Top
SCID-X1 - Healing Genes
703
post-template-default,single,single-post,postid-703,single-format-standard,mkdf-bmi-calculator-1.0,mkd-core-1.1.1,tribe-no-js,wellspring-ver-1.8,mkdf-smooth-page-transitions,mkdf-ajax,mkdf-grid-1300,mkdf-blog-installed,mkdf-header-standard,mkdf-no-behavior,mkdf-default-mobile-header,mkdf-sticky-up-mobile-header,mkdf-dropdown-slide-from-bottom,mkdf-full-width-wide-menu,mkdf-search-dropdown,elementor-default,elementor-template-full-width,elementor-kit-3486,elementor-page-2540

SCID-X1

Share This Post

Share on facebook
Share on linkedin
Share on twitter
Share on email

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


Phase 1 / 2

DESCRIPTION:

Researchers at Mattel Children’s Hospital of UCLA and Boston Children’s Hospital seek patients up to 5 years of age with SCID-X1 ) caused by mutations in the IL2RG gene. Researchers will withdraw stem cells, gene-modify them to deliver a normal copy of the IL2RG gene, and return them to the patient after a low dose of chemotherapy. It is hoped this will provide the patients with a normal immune system.

Study participants will undergo a blood draw treatment that sorts out specific cells and returns the blood to the patient. After the gene modification in the lab, the cells are infused to the patient post chemotherapy and the patient will be followed for 2 years after infusion of the drug. Required long-term monitoring for a total of 15 years after infusion will be performed on a separate protocol.


PATIENT MUST:

  • Be 2 months of age or older
  • Have a diagnosis of SCID-X1 based on immunophenotype and lack of T cell function
  • Meet lab assessments for minimal levels of the type of immune cells targeted by the gene therapy
  • Not have encephalopathy

THE STUDY INVOLVES:

  1. Screening before the treatment.
  2. Leukapheresis will draw the needed immune cells but return blood cells to the body.
  3. Researchers will use a virus in the lab to deliver the gene to the immune cells.
  4. A low dose of chemotherapy will be delivered.
  5. The gene-modified immune cells will be re-infused to the patient.
  6. The patient will be closely followed for 2 years, with less intensive follow up for 15 total years from beginning of study.

LOCATIONS AND CONTACTS:

Trials will take place at the Mattel Children’s Hospital – UCLA, Map, and Boston Children’s Hospital, Map.

Contact: Colleen Dansereau  |  6179197008  |  [email protected]

 

SPONSOR INFORMATION:

David Williams

University of California, Los Angeles

 

Or go online:

https://clinicaltrials.gov/ct2/show/NCT03311503

 

Subscribe To Our Newsletter

Get updates and learn from the best

More To Explore

Vanquishing the Virus: 160+ COVID-19 Drug and Vaccine Candidates in Development

While President Trump continues to promote antimalarial drugs such as hydroxychloroquine sulfate and azithromycin to treat COVID-19—“What do you have to lose?” he exclaimed during the April 4 Coronavirus Task Force Briefing—the global community of drug discovery researchers and biopharmas is developing a much broader