EZN-2279 in Patients With ADA-SCID

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Brief Title

EZN-2279 in Patients With ADA-SCID

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

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.

Study Phase

Phase 3

Study Type


Primary Outcome

total erythrocyte dAXP concentration from a trough blood sample

Secondary Outcome

 plasma ADA activity





Study Arms / Comparison Groups

Description:  Patients crossed over to receive EZN-2279 following an Adagen lead-in period


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

December 2013

Completion Date

May 29, 2019

Primary Completion Date

May 29, 2019

Eligibility Criteria

        Inclusion Criteria:

          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:

               1. Trough plasma ADA activity >15 μmol/h/mL while receiving Adagen®

               2. Total erythrocyte dAXP ≤0.02 μmol/mL from a trough blood sample

          4. Patients or parent/guardian must be capable of understanding the protocol requirements
             and risks and providing written informed assent/consent

        Exclusion Criteria:

          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

          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

          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

          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




N/A - N/A

Accepts Healthy Volunteers



Elie Haddad, MD, PhD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Responsible Party


Study Sponsor

Leadiant Biosciences, Inc.

Study Sponsor

Elie Haddad, MD, PhD, Principal Investigator, Université de Montréal

Verification Date

August 2019