Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease

• Status: Completed
• Trial end date: 2015-07-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of the study is to determine if triheptanoin is an effective treatment for the symptoms of Adult Polyglucosan Body Disease.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Baylor Research Institute
• Collaborator: Ultragenyx Pharmaceutical Inc

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