Ignite Creation Date:
2024-05-06 @ 3:51 PM
Last Modification Date:
2024-10-26 @ 1:58 PM
Study NCT ID:
NCT04781309
Status:
RECRUITING
Last Update Posted:
2024-02-14
First Post:
2021-03-03
Brief Title:
NT-I7 a Long-Acting Recombinant IL-7 Molecule as an Immune Reconstitution Strategy for Lymphopenia in Patients With Progressive Multifocal Leukoencephalopathy
Sponsor:
National Institute of Neurological Disorders and Stroke NINDS
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
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
Status:
RECRUITING
Status Verified Date:
2024-10-18
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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
Detailed Description:
Study Description
This protocol will test whether NT-I7 is a viable strategy for promoting immune reconstitution in lymphopenic patients with PML Twelve adults with PML and lymphopenia CD4 or CD8 T cell count of less than or equal to 200 cellsdL from all causes will complete this pilot study Patients will be observed as inpatient for the first 7 days following any experimental drug dosing To follow patients will return to NIH for a second 7-day inpatient stay by Day 21 and then for scheduled outpatient visits at NIH at month 2 3 6 9 and 12 following any drug dosing Follow up phone calls will be conducted at month 4 5 7 and 8 Patients may be eligible for a second dose of NT-I7 at higher dose if target ALC 1000microliter not reached or at same dose if initial response is adequate but not sustained for a maximum of 3 doses The primary outcome measure is change in absolute lymphocyte counts ALC Secondary outcomes include safety and tolerability Exploratory clinical radiological and laboratory measures will be obtained to investigate mechanism of action of NT-I7 and for biomarker development
Objectives
Primary Objective To determine the kinetics and magnitude of effect of NT-I7 on absolute lymphocyte counts in patients with PML and underlying lymphopenia from various causes in order to inform appropriate design of a future study
Secondary Objectives 1 To assess safety and tolerability of NT-I7 2 To determine kinetics and magnitude of effect of NT-I7 on lymphocyte subsets 3 To investigate effect of NT-I7 on PML disease course 4 To investigate mechanism of action of NT-I7
Endpoints
Primary Endpoint Change in absolute lymphocyte counts ALC over 6 months
Secondary Endpoints 1 Adverse event tables 2 Change in lymphocyte subset counts including CD4 CD8 and CD19 positive cells 3 Change in standardized disability rating scales PML lesion extension by brain MRI viral quantification in CSF and survival 4 Exploratory serological and CSF measures to investigate immune response to JCV and mechanism of action of NT-I7
This protocol will test whether NT-I7 is a viable strategy for promoting immune reconstitution in lymphopenic patients with PML Twelve adults with PML and lymphopenia CD4 or CD8 T cell count of less than or equal to 200 cellsdL from all causes will complete this pilot study Patients will be observed as inpatient for the first 7 days following any experimental drug dosing To follow patients will return to NIH for a second 7-day inpatient stay by Day 21 and then for scheduled outpatient visits at NIH at month 2 3 6 9 and 12 following any drug dosing Follow up phone calls will be conducted at month 4 5 7 and 8 Patients may be eligible for a second dose of NT-I7 at higher dose if target ALC 1000microliter not reached or at same dose if initial response is adequate but not sustained for a maximum of 3 doses The primary outcome measure is change in absolute lymphocyte counts ALC Secondary outcomes include safety and tolerability Exploratory clinical radiological and laboratory measures will be obtained to investigate mechanism of action of NT-I7 and for biomarker development
Objectives
Primary Objective To determine the kinetics and magnitude of effect of NT-I7 on absolute lymphocyte counts in patients with PML and underlying lymphopenia from various causes in order to inform appropriate design of a future study
Secondary Objectives 1 To assess safety and tolerability of NT-I7 2 To determine kinetics and magnitude of effect of NT-I7 on lymphocyte subsets 3 To investigate effect of NT-I7 on PML disease course 4 To investigate mechanism of action of NT-I7
Endpoints
Primary Endpoint Change in absolute lymphocyte counts ALC over 6 months
Secondary Endpoints 1 Adverse event tables 2 Change in lymphocyte subset counts including CD4 CD8 and CD19 positive cells 3 Change in standardized disability rating scales PML lesion extension by brain MRI viral quantification in CSF and survival 4 Exploratory serological and CSF measures to investigate immune response to JCV and mechanism of action of NT-I7
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 000126-N | None | None | None |