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

Interventional


Primary Outcome

total erythrocyte dAXP concentration from a trough blood sample

Secondary Outcome

 plasma ADA activity

Condition

ADA-SCID

Intervention

EZN-2279

Study Arms / Comparison Groups

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

Publications

* 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

Biological

Estimated Enrollment

6

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
             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
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Elie Haddad, MD, PhD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01420627

Organization ID

STP-2279-002


Responsible Party

Sponsor

Study Sponsor

Leadiant Biosciences, Inc.


Study Sponsor

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


Verification Date

August 2019