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Location
at San Francisco, California and other locations
Dates
study started
estimated completion:
Principal Investigator

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

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

  • Children's Hospital Los Angeles accepting new patients
    Los Angeles, California, 90027, USA

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Leadiant Biosciences, Inc.
ID
NCT01420627
Phase
Phase 3
Lead Scientist
Morna Dorsey
Study Type
Interventional
Last Updated
October 2016
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