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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.
 
ClinicalTrials.gov Identifier: NCT01420627
Recruitment Status : Completed
First Posted : August 19, 2011
Results First Posted : April 16, 2020
Last Update Posted : April 16, 2020
Sponsor:
Information provided by (Responsible Party):
Leadiant Biosciences, Inc.

Study Description
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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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Allocation: N/A
Intervention Model: Single Group 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
Actual Study Start Date : January 24, 2014
Actual Primary Completion Date : April 10, 2018
Actual Study Completion Date : May 29, 2019

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Adagen/EZN-2279
Patients started on Adagen and crossed over to experimental EZN-2279 treatment after at least a 3 week Lead-in Period on Adagen
 Biological: EZN-2279
Weekly administration of EZN-2279 via IM injection
Other Name: rADA

Biological: Adagen


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
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 6 months prior to study entry.

  3. Have both of the following during the Adagen® Lead-in phase of the study prior to EZN-2279 transition:

    1. Trough plasma ADA activity >15 μmol/h/mL while receiving Adagen® and
    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 detectable neutralizing anti-Adagen® antibodies at screening evaluation
  3. Severe thrombocytopenia (platelet count <50 x 10^9/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, 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
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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 ClinicalTrials.gov identifier (NCT number): NCT01420627


Locations
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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, 94158
United States, Colorado
National Jewish Health
Denver, Colorado, United States, 80206-2761
United States, New York
Albert Einstein College of Medicine
Bronx, New York, United States, 10461
UBMD
Buffalo, New York, United States, 14203
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.
Investigators
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Principal Investigator: Elie Haddad, MD, PhD Université de Montréal
  Study Documents (Full-Text)

Documents provided by Leadiant Biosciences, Inc.:
Statistical Analysis Plan  [PDF] April 4, 2019
Study Protocol  [PDF] August 23, 2017

More Information
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Responsible Party: Leadiant Biosciences, Inc.
ClinicalTrials.gov Identifier: NCT01420627    
Other Study ID Numbers: STP-2279-002
First Posted: August 19, 2011    Key Record Dates
Results First Posted: April 16, 2020
Last Update Posted: April 16, 2020
Last Verified: April 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Severe Combined Immunodeficiency
Immunologic Deficiency Syndromes
Immune System Diseases
Primary Immunodeficiency Diseases
 Genetic Diseases, Inborn
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases