EZN-2279 in Patients With ADA-SCID
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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
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
ADA-SCID Adenosine Deaminase Deficiency Severe Combined Immunodeficiency | Biological: EZN-2279 Biological: Adagen | Phase 3 |
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 |

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
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Biological: EZN-2279
Weekly administration of EZN-2279 via IM injection
Other Name: rADA Biological: Adagen |
- 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
- Safety Summary Data [ Time Frame: Through end of EZN-2279 study treatment, up to 203 weeks ]Summary of adverse events and serious adverse events
- 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
- 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
- Summary of Trough dAXP Levels in EZN-2279 Treatment Period [ Time Frame: From Baseline through Week T-21 ]Trough dAXP levels, mmol/L
- 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
- 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
- Duration of Hospitalization [ Time Frame: Through end of EZN-2279 study treatment, up to 203 weeks ]
- 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

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Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.
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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

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
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 |
Principal Investigator: | Elie Haddad, MD, PhD | Université de Montréal |
Documents provided by Leadiant Biosciences, Inc.:
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 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Severe Combined Immunodeficiency Immunologic Deficiency Syndromes Immune System Diseases |
Infant, Newborn, Diseases DNA Repair-Deficiency Disorders Metabolic Diseases |