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Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease

NCT00947960 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 23

Last updated 2018-03-05

No results posted yet for this study

Summary

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

Conditions

  • Adult Polyglucosan Body Disease
  • Glycogen Brancher Enzyme Deficiency
  • Glycogen Storage Disease Type IV

Interventions

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.

Sponsors & Collaborators

Principal Investigators

  • Raphael Schiffmann, M.D, M.H.Sc · Institute of Metabolic Disease

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
CROSSOVER

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-06-30
Primary Completion
2015-07-31
Completion
2015-07-31

Countries

  • France

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00947960 on ClinicalTrials.gov