Safety and Survival of Genetically Modified White Blood Cells in HIV-infected Twins The Gemini Study

NCT04799483 | Active Not Recruiting

• 2 other identifiers: 02008002-I-0080
• Study Type: observational
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

This study will consolidate under one umbrella protocol all NIH studies of the safety and survival of gene-modified lymphocytes in HIV-infected twins. The major purposes of the current study are to:

• place subjects from the various identical twin cell transfer/gene therapy studies on a single, more uniform schedule of visits;
• examine the long-term safety of apheresis procedures in non-HIV-infected lymphocyte donor twins;
• examine the long-term safety of lymphocytes in HIV-infected lymphocyte recipient twins. Identical twins enrolled in NIH protocols 93-I-0110, 94-I-0206, 96-HG-0051, and 97-I-0165 involving the transfer of lymphocytes for the treatment of HIV infection are eligible to continue their research participation under the current protocol, 02-I-0080. Participants will not undergo any additional research procedures in this study. They will continue to take their antiretroviral therapy and will be followed at least once a year, or more often if medically indicated, with a medical history, physical examination and blood tests for routine studies and to determine the number of lymphocytes in the blood. Recipient twins will also have blood levels of gene-modified lymphocytes checked. Recipient twins will have some blood stored for RCR (replication competent retrovirus) testing, should it become necessary. A special virus was used to insert genes into the donated lymphocytes and, although the virus was rendered incapable of growing in the body, a theoretical risk exists that the virus could mix with other viruses and possibly begin growing or reproducing. If a participant subsequently develops medical problems, the stored blood samples can be checked for RCR to see if it may be a cause of the problem.

Conditions

HIV

Keywords

Gene Therapy; HIV; Immunotherapy; Syngeneic Cells; Gemini Study

Outcome Measures

Primary Outcomes (1)

• Evaluate the Long-term safety of gene-modified and unmodified infusion

  Annually

Eligibility Criteria

• Age: 21 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients who participated in the long-term follow-up phase of previous NIAID/NHGRI intramural research protocols in which unmodified cells, gene marked cells or cells with therapeutic genes were infused.

You may qualify if:

• Activet participation, in an NIAID/CCMD/NHGRI protocol involving the transfer of unmodified lymphocytes, gene-marked lymphocytes or cells containing potentially therapeutic genes in the treatment of HIV-1 infection (cell donors and recipients were eligible).
• Ongoing follow-up with a primary medical care provider who manages the subject's HIV-related and/or other medical conditions.
• Agree to comply with study requirements and clinical policies.
• Able to provide informed consent.

You may not qualify if:

• A history of gross noncompliance with prior NIAID/CCMD protocol requirements that, in the opinion of the Principal Investigator, is likely to seriously compromise the safety or value of an individual's participation in the present protocol.
• Refusal to follow Clinical Center policy on partner notification (if HIV-positive).
• Refusal to permit research blood specimens to be stored (frozen) for potential future studies, including RCR testing will be excluded (participants who have received gene-modified cells may refuse to have specimens stored for research purposes other than RCR).
• Any medical condition that, in the opinion of the Principal Investigator, would make the subject inappropriate for protocol participation (such as anemia or inadequate venous access).
• Refusal to allow the NIH research team to exchange pertinent medical information with the subjects primary medical care provider.

Sponsors & Collaborators

• National Institute of Allergy and Infectious Diseases (NIAID): lead

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• Donahue RE, Kessler SW, Bodine D, McDonagh K, Dunbar C, Goodman S, Agricola B, Byrne E, Raffeld M, Moen R, et al. Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J Exp Med. 1992 Oct 1;176(4):1125-35. doi: 10.1084/jem.176.4.1125.

  PMID: 1383375 BACKGROUND

• Carr A, Cooper DA. Adverse effects of antiretroviral therapy. Lancet. 2000 Oct 21;356(9239):1423-30. doi: 10.1016/S0140-6736(00)02854-3.

  PMID: 11052597 BACKGROUND

• Morgan RA. Genetic strategies to inhibit HIV. Mol Med Today. 1999 Oct;5(10):454-8. doi: 10.1016/s1357-4310(99)01542-7.

  PMID: 10498914 BACKGROUND

Related Links

• NIH Clinical Center Detailed Web Page

Study Officials

• Mary E Wright, M.D.

  National Institute of Allergy and Infectious Diseases (NIAID)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 15, 2021
• First Posted: March 16, 2021
• Study Start: January 5, 2001
• Primary Completion (Estimated): January 1, 2030
• Study Completion (Estimated): January 30, 2030
• Last Updated: March 22, 2024

Record last verified: 2024-03

Locations

US (1)