EZN-2279 in Patients With ADA-SCID



Status:Active, not recruiting
Conditions:Other Indications, Infectious Disease, HIV / AIDS
Therapuetic Areas:Immunology / Infectious Diseases, Other
Healthy:No
Age Range:Any
Updated:1/27/2019
Start Date:December 2013
End Date:September 2019

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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

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.


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
We found this trial at
5
sites
Hershey, Pennsylvania 17033
Principal Investigator: Tracy Fausnight, MD
Phone: 717-531-7765
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Principal Investigator: Neena Kapoor, MD
Phone: 323-361-8569
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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1300 Morris Park Ave
Bronx, New York 10461
(718) 430-2000
Albert Einstein College of Medicine The Albert Einstein College of Medicine of Yeshiva University is...
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Bronx, NY
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Buffalo, New York 14222
Principal Investigator: Heather Lehman, MD
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Buffalo, NY
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San Francisco, California 94143
Principal Investigator: Morna Dorsey, MD
Phone: 415-502-6057
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San Francisco, CA
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