that this medication can be used safely."

Answered by AI

How many participants can enroll in this experiment?

"Indeed, information accessible on clinicaltrials.gov reveals that this medical experiment is currently searching for volunteers. It was initially published on October 30th 2023 and most recently edited on September 27th 2023. The investigation requires 8 participants to be enrolled from a single site."

Answered by AI

Are there any open slots for participation in this scientific investigation?

"Indeed, the clinicaltrials.gov webpage confirms that this trial is actively enrolling subjects. The initial post was on October 30th 2023 and most recently updated September 27th 2023. 8 patients are required for admission to one medical center."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Jerry Vockley, MD, PhD 2024. "Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06067802.

All > Hypoglycemia