Safety of Belimumab in People With Idiopathic CD4 Lymphopenia and Autoantibodies (Phoebe)

Description

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).

Conditions

Idiopathic CD4 Lymphopenia

Talk to us

Study Overview

On this page

Study Details

Study overview

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).

A Phase 1 Evaluation of the Safety of Belimumab in People With Idiopathic CD4 Lymphopenia and Autoantibodies (Phoebe)

Safety of Belimumab in People With Idiopathic CD4 Lymphopenia and Autoantibodies (Phoebe)

Condition
Idiopathic CD4 Lymphopenia
Intervention / Treatment

-

Contacts and Locations

Bethesda

National Institutes of Health Clinical Center, Bethesda, Maryland, United States, 20892

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * 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 adequate contraception when engaging in sexual activities that can result in pregnancy, beginning at day 0 (or day 30 for hormonal contraception) until 4 months after the last dose of belimumab.
  • * Hormonal contraception.
  • * Male or female condom.
  • * Diaphragm or cervical cap with a spermicide.
  • * Intrauterine device.
  • * Able to provide informed consent.
  • * Willing to allow samples to be stored for future research.
  • * Prior receipt of belimumab for any reason.
  • * Allergy to any component of belimumab formulation.
  • * HIV infection or other recognized congenital or acquired immunodeficiency.
  • * Current moderate or severe acute illness (eg, febrile illness, seizure, myocardial infarction, cerebrovascular accident, pulmonary embolism) or progressive serious infection related to ICL that, in the opinion of the principal investigator, would make the participant unsuitable for the study. Pre-existing infections that have been stable both clinically and with laboratory (eg, cryptococcal antigen titer or histoplasma antigen level) and radiographic evaluations on maintenance therapy over at least a year will be eligible.
  • * Untreated hepatitis B or C (acute or chronic).
  • * Active tuberculosis infection.
  • * Serum creatinine \> 1.5 times the upper limit of normal (ULN).
  • * Hemoglobin \< 8 g/dL.
  • * Alanine transaminase or aspartate transaminase \> 2 times ULN.
  • * Serum IgG \< 400 mg/L.
  • * Current use of systemic glucocorticosteroids, with the exception of corticosteroid nasal spray or inhaler and topical steroids.
  • * Any cancer diagnosis or autoimmune condition requiring systemic chemotherapy or immunomodulant-affecting antibody responses (eg, rituximab, ibrutinib), IV or SC Ig supplementation, radiation therapy, or any such treatment within the previous 6 months. Apremilast, Plaquenil, or nonsteroidal anti-inflammatory drugs will not be exclusionary.
  • * Severe depression. Psychiatry may be consulted prior to final eligibility decision.
  • * Infections (recently acquired or exacerbation of a chronic infection) that required new medications for management within the past 60 days.
  • * Receipt of any vaccination within the past 30 days.
  • * Pregnant.
  • * Breastfeeding.
  • * Any behavioral or substance use issue that would compromise appropriate follow up and participation in this study.

Ages Eligible for Study

18 Years to 70 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

National Institute of Allergy and Infectious Diseases (NIAID),

Irini Sereti, M.D., PRINCIPAL_INVESTIGATOR, National Institute of Allergy and Infectious Diseases (NIAID)

Study Record Dates

2025-12-29