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Immunodeficiency clinical trials at UCLA

16 in progress, 7 open to eligible people

Showing trials for
  • Autologous Mobilized Peripheral Blood CD34+ Hematopoietic Stem and Progenitor Cells (HSPC) Transduced With the Elongation Factor Alpha Short Promoter (EFS) - Adenosine Deaminase (ADA) Gene (EFS-ADA) Lentiviral Vector for Adenosine Deaminase Severe Combined Immune Deficiency (ADA SCID)

    open to eligible people ages 1 month and up

    The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.

    Los Angeles, California

  • Conditioning SCID Infants Diagnosed Early

    open to eligible people ages 0-2

    The investigators want to study if lower doses of chemotherapy will help babies with SCID to achieve good immunity with less short and long-term risks of complications after transplantation. This trial identifies babies with types of immune deficiencies that are most likely to succeed with this approach and offers them transplant early in life before they get severe infections or later if their infections are under control. It includes only patients receiving unrelated or mismatched related donor transplants. The study will test if patients receiving transplant using either a low dose busulfan or a medium dose busulfan will have immune recovery of both T and B cells, measured by the ability to respond to immunizations after transplant. The exact regimen depends on the subtype of SCID the patient has. Donors used for transplant must be unrelated or half-matched related (haploidentical) donors, and peripheral blood stem cells must be used. To minimize the chance of graft-versus-host disease (GVHD), the stem cells will have most, but not all, of the T cells removed, using a newer, experimental approach of a well-established technology. Once the stem cell transplant is completed, patients will be followed for 3 years. Approximately 9-18 months after the transplant, vaccinations will be administered, and a blood test measuring whether your child's body has responded to the vaccine will be collected.

    Los Angeles, California and other locations

  • JSP191 Antibody Targeting Conditioning in SCID Patients

    open to eligible people ages 3 months and up

    A Phase 1/2 study to evaluate the safety, tolerability, and efficacy of an antibody conditioning regimen, known as JSP191, in patients with Severe Combined Immune Deficiency undergoing blood stem cell transplantation

    Los Angeles, California and other locations

  • Tabelecleucel in Participants With Epstein-barr Virus-associated Diseases

    open to all eligible people

    The purpose of this study is to assess the efficacy and safety of tabelecleucel in participants with Epstein-Barr virus (EBV) associated diseases.

    Los Angeles, California and other locations

  • Adenovirus-specific Cytotoxic T-lymphocytes for Refractory Adenovirus Infection

    open to eligible people ages 1 month to 79 years

    Related donor Adenovirus (ADV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered intravenously in in children, adolescents and young adults with refractory ADV infection post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT), with primary immunodeficiencies (PID) or post solid organ transplant. Funding Source: FDA OOPD

    Los Angeles, California and other locations

  • EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection

    open to eligible people ages 1 month to 79 years

    Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults with refractory EBV infection post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT), with primary immunodeficiencies (PID) or post solid organ transplant. Funding Source: FDA OOPD

    Los Angeles, California and other locations

  • Adolescent Master Protocol for Participants 18 Years of Age or Older - Lite

    open to eligible people ages 18 years and up

    This is a prospective cohort study designed to define the impact of HIV infection and antiretroviral therapy (ART) on young adults with perinatal HIV infection as they transition to adulthood.

    Los Angeles, California and other locations

  • Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

    Sorry, in progress, not accepting new patients

    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

    Los Angeles, California and other locations

  • Lenacapavir for HIV Pre-Exposure Prophylaxis in People Who Are at Risk for HIV Infection

    Sorry, in progress, not accepting new patients

    The goal of this clinical study is to test how well the study drug, lenacapavir (LEN), works in preventing the risk of HIV.

    Los Angeles, California and other locations

  • Combination HIV-Specific Broadly Neutralizing Monoclonal Antibodies Combined With ART Initiation During Acute HIV Infection to Induce HIV Remission

    Sorry, not yet accepting patients

    A5388 is a phase II, two-arm, randomized, double-blind, placebo-controlled study that will enroll 48 antiretroviral therapy (ART)-naïve adults with acute HIV infection (AHI) in order to determine whether: - Administration of combination HIV-specific broadly neutralizing antibody (bNAb) therapy in addition to ART during acute HIV infection (AHI) will be safe. - Participants who receive combination bNAb therapy in addition to ART during AHI will be more likely to demonstrate a delay in time to HIV-1 RNA ≥1,000 copies/mL for 4 consecutive weeks compared to participants who receive placebo plus ART. - Participants who receive combination bNAb therapy in addition to ART during AHI will demonstrate lower viral reservoirs and enhanced HIV-specific immunity compared to participants who receive placebo plus ART.

    Los Angeles, California and other locations

  • Cobicistat-Boosted Atazanavir (ATV/co), Cobicistat-Boosted Darunavir (DRV/co) and Emtricitabine/Tenofovir Alafenamide (F/TAF) in Children With HIV

    Sorry, not currently recruiting here

    The goal of this clinical study is to learn more about the safety and dosing of study drugs, cobicistat-boosted Atazanavir (ATV/co), cobicistat-boosted darunavir (DRV/co) and emtricitabine/tenofovir alafenamide (F/TAF), in children (age ≥ 4 weeks to < 18 years) with HIV.

    Los Angeles, California and other locations

  • Emtricitabine/Tenofovir Alafenamide (F/TAF) in Human Immunodeficiency Virus 1 (HIV-1) Infected Children and Adolescents Virologically Suppressed on a 2- Nucleoside/Nucleotide Reverse Transcriptase Inhibitor (2-NRTI)-Containing Regimen

    Sorry, in progress, not accepting new patients

    The primary objective of this study is to confirm the TAF dose and to evaluate the pharmacokinetics (PK) of TAF, safety, and tolerability of F/TAF in HIV-1 infected children and adolescents virologically suppressed (defined as having < 50 copies/mL of HIV-1 ribonucleic acid [RNA] for a period of at least 6 months) while on a stable NRTI containing regimen.

    Los Angeles, California and other locations

  • Immunogenicity of a Modified Vaccinia Ankara (MVA)-Based Anti-Cytomegalovirus (CMV) Vaccine (Triplex®)

    Sorry, in progress, not accepting new patients

    Participants will be randomized in a 2:1 ratio to receive either two injections of CMV-MVA Triplex® or placebo administered at study Entry/Day 0 and week 4. Vaccine Group: 60 participants will receive CMV-MVA Triplex® containing 5 x 10^8 plaque-forming unit (pfu) ±0.5 x 10^8 pfu of MVA Vaccine Encoding CMV Antigens by intramuscular (IM) deltoid injections. Placebo Group: 30 participants will receive a volume of placebo (7.5% Lactose in phosphate-buffered saline [PBS]) that matches the volume of the active vaccine injection by IM deltoid injections.

    Los Angeles, California and other locations

  • Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

    Sorry, accepting new patients by invitation only

    This observational long-term follow-up study is designed to collect safety and efficacy data from ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-ADA LV encoding for human adenosine deaminase (ADA) gene (EFS-ADA LV), as part of the OTL-101 clinical development program. No investigational medicinal product will be administered to these patients as part of the OTL-101-6 study.

    Los Angeles, California and other locations

  • Natural History Study of SCID Disorders

    Sorry, accepting new patients by invitation only

    This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions. In order to determine the patient, recipient and transplant-related variables that are most important in determining outcome, study investigators will uniformly collect pre-, post- and peri-transplant (or other treatment) information on all children enrolled into this study. Children will be divided into three strata: - Stratum A: Typical SCID with virtual absence of autologous T cells and poor T cell function - Stratum B: Atypical SCID (leaky SCID, Omenn syndrome and reticular dysgenesis with limited T cell diversity or number and reduced function), and - Stratum C: ADA deficient SCID and XSCID patients receiving alternative therapy including PEG-ADA ERT or gene therapy. Each Group/Cohort Stratum will be analyzed separately.

    Los Angeles, California and other locations

  • Prevention and Treatment Continuum for Youth at HIV Risk, Acutely Infected and With Established HIV Infection

    Sorry, in progress, not accepting new patients

    This is a strategic prospective cohort study which will measure the effects of early intensive antiretroviral therapy (ART) on the establishment and persistence of HIV-1 reservoirs and HIV-1-specific immunity in acutely /recently HIV infected youth aged 12 to 24 years as compared to newly diagnosed youth with established infection > 6 months. Participants with newly diagnosed acute /recent HIV-1 infection will be offered enrollment into the study with immediate initiation of ART which is the current standard of care.

    Los Angeles, California and other locations

Our lead scientists for Immunodeficiency research studies include .

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