Actively Recruiting

Phase 1
Phase 2
Age: 18Years - 75Years
All Genders
ID05600920

An Open-Label, Single-Arm, Dose-Escalation Trial of Long-Acting Recombinant Human IL-7 (NT-I7, Efineptakin Alfa) for Idiopathic CD4 Lymphopenia

Led by National Institute of Allergy and Infectious Diseases (NIAID) ยท Updated on 2026-03-09

60

Participants Needed

1

Research Sites

N/A

Total Duration

On this page

Sponsors

N

National Institute of Allergy and Infectious Diseases (NIAID)

Lead Sponsor

N

NeoImmuneTech

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are studying idiopathic CD4 lymphopenia (ICL), a condition where people have low levels of CD4 T cells, making them vulnerable to infections, autoimmune diseases, and cancers. The study aims to test a new drug called NT-I7 (efineptakin alfa) that may increase CD4 T cell counts in people with ICL. This open-label trial is sponsored by the National Institute of Allergy and Infectious Diseases and focuses on evaluating the safety and immune effects of NT-I7 in adult participants with ICL. Participants will receive three doses of NT-I7 by injection into the muscle of the upper arm, thigh, or buttock, spaced about 12 weeks apart over a 24-week period. The study involves three dose levels based on body weight: 240, 480, or 720 micrograms per kilogram. Alongside dosing, participants will undergo leukapheresis three times to collect and separate white blood cells. Some visits may include rectal swabs, skin exams, biopsies, and medical imaging as part of the assessments. Throughout the study, participants will have physical exams and blood tests before each dose and at follow-up visits up to week 60. Blood samples will be collected regularly to monitor immune cell counts and safety. Questionnaires will assess patients' health perceptions and quality of life. After the final dose, participants will have three follow-up visits every three months to continue monitoring safety and immune responses. The primary outcome is the number and severity of side effects related to NT-I7, assessed at week 60.

CONDITIONS

Brief Title

A Single-arm, Dose-escalation Trial of Long-acting Recombinant Human IL-7 (NT-I7, Efineptakin Alfa) for Idiopathic CD4 Lymphopenia

Who Can Participate

Age: 18Years - 75Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Aged 18 to 75 years
  • Able to provide informed consent
  • Co-enrolled in NIH protocol 09-I-0102
  • Documented idiopathic CD4 lymphopenia with CD4 T-cell count below 300 cells/microliter on at least two occasions 6 weeks apart
  • Agree to use two effective methods of contraception, including one barrier method, if able to become pregnant or impregnate a partner (unless post-menopausal)
Not Eligible

You will not qualify if you...

  • Current moderate or severe acute illness
  • Active progressive cryptococcal CNS disease or nontuberculous mycobacterial infections within the last year
  • Pregnant or breastfeeding
  • HIV infection, chronic hepatitis B or C, or other known immunodeficiencies
  • Abnormal blood test results including creatinine, platelets, hemoglobin, liver enzymes, bilirubin, or immunoglobulin G levels
  • Recent use of systemic glucocorticosteroids or immunomodulants (except nasal spray, inhaler, topical steroids)
  • Autoimmune diseases requiring systemic treatment except vitiligo or controlled endocrine diseases
  • Recent malignancy requiring chemotherapy or immunotherapy
  • Use of other investigational agents within 3 months
  • Any condition that contraindicates participation according to the study team

AI-Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 screening and enrollment visit

Treatment

Duration - 24 weeks

Participants receive intramuscular injections of NT-I7 once every 12 weeks for a total of 3 doses, with the final dose at week 24.

3 dose visits with blood draws at each visit, plus additional blood draws 5 days before, and 14 and 30 days after each dose visit

Follow-up

Duration - Up to 36 weeks after treatment ends

Participants are monitored for safety and immunologic effects of NT-I7 up to week 60 after the first dose.

Periodic visits including blood draws and questionnaires until week 60

Trial Site Locations

Total: 1 location

1

National Institutes of Health Clinical Center

Bethesda, Maryland, United States, 20892

Actively Recruiting

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Research Team

A

Andrea Lisco, M.D.

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

1

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