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CompletedNCT01420627Updated Apr 16, 2020Results posted

EZN-2279 in Patients With ADA-SCID

A Phase 3 interventional study of EZN-2279 and Adagen in ADA-SCID, Adenosine Deaminase Deficiency and Severe Combined Immunodeficiency, sponsored by Leadiant Biosciences, Inc.. Completed at 6 sites in United States. Per ClinicalTrials.gov, last updated 2020-04-16.

Sponsored by Leadiant Biosciences, Inc. · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
7
Allocation
Not applicable
Sex
All
01

Study 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.

02

Conditions studied

  • ADA-SCID
  • Adenosine Deaminase Deficiency
  • Severe Combined Immunodeficiency
03

In context

Severe Combined Immunodeficiency

64 studies on the registry are indexed under Severe Combined Immunodeficiency; 15 are open to participants now.

This study's enrollment of 7 is below the median of 9 across 34 interventional studies indexed under Severe Combined Immunodeficiency.

Browse Severe Combined Immunodeficiency studies →

Lead sponsor

Leadiant Biosciences, Inc. is the lead sponsor of 5 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts 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

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
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
7 participants (actual)

Study arms

  • Experimental
    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

    Biological: EZN-2279 · Biological: Adagen

Interventions

  • BiologicalEZN-2279

    Weekly administration of EZN-2279 via IM injection

    Also known as: rADA

  • BiologicalAdagen
06

What researchers measure

Primary outcomes

  1. 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

Secondary outcomes

  1. Safety Summary Data

    Summary of adverse events and serious adverse events

    Time frame: Through end of EZN-2279 study treatment, up to 203 weeks

  2. 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

  3. 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

  4. Summary of Trough dAXP Levels in EZN-2279 Treatment Period

    Trough dAXP levels, mmol/L

    Time frame: From Baseline through Week T-21

  5. 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

  6. 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

  7. Duration of Hospitalization

    Time frame: Through end of EZN-2279 study treatment, up to 203 weeks

  8. 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

07

Results

Posted Apr 16, 2020

Participant flow

Adagen Lead-in Period
Participant flow — Adagen Lead-in Period
MilestoneAdagen/EZN-2279
Started7
Completed7
Not completed0
EZN-2279 Treatment Period
Participant flow — EZN-2279 Treatment Period
MilestoneAdagen/EZN-2279
Started7
Completed6
Not completed1
Withdrew: Adverse event1
EZN-2279 Maintenance Period
Participant flow — EZN-2279 Maintenance Period
MilestoneAdagen/EZN-2279
Started6
Completed6
Not completed0

Outcome measures

PrimaryNumber 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
Reported as:
Count of participants · Participants
Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period
ParticipantsEZN-2279
Baseline6
Week T-16
Week T-36
Week T-55
Week T-76
Week T-86
Week T-93
Week T-105
Week T-116
Week T-136
Week T-156
Week T-176
Week T-196
Week T-216
SecondarySafety Summary Data

Summary of adverse events and serious adverse events

Time frame:
Through end of EZN-2279 study treatment, up to 203 weeks
Reported as:
Count of participants · Participants
Safety Summary Data
ParticipantsAdagenEZN-2279
Patients with treatment-emergent adverse events67
Mild TEAE41
Moderate TEAE23
Severe TEAE03
Treatment-related adverse event12
Mild related AE11
Moderate related AE00
Severe related AE01
Patients with any SAE04
Patients discontinued treatment due to AE01
Non-serious AEs67
SecondarySummary of Trough ADA Activity Levels in EZN-2279 Treatment Period

Trough ADA activity, mmol/h/L

Time frame:
From Baseline through Week T-21
Reported as:
Median · mmol/h/L
Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period
mmol/h/LEZN-2279 Treatment Period
Baseline15.7800 (13.200 to 22.900)
Week T-114.0950 (9.130 to 24.600)
Change from Baseline at Week T-1-1.7450 (-8.000 to 5.000)
Week T-323.3850 (11.860 to 30.200)
Change from Baseline at Week T-37.6500 (-4.060 to 9.360)
Week T-528.5600 (25.900 to 33.040)
Change from Baseline at Week T-513.3800 (3.000 to 17.400)
Week T-734.8000 (29.240 to 42.590)
Change from Baseline at Week T-719.000 (11.000 to 26.950)
Week T-832.9300 (29.200 to 48.700)
Change from Baseline at Week T-818.7400 (6.300 to 29.100)
Week T-938.8750 (26.160 to 52.800)
Change from Baseline at Week T-924.6840 (9.600 to 33.200)
Week T-1037.7000 (23.480 to 46.200)
Change from baseline at Week T-1024.5000 (6.100 to 28.280)
Week T-1136.4350 (19.290 to 44.850)
Change from Baseline at Week T-1121.1950 (3.370 to 29.210)
Week T-1336.1000 (18.930 to 55.180)
Change from Baseline at Week T-1321.1150 (3.010 to 39.540)
Week T-1535.7850 (26.150 to 43.300)
Change from Baseline at Week T-1520.3950 (10.230 to 26.970)
Week T-1731.1000 (24.040 to 63.590)
Change from Baseline at Week T-1712.3500 (8.120 to 47.950)
Week T-1932.4200 (24.350 to 55.030)
Change from Baseline at Week T-1914.4300 (6.500 to 39.390)
Week T-2130.1500 (23.860 to 43.930)
Change from Baseline at Week T-2113.7500 (5.300 to 28.290)
SecondarySummary 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
Reported as:
Median · mmol/h/L
Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period
mmol/h/LEZN-2279 Treatment Period
Baseline15.7800 (13.200 to 22.900)
Week 3431.1000 (16.400 to 51.800)
Change from Baseline at Week 3414.0350 (0.480 to 36.160)
Week 4737.9000 (16.170 to 42.230)
Change from Baseline at Week 4721.2600 (0.250 to 26.590)
Week 6035.3100 (13.440 to 50.770)
Change from Baseline at Week 6020.1300 (-2.480 to 35.130)
Week 7342.1900 (31.250 to 47.550)
Change from Baseline at Week 7322.5900 (15.330 to 34.350)
Week 8641.2000 (24.870 to 54.960)
Change from Baseline at Week 8625.5600 (8.950 to 41.760)
Week 9942.200 (27.330 to 57.090)
Change from Baseline at Week 9926.5800 (4.430 to 37.490)
Week 11231.6750 (23.340 to 44.840)
Change from Baseline at Week 11210.4250 (7.420 to 31.640)
Week 12539.2700 (33.300 to 44.990)
Change from Baseline at Week 12521.1150 (11.190 to 31.790)
Week 13846.0600 (31.850 to 51.820)
Change from Baseline at Week 13826.4600 (8.950 to 38.620)
Week 15136.6700 (30.370 to 44.890)
Change from Baseline at Week 15117.070 (7.470 to 31.690)
Week 16434.4500 (27.950 to 41.990)
Change from Baseline at Week 16411.5500 (8.350 to 28.790)
Week 17733.1300 (26.980 to 47.850)
Change from Baseline at Week 17713.5300 (4.080 to 34.650)
Week 19032.5000 (31.180 to 38.920)
Change from Baseline at Week 19011.5800 (9.600 to 25.720)
Week 20336.3300 (33.890 to 43.310)
Change from Baseline at Week 20316.7300 (10.990 to 30.110)
End of Study/Early Discontinuation39.7700 (33.740 to 46.170)
Change from Baseline at End of Study/Early Discont22.3800 (10.840 to 30.530)
SecondarySummary of Trough dAXP Levels in EZN-2279 Treatment Period

Trough dAXP levels, mmol/L

Time frame:
From Baseline through Week T-21
Reported as:
Median · mmol/L
Summary of Trough dAXP Levels in EZN-2279 Treatment Period
mmol/LEZN-2279 Treatment Period
Baseline0.0020 (0.002 to 0.002)
Week T-10.0020 (0.002 to 0.002)
Change from Baseline at Week T-10.0000 (0.000 to 0.000)
Week T-30.0020 (0.002 to 0.002)
Change from Baseline at Week T-30.0000 (0.000 to 0.000)
Week T-50.0020 (0.002 to 0.002)
Change from Baseline at Week T-50.0000 (0.000 to 0.000)
Week T-70.0020 (0.002 to 0.002)
Change from Baseline at Week T-70.0000 (0.000 to 0.000)
Week T-80.0020 (0.002 to 0.002)
Change from Baseline at Week T-80.0000 (0.000 to 0.000)
Week T-90.0020 (0.002 to 0.002)
Change from Baseline at Week T-90.0000 (0.000 to 0.000)
Week T-100.0020 (0.002 to 0.002)
Change from Baseline at Week T-100.0000 (0.000 to 0.000)
Week T-110.0020 (0.002 to 0.002)
Change from Baseline at Week T-110.0000 (0.000 to 0.000)
Week T-130.0020 (0.002 to 0.032)
Change from Baseline at Week T-130.0000 (0.000 to 0.030)
Week T-150.0020 (0.002 to 0.002)
Change from Baseline at Week T-150.0000 (0.000 to 0.000)
Week T-170.0020 (0.002 to 0.047)
Change from Baseline at Week T-170.0000 (0.000 to 0.045)
Week T-190.0020 (0.002 to 0.002)
Change from Baseline at Week T-190.0000 (0.000 to 0.000)
Week T-210.0020 (0.002 to 0.002)
Change from Baseline at Week T-210.0000 (0.000 to 0.000)
SecondarySummary 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
Reported as:
Median · mmol/L
Summary of Trough dAXP Levels in EZN-2279 Maintenance Period
mmol/LEZN-2279 Treatment Period
Baseline0.0020 (0.002 to 0.002)
Week 340.0020 (0.002 to 0.002)
Change from Baseline at Week 340.0000 (0.000 to 0.000)
Week 470.0020 (0.002 to 0.008)
Change from Baseline at Week 470.0000 (0.000 to 0.006)
Week 600.0020 (0.002 to 0.008)
Change from Baseline at Week 600.0000 (0.000 to 0.006)
Week 730.0020 (0.002 to 0.006)
Change from Baseline at Week 730.0000 (0.000 to 0.004)
Week 860.0020 (0.002 to 0.002)
Change from Baseline at Week 860.0000 (0.000 to 0.000)
Week 990.0020 (0.002 to 0.002)
Change from Baseline at Week 990.000 (0.000 to 0.000)
Week 1120.0020 (0.002 to 0.002)
Change from Baseline at Week 1120.0000 (0.000 to 0.000)
Week 1250.0020 (0.002 to 0.002)
Change from Baseline at Week 1250.0000 (0.000 to 0.000)
Week 1380.0020 (0.002 to 0.002)
Change from Baseline at Week 1380.0000 (0.000 to 0.000)
Week 1510.0020 (0.002 to 0.005)
Change from Baseline at Week 1510.0000 (0.000 to 0.003)
Week 1640.0020 (0.002 to 0.002)
Change from Baseline at Week 1640.0000 (0.000 to 0.000)
Week 1770.0020 (0.002 to 0.002)
Change from Baseline at Week 1770.0000 (0.000 to 0.000)
Week 1900.0020 (0.002 to 0.002)
Change from Baseline at Week 1900.0000 (0.000 to 0.000)
Week 2030.0020 (0.002 to 0.008)
Change from Baseline at Week 2030.0000 (0.000 to 0.006)
End of Study/Early Discontinuation0.0020 (0.002 to 0.010)
Change from Baseline at End of Study/Early Discont0.0000 (0.000 to 0.008)
SecondaryNumber 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
Reported as:
Count of participants · Participants
Number of Patients With Infections and Hospitalizations
ParticipantsEZN-2279
Patients with infection5
Patients with hospitalizations3
SecondaryDuration of Hospitalization
Time frame:
Through end of EZN-2279 study treatment, up to 203 weeks
Reported as:
Mean · Days
Duration of Hospitalization
DaysEZN-2279
Duration of Hospitalization5.0 ± 2.16
SecondaryNumber 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
Reported as:
Count of participants · Participants
Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period
ParticipantsEZN-2279 Treatment Period
Baseline6
Week 346
Week 476
Week 603
Week 735
Week 865
Week 995
Week 1124
Week 1254
Week 1383
Week 1513
Week 1643
Week 1773
Week 1903
Week 2033
End of Study/Early Discontinuation3

Adverse events

Collected over Through end of EZN-2279 study treatment, up to 203 weeks. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Adagen0/7 (0%)0/7 (0%)6/7 (85.7%)
EZN-22790/7 (0%)4/7 (57.1%)7/7 (100%)
Most frequent serious events
Most frequent serious events
EventAdagenEZN-2279
Respiratory tract infectionInfections and infestations0/71/7
Tooth abscessInfections and infestations0/71/7
Injection site painGeneral disorders0/71/7
DehydrationMetabolism and nutrition disorders0/71/7
Vestibular migraineNervous system disorders0/71/7
HaemoptysisRespiratory, thoracic and mediastinal disorders0/71/7
Tooth extractionSurgical and medical procedures0/71/7
Most frequent other events
Showing 10 of 90
Most frequent other events
EventAdagenEZN-2279
VomitingGastrointestinal disorders1/73/7
CoughRespiratory, thoracic and mediastinal disorders0/73/7
Non-cardiac chest painGeneral disorders0/72/7
PyrexiaGeneral disorders0/72/7
DiarrhoeaGastrointestinal disorders0/72/7
NauseaGastrointestinal disorders0/72/7
Upper respiratory tract infectionInfections and infestations0/72/7
HaemoptysisRespiratory, thoracic and mediastinal disorders0/72/7
Oropharyngeal painRespiratory, thoracic and mediastinal disorders0/72/7
Productive coughRespiratory, thoracic and mediastinal disorders0/72/7

Baseline characteristics

As-treated

Age, Continuous
Age, Continuous(years)Adagen/EZN-2279
Mean21.3 ± 9.52
Sex: Female, Male
Sex: Female, Male(Participants)Adagen/EZN-2279
Female2
Male5
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Adagen/EZN-2279
Hispanic or Latino3
Not Hispanic or Latino4
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Adagen/EZN-2279
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American1
White4
More than one race0
Unknown or Not Reported2
Region of Enrollment
Region of Enrollment(participants)Adagen/EZN-2279
United States7
08

Study locations

6 sites
  • Children's Hospital Los Angeles
    Los Angeles, California 90027, United States
  • University of California San Francisco
    San Francisco, California 94158, United States
  • National Jewish Health
    Denver, Colorado 80206-2761, United States
  • Albert Einstein College of Medicine
    Bronx, New York 10461, United States
  • UBMD
    Buffalo, New York 14203, United States
  • Penn State College of Medicine The Milton S. Hershey Medical Center
    Hershey, Pennsylvania 17033, United States
09

References and documents

Study documents

  • Statistical analysis plan · Apr 4, 2019
  • Study protocol · Aug 23, 2017

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Apr 16, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT01420627
Lead sponsor
Leadiant Biosciences, Inc.
Responsible party
Sponsor
First posted
Aug 19, 2011
Start date
Jan 24, 2014
Primary completion
Apr 10, 2018
Completion
May 29, 2019
Results posted
Apr 16, 2020
Last update
Apr 16, 2020

Study contacts

Elie Haddad, MD, PhD
principal investigator · Université de Montréal

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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