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

SexALL

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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

What they're measuring

Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period

Timeframe: Baseline through Week T-21

Trial details

NCT IDNCT01420627

SponsorLeadiant Biosciences, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2018-04-10

Results submitted2020-03-19

View on ClinicalTrials.gov More ADA-SCID trials