Clinical Trial: Interleukin-7 (CYT107) Treatment of Idiopathic CD4 Lymphocytopenia: Expansion of CD4 T Cells (ICICLE)

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Interleukin-7 (CYT107) Treatment of Idiopathic CD4 Lymphocytopenia: Expansion of CD4 T Cells (ICICLE)

Brief Summary:

Background:

  • Idiopathic CD4 lymphocytopenia (ICL) is a condition in which patients have low levels of T cells, a type of white blood cell that helps fight infection. Animal studies have shown that an experimental drug Interleukin 7 (IL-7), which is named CYT107, can increase the number and function of T cells. CYT107, however, has not been used in people with ICL.

Objectives:

  • To determine the safety of CYT107 in people with ICL.
  • To determine whether CYT107 will increase the number and function of T cells in people with ICL.

Eligibility:

  • Patients 18 years of age and older diagnosed with ICL and who are at risk of becoming sick because of this condition are eligible for this study. In addition, patients must not be pregnant, or have other illnesses that would cause low CD4 T cell counts, such as human immunodeficiency virus (HIV) or human T-lymphotrophic virus (HTLV) infection.

Design:

  • The initial screening visit will include the following examinations and tests:
  • A complete physical exam and medical history
  • Blood analysis, including CD4 T cell count; complete blood count and additional blood tests to determine clotting ability and blood composition; thyroid, liver, kidney, and pancreatic function tests; HIV and HTLV tests; and tests for anti-IL-7 antibodies that block normal IL-7 activity
  • Routine

    Detailed Summary:

    Interleukin-7 (CYT107) Treatment of Idiopathic CD4 Lymphocytopenia: Expansion of CD4 T Cells (ICICLE) is a Phase I/IIa open-label, single arm clinical trial evaluating the safety profile of glycosylated recombinant human interleukin-7 (rhIL-7) as an immunostimulatory therapy in patients with idiopathic CD4 T cell lymphocytopenia (ICL) at risk of disease progression. Secondary analyses will assess the immunostimulatory effects of rhIL-7 on T cell number and function.

    ICL was first characterized in the early 1990 s and is a primary immune disorder of CD4 T cell lymphocytopenia (less than 300 cells/microL or less than 20% of lymphocytes), which is not due to any known infectious process, exogenous medication, autoimmune cytopenia, or other underlying disorder associated with lymphocytopenia. ICL patients are at risk for a wide spectrum of opportunistic and other serious infections, autoimmune disorders, and other types of lymphocytopenia. At present, no validated treatment exists for ICL, and treatment is directed primarily toward infectious complications once they arise. A first-generation form of rhIL-7 was shown in pre-clinical and Phase I studies in oncology and human immunodeficiency virus (HIV)-infected patients to be well tolerated in repeated dose trials, with long-lasting increases in both CD4 and CD8 T cells. CYT107 is a second-generation rhIL-7 product made by Cytheris via a recombinant mammalian cell culture system.

    DESIGN - Open-label, single-arm, Phase I/IIa interventional clinical trial. Participants will be evaluated at baseline (prior to study treatment) and according to the protocol follow-up schedule, receiving a total of 2 cycles of rhIL-7 (CYT107) during the induction phase and up to 8 cycles during the maintenance phase. Safety assessments of rhIL-7 will be the primary focus at each study visit, with sec
    Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

    Current Primary Outcome: Adverse Events and Toxicities Associated With CYT107. [ Time Frame: 48 weeks per patient with a 3-4 year enrollment period ]

    Original Primary Outcome: Adverse events and toxicities associated with CYT107; changes in CD4/CD8 T cell counts after CYT107.

    Current Secondary Outcome:

    Original Secondary Outcome: Changes in immunophenotype (naive, memory, regulatory T cell subsets) and antigen-specific T cell function after CYT107, changes in T cell activation/proliferation status and TCR repertoire after CYT107 immunogenicity of CYT107.

    Information By: National Institutes of Health Clinical Center (CC)

    Dates:
    Date Received: February 6, 2009
    Date Started: February 2009
    Date Completion:
    Last Updated: October 14, 2015
    Last Verified: October 2015