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ADA Deficiency clinical trials at UCSF

3 in progress, 0 open to eligible people

Showing trials for
  • 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.

    San Francisco, California and other locations

  • Patients Treated for SCID (1968-Present)

    Sorry, accepting new patients by invitation only

    Individuals with a past diagnosis of severe combined immune deficiency (including many cases of "leaky SCID", Omenn syndrome, and reticular dysgenesis) who have undergone blood and marrow transplant, gene therapy, or enzyme replacement in the past may be eligible for this study. The purpose of study is to look backwards at what has already been done in the. Over 800 patients with SCID are expected to be enrolled, making this one of the largest studies ever to describe outcomes for patients with SCID treated at many different hospitals around North America.

    San Francisco, California and other locations

  • Registry Study of Revcovi Treatment in Patients With ADA-SCID

    Sorry, not currently recruiting here

    The objective of this study is to develop a registry of patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) treated with Revcovi™ that contains clinical and biochemical assessments for safety and dose adjustment based on adenosine deaminase (ADA) activity and erythrocyte deoxyadenosine nucleotide (dAXP) levels as well as immunologic monitoring.

    San Francisco, California and other locations

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