EZN-2279 in Patients With ADA-SCID

Severe Combined Immunodeficiency Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01420627
• Other study ID #: STP-2279-002
• Status: Completed
• Phase: Phase 3
• Start date: January 24, 2014
• Est. completion date: May 29, 2019

Study information

• Verified date: April 2020
• Source: Leadiant Biosciences, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 7
• Est. completion date: May 29, 2019
• Est. primary completion date: April 10, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• ADA-SCID
• Adenosine Deaminase Deficiency
• Immunologic Deficiency Syndromes
• Severe Combined Immunodeficiency

Intervention

Biological:
• EZN-2279
Weekly administration of EZN-2279 via IM injection
• Adagen

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Leadiant Biosciences, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Safety Summary Data	Summary of adverse events and serious adverse events	Through end of EZN-2279 study treatment, up to 203 weeks
Secondary	Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period	Trough ADA activity, mmol/h/L	From Baseline through Week T-21
Secondary	Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period	Trough ADA activity levels, mmol/h/L	Through end of EZN-2279 study treatment, up to 203 weeks
Secondary	Summary of Trough dAXP Levels in EZN-2279 Treatment Period	Trough dAXP levels, mmol/L	From Baseline through Week T-21
Secondary	Summary of Trough dAXP Levels in EZN-2279 Maintenance Period	Trough dAXP levels, mmol/L	Through end of EZN-2279 study treatment, up to 203 weeks
Secondary	Number of Patients With Infections and Hospitalizations	Infections were documented clinically with signs and symptoms without microbiologic cultures or with positive viral or bacterial cultures	Through end of EZN-2279 study treatment, up to 203 weeks
Secondary	Duration of Hospitalization		Through end of EZN-2279 study treatment, up to 203 weeks
Secondary	Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period	Number of patients with total erythrocyte dAXP concentration from a trough blood sample <0.02 mmol/L	From Week 34 to End of Study/Early Discontinuation, up to 203 weeks