EZN-2279 in Patients With ADA-SCID

Completed · Phase 3

Conditions studied: ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency

In brief

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.

Key facts

Study ID

NCT01420627

Run by

Leadiant Biosciences, Inc.

People needed

7

Starts

2014-01-24

Expected to finish

2019-05-29

Last updated by the study team

2020-04-16

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Children's Hospital Los Angeles — Los Angeles, California, United States
• University of California San Francisco — San Francisco, California, United States
• National Jewish Health — Denver, Colorado, United States
• UBMD — Buffalo, New York, United States
• Albert Einstein College of Medicine — The Bronx, New York, United States
• Penn State College of Medicine The Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States

Full record on ClinicalTrials.gov

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