Skip to main content
OpenTrials
Completed

NCT Number: NCT01420627

EZN-2279 in Patients With ADA-SCID

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

ADA-SCID Adenosine Deaminase Deficiency Congenital, Hereditary, and Neonatal Diseases and Abnormalities DNA Repair-Deficiency Disorders Genetic Diseases, Inborn Immune System Diseases Immunologic Deficiency Syndromes Infant, Newborn, Diseases Metabolic Diseases Nutritional and Metabolic Diseases Primary Immunodeficiency Diseases Severe Combined Immunodeficiency Severe combined immunodeficiency due to adenosine deaminase deficiency

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Children's Hospital Los Angeles, Los Angeles, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of ADA-deficient combined immunodeficiency
  • 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 6 months prior to study entry.
  • Have both of the following during the Adagen® Lead-in phase of the study prior to EZN-2279 transition:
  • Trough plasma ADA activity >15 μmol/h/mL while receiving Adagen® and
  • Total erythrocyte dAXP ≤0.02 μmol/mL from a trough blood sample
  • Patients or parent/guardian must be capable of understanding the protocol requirements and risks and providing written informed assent/consent

Exclusion criteria

  • Autoimmunity requiring immunosuppressive treatment
  • Patients with detectable neutralizing anti-Adagen® antibodies at screening evaluation
  • Severe thrombocytopenia (platelet count <50 x 10^9/L)
  • Current participation in other therapeutic protocols for ADA-deficient combined immunodeficiency
  • 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
  • Known planned participation in a gene-therapy study for the planned duration of this study
  • Any condition that, in the opinion of the PI, makes the patient unsuitable for the study
  • Inability or unwillingness to administer Adagen® or EZN-2279 on a one-time-per-week regimen
  • Inability to comply with the study protocol
  • Female patients who are pregnant or lactating
  • Female patients who are breast-feeding
  • Female subjects of childbearing potential who are not using an FDA approved birth control method

Treatment and study plan

EZN-2279

Biological

Weekly administration of EZN-2279 via IM injection

Other names: rADA

Adagen

Biological

Primary outcomes

  1. Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period

    Time frame: Baseline through Week T-21

    Number of patients with total erythrocyte dAXP concentration from a trough blood sample <0.02 mmol/L

Secondary outcomes

  1. Safety Summary Data

    Time frame: Through end of EZN-2279 study treatment, up to 203 weeks

    Summary of adverse events and serious adverse events

  2. Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period

    Time frame: From Baseline through Week T-21

    Trough ADA activity, mmol/h/L

  3. Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period

    Time frame: Through end of EZN-2279 study treatment, up to 203 weeks

    Trough ADA activity levels, mmol/h/L

  4. Summary of Trough dAXP Levels in EZN-2279 Treatment Period

    Time frame: From Baseline through Week T-21

    Trough dAXP levels, mmol/L

  5. Summary of Trough dAXP Levels in EZN-2279 Maintenance Period

    Time frame: Through end of EZN-2279 study treatment, up to 203 weeks

    Trough dAXP levels, mmol/L

  6. Number of Patients With Infections and Hospitalizations

    Time frame: Through end of EZN-2279 study treatment, up to 203 weeks

    Infections were documented clinically with signs and symptoms without microbiologic cultures or with positive viral or bacterial cultures

  7. Duration of Hospitalization

    Time frame: Through end of EZN-2279 study treatment, up to 203 weeks

  8. Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period

    Time frame: From Week 34 to End of Study/Early Discontinuation, up to 203 weeks

    Number of patients with total erythrocyte dAXP concentration from a trough blood sample <0.02 mmol/L

Sponsors and collaborators

Lead sponsor

Leadiant Biosciences, Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2014
Primary completion
2018
Study completion
2019
First posted
Aug 19, 2011
Registry last updated
Apr 16, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.