Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00947960
• Date of registration: 24/07/2009
• Prospective Registration: No
• Primary sponsor: Baylor Research Institute
• Public title: Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease
• Scientific title: A Treatment Trial of Triheptanoin in Patients With Adult Polyglucosan Body Disease - A Randomized Controlled Study
• Date of first enrolment: June 2009
• Target sample size: 23
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00947960
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2

Countries of recruitment

France, United States

Contacts

• Name: Raphael Schiffmann, M.D, M.H.Sc
• Affiliation: Institute of Metabolic Disease

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Adult Polyglucosan Body Disease

Glycogen Storage Disease Type IV

Glycogen Brancher Enzyme Deficiency

Intervention(s)

Drug: Triheptanoin

Other: Vegetable Oil

Primary Outcome(s)

Distance traveled in six minute walk test [Time Frame: every three months]

Secondary ID(s)

009-103

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ultragenyx Pharmaceutical Inc