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.
William K Lohr, Phone: 301-670-2499, Email: firstname.lastname@example.org
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
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
plasma ADA activity
Minimum age: N/A.
Maximum age: N/A.
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
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
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
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
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
Locations and Contacts
William K Lohr, Phone: 301-670-2499, Email: email@example.comAdditional Information
Starting date: May 2012
Last updated: January 11, 2012