Idiopathic CD4 Lymphocytopenia
Idiopathic CD4 lymphocytopenia (ICL) is a rare, orphan disease characterized by abnormally low CD4 T cell counts without evidence of human immunodeficiency virus (HIV-1 or HIV-2) infection.
The study is sponsored, conducted and partially funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the US National Institutes of Health (NIH). Under the direction of Irini Sereti, M.D., M.H.S., NIAID/NIH Study Investigator, the trial is designed to assess the safety and biological effects of repeated administration of CYT107 and will be conducted at the NIH Clinical Center in Bethesda, Maryland, the largest US hospital devoted entirely to clinical research.
Title: A Phase I/IIa open-label, single arm clinical trial evaluating the safety profile of glycosylated recombinant human interleukin-7 (rhIL-7) as an immune modulator in patients with ICL at risk of disease progression.
Trial Name: ICICLE (Interleukin-7 (CYT107) Treatment of Idiopathic CD4 Lymphocytopenia: Expansion of CD4 T Cells)
Countries: United States
Status: Ongoing
About the ICICLE Study
This is a single center, open-label, single-arm, Phase I/IIa interventional clinical trial. The study population is defined as men and women, aged >18 years, with a confirmed diagnosis of ICL (CD4 <300 cells/µL or <20% of lymphocytes on two occasions) deemed at risk for complications due to concurrent CD8 T cell lymphocytopenia and/or a history of opportunistic or otherwise serious infection, without autoimmunity or hematologic or lymphoid malignancy.
Participants will be evaluated at baseline (prior to study treatment) and according to the protocol follow-up schedule, receive a total of 2 cycles of subcutaneous rhIL-7 (CYT107) dosed once weekly for 3 weeks in a dose escalation fashion: 3 µg/kg (first 5 volunteers), 10 µg/kg (next 5 volunteers) and 20 µg/kg (last 5 volunteers), with an additional 5 study participants at the highest achieved dose level. Cycles of rhIL-7 will be administered starting at Week 1 and Week 24.
Approximately 35-40 patients will be screened over a 3-year period to achieve the desired sample of 20 ICL patients. Safety assessments of rhIL-7 will be the primary focus at each study visit, with secondary analyses of immune parameters, including changes from baseline in T cell numbers and function, at Weeks 24 and 48. Enrollment is expected to take 3 to 4 years. Each volunteer will be followed for 48 weeks. Thus, total duration of the study will be approximately 5 years.