EZN-2279 in Patients With ADA-SCID

This study is not yet open for participant recruitment.

Verified May 2013 by Sigma Tau Pharmaceuticals, Inc.

Sponsor:

Information provided by (Responsible Party):

Sigma Tau Pharmaceuticals, Inc.

ClinicalTrials.gov Identifier:

NCT01420627

First received: August 18, 2011

Last updated: May 16, 2013

Last verified: May 2013

History of Changes

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.

Condition	Intervention	Phase
ADA-SCID Adenosine Deaminase Deficiency Severe Combined Immunodeficiency	Biological: EZN-2279	Phase 3

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Open Label Primary Purpose: Treatment
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

Resource links provided by NLM:

Genetics Home Reference related topics: adenosine deaminase deficiency complement factor I deficiency purine nucleoside phosphorylase deficiency X-linked severe combined immunodeficiency ZAP70-related severe combined immunodeficiency

U.S. FDA Resources

Further study details as provided by Sigma Tau Pharmaceuticals, Inc.:

Primary Outcome Measures:

total erythrocyte dAXP concentration from a trough blood sample [ Time Frame: through 21 weeks of EZN-2279 treatment ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

plasma ADA activity [ Time Frame: through 21 weeks of EZN-2279 treatment ] [ Designated as safety issue: No ]
Immune status [ Time Frame: through end of EZN-2279 treatment ] [ Designated as safety issue: No ]
includes absolute lymphocyte counts, lymphocyte subset analysis, quantitative immunoglobulin concentration
Safety [ Time Frame: through end of EZN-2279 study treatment ] [ Designated as safety issue: Yes ]
adverse events, serious adverse events, physical examinations, laboratory evaluations and immunogenicity
Clinical Status [ Time Frame: through end of EZN-2279 treatment ] [ Designated as safety issue: No ]
infection rate, incidence and duration of hospitalizations, overall survival, performance status

Estimated Enrollment:	9
Study Start Date:	October 2013
Estimated Study Completion Date:	August 2017
Estimated Primary Completion Date:	August 2016 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: EZN-2279 Patients crossed over to receive EZN-2279 following an Adagen lead-in period	Biological: EZN-2279 weekly administration of EZN-2279 via IM injection Other Name: rADA

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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 3 months prior to study entry.
Have both during the Adagen® Lead-in phase of the study:
1. Trough plasma ADA activity >15 μmol/h/mL while receiving Adagen®
2. 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 neutralizing anti-Adagen® antibodies at screening evaluation.
Severe thrombocytopenia (platelet count <50 x 109/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 or Sigma-Tau, 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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01420627

Contacts

Contact: William K Lohr	301-670-2499	william.lohr@sigmatau.com
Contact: Thomas Genna	301-670-1516	thomas.genna@sigmatau.com

Sponsors and Collaborators

Sigma Tau Pharmaceuticals, Inc.

More Information

No publications provided

Responsible Party:	Sigma Tau Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:	NCT01420627 History of Changes
Other Study ID Numbers:	STP-2279-002
Study First Received:	August 18, 2011
Last Updated:	May 16, 2013
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Severe Combined Immunodeficiency
Immunologic Deficiency Syndromes
Agammaglobulinemia
Immune System Diseases
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders

Metabolic Diseases
Blood Protein Disorders
Hematologic Diseases
Lymphoproliferative Disorders
Lymphatic Diseases

ClinicalTrials.gov processed this record on May 19, 2013

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