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Clinical Trials/NCT01420627
NCT01420627CompletedPhase 3

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

Leadiant Biosciences, Inc.12 sites in 1 country7 target enrollmentStarted: January 24, 2014Last updated:
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Conditions

Trial Snapshot

Phase
Phase 3
Status
Completed
Enrollment
7
Locations
12
Primary Endpoint
Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period
Study OverviewStudy DesignEligibility CriteriaOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

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.

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Sex
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.
  • •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

Outcomes

Primary Outcomes

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

Secondary Outcomes

  • Safety Summary Data(Through end of EZN-2279 study treatment, up to 203 weeks)
  • Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period(From Baseline through Week T-21)
  • Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period(Through end of EZN-2279 study treatment, up to 203 weeks)
  • Duration of Hospitalization(Through end of EZN-2279 study treatment, up to 203 weeks)
  • Summary of Trough dAXP Levels in EZN-2279 Treatment Period(From Baseline through Week T-21)
  • Summary of Trough dAXP Levels in EZN-2279 Maintenance Period(Through end of EZN-2279 study treatment, up to 203 weeks)
  • Number of Patients With Infections and Hospitalizations(Through end of EZN-2279 study treatment, up to 203 weeks)
  • Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period(From Week 34 to End of Study/Early Discontinuation, up to 203 weeks)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (12)

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