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EZN-2279 in Patients With ADA-SCID

Information source: Sigma Tau Pharmaceuticals, Inc.
Information obtained from ClinicalTrials.gov on February 07, 2013
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: ADA-SCID; Adenosine Deaminase Deficiency; Severe Combined Immunodeficiency

Intervention: EZN-2279 (Biological)

Phase: Phase 3

Status: Not yet recruiting

Sponsored by: Sigma Tau Pharmaceuticals, Inc.

Overall contact:
William K Lohr, Phone: 301-670-2499, Email: william.lohr@sigmatau.com

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.

Clinical Details

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

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

Primary outcome: total erythrocyte dAXP concentration from a trough blood sample

Secondary outcome:

plasma ADA activity

Immune status

Safety

Clinical Status

Eligibility

Minimum age: N/A. Maximum age: N/A. Gender(s): Both.

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 3 months prior to study entry.

3. 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

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 neutralizing anti-Adagen® antibodies at screening evaluation.

3. Severe thrombocytopenia (platelet count <50 x 109/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 or Sigma-Tau, 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

Locations and Contacts

William K Lohr, Phone: 301-670-2499, Email: william.lohr@sigmatau.com

Additional Information

Starting date: May 2012
Last updated: January 11, 2012

Page last updated: February 07, 2013

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