EZN-2279 in Patients With ADA-SCID (NCT01420627) | Clinical Trial Compass
CompletedPhase 3
EZN-2279 in Patients With ADA-SCID
United States7 participantsStarted 2014-01-24
Plain-language 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.
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
. Patients with detectable neutralizing anti-Adagen® antibodies at screening evaluation
. Severe thrombocytopenia (platelet count \<50 x 10\^9/L)
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period
. 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