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EZN-2279 in Patients With ADA-SCID

This study is currently recruiting participants.
See Contacts and Locations
Verified May 2017 by Leadiant Biosciences, Inc.
Sponsor:
Information provided by (Responsible Party):
Leadiant Biosciences, Inc.
ClinicalTrials.gov Identifier:
NCT01420627
First received: August 18, 2011
Last updated: May 9, 2017
Last verified: May 2017
  Purpose
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
ADA-SCID 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: 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

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 2019
Estimated Primary Completion Date: September 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

  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  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 ClinicalTrials.gov identifier: NCT01420627

Contacts
Contact: Vincent Elliott 301-670-1525 vincent.elliott@sigmatau.com
Contact: Scott Rodgers 301-670-1565 scott.rodgers@sigmatau.com

Locations
United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Greg Balagot    323-361-8569    gbalagot@chla.edu   
Principal Investigator: Neena Kapoor, MD         
University of California San Francisco Recruiting
San Francisco, California, United States, 94143
Contact: Heidi Shannon    415-502-6057    heidi.shannon@ucsf.edu   
Principal Investigator: Morna Dorsey, MD         
United States, Colorado
National Jewish Health Withdrawn
Denver, Colorado, United States, 80206
United States, New York
Women and Children's Hospital of Buffalo Recruiting
Buffalo, New York, United States, 14222
Contact: Helena McClenahan       hmcclenahan@upa.chob.edu   
Principal Investigator: Heather Lehman, MD         
Albert Einstein College of Medicine Recruiting
The Bronx, New York, United States, 10461
Contact: Gayle Krenik    718-405-8830    gkreinik@montefiore.org   
Principal Investigator: Arye Rubinstein, MD, PhD         
United States, Pennsylvania
Penn State College of Medicine The Milton S. Hershey Medical Center Not yet recruiting
Hershey, Pennsylvania, United States, 17033
Contact: Jennifer Stokes    717-531-7765    jstokes1@hmc.psu.edu   
Principal Investigator: Tracy Fausnight, MD         
United States, Virginia
Virginia Commonweath University Not yet recruiting
Richmond, Virginia, United States, 23298
Contact: Kimberly Bradley    804-518-8843    Kbbradley@vcu.edu   
Principal Investigator: Brant Ward, MD, PhD         
Sponsors and Collaborators
Leadiant Biosciences, Inc.
  More Information

Responsible Party: Leadiant Biosciences, Inc.
ClinicalTrials.gov Identifier: NCT01420627     History of Changes
Other Study ID Numbers: STP-2279-002
Study First Received: August 18, 2011
Last Updated: May 9, 2017

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Severe Combined Immunodeficiency
Agammaglobulinemia
Immune System Diseases
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases
Blood Protein Disorders
Hematologic Diseases
Lymphoproliferative Disorders
Lymphatic Diseases

ClinicalTrials.gov processed this record on September 21, 2017