ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency
Conditions
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.
Interventions
BIOLOGICALEZN-2279
Weekly administration of EZN-2279 via IM injection
BIOLOGICALAdagen
Sponsors
Leadiant Biosciences, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Baseline through Week T-21 | Number of patients with total erythrocyte dAXP concentration from a trough blood sample \<0.02 mmol/L |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | From Baseline through Week T-21 | Trough ADA activity, mmol/h/L |
| Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Through end of EZN-2279 study treatment, up to 203 weeks | Trough ADA activity levels, mmol/h/L |
| Summary of Trough dAXP Levels in EZN-2279 Treatment Period | From Baseline through Week T-21 | Trough dAXP levels, mmol/L |
| Safety Summary Data | Through end of EZN-2279 study treatment, up to 203 weeks | Summary of adverse events and serious adverse events |
| Number of Patients With Infections and Hospitalizations | Through end of EZN-2279 study treatment, up to 203 weeks | Infections were documented clinically with signs and symptoms without microbiologic cultures or with positive viral or bacterial cultures |
| Duration of Hospitalization | Through end of EZN-2279 study treatment, up to 203 weeks | — |
| Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | From Week 34 to End of Study/Early Discontinuation, up to 203 weeks | Number of patients with total erythrocyte dAXP concentration from a trough blood sample \<0.02 mmol/L |
| Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Through end of EZN-2279 study treatment, up to 203 weeks | Trough dAXP levels, mmol/L |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Adagen/EZN-2279 Patients started on Adagen and crossed over to experimental EZN-2279 treatment after at least a 3 week Lead-in Period on Adagen
EZN-2279: Weekly administration of EZN-2279 via IM injection
Adagen | 7 |
| Total | 7 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| EZN-2279 Treatment Period | Adverse Event | 1 |
Baseline characteristics
| Characteristic | Adagen/EZN-2279 |
|---|---|
| Age, Continuous | 21.3 years STANDARD_DEVIATION 9.52 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 3 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 4 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants |
| Race (NIH/OMB) White | 4 Participants |
| Region of Enrollment United States | 7 participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 7 | 0 / 7 |
| other Total, other adverse events | 6 / 7 | 7 / 7 |
| serious Total, serious adverse events | 0 / 7 | 4 / 7 |
Outcome results
Primary
Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period
Number of patients with total erythrocyte dAXP concentration from a trough blood sample \<0.02 mmol/L
Time frame: Baseline through Week T-21
Population: Completer
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Baseline | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-1 | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-3 | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-5 | 5 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-7 | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-8 | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-9 | 3 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-10 | 5 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-11 | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-13 | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-15 | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-17 | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-19 | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period | Week T-21 | 6 Participants |
Secondary
Duration of Hospitalization
Time frame: Through end of EZN-2279 study treatment, up to 203 weeks
Population: As-treated population who were hospitalized
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| EZN-2279 | Duration of Hospitalization | 5.0 Days | Standard Deviation 2.16 |
Secondary
Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period
Number of patients with total erythrocyte dAXP concentration from a trough blood sample \<0.02 mmol/L
Time frame: From Week 34 to End of Study/Early Discontinuation, up to 203 weeks
Population: Completer
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Baseline | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 34 | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 47 | 6 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 60 | 3 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 73 | 5 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 86 | 5 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 99 | 5 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 112 | 4 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 125 | 4 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 138 | 3 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 151 | 3 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 164 | 3 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 177 | 3 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 190 | 3 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | Week 203 | 3 Participants |
| EZN-2279 | Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period | End of Study/Early Discontinuation | 3 Participants |
Secondary
Number of Patients With Infections and Hospitalizations
Infections were documented clinically with signs and symptoms without microbiologic cultures or with positive viral or bacterial cultures
Time frame: Through end of EZN-2279 study treatment, up to 203 weeks
Population: As-treated
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| EZN-2279 | Number of Patients With Infections and Hospitalizations | Patients with infection | 5 Participants |
| EZN-2279 | Number of Patients With Infections and Hospitalizations | Patients with hospitalizations | 3 Participants |
Secondary
Safety Summary Data
Summary of adverse events and serious adverse events
Time frame: Through end of EZN-2279 study treatment, up to 203 weeks
Population: As-treated
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| EZN-2279 | Safety Summary Data | Severe TEAE | 0 Participants |
| EZN-2279 | Safety Summary Data | Moderate related AE | 0 Participants |
| EZN-2279 | Safety Summary Data | Moderate TEAE | 2 Participants |
| EZN-2279 | Safety Summary Data | Severe related AE | 0 Participants |
| EZN-2279 | Safety Summary Data | Treatment-related adverse event | 1 Participants |
| EZN-2279 | Safety Summary Data | Patients with any SAE | 0 Participants |
| EZN-2279 | Safety Summary Data | Mild TEAE | 4 Participants |
| EZN-2279 | Safety Summary Data | Patients discontinued treatment due to AE | 0 Participants |
| EZN-2279 | Safety Summary Data | Mild related AE | 1 Participants |
| EZN-2279 | Safety Summary Data | Non-serious AEs | 6 Participants |
| EZN-2279 | Safety Summary Data | Patients with treatment-emergent adverse events | 6 Participants |
| EZN-2279 | Safety Summary Data | Non-serious AEs | 7 Participants |
| EZN-2279 | Safety Summary Data | Patients with treatment-emergent adverse events | 7 Participants |
| EZN-2279 | Safety Summary Data | Mild TEAE | 1 Participants |
| EZN-2279 | Safety Summary Data | Moderate TEAE | 3 Participants |
| EZN-2279 | Safety Summary Data | Severe TEAE | 3 Participants |
| EZN-2279 | Safety Summary Data | Treatment-related adverse event | 2 Participants |
| EZN-2279 | Safety Summary Data | Mild related AE | 1 Participants |
| EZN-2279 | Safety Summary Data | Moderate related AE | 0 Participants |
| EZN-2279 | Safety Summary Data | Severe related AE | 1 Participants |
| EZN-2279 | Safety Summary Data | Patients with any SAE | 4 Participants |
| EZN-2279 | Safety Summary Data | Patients discontinued treatment due to AE | 1 Participants |
Secondary
Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period
Trough ADA activity levels, mmol/h/L
Time frame: Through end of EZN-2279 study treatment, up to 203 weeks
Population: Completer
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 60 | 35.3100 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 60 | 20.1300 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Baseline | 15.7800 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 34 | 31.1000 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 34 | 14.0350 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 47 | 37.9000 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 47 | 21.2600 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 73 | 42.1900 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 73 | 22.5900 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 86 | 41.2000 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 86 | 25.5600 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 99 | 42.200 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 99 | 26.5800 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 112 | 31.6750 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 112 | 10.4250 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 125 | 39.2700 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 125 | 21.1150 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 138 | 46.0600 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 138 | 26.4600 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 151 | 36.6700 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 151 | 17.070 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 164 | 34.4500 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 164 | 11.5500 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 177 | 33.1300 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 177 | 13.5300 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 190 | 32.5000 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 190 | 11.5800 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Week 203 | 36.3300 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 203 | 16.7300 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | End of Study/Early Discontinuation | 39.7700 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period | Change from Baseline at End of Study/Early Discont | 22.3800 mmol/h/L |
Secondary
Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period
Trough ADA activity, mmol/h/L
Time frame: From Baseline through Week T-21
Population: Completer
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Baseline | 15.7800 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-1 | 14.0950 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-1 | -1.7450 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-3 | 23.3850 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-3 | 7.6500 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-5 | 28.5600 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-5 | 13.3800 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-7 | 34.8000 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-7 | 19.000 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-8 | 32.9300 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-8 | 18.7400 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-9 | 38.8750 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-9 | 24.6840 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-10 | 37.7000 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from baseline at Week T-10 | 24.5000 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-11 | 36.4350 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-11 | 21.1950 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-13 | 36.1000 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-13 | 21.1150 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-15 | 35.7850 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-15 | 20.3950 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-17 | 31.1000 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-17 | 12.3500 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-19 | 32.4200 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-19 | 14.4300 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Week T-21 | 30.1500 mmol/h/L |
| EZN-2279 | Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-21 | 13.7500 mmol/h/L |
Secondary
Summary of Trough dAXP Levels in EZN-2279 Maintenance Period
Trough dAXP levels, mmol/L
Time frame: Through end of EZN-2279 study treatment, up to 203 weeks
Population: Completer
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Baseline | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 34 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 34 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 47 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 47 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 60 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 60 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 73 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 73 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 86 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 86 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 99 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 99 | 0.000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 112 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 112 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 125 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 125 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 138 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 138 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 151 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 151 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 164 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 164 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 177 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 177 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 190 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 190 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Week 203 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at Week 203 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | End of Study/Early Discontinuation | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Maintenance Period | Change from Baseline at End of Study/Early Discont | 0.0000 mmol/L |
Secondary
Summary of Trough dAXP Levels in EZN-2279 Treatment Period
Trough dAXP levels, mmol/L
Time frame: From Baseline through Week T-21
Population: Completer
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Baseline | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-1 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-1 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-3 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-3 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-5 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-5 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-7 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-7 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-8 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-8 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-9 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-9 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-10 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-10 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-11 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-11 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-13 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-13 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-15 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-15 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-17 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-17 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-19 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-19 | 0.0000 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Week T-21 | 0.0020 mmol/L |
| EZN-2279 | Summary of Trough dAXP Levels in EZN-2279 Treatment Period | Change from Baseline at Week T-21 | 0.0000 mmol/L |