NT-I7, a Long-Acting Recombinant IL-7 Molecule, as an Immune Reconstitution Strategy for Lymphopenia in Patients With Progressive Multifocal Leukoencephalopathy

NCT04781309 | Completed Early Phase 1 FDA Drug

• 2 other identifiers: 10000126000126-N
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

Background: Progressive multifocal leukoencephalopathy (PML) is a brain infection. It is caused by a virus. PML can happen in people with a weakened immune system. PML is associated with cognitive and visual impairment as well as motor and speech disturbances. There is no treatment for PML. Researchers want to see if a new drug can help. Objective: To see if the drug NT-I7 can help increase lymphocyte numbers, which may help control PML infection. Eligibility: Adults ages 18 and older with PML who are enrolled in Protocol #13-N-0017. Design: Participants will be screened under Protocol #13-N-0017. Participants will have a 7-day inpatient stay, outpatient visits, and follow-up phone calls. Participants will have a medical history and physical exam. They will give urine samples. Blood will be drawn from an arm vein or through an intravenous (IV) catheter. Participants will get up to 3 doses of NT-I7. It will be given by injection into the muscle. Participants will have lumbar punctures ( spinal taps ). A thin needle will be inserted into the spinal canal in the lower back. Cerebrospinal fluid will be removed. X-ray may be used to guide the procedure. Participants will have magnetic resonance imaging (MRI) of the brain. The MRI scanner is a metal cylinder surrounded by a magnetic field. During MRIs, participants will lie on a table that slides in and out of the scanner. Soft padding or a coil will be placed around their head. They will get gadolinium, a contrast agent, through an IV catheter. Participation will last for 12 to 19 months. ...

Conditions

Progressive Multifocal Leukoencephalopathy

Keywords

Absolute Lymphocyte Count; safety and tolerability of NT-I7

Outcome Measures

Primary Outcomes (1)

• the longitudinal change in absolute lymphocyte count over 6 months following study drug administration

  determine the kinetics and magnitude of effect of NT-I7 on absolute lymphocyte counts (ALC) in patients with PML and underlying lymphopenia from various causes, in order to inform appropriate design of a future study

  over 6months following study drug administration

Secondary Outcomes (3)

• Safety

  at each study timepoint

• Change in lymphocyte subsets, including CD4, CD8, and CD19 positive cells

  at each study timepoint

• Disease course

  at each study timepoint

Study Arms (1)

NT-I7

EXPERIMENTAL

480 microgram/kg IM (initial dose)

Drug: NT-I7

Interventions

NT-I7 DRUG

NT-I7 is a recombinant human interleukin-7 fused to a hybrid fragment crystallizable region of a human antibody (hyFc). NT-I6 has a molecular weight of 104 kD. The active moiety of NT-I7 is recombinant human interleukin-7 (rhIL-7), containing human IL-7 (amino acids 4 through 155) and exhibiting all known functions of endogenous human IL-7.

NT-I7

Eligibility Criteria

• Age: 18 Years - 120 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adults (18 years of age or older)
• Definite or Probable PML (2013 AAN Consensus Diagnostic Criteria)
• CD4 and/or CD8 lymphopenia \<= 200 cells/dL that is not readily reversible within one month
• Enrolled in 13-N-0017
• Ability to provide own consent at study entry
• Ability to travel to NIH for study visits
• Willingness to comply with all study procedures
• If able to become pregnant or to father a child, patient must agree to commit to the use of a reliable/accepted method of birth control (i.e. hormonal contraception (birth control pills, injected hormones, vaginal ring), intrauterine device, barrier methods with spermicide (diaphragm with spermicide, condom with spermicide) or surgical sterilization (hysterectomy, tubal ligation, or vasectomy) for the duration of the study

You may not qualify if:

• Age \< 18 years of age
• Ongoing treatment with immune-suppressive medications (exception: topical steroid use and all forms of systemic steroids with durations less than 2 weeks)
• History of immune-mediated disease affecting vital organ
• Concurrent treatment with experimental therapies for PML that would interfere with or confound assessment of study outcomes
• History of underlying autoimmune disease involving the CNS
• Contraindication to any study procedures that would compromise ability to safely monitor the patient
• History or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial or interfere with study participation; or not in the best interest of the subject to participate, in the opinion of the treating investigator
• Women who are pregnant or breastfeeding
• Unwilling to have coded samples and/or data saved or used in other studies

Sponsors & Collaborators

• National Institute of Neurological Disorders and Stroke (NINDS): lead
• NeoImmuneTech: collaborator

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Leukoencephalopathy, Progressive Multifocal

Interventions

efineptakin alfa

Condition Hierarchy (Ancestors)

Encephalitis, Viral; Central Nervous System Viral Diseases; Central Nervous System Infections; Infections; Infectious Encephalitis; Virus Diseases; Polyomavirus Infections; DNA Virus Infections; Slow Virus Diseases; Encephalitis; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Leukoencephalopathies; Demyelinating Diseases; Neuroinflammatory Diseases

Study Officials

• Irene CM Cortese, M.D.

  National Institute of Neurological Disorders and Stroke (NINDS)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 3, 2021
• First Posted: March 4, 2021
• Study Start: May 5, 2021
• Primary Completion: November 7, 2025
• Study Completion: November 7, 2025
• Last Updated: November 18, 2025

Record last verified: 2025-11-14

Locations

US (1)