NT-I7 for Leukoencephalopathy, Progressive Multifocal

Phase-Based Estimates
1
Effectiveness
1
Safety
National Institutes of Health Clinical Center, Bethesda, MD
Leukoencephalopathy, Progressive Multifocal+3 More
NT-I7 - Drug
Eligibility
18+
All Sexes
Eligible conditions
Leukoencephalopathy, Progressive Multifocal

Study Summary

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

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Eligible Conditions

  • Leukoencephalopathy, Progressive Multifocal
  • Leukoencephalopathies
  • Lymphopenia
  • Progressive Multifocal Leucoencephalopathy (PML)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether NT-I7 will improve 1 primary outcome and 3 secondary outcomes in patients with Leukoencephalopathy, Progressive Multifocal. Measurement will happen over the course of at each study timepoint.

at each study timepoint
Change in lymphocyte subsets, including CD4, CD8, and CD19 positive cells
Disease course
Safety
over 6months following study drug administration
the longitudinal change in absolute lymphocyte count over 6 months following study drug administration

Trial Safety

Safety Estimate

1 of 3

Trial Design

2 Treatment Groups

Control
NT-I7

This trial requires 12 total participants across 2 different treatment groups

This trial involves 2 different treatments. NT-I7 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase < 1 and are in the first stage of evaluation with people.

NT-I7
Drug
480 microgram/kg IM (initial dose)
ControlNo treatment in the control group

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: over 6months following study drug administration
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly over 6months following study drug administration for reporting.

Closest Location

National Institutes of Health Clinical Center - Bethesda, MD

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 8 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
CD4 and/or CD8 lymphopenia less than or equal to 200 cells/dL from any cause that is not readily reversible within one month
Enrolled in 13-N-0017
Adults (18 years of age or older)
Definite or Probable PML (2013 AAN Consensus Diagnostic Criteria)
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

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many people get leukoencephalopathy, progressive multifocal a year in the United States?

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Despite widespread surveillance to identify patients and provide treatment, cases of leukoencephalopathy, progressive multifocal a year still occur in this population. Although the clinical presentation, course, and age of the patients seem to be distinct from those of the progressive multifocal leukoencephalopathy observed in other countries, the development of a more sensitive assay to detect the presence of the organism is essential.

Unverified Answer

Can leukoencephalopathy, progressive multifocal be cured?

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It is now recommended that patients suffering from PMB should be treated with the drug eculizumab despite the lack of cure. However, it is still a challenge that researchers will try to cure PMB at an early stage.

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What are common treatments for leukoencephalopathy, progressive multifocal?

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Patients with progressive multifocal leukoencephalopathy, leukodystrophy, and leukorachnia generally have long histories of immunosuppressive treatment, often prior to diagnosis of the disease. The diagnosis of a disorder such as leukoencephalopathy, progressive multifocal leukoencephalopathy, or leukoencephalopathy, progressive multifocal leukoencephalopathy often depends on the history of prior treatment. When no other precipitating factor for the leukoencephalopathy, progressive multifocal leukoencephalopathy, is known, the disorder has usually been present in all of the family members.

Unverified Answer

What causes leukoencephalopathy, progressive multifocal?

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We identified only one case of prion disease for leukoencephalopathy in the literature. The clinical features are similar to other types of encephalopathy, and histological features are discussed. We speculate that the etiopathogenic mechanism in leukoencephalopathy is similar to that in Creutzfeldt-Jakob disease and fatal familial insomnia.

Unverified Answer

What are the signs of leukoencephalopathy, progressive multifocal?

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The diagnosis of PMP was not made earlier in this series, because the symptoms were atypical and progressive when they appeared. Most likely, the symptoms were misdiagnosed as typical Parkinson's disease. The clinical course and progression of the disease were also variable.

Unverified Answer

What is leukoencephalopathy, progressive multifocal?

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PMF, also known as progressive multifocal leukoencephalopathy, is a progressive and potentially fatal condition characterised by subcortical white matter lesions. The underlying diagnosis of PMF is typically made post-mortem by post mortem examination, which is also required to diagnose other leukodystropheic conditions such as metachromatic leukodystrophy. PMF's post mortem differential diagnosis includes vascular dementia, Creutzfeldt–Jakob disease and leukoencephalopathy, chronic B encephalopathy, and Creutzfeldt–Jakob variants.

Unverified Answer

Who should consider clinical trials for leukoencephalopathy, progressive multifocal?

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No clinical trials for PM (a.k.a. subacute sclerosing leucocytoclastic vasculitis, leukoencephalopathy, subacute sclerosing scleroclastic vasculitis, subacute sclerosing vasculitis of the white matter, acute demyelinating subacute myelinating leukoencephalopathy, acute multifocal subacute progressive leukoencephalopathy and subacute demyelinating leukoencephalopathy of the white matter) in PMF patients seem promising in clinical trials in regards to the treatment options.

Unverified Answer

Have there been any new discoveries for treating leukoencephalopathy, progressive multifocal?

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Recent research has identified a number of new treatment strategies that may be useful in combating leukoencephalia, although long-term studies are needed to show that they prevent disease progression. However, the most promising treatments for this disease include a new class of drugs called glutaminase inhibitors, such as aciclovir, which is currently being researched in a phase three trial. In addition researchers are exploring the possibility of using monotherapy, which effectively suppresses the virus, for the long-term goal of preventing disease progression. Unfortunately, treatments like these are not available in North America.

Unverified Answer

What is nt-i7?

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It is important to know that there are many variants in nt-i7 and this number is expected to grow larger in the near future due to the high throughput sequencing method.\nThe use of nucleotides is more common than expected after it is understood that every nucleotide contains four components, three bases that are in the purine and pyrimidine family and a sugar. The types of sugars that can combine with the four nucleic acid bases are a purine or pyrimidine.

Unverified Answer

What is the primary cause of leukoencephalopathy, progressive multifocal?

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Although the precise mechanism is yet unknown, [leukoencephalopathy, progressive multifocal (PML)] seems to result from an accumulation of [white matter (WM)] and [pia mater (PM)] that are often not fully developed. An [intrinsic defect of WM cells during development is hypothesized to cause this white matter disease. The primary cause is unknown, but the disease seems to occur with a high risk in families or [environmental variables associated with aging]. The white matter disease is most likely diagnosed by neurological exam of patients with cognitive impairment. Some [white matter lesions found in the brains of deceased members of the disease's family appear to occur early in life and have a progressive course.

Unverified Answer

How does nt-i7 work?

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Data from a recent study are consistent with the hypothesis that disruption of the mitochondrial complex III activity alters the mitochondrial lipid composition, resulting in activation of caspase-3.

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Has nt-i7 proven to be more effective than a placebo?

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There was no significant difference between the two medicines when measured by a questionnaire and motor function tests. If patients have the chance of a good response, an additional placebo group should be added and measured again in a following month to be sure that there is no placebo effect.

Unverified Answer
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