EZN-2279 in Patients With ADA-SCID

NCT ID: NCT01420627

Last Updated: 2020-04-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-01-24
• Study Completion Date: 2019-05-29

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.

Conditions

ADA-SCID; Adenosine Deaminase Deficiency; Severe Combined Immunodeficiency

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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

Group Type: EXPERIMENTAL

EZN-2279

Intervention Type: BIOLOGICAL

Weekly administration of EZN-2279 via IM injection

Adagen

Intervention Type: BIOLOGICAL

Interventions

EZN-2279

Weekly administration of EZN-2279 via IM injection

Intervention Type: BIOLOGICAL

Adagen

Intervention Type: BIOLOGICAL

Other Intervention Names

rADA

Eligibility Criteria

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

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Leadiant Biosciences, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Elie Haddad, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Université de Montréal

Locations

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

Countries

United States

Provided Documents

Document Type: Statistical Analysis Plan

View Document

Document Type: Study Protocol

View Document

Other Identifiers

STP-2279-002

Identifier Type: -

Identifier Source: org_study_id