A Phase 1 Evaluation of the Safety of Belimumab in People With Idiopathic CD4 Lymphopenia and Autoantibodies (Phoebe)
National Institute of Allergy and Infectious Diseases (NIAID)
Summary
Background: People with Idiopathic CD4 lymphopenia (ICL) have lower numbers of a type of white blood cell called CD4 cells. White blood cells fight against infections. Low levels of CD4 cells may make a person more likely to get sick. There are no approved treatments for ICL. Researchers think a drug called belimumab may be able to help in specific situations. Objective: To see if belimumab is safe for people with ICL. Eligibility: People ages 18-70 who have ICL and are participating in NIH protocol 09-I-0102 (EPIC) Design: Participants will be screened with: Medical and medication history Physical exam Questionnaire about mental health and depression Blood and urine tests Participants will have a baseline visit. This will include some repeats of the screening tests. They may also have leukapheresis: Blood will be taken from a needle in one arm and passed through a machine that separates out the white blood cells. The rest of the blood will be returned through a needle in the other arm. Participants will receive 8 doses of belimumab through IV: A needle will insert a thin plastic tube into an arm vein. Belimumab will be given through the IV line. The first 3 doses will be given every 2 weeks. The other 5 will be given once every 4 weeks. Participants will have a physical exam and blood and urine tests at each dosing visit. They will be monitored for up to 4 hours after the infusion. Participants will have 3 follow-up visits, at around 8, 16, and 24 weeks after the last dose of belimumab. They will have a physical exam and blood and urine tests. Once they finish this protocol and they will continue to be followed under 09-I-0102 (EPIC study).
Description
Idiopathic CD4 lymphopenia (ICL) is characterized by persistent low CD4 counts, frequently in combination with CD8, natural killer, or B cell lymphopenia. It is considered a heterogeneous disorder with manifestations that can include autoimmunity, invasive fungal infections or infections with human papillomavirus, and malignancies typically related to infections. The exact etiology of ICL remains unclear and there is no specific treatment. Recent data from our group revealed a high prevalence of antilymphocyte antibodies in many of the ICL patients evaluated. The targets of these antibodies r…
Eligibility
- Age range
- 18–70 years
- Sex
- All
- Healthy volunteers
- No
* INCLUSION CRITERIA: Individuals must meet all of the following criteria to be eligible for study participation: * Aged 18-70 years. * Enrolled in study 09-I-0102. * Has a documented diagnosis of ICL, defined as the following: * CD4 count \< 300 cells/microliter in at least 2 separate measurements 6 weeks apart at any point in the past, AND * CD4 count \< 300 cells/microliter within previous 90 days. * Evidence for autoantibody positivity (eg, ANA or in the research flow method looking for antilymphocyte antibodies). * Female participants of childbearing potential must agree to use ade…
Interventions
- BiologicalBelimumab
Belimumab 10 mg/kg once every 2 weeks for 3 doses, and then once every 4 weeks for 5 doses, delivered via 1-hour intravenous (IV) infusion.
Location
- National Institutes of Health Clinical CenterBethesda, Maryland