Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

ConditionsSevere Combined Immunodeficiency, X Linked Gene Therapy

Trial statusRecruiting

Trial phasePhase 1, Phase 2

Trial typeInterventional

Biological sexMale

Age0-5

SponsorDavid Williams

About this trial

This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector. Subjects will be enrolled over 3 years and be followed for 2 years post-infusion on this protocol, then followed long-term on a separate long-term follow-up protocol.

Enrollment of subjects will be agreed upon by representatives of both sites. Data will be collected uniformly from both sites through an electronic capture system and key laboratory studies will be centralized.

Harvest, cellular manufacturing and infusion will occur at each site using the same SOPs. Key aspects of cellular product characterization will be centralized

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

None

Disqualifiers

Patients with an active, therapy-resistant infection. Infections that are known to be highly morbid in SCID patients will be considered active and therapy-resistant if the infectious agent is repeatedly isolated despite a minimum of 2 weeks of appropriate therapy and is associated with significant organ dysfunction (including but not limited to abnormalities listed below).

Mechanical ventilation including continuous positive airway pressure

Abnormal liver function defined by AST and ALT >10 times the upper range of normal OR Bilirubin >2 mg/dL

Shortening fraction on echocardiogram <25% or ejection fraction <50%

Trial design

Design model

Single group

Treatments tested in this trial

autologous CD34+ cell transduced with G2SCID vector
Biological/Vaccine
single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) lentiviral vector G2SCID

Treatment groups

12 Participants

are divided into 1 treatment group

Group A: Treatment armExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

The primary objective is to measure event free survival

Events will include death, infusion of unmanipulated back-up product for failure of hematopoietic recovery, and allogeneic transplant performed for poor immune reconstitution

Time frame

1 year post infusion

T cell reconstitution

* CD3+ T cell count ≥300 cells/microliter in peripheral blood * Gene marking ≥0.1 copies/cell in sorted CD3+ T cells

Time frame

1 year post infusion

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

David Williams

Lead sponsor

Boston Children's Hospital

Sponsor institution

Source ClinicalTrials.gov

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