Back to resultsEZN-2279 in Patients With ADA-SCID
Primary Purpose
ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency
Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
EZN-2279
Adagen
Sponsored by
About this trial
This is an interventional treatment trial for ADA-SCID
Eligibility Criteria
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
Sites / Locations
- Children's Hospital Los Angeles
- University of California San Francisco
- National Jewish Health
- Albert Einstein College of Medicine
- UBMD
- Penn State College of Medicine The Milton S. Hershey Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Adagen/EZN-2279
Arm Description
Patients started on Adagen and crossed over to experimental EZN-2279 treatment after at least a 3 week Lead-in Period on Adagen
Outcomes
Primary Outcome Measures
Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period
Number of patients with total erythrocyte dAXP concentration from a trough blood sample <0.02 mmol/L
Secondary Outcome Measures
Safety Summary Data
Summary of adverse events and serious adverse events
Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period
Trough ADA activity, mmol/h/L
Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period
Trough ADA activity levels, mmol/h/L
Summary of Trough dAXP Levels in EZN-2279 Treatment Period
Trough dAXP levels, mmol/L
Summary of Trough dAXP Levels in EZN-2279 Maintenance Period
Trough dAXP levels, mmol/L
Number of Patients With Infections and Hospitalizations
Infections were documented clinically with signs and symptoms without microbiologic cultures or with positive viral or bacterial cultures
Duration of Hospitalization
Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period
Number of patients with total erythrocyte dAXP concentration from a trough blood sample <0.02 mmol/L
Full Information
NCT ID
NCT01420627
First Posted
August 18, 2011
Last Updated
April 6, 2020
Sponsor
Leadiant Biosciences, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT01420627
Brief Title
EZN-2279 in Patients With ADA-SCID
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
Study Type
Interventional
2. Study Status
Record Verification Date
April 2020
Overall Recruitment Status
Completed
Study Start Date
January 24, 2014 (Actual)
Primary Completion Date
April 10, 2018 (Actual)
Study Completion Date
May 29, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Leadiant Biosciences, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
7 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Adagen/EZN-2279
Arm Type
Experimental
Arm Description
Patients started on Adagen and crossed over to experimental EZN-2279 treatment after at least a 3 week Lead-in Period on Adagen
Intervention Type
Biological
Intervention Name(s)
EZN-2279
Other Intervention Name(s)
rADA
Intervention Description
Weekly administration of EZN-2279 via IM injection
Intervention Type
Biological
Intervention Name(s)
Adagen
Primary Outcome Measure Information:
Title
Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period
Description
Number of patients with total erythrocyte dAXP concentration from a trough blood sample <0.02 mmol/L
Time Frame
Baseline through Week T-21
Secondary Outcome Measure Information:
Title
Safety Summary Data
Description
Summary of adverse events and serious adverse events
Time Frame
Through end of EZN-2279 study treatment, up to 203 weeks
Title
Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period
Description
Trough ADA activity, mmol/h/L
Time Frame
From Baseline through Week T-21
Title
Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period
Description
Trough ADA activity levels, mmol/h/L
Time Frame
Through end of EZN-2279 study treatment, up to 203 weeks
Title
Summary of Trough dAXP Levels in EZN-2279 Treatment Period
Description
Trough dAXP levels, mmol/L
Time Frame
From Baseline through Week T-21
Title
Summary of Trough dAXP Levels in EZN-2279 Maintenance Period
Description
Trough dAXP levels, mmol/L
Time Frame
Through end of EZN-2279 study treatment, up to 203 weeks
Title
Number of Patients With Infections and Hospitalizations
Description
Infections were documented clinically with signs and symptoms without microbiologic cultures or with positive viral or bacterial cultures
Time Frame
Through end of EZN-2279 study treatment, up to 203 weeks
Title
Duration of Hospitalization
Time Frame
Through end of EZN-2279 study treatment, up to 203 weeks
Title
Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period
Description
Number of patients with total erythrocyte dAXP concentration from a trough blood sample <0.02 mmol/L
Time Frame
From Week 34 to End of Study/Early Discontinuation, up to 203 weeks
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Elie Haddad, MD, PhD
Organizational Affiliation
Université de Montréal
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
University of California San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94158
Country
United States
Facility Name
National Jewish Health
City
Denver
State/Province
Colorado
ZIP/Postal Code
80206-2761
Country
United States
Facility Name
Albert Einstein College of Medicine
City
Bronx
State/Province
New York
ZIP/Postal Code
10461
Country
United States
Facility Name
UBMD
City
Buffalo
State/Province
New York
ZIP/Postal Code
14203
Country
United States
Facility Name
Penn State College of Medicine The Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
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EZN-2279 in Patients With ADA-SCID
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