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Purpose

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.

Conditions

Eligibility

Eligible Ages
All ages
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
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 names:
    • rADA
  • Biological: Adagen

More Details

Status
Completed
Sponsor
Leadiant Biosciences, Inc.

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.