NCT01379625

Brief Summary

Humans eat long-chain fat in their diet and use it for energy during exercise and during periods of fasting. Patients with long-chain fatty acid oxidation disorders cannot use dietary fat for energy. They sometimes develop muscle breakdown, and severe pain with exercise or illness. They can also develop a heart that does not function properly. These patients are tired and expend less energy than people who do not have a long-chain fatty acid oxidation disorder. However, they can use a supplement oil called medium chain triglyceride or MCT. This study will determine if a new experimental oil called Triheptanoin can decrease the muscle pain and increase the heart function and the amount of energy in patients with long-chain fatty acid oxidation disorders. Funding source - FDA's OOPD

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
32

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Sep 2011

Typical duration for phase_2

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 21, 2011

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 23, 2011

Completed
2 months until next milestone

Study Start

First participant enrolled

September 1, 2011

Completed
3.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2015

Completed
2 years until next milestone

Results Posted

Study results publicly available

March 23, 2017

Completed
Last Updated

March 23, 2017

Status Verified

March 1, 2017

Enrollment Period

3.6 years

First QC Date

June 21, 2011

Results QC Date

July 12, 2016

Last Update Submit

March 21, 2017

Conditions

Very Long-chain acylCoA Dehydrogenase (VLCAD) DeficiencyCarnitine Palmitoyltransferase 2 (CPT2) DeficiencyMitochondrial Trifunctional Protein (TFP) DeficiencyLong-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency

Keywords

fatty acid oxidationtriheptanoinlong-chain fatty acid oxidation disordersLCHADDVLCADDCPT2TFP

Outcome Measures

Primary Outcomes (2)

  • Energy Expenditure

    Total energy expenditure will be measured by doubly labeled water and resting energy expenditure will be measured by indirect calorimetry at baseline and again after 4 months of either MCT or trihpetanoin treatment.

    change from baseline after 4 months of treatment

  • Ejection Fraction

    Change in resting ejection fraction over 4 month treatment period

    4 months

Secondary Outcomes (1)

  • Exercise Heart Rate

    change from baseline to 4 months of treatment

Study Arms (2)

Medium Chain Triglyceride (MCT)

ACTIVE COMPARATOR

Subjects randomized to consume 20% of energy from MCT

Drug: Triheptanoin

Triheptanoin

EXPERIMENTAL

Subject randomized to consume 20% of energy from triheptanoin.

Drug: Triheptanoin

Interventions

TriheptanoinDRUG

Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone

Also known as: IND 113386, C7
Medium Chain Triglyceride (MCT)Triheptanoin

Eligibility Criteria

Age7 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Confirmed diagnosis of Very long-chain acylCoA dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency, or Long-chain 3 hydroxyacylCoA dehydrogenase (LCHAD) deficiency
  • \> 7 years
  • Ability to travel to CRC to participate
  • Ability to follow protocol

You may not qualify if:

  • Hgb \< 10 g/dl
  • Peripheral neuropathy that limits ability to complete treadmill studies
  • Pregnant females
  • history of myocardial infarction or cardiovascular disease

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Related Publications (1)

  • Gillingham MB, Heitner SB, Martin J, Rose S, Goldstein A, El-Gharbawy AH, Deward S, Lasarev MR, Pollaro J, DeLany JP, Burchill LJ, Goodpaster B, Shoemaker J, Matern D, Harding CO, Vockley J. Triheptanoin versus trioctanoin for long-chain fatty acid oxidation disorders: a double blinded, randomized controlled trial. J Inherit Metab Dis. 2017 Nov;40(6):831-843. doi: 10.1007/s10545-017-0085-8. Epub 2017 Sep 4.

    PMID: 28871440DERIVED

MeSH Terms

Conditions

VLCAD deficiency

Interventions

triheptanoin

Results Point of Contact

Title
Dr. Melanie Gillingham
Organization
Oregon Health & Science University
gillingm@ohsu.edu
503-494-1682

Study Officials

  • Melanie B Gillingham, PhD

    Oregon Health and Science University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Assistant Professor

Study Record Dates

First Submitted

June 21, 2011

First Posted

June 23, 2011

Study Start

September 1, 2011

Primary Completion

April 1, 2015

Study Completion

April 1, 2015

Last Updated

March 23, 2017

Results First Posted

March 23, 2017

Record last verified: 2017-03

Locations

US(2)