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EZN-2279 in Patients With ADA-SCID

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2016 by Leadiant Biosciences, Inc.
Information provided by (Responsible Party):
Leadiant Biosciences, Inc. Identifier:
First received: August 18, 2011
Last updated: October 24, 2016
Last verified: October 2016
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.

Condition Intervention Phase
Adenosine Deaminase Deficiency
Severe Combined Immunodeficiency
Biological: EZN-2279
Biological: Adagen
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Leadiant Biosciences, Inc.:

Primary Outcome Measures:
  • total erythrocyte dAXP concentration from a trough blood sample [ Time Frame: through 21 weeks of EZN-2279 treatment ]

Secondary Outcome Measures:
  • plasma ADA activity [ Time Frame: through 21 weeks of EZN-2279 treatment ]
  • Immune status [ Time Frame: through end of EZN-2279 treatment ]
    includes absolute lymphocyte counts, lymphocyte subset analysis, quantitative immunoglobulin concentration

  • Safety [ Time Frame: through end of EZN-2279 study treatment ]
    adverse events, serious adverse events, physical examinations, laboratory evaluations and immunogenicity

  • Clinical Status [ Time Frame: through end of EZN-2279 treatment ]
    infection rate, incidence and duration of hospitalizations, overall survival, performance status

Estimated Enrollment: 6
Study Start Date: December 2013
Estimated Study Completion Date: March 2018
Estimated Primary Completion Date: March 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: EZN-2279
Patients crossed over to receive EZN-2279 following an Adagen lead-in period
Biological: EZN-2279
weekly administration of EZN-2279 via IM injection
Other Name: rADA
Active Comparator: Adagen
Patients start on Adagen and cross over to experimental EZN-2279 treatment
Biological: Adagen


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01420627

Contact: Vincent Elliott 301-670-1525
Contact: Scott Rodgers 301-670-1565

United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Robert LaFerte    323-361-8569   
Principal Investigator: Neena Kapoor, MD         
University of California San Francisco Recruiting
San Francisco, California, United States, 94143
Contact: Angel Hu    415-476-6997   
Principal Investigator: Morna Dorsey, MD         
United States, Colorado
National Jewish Health Withdrawn
Denver, Colorado, United States, 80206
United States, New York
Albert Einstein College of Medicine Recruiting
Bronx, New York, United States, 10461
Contact: Gayle Krenik    718-405-8830   
Principal Investigator: Arye Rubinstein, MD, PhD         
Sponsors and Collaborators
Leadiant Biosciences, Inc.
  More Information

Responsible Party: Leadiant Biosciences, Inc. Identifier: NCT01420627     History of Changes
Other Study ID Numbers: STP-2279-002
Study First Received: August 18, 2011
Last Updated: October 24, 2016

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Severe Combined Immunodeficiency
Immune System Diseases
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases
Blood Protein Disorders
Hematologic Diseases
Lymphoproliferative Disorders
Lymphatic Diseases processed this record on April 21, 2017