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Trial record 3 of 76 for:    Long-chain fatty acids

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

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ClinicalTrials.gov Identifier: NCT01379625
Recruitment Status : Completed
First Posted : June 23, 2011
Results First Posted : March 23, 2017
Last Update Posted : March 23, 2017
Sponsor:
Collaborator:
University of Pittsburgh
Information provided by (Responsible Party):
Melanie B Gillingham, Oregon Health and Science University

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
Enrollment 32
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Medium Chain Triglyceride (MCT) Triheptanoin
Hide Arm/Group Description

Subjects randomized to consume 20% of energy from MCT

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

Subject randomized to consume 20% of energy from triheptanoin.

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

Period Title: Overall Study
Started 16 16
Completed 16 16
Not Completed 0 0
Arm/Group Title Medium Chain Triglyceride (MCT) Triheptanoin Total
Hide Arm/Group Description

Subjects randomized to consume 20% of energy from MCT

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

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 of all reporting groups
Overall Number of Baseline Participants 16 16 32
Hide Baseline Analysis Population Description
Subjects were 7 years of age or greater with one of 3 fatty acid oxidation disorders: CPT2, VLCAD or LCHAD/TFP.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 16 participants 16 participants 32 participants
22.3  (12.7) 27.2  (15.9) 24.8  (14.4)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 16 participants 32 participants
Female
10
  62.5%
10
  62.5%
20
  62.5%
Male
6
  37.5%
6
  37.5%
12
  37.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 16 participants 32 participants
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)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 16 participants 32 participants
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  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 16 participants 16 participants 32 participants
16 16 32
Body Weight  
Mean (Standard Deviation)
Unit of measure:  Kilograms
Number Analyzed 16 participants 16 participants 32 participants
67.1  (26.8) 62.1  (23.5) 64.6  (24.9)
Ejection Fraction   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Percent
Number Analyzed 11 participants 10 participants 21 participants
63  (5.3) 57  (9.2) 59.7  (8.0)
[1]
Measure Description: 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]
Measure Analysis Population Description: 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] 
Mean (Standard Deviation)
Unit of measure:  Beats per minute
Number Analyzed 16 participants 16 participants 32 participants
132  (7) 125  (12) 128  (10)
[1]
Measure Description: 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] 
Mean (Standard Deviation)
Unit of measure:  Kcal/day
Number Analyzed 15 participants 15 participants 30 participants
2567  (604) 2230  (512) 2392  (572)
[1]
Measure Description: 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]
Measure Analysis Population Description: 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.
1.Primary Outcome
Title Energy Expenditure
Hide Description 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.
Time Frame change from baseline after 4 months of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
One subject in each group did not complete the doubly labeled water measures. A total of 15 in each group measured total energy expenditure at baseline and at the end of the study. This value compares the change over treatment.
Arm/Group Title Medium Chain Triglyceride (MCT) Triheptanoin
Hide Arm/Group Description:

Subjects randomized to consume 20% of energy from MCT

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

Subject randomized to consume 20% of energy from triheptanoin.

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

Overall Number of Participants Analyzed 15 15
Mean (Standard Deviation)
Unit of Measure: kcal/day
-73  (335) 107  (387)
2.Primary Outcome
Title Ejection Fraction
Hide Description Change in resting ejection fraction over 4 month treatment period
Time Frame 4 months
Hide Outcome Measure Data
Hide Analysis Population Description
Change in resting ejection fraction over 4 month treatment period calculated as: 4 month ejection fraction-baseline ejection fraction. Only participants with available data are included in this analysis.
Arm/Group Title Medium Chain Triglyceride (MCT) Triheptanoin
Hide Arm/Group Description:

Subjects randomized to consume 20% of energy from MCT

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

Subject randomized to consume 20% of energy from triheptanoin.

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

Overall Number of Participants Analyzed 11 10
Mean (Standard Deviation)
Unit of Measure: percent
-1.91  (4.16) 2.14  (4.43)
3.Secondary Outcome
Title Exercise Heart Rate
Hide Description Subjects will complete a submaximal treadmill exercise study at baseline. Exercise heart heart, ventilation and perceived exertion will be measured. Subjects will be randomized to MCT or triheptanoin supplementation for 4 months. At the end of treatment, the exercise test will be repeated keeping work performed constant. Change in exercise heart rate, ventilation and exertion will be compared between groups.
Time Frame change from baseline to 4 months of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Medium Chain Triglyceride (MCT) Triheptanoin
Hide Arm/Group Description:

Subjects randomized to consume 20% of energy from MCT

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

Subject randomized to consume 20% of energy from triheptanoin.

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

Overall Number of Participants Analyzed 16 16
Mean (Standard Deviation)
Unit of Measure: beats per minute
-0.1  (9.6) -12.6  (34)
Time Frame Four months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Medium Chain Triglyceride (MCT) Triheptanoin
Hide Arm/Group Description

Subjects randomized to consume 20% of energy from MCT

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

Subject randomized to consume 20% of energy from triheptanoin.

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

All-Cause Mortality
Medium Chain Triglyceride (MCT) Triheptanoin
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Medium Chain Triglyceride (MCT) Triheptanoin
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/16 (0.00%)      0/16 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Medium Chain Triglyceride (MCT) Triheptanoin
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   12/16 (75.00%)      11/16 (68.75%)    
Gastrointestinal disorders     
Gastrointestinal Upset  1 [1]  12/16 (75.00%)  38 11/16 (68.75%)  24
Diarrhea/Loose stools  1 [2]  6/16 (37.50%)  12 5/16 (31.25%)  9
Musculoskeletal and connective tissue disorders     
Rhabdomyolysis  1 [3]  4/16 (25.00%)  7 5/16 (31.25%)  7
muscle pain/ elevated CPK  1 [4]  10/16 (62.50%)  18 11/16 (68.75%)  16
Indicates events were collected by systematic assessment
1
Term from vocabulary, 21 CRF 312
[1]
Gastrointestinal Upset including stomach pain, burning, or cramping.
[2]
Report of diarrhea or loose stools during the treatment period.
[3]
Acute rhabdomyolysis requiring hospitalization is expected. This is a known complication of fatty acid oxidation disorders.
[4]
This is an anticipated adverse event because muscle pain with elevated CPK is a complication of a fatty acid oxidation disorder.
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
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