NCT04097561 Safety of Belimumab in People With Idiopathic CD4 Lymphopenia and Autoantibodies (Phoebe)
| NCT ID | NCT04097561 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | National Institute of Allergy and Infectious Diseases (NIAID) |
| Condition | Idiopathic CD4 Lymphopenia |
| Study Type | INTERVENTIONAL |
| Enrollment | 20 participants |
| Start Date | 2020-01-13 |
| Primary Completion | 2027-03-25 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 20 participants in total. It began in 2020-01-13 with a primary completion date of 2027-03-25.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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).
Eligibility Criteria
* 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 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. Acceptable methods of contraception include the following: * 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. EXCLUSION CRITERIA: Individuals meeting any of the following criteria will be excluded from study participation: * Prior receipt of belimumab. Exception: If an individual had been previously enrolled in this study and received up to 3 doses of belimumab but had to withdraw for reasons that were not related to safety or clinical concerns, they may be re-enrolled. In this case, at least 6 months must have passed from the last prior infusion and they will restart the protocol and drug administration from the beginning. * 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. Concomitant diagnosis of SLE will not be exclusionary. Although SLE can be associated with lymphopenia, patients with lupus and extreme lymphopenia in the absence of cytotoxic or immunosuppressive medications and history of unusual infections will be eligible if they are participants of study 09-I-0102.
Contact & Investigator
Irini Sereti, M.D.
PRINCIPAL INVESTIGATOR
National Institute of Allergy and Infectious Diseases (NIAID)
Frequently Asked Questions
Who can join the NCT04097561 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 70 Years, studying Idiopathic CD4 Lymphopenia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04097561 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT04097561 currently recruiting?
Yes, NCT04097561 is actively recruiting participants. Contact the research team at isereti@niaid.nih.gov for enrollment information.
Where is the NCT04097561 trial being conducted?
This trial is being conducted at Bethesda, United States.
Who is sponsoring the NCT04097561 clinical trial?
NCT04097561 is sponsored by National Institute of Allergy and Infectious Diseases (NIAID). The principal investigator is Irini Sereti, M.D. at National Institute of Allergy and Infectious Diseases (NIAID). The trial plans to enroll 20 participants.