EZN-2279 in Patients With ADA-SCID

This study is currently recruiting participants.
Verified December 2013 by Sigma Tau Pharmaceuticals, Inc.
Sponsor:
Information provided by (Responsible Party):
Sigma Tau Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT01420627
First received: August 18, 2011
Last updated: December 17, 2013
Last verified: December 2013
  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
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
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 Sigma Tau Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • total erythrocyte dAXP concentration from a trough blood sample [ Time Frame: through 21 weeks of EZN-2279 treatment ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • plasma ADA activity [ Time Frame: through 21 weeks of EZN-2279 treatment ] [ Designated as safety issue: No ]
  • Immune status [ Time Frame: through end of EZN-2279 treatment ] [ Designated as safety issue: No ]
    includes absolute lymphocyte counts, lymphocyte subset analysis, quantitative immunoglobulin concentration

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

  • Clinical Status [ Time Frame: through end of EZN-2279 treatment ] [ Designated as safety issue: No ]
    infection rate, incidence and duration of hospitalizations, overall survival, performance status


Estimated Enrollment: 6
Study Start Date: December 2013
Estimated Study Completion Date: April 2017
Estimated Primary Completion Date: April 2015 (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

  Eligibility

Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01420627

Contacts
Contact: Thomas Genna 301-670-1516 thomas.genna@sigmatau.com

Locations
United States, Colorado
National Jewish Health Recruiting
Denver, Colorado, United States, 80206
Contact: Cathy Hancock    303-398-1442      
Principal Investigator: Erwin W Gelfand, MD         
Sponsors and Collaborators
Sigma Tau Pharmaceuticals, Inc.
  More Information

No publications provided

Responsible Party: Sigma Tau Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT01420627     History of Changes
Other Study ID Numbers: STP-2279-002
Study First Received: August 18, 2011
Last Updated: December 17, 2013
Health Authority: United States: Food and Drug Administration

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 April 16, 2014