We are updating the design of this site. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder (Triheptanoin)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01379625
First Posted: June 23, 2011
Last Update Posted: March 23, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
University of Pittsburgh
Information provided by (Responsible Party):
Melanie B Gillingham, Oregon Health and Science University
Results First Submitted: July 12, 2016  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Conditions: Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency
Carnitine Palmitoyltransferase 2 (CPT2) Deficiency
Mitochondrial Trifunctional Protein (TFP) Deficiency
Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency
Intervention: Drug: Triheptanoin

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Medium Chain Triglyceride (MCT)

Subjects randomized to consume 20% of energy from MCT

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

Triheptanoin

Subject randomized to consume 20% of energy from triheptanoin.

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


Participant Flow:   Overall Study
    Medium Chain Triglyceride (MCT)   Triheptanoin
STARTED   16   16 
COMPLETED   16   16 
NOT COMPLETED   0   0 



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Subjects were 7 years of age or greater with one of 3 fatty acid oxidation disorders: CPT2, VLCAD or LCHAD/TFP.

Reporting Groups
  Description
Medium Chain Triglyceride (MCT)

Subjects randomized to consume 20% of energy from MCT

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

Triheptanoin

Subject randomized to consume 20% of energy from triheptanoin.

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

Total Total of all reporting groups

Baseline Measures
   Medium Chain Triglyceride (MCT)   Triheptanoin   Total 
Overall Participants Analyzed 
[Units: Participants]
 16   16   32 
Age 
[Units: Years]
Mean (Standard Deviation)
     
Participants Analyzed 
[Units: Participants]
 16   16   32 
   22.3  (12.7)   27.2  (15.9)   24.8  (14.4) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Participants Analyzed 
[Units: Participants]
 16   16   32 
Female      10  62.5%      10  62.5%      20  62.5% 
Male      6  37.5%      6  37.5%      12  37.5% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Participants Analyzed 
[Units: Participants]
 16   16   32 
Hispanic or Latino      1   6.3%      0   0.0%      1   3.1% 
Not Hispanic or Latino      15  93.8%      16 100.0%      31  96.9% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Participants Analyzed 
[Units: Participants]
 16   16   32 
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0% 
Asian      0   0.0%      0   0.0%      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      0   0.0%      0   0.0%      0   0.0% 
White      16 100.0%      16 100.0%      32 100.0% 
More than one race      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
Region of Enrollment 
[Units: Participants]
     
United States       
Participants Analyzed 
[Units: Participants]
 16   16   32 
United States   16   16   32 
Body Weight 
[Units: Kilograms]
Mean (Standard Deviation)
     
Participants Analyzed 
[Units: Participants]
 16   16   32 
   67.1  (26.8)   62.1  (23.5)   64.6  (24.9) 
Ejection Fraction [1] [2] 
[Units: Percent]
Mean (Standard Deviation)
     
Participants Analyzed 
[Units: Participants]
 11   10   21 
   63  (5.3)   57  (9.2)   59.7  (8.0) 
[1] Echocardiogram ejection fraction as a %. This was not completed for all the participant. There were 11 participants in the MCT group and 10 participants in the Triheptanoin group who are included in this baseline measure.
[2] There was a change in the protocol from cardiac MRI to echo after the 1st 7 participants and some of the echocardiogram results were unreadable due to technical errors. A total of 11 subjects in the MCT and 10 subjects in triheptanoin group were analyzed.
Exercise Heart Rate [1] 
[Units: Beats per minute]
Mean (Standard Deviation)
     
Participants Analyzed 
[Units: Participants]
 16   16   32 
   132  (7)   125  (12)   128  (10) 
[1] All participants performed a moderate intensity exercise treadmill walking at an estimated 60% of their estimated max heart rate for 40 minutes. The exercise heart rate at minute 30 when the exercise was steady is reported.
Total Energy Expenditure [1] [2] 
[Units: Kcal/day]
Mean (Standard Deviation)
     
Participants Analyzed 
[Units: Participants]
 15   15   30 
   2567  (604)   2230  (512)   2392  (572) 
[1] Total energy expenditure in kcal/day was measured by doubly labled water in 12 subjects in the MCT group and in 13 of the subjects in the Triheptanoin group.
[2] One participant in the MCT group and one participant in the Triheptanoin group did not return their urine samples for the doubly labeled water analysis. A total of 15 in each group were analyzed.


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Energy Expenditure   [ Time Frame: change from baseline after 4 months of treatment ]

2.  Primary:   Ejection Fraction   [ Time Frame: 4 months ]

3.  Secondary:   Exercise Heart Rate   [ Time Frame: change from baseline to 4 months of treatment ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
  Hide More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr. Melanie Gillingham
Organization: Oregon Health & Science University
phone: 503-494-1682
e-mail: gillingm@ohsu.edu



Responsible Party: Melanie B Gillingham, Oregon Health and Science University
ClinicalTrials.gov Identifier: NCT01379625     History of Changes
Other Study ID Numbers: FD003895
First Submitted: June 21, 2011
First Posted: June 23, 2011
Results First Submitted: July 12, 2016
Results First Posted: March 23, 2017
Last Update Posted: March 23, 2017