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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT01420627
Date of registration: 18/08/2011
Prospective Registration: Yes
Primary sponsor: Leadiant Biosciences, Inc.
Public title: EZN-2279 in Patients With ADA-SCID
Scientific 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
Date of first enrolment: January 24, 2014
Target sample size: 7
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT01420627
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 3
Countries of recruitment
United States
Contacts
Name:     Elie Haddad, MD, PhD
Address: 
Telephone:
Email:
Affiliation:  Université de Montréal
Key inclusion & exclusion 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



Age minimum: N/A
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
ADA-SCID
Adenosine Deaminase Deficiency
Severe Combined Immunodeficiency
Intervention(s)
Biological: Adagen
Biological: EZN-2279
Primary Outcome(s)
Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period [Time Frame: Baseline through Week T-21]
Secondary Outcome(s)
Duration of Hospitalization [Time Frame: Through end of EZN-2279 study treatment, up to 203 weeks]
Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period [Time Frame: From Week 34 to End of Study/Early Discontinuation, up to 203 weeks]
Number of Patients With Infections and Hospitalizations [Time Frame: Through end of EZN-2279 study treatment, up to 203 weeks]
Safety Summary Data [Time Frame: Through end of EZN-2279 study treatment, up to 203 weeks]
Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period [Time Frame: Through end of EZN-2279 study treatment, up to 203 weeks]
Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period [Time Frame: From Baseline through Week T-21]
Summary of Trough dAXP Levels in EZN-2279 Maintenance Period [Time Frame: Through end of EZN-2279 study treatment, up to 203 weeks]
Summary of Trough dAXP Levels in EZN-2279 Treatment Period [Time Frame: From Baseline through Week T-21]
Secondary ID(s)
STP-2279-002
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available: Yes
Date Posted: 16/04/2020
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT01420627
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