Summary

at San Francisco, California and other locations
study started
estimated completion:
Morna Dorsey

Description

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.

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 Immunodeficiency

Keywords

ADA-SCID Adenosine Deaminase Deficiency Severe Combined Immunodeficiency Immunologic Deficiency Syndromes Agammaglobulinemia EZN-2279 Adagen

Eligibility

You can join if…

  1. Diagnosis of ADA-deficient combined immunodeficiency
  2. Stable clinical status while receiving therapy with Adagen®. Patients previously receiving gene therapy or undergoing hematopoietic stem cell transplantation who still require Adagen® treatment are eligible. The dose of Adagen® must be stable for at least 3 months prior to study entry.
  3. Have both during the Adagen® Lead-in phase of the study:
  4. Trough plasma ADA activity >15 μmol/h/mL while receiving Adagen®
  5. Total erythrocyte dAXP ≤0.02 μmol/mL from a trough blood sample
  6. Patients or parent/guardian must be capable of understanding the protocol requirements and risks and providing written informed assent/consent

You CAN'T join if...

  1. Autoimmunity requiring immunosuppressive treatment
  2. Patients with neutralizing anti-Adagen® antibodies at screening evaluation.
  3. Severe thrombocytopenia (platelet count <50 x 109/L)
  4. Current participation in other therapeutic protocols for ADA-deficient combined immunodeficiency
  5. Current or prior participation in another clinical study with an investigational agent and/or use of an investigational drug in the 30 days before study entry.
  6. Known planned participation in a gene-therapy study for the planned duration of this study
  7. Any condition that, in the opinion of the PI or Sigma-Tau, makes the patient unsuitable for the study
  8. Inability or unwillingness to administer Adagen® or EZN-2279 on a one time per week regimen
  9. Inability to comply with the study protocol
  10. . Female patients who are pregnant or lactating
  11. . Female patients who are breast-feeding
  12. . Female subjects of childbearing potential who are not using an FDA approved birth control method

Locations

  • University of California San Francisco accepting new patients
    San Francisco California 94143 United States
  • Children's Hospital Los Angeles accepting new patients
    Los Angeles California 90027 United States

Lead Scientist

  • Morna Dorsey
    1. Optimizing diagnosis and care of patients with primary immunodeficiency 2. Investigating the psychosocial impact of primary immunodeficiency on patients and families 3. Establishing treatment for patients with food allergy and eosinophilic disorders

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Leadiant Biosciences, Inc.
ID
NCT01420627
Phase
Phase 3
Study Type
Interventional
Last Updated