Clinical trial NCT01420627: EZN-2279 in Patients With ADA-SCID
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: ADA-SCID
Adenosine Deaminase Deficiency
Severe Combined Immunodeficiency
Lead sponsor: Leadiant Biosciences, Inc.
Sponsor class: INDUSTRY
First posted: 2011-08-19
Enrollment: 7
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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 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

Primary outcomes:
measure: Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period
timeFrame: Baseline through Week T-21
Secondary outcomes:
measure: Safety Summary Data
timeFrame: Through end of EZN-2279 study treatment, up to 203 weeks
measure: Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period
timeFrame: From Baseline through Week T-21
measure: Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period
timeFrame: Through end of EZN-2279 study treatment, up to 203 weeks
measure: Summary of Trough dAXP Levels in EZN-2279 Treatment Period
timeFrame: From Baseline through Week T-21
measure: Summary of Trough dAXP Levels in EZN-2279 Maintenance Period
timeFrame: Through end of EZN-2279 study treatment, up to 203 weeks
measure: Number of Patients With Infections and Hospitalizations
timeFrame: Through end of EZN-2279 study treatment, up to 203 weeks
measure: Duration of Hospitalization
timeFrame: Through end of EZN-2279 study treatment, up to 203 weeks
measure: Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period
timeFrame: From Week 34 to End of Study/Early Discontinuation, up to 203 weeks
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
city: Bronx
country: United States
facility: Albert Einstein College of Medicine
state: New York
city: Buffalo
country: United States
facility: UBMD
state: New York
city: Hershey
country: United States
facility: Penn State College of Medicine The Milton S. Hershey Medical Center
state: Pennsylvania
Study officials:
affiliation: Université de Montréal
name: Elie Haddad, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: