Phase 3
N=7
EZN-2279 in Patients With ADA-SCID
ADA-SCID · Adenosine Deaminase Deficiency · Severe Combined Immunodeficiency
Bottom Line
View on ClinicalTrials.gov: NCT01420627 ↗Enrolled (actual)
7
Serious AEs
28.6%
Results posted
Apr 2020
Primary outcome: Primary: Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period — 6; 6; 6; 5 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- EZN-2279 (Biological); Adagen (Biological)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Leadiant Biosciences, Inc.
- Primary completion
- Apr 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period |
6; 6; 6; 5; 6; 6 | — |
| SECONDARY Safety Summary Data |
6; 7; 4; 1; 2; 3 | — |
| SECONDARY Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period |
15.7800; 14.0950; -1.7450; 23.3850; 7.6500; 28.5600 | — |
| SECONDARY Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period |
15.7800; 31.1000; 14.0350; 37.9000; 21.2600; 35.3100 | — |
| SECONDARY Summary of Trough dAXP Levels in EZN-2279 Treatment Period |
0.0020; 0.0020; 0.0000; 0.0020; 0.0000; 0.0020 | — |
| SECONDARY Summary of Trough dAXP Levels in EZN-2279 Maintenance Period |
0.0020; 0.0020; 0.0000; 0.0020; 0.0000; 0.0020 | — |
| SECONDARY Number of Patients With Infections and Hospitalizations |
5; 3 | — |
| SECONDARY Duration of Hospitalization |
5.0 | — |
| SECONDARY Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period |
6; 6; 6; 3; 5; 5 | — |
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
- 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 6 months prior to study entry.
- Have both of the following during the Adagen® Lead-in phase of the study prior to EZN-2279 transition:
- Trough plasma ADA activity >15 μmol/h/mL while receiving Adagen® and
- 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 detectable neutralizing anti-Adagen® antibodies at screening evaluation
- Severe thrombocytopenia (platelet count <50 x 10^9/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, 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
Data sourced from ClinicalTrials.gov (NCT01420627). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.