Using NT-I7 to boost immune response in patients with a brain infection called PML
A Pilot Study of NT-I7, a Long-Acting Recombinant IL-7 Molecule, as an Immune Reconstitution Strategy for Lymphopenia in Patients With Progressive Multifocal Leukoencephalopathy
This study is testing if a new treatment called NT-I7 can help boost the immune system in adults with a brain infection called PML.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 18 Years to 120 Years |
| Sex | All |
| Sponsor | National Institutes of Health Clinical Center (CC) NIH |
| Locations | 1 site (Bethesda, Maryland) |
| Trial ID | NCT04781309 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the effectiveness of NT-I7, a long-acting recombinant IL-7 molecule, in increasing lymphocyte counts in adults suffering from progressive multifocal leukoencephalopathy (PML) and lymphopenia. The study will involve a 7-day inpatient stay followed by outpatient visits and phone follow-ups, where participants will receive up to three doses of NT-I7 via muscle injection. The primary goal is to measure changes in absolute lymphocyte counts, while secondary objectives include assessing the safety and tolerability of the treatment. Participants will also undergo various clinical and laboratory assessments to explore the drug's mechanism of action.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older diagnosed with PML and exhibiting lymphopenia.
Not a fit: Patients under 18 years old or those currently on immune-suppressive medications may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could enhance immune function in patients with PML, potentially improving their health outcomes.
How similar studies have performed: While this approach is novel in the context of PML, similar immune reconstitution strategies have shown promise in other conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
* INCLUSION CRITERIA: * 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 EXCLUSION CRITERIA: * 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
Where this trial is running
Bethesda, Maryland
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States (Recruiting)
Study contacts
- Principal investigator: Irene CM Cortese, M.D. — National Institute of Neurological Disorders and Stroke (NINDS)
- Study coordinator: Irene CM Cortese, M.D.
- Email: corteseir@ninds.nih.gov
- Phone: (301) 496-9175
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.