Clinical Trial: EDS in Ataxia Telangiectasia Patients

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients With All patients who complete the assessments as designed over the initial 6 months of the trial will be eligible to continue in an additional 6-month, double-blind, placebo-controlled extension designed to collect information on the long-term safety and efficacy of the trial treatments.

Upon completion of all screening assessments for eligibility patients meeting all selection criteria at baseline will be randomized in a 1:1:1 fashion to one of the two EDS-EP dose levels or placebo. A minimization procedure will be employed to ensure that the proportions of male and female, and younger (6 to <10 years) and older (≥10 years), patients are comparable across the three treatment groups. Every attempt will be made to ensure the same balance is achieved across different regions.

A minimum of 180 patients will be enrolled, hence, each group will consist of 60 patients randomly assigned to receive one of the two doses of EDS-EP or placebo, as follows:

Group 1: EDS-EP dose range of ~5-10 mg D SP/infusion, Group 2: EDS-EP dose range of ~14-22 mg D SP/infusion, Group 3: Placebo EDS infusion.

The initial 6-month treatment period will be considered complete when the endpoint assessment (at Visit 9/Month 6 or at early discontinuation) has been performed for all patients. Patients who are not experiencing severe side-effects, or have deteriorated significantly while on the treatment and provide informed consent will be eligible to continue treatment for an additional 6 months in a double-blind, placebo-controlled extension treatment period. Patients meeting all entry criteria will be treated as follows:

Patients originally randomized to EDS-EP treatment groups (Group 1 or Group 2) wil
Sponsor: Erydel

Current Primary Outcome: Modified International Cooperative Ataxia Rating Scale (mICARS) [ Time Frame: 3 months, 6 months, 9 months, 12 months ]

Change from baseline analyzed using a Mixed Model Repeated Measures (MMRM) approach


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Clinical Global Impression of Change (CGI-C) key secondary outcome measure [ Time Frame: 3 months, 6 months, 9 months, 12 months ]
    Change from baseline analyzed using ANCOVA
  • Clinical Global Impression of Severity (CGI-S) of neurological symptoms of AT [ Time Frame: 3 months, 6 months, 9 months, 12 months ]
    Change from baseline
  • Vineland Adaptive Behavior Scales (VABS) [ Time Frame: 3 months, 6 months, 9 months, 12 months ]
    Change from baseline


Original Secondary Outcome:

  • Clinical Global Impression of Change (CGI-C) [ Time Frame: 3 months, 6 months, 9 months, 12 months ]
    Change from baseline analyzed using ANCOVA
  • Clinical Global Impression of Severity (CGI-S) of neurological symptoms of AT [ Time Frame: 3 months, 6 months, 9 months, 12 months ]
    Change from baseline
  • Vineland Adaptive Behavior Scales [ Time Frame: 3 months, 6 months, 9 months, 12 months ]
    Change from baseline


Information By: Erydel

Dates:
Date Received: May 2, 2016
Date Started: March 2, 2017
Date Completion: March 2019
Last Updated: March 28, 2017
Last Verified: March 2017