Clinical Trial: Study to Assess the Long Term Safety and Efficacy of UX007 in Subjects With Glucose Type 1 DS

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional

Official Title: An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects With Glucose Transporter Type 1 Deficiency Syndrome

Brief Summary: UX007G-CL202 is an open-label, single-arm, multi center extension study to assess the long-term safety and efficacy of UX007 in Glut 1 DS. The study will enroll up to 40 pediatric, adolescent and adult Glut 1 DS subjects who have completed the UX007G-CL201 study or participated in qualified ISTs.

Detailed Summary:

A Phase 2 study (UX007G-CL-201) is currently ongoing to assess safety and efficacy of UX007 in reducing the frequency of seizures in Glut 1 DS patients. ISTs and other treatment programs are also evaluating UX007 as a treatment for Glut 1 DS.

This open label long term safety and efficacy study (UX007G-CL202) will provide an opportunity for Glut 1 DS patients to be treated with UX007 for up to 3 years (or until market approval) under a single standardized protocol for maintenance therapy and consistent safety monitoring. The study is designed to obtain long-term safety information and evaluate maintenance of efficacy in a pediatric and adult Glut 1 DS population.


Sponsor: Ultragenyx Pharmaceutical Inc

Current Primary Outcome: Frequency of AEs and SAEs [ Time Frame: 3 years ]

Frequency of Adverse Events and Serious Adverse Events assessed as related to study drug


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Maintenance of reduction in seizure frequency [ Time Frame: 3 years ]
  • Long term Efficacy of UX007 [ Time Frame: 3 years ]
    Long term efficacy in measures of neurological function and development delay using the Cambridge Neuropsychological Test Automated Battery (CANTAB)
  • Long term Efficacy of UX007 [ Time Frame: 3 years ]
    Long term efficacy in measures of neurological function and development delay using the Columbia Neurological Score
  • Maintenance of UX007 treatment effect on gross motor function and movement disorders using the 6 Minute Walk Test [ Time Frame: 3 years ]
  • Maintenance of UX007 treatment effect on gross motor function and movement disorders using Paroxymal Exertional Dyskinesia (PED) [ Time Frame: 3 years ]
  • Continued impact of UX007 treatment on clinical status and health related quality of life using the Clinical Global Impression Scales [ Time Frame: 3 years ]
  • Continued impact of UX007 treatment on clinical status and health related quality of life using the Short Form Health Surveys [ Time Frame: 3 years ]
  • Continued impact of UX007 treatment on clinical status and health related quality of life using thePediatric Evaluations of Disability Inventory Computer Adaptive Test (PEDI-CAT) [ Time Frame: 3 years ]


Original Secondary Outcome: Same as current

Information By: Ultragenyx Pharmaceutical Inc

Dates:
Date Received: November 3, 2015
Date Started: September 2015
Date Completion: September 2018
Last Updated: April 18, 2017
Last Verified: April 2017