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EZN-2279 in Patients With ADA-SCID

Information source: Sigma Tau Pharmaceuticals, Inc.
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: ADA-SCID; Adenosine Deaminase Deficiency; Severe Combined Immunodeficiency

Intervention: EZN-2279 (Biological)

Phase: Phase 3

Status: Recruiting

Sponsored by: Sigma Tau Pharmaceuticals, Inc.

Official(s) and/or principal investigator(s):
Luigi Notarangelo, MD, Principal Investigator, Affiliation: Children's Hospital Boston

Overall contact:
Melissa Jameson, Phone: 301-670-5447, Email: melissa.jameson@sigmatau.com


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.

Clinical Details

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

Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: total erythrocyte dAXP concentration from a trough blood sample

Secondary outcome:

plasma ADA activity

Immune status


Clinical Status


Minimum age: N/A. Maximum age: N/A. Gender(s): Both.


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

Locations and Contacts

Melissa Jameson, Phone: 301-670-5447, Email: melissa.jameson@sigmatau.com

Children's Hospital Los Angeles, Los Angeles, California 90027, United States; Recruiting
Robert LaFerte, Phone: 323-361-8569, Email: rlaferte@chla.usc.edu
Neena Kapoor, MD, Principal Investigator

University of California San Francisco, San Francisco, California 94143, United States; Recruiting
Angel Hu, Phone: 415-476-6997, Email: angel.hu@ucsf.edu
Morna Dorsey, MD, Principal Investigator

National Jewish Health, Denver, Colorado 80206, United States; Recruiting
Cathy Hancock, Phone: 303-398-1442
Erwin W Gelfand, MD, Principal Investigator

Albert Einstein College of Medicine, Bronx, New York 10461, United States; Recruiting
Gayle Krenik, Phone: 718-405-8830, Email: gkreinik@montefiore.org
Arye Rubinstein, MD, PhD, Principal Investigator

Additional Information

Starting date: December 2013
Last updated: February 9, 2015

Page last updated: August 20, 2015

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