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EZN-2279 in Patients With ADA-SCID

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01420627
Recruitment Status : Active, not recruiting
First Posted : August 19, 2011
Last Update Posted : January 25, 2019
Information provided by (Responsible Party):
Leadiant Biosciences, Inc.

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.

Condition or disease Intervention/treatment Phase
ADA-SCID Adenosine Deaminase Deficiency Severe Combined Immunodeficiency Biological: EZN-2279 Biological: Adagen Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Allocation: Non-Randomized
Intervention Model: Crossover Assignment
Masking: None (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
Study Start Date : December 2013
Estimated Primary Completion Date : September 2019
Estimated Study Completion Date : September 2019

Arm Intervention/treatment
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

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

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

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

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

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01420627

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United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
University of California San Francisco
San Francisco, California, United States, 94143
United States, New York
Albert Einstein College of Medicine
Bronx, New York, United States, 10461
Women and Children's Hospital of Buffalo
Buffalo, New York, United States, 14222
United States, Pennsylvania
Penn State College of Medicine The Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
Sponsors and Collaborators
Leadiant Biosciences, Inc.
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Principal Investigator: Elie Haddad, MD, PhD Université de Montréal

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Responsible Party: Leadiant Biosciences, Inc. Identifier: NCT01420627     History of Changes
Other Study ID Numbers: STP-2279-002
First Posted: August 19, 2011    Key Record Dates
Last Update Posted: January 25, 2019
Last Verified: January 2019

Additional relevant MeSH terms:
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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