Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease
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ClinicalTrials.gov Identifier: NCT00947960 |
Recruitment Status
:
Completed
First Posted
: July 28, 2009
Last Update Posted
: March 5, 2018
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Adult Polyglucosan Body Disease Glycogen Brancher Enzyme Deficiency Glycogen Storage Disease Type IV | Drug: Triheptanoin Other: Vegetable Oil | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 23 participants |
Allocation: | Randomized |
Intervention Model: | Crossover Assignment |
Masking: | Double (Participant, Investigator) |
Primary Purpose: | Treatment |
Official Title: | A Treatment Trial of Triheptanoin in Patients With Adult Polyglucosan Body Disease - A Randomized Controlled Study |
Study Start Date : | June 2009 |
Actual Primary Completion Date : | July 2015 |
Actual Study Completion Date : | July 2015 |

Arm | Intervention/treatment |
---|---|
Active/Placebo
Subjects receive 1-2 grams/kilogram body weight triheptanoin divided into 4 equal doses taken with meals and snack for 6 months crossing over to receive placebo vegetable oil at the same dose and frequency for the next 6 months during the randomization phase.
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Drug: Triheptanoin
1-2 grams triheptanoin (drug)/kilogram body weight divided into 4 equal doses per day taken with meals and a snack for 6 months followed by 1-2 grams vegetable oil (placebo)/kilogram body weight divided into 4 equal doses per day taken with meals and a snack for 6 months.
Other: Vegetable Oil
1-2 grams vegetable oil (placebo)/kilogram body weight divided into 4 equal doses per day taken with meals and a snack for 6 months followed by 1-2 g triheptanoin (drug)/kilogram body weight for 6 months.
Other Name: Placebo
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Placebo/Active
Subjects receive 1-2 grams/kilogram body weight placebo vegetable oil divided into 4 equal doses taken with meals and snack for 6 months crossing over to receive triheptanoin at the same dose and frequency for the next 6 months during the randomization phase.
|
Drug: Triheptanoin
1-2 grams triheptanoin (drug)/kilogram body weight divided into 4 equal doses per day taken with meals and a snack for 6 months followed by 1-2 grams vegetable oil (placebo)/kilogram body weight divided into 4 equal doses per day taken with meals and a snack for 6 months.
Other: Vegetable Oil
1-2 grams vegetable oil (placebo)/kilogram body weight divided into 4 equal doses per day taken with meals and a snack for 6 months followed by 1-2 g triheptanoin (drug)/kilogram body weight for 6 months.
Other Name: Placebo
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- Distance traveled in six minute walk test [ Time Frame: every three months ]

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Ages Eligible for Study: | 18 Years to 75 Years (Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Confirmed diagnosis of APBD by the presence of mutations of the GBE1 gene in both alleles or brancher enzyme deficiency
- Willing and able to travel to Dallas TX
- Able to tolerate dietary oil
- Able to provide informed consent
Exclusion Criteria:
- Intercurrent medical conditions that would confound the assessment of efficacy, such as HIV or diabetes
- Patients who are wheelchair bound
- Patients deemed unsuitable for the study by the investigator

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00947960
France | |
Department of Genetics, Groupe Hospitalier Pitié-Salpêtrière | |
Paris, France, 75013 |
Principal Investigator: | Raphael Schiffmann, M.D, M.H.Sc | Institute of Metabolic Disease |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Baylor Research Institute |
ClinicalTrials.gov Identifier: | NCT00947960 History of Changes |
Other Study ID Numbers: |
009-103 |
First Posted: | July 28, 2009 Key Record Dates |
Last Update Posted: | March 5, 2018 |
Last Verified: | March 2018 |
Keywords provided by Baylor Research Institute:
Adult Polyglucosan Body Disease (APBD) Glycogen Brancher Enzyme (GBE1) Deficiency |
Additional relevant MeSH terms:
Glycogen Storage Disease Glycogen Storage Disease Type IV Nervous System Diseases Carbohydrate Metabolism, Inborn Errors |
Metabolism, Inborn Errors Genetic Diseases, Inborn Metabolic Diseases |