Clinical Trial: EZN-2279 in Patients With ADA-SCID

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

Official Title: A Study of EZN-2279 (Polyethylene Glycol Recombinant Adenosine Deaminase [PEG-rADA]) Administered as a Weekly Intramuscular Injection in Patients With Adenosine Deaminase (ADA)-Deficient Combined Immu

Brief Summary: The purpose of this study is to evaluate the safety, efficacy, and pharmacokinetics of EZN-2279 in patients with ADA-deficient combined immunodeficiency currently being treated with Adagen.

Detailed Summary:
Sponsor: Leadiant Biosciences, Inc.

Current Primary Outcome: total erythrocyte dAXP concentration from a trough blood sample [ Time Frame: through 21 weeks of EZN-2279 treatment ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

• plasma ADA activity [ Time Frame: through 21 weeks of EZN-2279 treatment ]
• Immune status [ Time Frame: through end of EZN-2279 treatment ]
  includes absolute lymphocyte counts, lymphocyte subset analysis, quantitative immunoglobulin concentration
• Safety [ Time Frame: through end of EZN-2279 study treatment ]
  adverse events, serious adverse events, physical examinations, laboratory evaluations and immunogenicity
• Clinical Status [ Time Frame: through end of EZN-2279 treatment ]
  infection rate, incidence and duration of hospitalizations, overall survival, performance status

Original Secondary Outcome:

• plasma ADA activity [ Time Frame: through 21 weeks of EZN-2279 treatment ]
• Immune status [ Time Frame: through 21 weeks of EZN-2279 treatment ]
  includes absolute lymphocyte counts, lymphocyte subset analysis, quantitative immunoglobulin concentration
• Safety [ Time Frame: through end of EZN-2279 study treatment ]
  adverse events, physical examinations, laboratory evaluations and immunogenicity

Information By: Leadiant Biosciences, Inc.

Dates:
Date Received: August 18, 2011
Date Started: December 2013
Date Completion: March 2019
Last Updated: May 9, 2017
Last Verified: May 2017