EZN-2279 in Patients With ADA-SCID

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Sigma Tau Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT01420627
First received: August 18, 2011
Last updated: July 25, 2014
Last verified: July 2014
  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
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

Locations
United States, California
University of California San Francisco
San Francisco, California, United States, 94143
United States, Colorado
National Jewish Health
Denver, Colorado, United States, 80206
United States, New York
Albert Einstein College of Medicine
Bronx, New York, United States, 10461
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: July 25, 2014
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 August 28, 2014