ADA-SCIDPhase 3NCT01420627
EZN-2279 in Patients With ADA-SCID
A Phase 3 study testing EZN-2279 and Adagen in people with ADA-SCID and Adenosine Deaminase Deficiency, run by Leadiant Biosciences, Inc. at 6 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency
- What is being tested?
- EZN-2279 and Adagen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 6 sitesUnited States
- Study dates
- 24 Jan 2014to 29 May 2019
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
UBMD
Buffalo, New York, 14203
National Jewish Health
Denver, Colorado, 80206-2761
Penn State College of Medicine The Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Children's Hospital Los Angeles
Los Angeles, California, 90027
University of California San Francisco
San Francisco, California, 94158
Albert Einstein College of Medicine
The Bronx, New York, 10461
About this study
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
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.
- Being tested
- EZN-2279, Adagen
- Medical subject headings
- Severe combined immunodeficiency due to adenosine deaminase deficiency, Severe Combined Immunodeficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EZN-2279Biological
Also called rADA
Weekly administration of EZN-2279 via IM injection
AdagenBiological
| Study group | Role | What they receive |
|---|---|---|
| Adagen/EZN-2279 | Experimental | Patients started on Adagen and crossed over to experimental EZN-2279 treatment after at least a 3 week Lead-in Period on Adagen Receives: Biological: EZN-2279; Biological: Adagen |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline through Week T-21
Secondary outcomes (8)
Safety Summary Data
Summary of adverse events and serious adverse events
Measured: Through end of EZN-2279 study treatment, up to 203 weeks
Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period
Trough ADA activity, mmol/h/L
Measured: From Baseline through Week T-21
Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period
Trough ADA activity levels, mmol/h/L
Measured: Through end of EZN-2279 study treatment, up to 203 weeks
Summary of Trough dAXP Levels in EZN-2279 Treatment Period
Trough dAXP levels, mmol/L
Measured: From Baseline through Week T-21
Summary of Trough dAXP Levels in EZN-2279 Maintenance Period
Trough dAXP levels, mmol/L
Measured: Through end of EZN-2279 study treatment, up to 203 weeks
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
Measured: Through end of EZN-2279 study treatment, up to 203 weeks
Duration of Hospitalization
Measured: Through end of EZN-2279 study treatment, up to 203 weeks
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
Measured: From Week 34 to End of Study/Early Discontinuation, up to 203 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2011 (estimated)
- Study start24 Jan 2014
- Primary completion10 Apr 2018
- Study completion29 May 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for ADA-SCIDStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Leadiant Biosciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Elie Haddad, MD, PhD, Principal investigator, Université de Montréal
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01420627
- Sponsor's protocol ID
- STP-2279-002
- Status last verified
- Apr 2020
- First submitted
- 18 Aug 2011
- Last updated
- 16 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01420627
- Acronym / sponsor ID
- STP-2279-002
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Leadiant Biosciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 24 Jan 2014 (actual)
- Primary completion
- 10 Apr 2018 (actual)
- Study completion
- 29 May 2019 (actual)
- First posted
- 19 Aug 2011 (estimated)
- Results
- Posted 16 Apr 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Adagen/EZN-2279 | Experimental | Biological: EZN-2279, Biological: AdagenPatients started on Adagen and crossed over to experimental EZN-2279 treatment after at least a 3 week Lead-in Period on Adagen |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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:
- Patients or parent/guardian must be capable of understanding the protocol requirements and risks and providing written informed assent/consent
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EZN-2279Also: rADAWeekly administration of EZN-2279 via IM injection | Biological | Adagen/EZN-2279 |
| Adagen | Biological | Adagen/EZN-2279 |
Primary outcome
Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period
Time frame: Baseline through Week T-21
Number of patients with total erythrocyte dAXP concentration from a trough blood sample <0.02 mmol/L
8 secondary outcomes
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 Treatment Period
Time frame: From Baseline through Week T-21
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 dAXP 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
Number of Patients With Infections and Hospitalizations
Time frame: Through end of EZN-2279 study treatment, up to 203 weeks
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Elie Haddad, MD, PhD principal investigator
Université de Montréal
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing EZN-2279 and Adagen in people with ADA-SCID and Adenosine Deaminase Deficiency, run by Leadiant Biosciences, Inc. at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Apr 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period (time frame: Baseline through Week T-21). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Apr 2020.