EZN-2279 in Patients With ADA-SCID

NCT01420627 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: STP-2279-002
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 6

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

Outcome Measures

Primary Outcomes (1)

• Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period

  Number of patients with total erythrocyte dAXP concentration from a trough blood sample \<0.02 mmol/L

  Baseline through Week T-21

Secondary Outcomes (8)

• 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

• 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

Study Arms (1)

Adagen/EZN-2279

EXPERIMENTAL

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; Biological: Adagen

Interventions

EZN-2279 BIOLOGICAL

Weekly administration of EZN-2279 via IM injection

Also known as: rADA

Adagen/EZN-2279

Adagen BIOLOGICAL

Adagen/EZN-2279

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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

Sponsors & Collaborators

• Leadiant Biosciences, Inc.: lead

Study Sites (6)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

University of California San Francisco

San Francisco, California, 94158, United States

Location

National Jewish Health

Denver, Colorado, 80206-2761, United States

Location

UBMD

Buffalo, New York, 14203, United States

Location

Albert Einstein College of Medicine

The Bronx, New York, 10461, United States

Location

Penn State College of Medicine The Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

MeSH Terms

Conditions

Severe combined immunodeficiency due to adenosine deaminase deficiency; Severe Combined Immunodeficiency

Interventions

pegademase bovine

Condition Hierarchy (Ancestors)

Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Immunologic Deficiency Syndromes; Immune System Diseases

Results Point of Contact

• Title: Scott Rodgers, MS, CCRA, ACRP-PM / Sr Director Clinical Operations
• Organization: Leadiant Biosciences, Inc.

scott.rodgers@leadiant.com

301-670-1565

Study Officials

• Elie Haddad, MD, PhD

  Université de Montréal

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 18, 2011
• First Posted: August 19, 2011
• Study Start: January 24, 2014
• Primary Completion: April 10, 2018
• Study Completion: May 29, 2019
• Last Updated: April 16, 2020
• Results First Posted: April 16, 2020

Record last verified: 2020-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)