Top
Sickle Cell Disease - Healing Genes
574
post-template-default,single,single-post,postid-574,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

Sickle Cell Disease

Share This Post

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

Gene Transfer for Patients With Sickle Cell Disease

Gene Transfer for Patients With Sickle Cell Disease Using a Gamma Globin Lentivirus Vector: An Open Label Phase I/II Pilot Study


Phase 1/2

DESCRIPTION:

Researchers at Cincinnati Children’s Hospital Medical Center are recruiting patients with Sickle Cell Disease / Sickle Cell Anemia for a genetic transfer investigational treatment. The goal of the treatment is to provide sickle cell patients with the gene needed to create normal red blood cells (RBCs).

This investigatory treatment requires surgical recovery of the patient’s bone marrow, which will then be gene-edited using a harmless virus to transfer the correct gene into the patient’s bone marrow. The patient will undergo chemotherapy prior to the treatment, then the gene-edited bone marrow will be transfused back into the patient. Follow up will be approximately 15 years after this important study.


PATIENT MUST:

  • Be 18 to 35 years of age
  • Able to undergo general or regional anesthesia
  • Willing to reside within 120 miles of Cincinnati Children’s Hospital for 3 to 6 months
  • Not be HIV-positive
  • Not be pregnant or breastfeeding
  • Not have alpha thalassemia

THE STUDY INVOLVES:

  1. Pre-screening at the study site to confirm eligibility of the patient to participate.
  2. A surgical bone marrow harvest to collect blood making cells.
  3. The gene-editing phase of the study will not require patient involvement.
  4. Chemotherapy treatment will reduce the patient’s bone marrow.
  5. One-time Infusion of the investigational drug, with close observation period.
  6. Patients will be followed for ~ 15 years.

LOCATIONS AND CONTACTS:

Cincinnati Children’s Hospital Medical Center in Cincinnati, OH, is recruiting participants for this trial. Map.

Primary contact: Amy Shova | [email protected] | 513-636-6770

SPONSOR INFORMATION:

Children’s Hospital Medical Center, Cincinnati & Doris Duke Charitable Foundation

Or go online:
https://clinicaltrials.gov/ct2/show/NCT02186418

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