Status:
COMPLETED
Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease
Lead Sponsor:
Baylor Research Institute
Collaborating Sponsors:
Ultragenyx Pharmaceutical Inc
Conditions:
Adult Polyglucosan Body Disease
Glycogen Brancher Enzyme Deficiency
Eligibility:
All Genders
18-75 years
Phase:
PHASE2
Brief Summary
The purpose of the study is to determine if triheptanoin is an effective treatment for the symptoms of Adult Polyglucosan Body Disease.
Detailed Description
Adult polyglucosan disease is a progressive neurogenetic disorder characterized by neurogenic bladder, progressive difficulty with walking, and sensory abnormalities in the lower extremities which typ...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
June 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2015
Estimated Enrollment :
23 Patients enrolled
Trial Details
Trial ID
NCT00947960
Start Date
June 1 2009
End Date
July 1 2015
Last Update
March 5 2018
Active Locations (1)
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1
Department of Genetics, Groupe Hospitalier Pitié-Salpêtrière
Paris, France, 75013