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

Part of paid clinical trials in Los Angeles, California.

Sponsor
David Williams
Study ID
NCT03311503
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Gene Therapy
  • Severe Combined Immunodeficiency, X Linked

Eligibility Criteria

Sex
MALE
Age
0 Years - 5 Years
Healthy Volunteers
Not accepted

Interventions

  • autologous CD34+ cell transduced with G2SCID vector — BIOLOGICAL
    single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) lentiviral vector G2SCID

Study Details

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

Key Dates

Start date
Feb 26, 2018
Status verified
Dec 2025
Primary completion
Jan 1, 2028
Completion
Jan 1, 2028

Study Design

Enrollment
12 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment arm
    single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) lentiviral vector G2SCID

Primary Outcome Measure

The primary objective is to measure event free survival [ Time Frame: 1 year post infusion ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
Mattel Children's Hospital - UCLALos AngelesCalifornia90095
Donald Kohn, MD
(310) 825-6708
Emory University/Childrens Healthcare of AtlantaAtlantaGeorgia30322
Shanmuganathan Chandrakasan, MD
[email protected]
404-727-8877
Shanmuganathan Chandrakasan, MD (PRINCIPAL_INVESTIGATOR)
Boston Childrens HospitalBostonMassachusetts02115
Susan Prockop, MD
[email protected]
617-355-3087
Susan Prockop, MD (PRINCIPAL_INVESTIGATOR)
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229
Sharat Chandra, M.D.
[email protected]
(513) 636-4266
Sharat Chandra, M.D. (PRINCIPAL_INVESTIGATOR)

Find similar trials in Los Angeles, CA

By research site
Mattel Children's Hospital - UCLA· Los Angeles, CAEmory University/Childrens Healthcare of Atlanta· Atlanta, GABoston Childrens Hospital· Boston, MACincinnati Children's Hospital Medical Center· Cincinnati, OH

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