RecruitingPhase 1/2

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

United States12 participantsStarted 2018-02-26

Plain-language summary

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

Who can participate

Age range0 Years – 5 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

✕. 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%
✕. Renal failure defined as glomerular filtration rate \<30 ml/min/1.73 m2 or dialysis dependence
✕. Uncontrolled seizure disorder
✕. Encephalopathy
✕. Documented coexistence of any disorder known to affect DNA repair

What they're measuring

The primary objective is to measure event free survival

Timeframe: 1 year post infusion

T cell reconstitution

Timeframe: 1 year post infusion

Trial details

NCT IDNCT03311503

SponsorDavid Williams

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2028-01-01

Contact for this trial

Colleen Dansereau

6179197008 colleen.dansereau@childrens.harvard.edu

View on ClinicalTrials.gov More Severe Combined Immunodeficiency, X Linked trials