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Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

NCT02696044 · Status: UNKNOWN · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2020-07-17

No results posted yet for this study

Summary

The aim of this study is to evaluate the safety and tolerability of triheptanoin in participants with Rett syndrome using laboratory values, electrocardiogram, rate of adverse events (AE), and physical exam.This study also seeks to evaluate the efficacy of UX007 (triheptanoin) in improving overall seizure frequency and dystonia.

Conditions

Interventions

DRUG

triheptanoin

Participants will begin a 2 week dose titration period to achieve study drug treatment comprising of 1-4 grams per kilogram of body weight (g/kg) daily depending on age. The age-related target dose will be mixed with food or formula and administered orally or by gastronomy tube. The total daily dose can be divided into 4 equal doses taken 4 times daily. Participants will maintain the age dependent dose treatment at the 1-4 g/kg daily for four months.

Sponsors & Collaborators

  • Ultragenyx Pharmaceutical Inc

    collaborator INDUSTRY

  • Rett Syndrome Research Trust

    collaborator OTHER

  • Center for Rare Neurological Diseases, Norcross, GA

    lead OTHER

Principal Investigators

  • Daniel Tarquinio, DO · Center for Rare Neurological Diseases

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-06-30
Primary Completion
2021-02-28
Completion
2022-02-28

Countries

  • United States

Study Locations

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Entities

Diseases
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 NCT02696044 on ClinicalTrials.gov