Status:
COMPLETED
EZN-2279 in Patients With ADA-SCID
Lead Sponsor:
Leadiant Biosciences, Inc.
Conditions:
ADA-SCID
Adenosine Deaminase Deficiency
Eligibility:
All Genders
Phase:
PHASE3
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.
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
January 24 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 29 2019
Estimated Enrollment :
7 Patients enrolled
Trial Details
Trial ID
NCT01420627
Start Date
January 24 2014
End Date
May 29 2019
Last Update
April 16 2020
Active Locations (6)
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1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
2
University of California San Francisco
San Francisco, California, United States, 94158
3
National Jewish Health
Denver, Colorado, United States, 80206-2761
4
UBMD
Buffalo, New York, United States, 14203