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Gene Therapy

Gene transfer for Severe Combined Immunodeficiency

Phase 1 & 2
Waitlist Available
Led By Jennifer Whangbo, MD
Research Sponsored by David Williams
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 15 years post gene transfer
Awards & highlights

Study Summary

This trial is testing a new way of doing gene transfer that may decrease the risk of leukemia. The new way uses a different virus to carry the normal gene into the stem cells. The new virus is called a lentivirus, and it has been used in other gene transfer trials in children with other diseases, but not in children with SCID-X1. The lentivirus has a lower risk of causing leukemia than the virus used in the previous two trials. Researchers also think that the new way of processing the stem cells, called "stimulation and transduction," may also decrease the risk of leukemia.

Eligible Conditions
  • Severe Combined Immunodeficiency (SCID)

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 15 years post gene transfer
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 15 years post gene transfer for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
CD3 cell count post transfusion
Incidence of life-threatening adverse reactions related to the gene therapy procedure.
Secondary outcome measures
Ability to mount antibody responses to vaccination.
Molecular characterization of gene transfer.
Normalization of nutritional status, growth, and development

Trial Design

1Treatment groups
Experimental Treatment
Group I: Gene TransferExperimental Treatment1 Intervention
open label single arm study
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Gene transfer
2010
Completed Phase 2
~20

Find a Location

Who is running the clinical trial?

Children's Hospital Medical Center, CincinnatiOTHER
815 Previous Clinical Trials
6,531,583 Total Patients Enrolled
Boston Children's HospitalOTHER
761 Previous Clinical Trials
5,579,722 Total Patients Enrolled
David WilliamsLead Sponsor
4 Previous Clinical Trials
50 Total Patients Enrolled
1 Trials studying Severe Combined Immunodeficiency
10 Patients Enrolled for Severe Combined Immunodeficiency

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there still vacancies available for participants in this research endeavor?

"As observed on clinicaltrials.gov, this research project is not currently enlisting participants. Although the trial was initially posted on April 1st 2010, with its last update occuring July 19th 2022, it does not appear to be actively seeking applicants at this moment in time. Fortunately for those wishing to join a medical study, there are 86 other studies presently accepting volunteers."

Answered by AI
Recent research and studies
New England Journal of MedicineJournal
A Modified Γ-retrovirus Vector for X-linked Severe Combined Immunodeficiency
Hacein-Bey-Abina, Salima, Sung-Yun Pai, H. Bobby Gaspar, Myriam Armant, Charles C. Berry, Stephane Blanche, Jack Bleesing, et al.. 2014. “A Modified Γ-retrovirus Vector for X-linked Severe Combined Immunodeficiency”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa1404588.
Genome MedicineJournal
T Cell Dynamics and Response of the Microbiota After Gene Therapy to Treat X-linked Severe Combined Immunodeficiency
Clarke, Erik L., A. Jesse Connell, Emmanuelle Six, Nadia A. Kadry, Arwa A. Abbas, Young Hwang, John K. Everett, et al.. 2018. “T Cell Dynamics and Response of the Microbiota After Gene Therapy to Treat X-linked Severe Combined Immunodeficiency”. Genome Medicine. Springer Science and Business Media LLC. doi:10.1186/s13073-018-0580-z.
clinicaltrials.govStudy
Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
David Williams 2010. "Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01129544.
~1 spots leftby Apr 2025
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