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| Name | Class |
|---|---|
| National Multiple Sclerosis Society | OTHER |
| Race to Erase MS | UNKNOWN |
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The goal of this clinical trial is to evaluate if the study drug will reduce brain and retinal atrophy by reducing inflammation and subsequently slowing neurodegeneration in people with Multiple Sclerosis. The main outcome for the trial is change in normalized brain parenchymal volume (nBPV), measured by magnetic resonance imaging (MRI).
Researchers will compare outcomes from participants randomized to the study drug, versus participants randomized to placebo, to see if there are signs of slowed neurodegeneration (i.e., reduction in brain and retinal atrophy).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NLY01 (Study Drug) | Active Comparator | 5 mg subcutaneous weekly for the first 4 weeks, then 10 mg subcutaneous weekly, with pre-planned dose adjustments if adverse events prevent full dosage. |
|
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NLY01 | Drug | NLY01 is a pegylated exenatide |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Change in normalized (for head size) brain parenchymal volume (nBPV) | Change in normalized (for head size) nBPV (mL). Measured from randomization to end of treatment (approximately 96 weeks). Presented as mean and standard deviation | Baseline, week 48, and week 96 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in normalized gray matter volume (mL) | Presented as mean and standard deviation Measured from randomization to end of treatment (approximately 96 weeks). | Baseline, week 48, and week 96 |
| Change in thalamic volume (mL) |
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Inclusion Criteria:
Exclusion Criteria:
No GLP-1RA or GIP/GLP-1 RA in past year; no known hypersensitivity to medication class
No known Barrett's esophagus/gastroesophageal reflux disease, pancreatitis (including past), or gastroparesis
No personal/family history of medullary thyroid carcinoma or history of multiple endocrine neoplasia syndrome type 2
No chronic kidney disease (estimated glomerular filtration rate ≤50 mL/min) in past year, type 1 diabetes, known diabetic retinopathy, use of insulin or insulin-inducing medications*, dipeptidyl peptidase IV inhibitors**, or warfarin; current/active alcohol or illicit substance abuse
No concerns about candidacy of individual on part of person's neurologist or study team clinicians
Current or planned (next 2 years) pregnancy/breastfeeding; if able to become pregnant, agree to reliable contraception (contraception requirements as discussed below)***
Contraceptive methods with a failure rate of < 1% per year includes the following:
Those who do not wish to use one of the above methods of contraception must use two methods. Options include:
Oral hormonal contraception plus one barrier method during sexual encounter with a person who has testes (below). While typically oral hormonal contraception has a low failure rate, it is possible that the absorption of contraceptive pills taken by mouth will be impacted by the study drug and thus lower contraceptive effectiveness. Thus, people using pills as primary contraception must, during asexual encounter with a person who has testes, use a second form of barrier contraceptive (below) or must change to one of the other contraceptive methods listed above.
Two forms of barrier contraception during sexual encounter with a person who has testes. Examples of barrier contraceptive methods include the following:
A condom with or without spermicide
A cap, diaphragm, or sponge with or without spermicide
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Study Manager | Contact | 667-306-8153 | mvieira4@jh.edu |
| Name | Affiliation | Role |
|---|---|---|
| Ellen M Mowry, MD, MCR | Johns Hopkins University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Johns Hopkins University | Recruiting | Baltimore | Maryland | 21287 | United States |
De-identified individual participant data (IPD) that underlie the results reported in this publication, along with the protocol and analysis plan, will be available upon reasonable request to qualified researchers. Requests must include a proposal and are contingent on meeting regulatory approvals, including a signed data use agreement.
Data will be available 6 months after publication and remain accessible for 12-24 months.
Qualified researchers who have completed and signed approvals for data sharing.
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| ID | Term |
|---|---|
| D009103 | Multiple Sclerosis |
| ID | Term |
|---|---|
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D003711 | Demyelinating Diseases |
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| Drug |
Placebo (saline solution) |
|
Measured from randomization to end of treatment (up to 96 weeks). Presented as mean and standard deviation.
| Baseline, week 48, and week 96 |
| Change in cortical thickness (mm) | Measured from randomization to end of treatment (approximately 96 weeks), Presented as mean and standard deviation. | Baseline, week 48, and week 96 |
| Change in retinal nerve fiber layer thickness | Measured in μm, from randomization to end of treatment (approximately 96 weeks), | Baseline, week 48, and week 96 |
| Change in ganglion cell/inner plexiform thickness | Measured in μm, from randomization to end of treatment (approximately 96 weeks), | Baseline, week 48, and week 96 |
| Disability progression as assessed by the Expanded Disability Status Scale (EDSS) | Expanded Disability Status Scale (EDSS). Scale range 0-10; higher score is worse disability progression | Approximately every 24 weeks, up to 96 weeks |
| Disability progression as assessed by the Multiple Sclerosis Functional Composite (MSFC) | The Multiple Sclerosis Functional Composite (MSFC) calculates a single Z-score from tests of ambulation, arm dexterity, and cognition. Scores are converted to a z score with a mean and standard deviation. A higher Z-score indicates better function. | Approximately every 24 weeks, up to 96 weeks |
| Disability progression as assessed by the Expanded Disability Status Scale-Plus | The EDSS-plus allows for documentation of progression as a composite value, considering progression as having occurred if any one of the following occurs: 20% increase in timed 25-foot walk or 9-hole peg test, or a 1.0-point increase in EDSS (if baseline is 5.5 or less) or a 0.5-point increase (if baseline is >5.5). | Approximately every 24 weeks, up to 96 weeks |
| Patient-reported disability progression as assessed by the Patient-Determined Disease Steps | Patient-Determined Disease Steps score range 0-8; higher is worse progression | Approximately every 24 weeks, up to 96 weeks |
| Change in self-reported anxiety as assessed by the Quality of Life in Neurological Disorders (Neuro-QOL) Anxiety Subscale | Quality of Life in Neurological Disorders (Neuro-QOL) Anxiety Subscale. Higher scores indicate more of the domain; distribution of T-Score has a mean of 50, standard deviation of 10. | Approximately every 24 weeks, up to 96 weeks |
| Change in self-reported depression as assessed by the Quality of Life in Neurological Disorders (Neuro-QOL) Depression Subscale | Quality of Life in Neurological Disorders (Neuro-QOL) Depression Subscale. Higher scores indicate more of the domain; distribution of T-Score has a mean of 50, standard deviation of 10. | Approximately every 24 weeks, up to 96 weeks |
| Change in self-reported fatigue as assessed by the Quality of Life in Neurological Disorders (Neuro-QOL) Fatigue Subscale | Quality of Life in Neurological Disorders (Neuro-QOL) Fatigue Subscale. Higher scores indicate more of the domain; distribution of T-Score has a mean of 50, standard deviation of 10. | Approximately every 24 weeks, up to 96 weeks |
| Change in self-reported upper extremity function as assessed by the Quality of Life in Neurological Disorders (Neuro-QOL) Upper Extremity Function Subscale | Quality of Life in Neurological Disorders (Neuro-QOL) Upper Extremity Function Subscale. Higher scores indicate more of the domain; distribution of T-Score has a mean of 50, standard deviation of 10. | Approximately every 24 weeks, up to 96 weeks |
| Change in self-reported lower extremity function as assessed by the Quality of Life in Neurological Disorders (Neuro-QOL) Lower Extremity Function Subscale | Quality of Life in Neurological Disorders (Neuro-QOL) Lower Extremity Function Subscale. Higher scores indicate more of the domain; distribution of T-Score has a mean of 50, standard deviation of 10. | Approximately every 24 weeks, up to 96 weeks |
| Change in self-reported cognitive function as assessed by the Quality of Life in Neurological Disorders (Neuro-QOL) Cognitive Function Subscale | Quality of Life in Neurological Disorders (Neuro-QOL) Cognitive Function Subscale. Higher scores indicate more of the domain; distribution of T-Score has a mean of 50, standard deviation of 10. | Approximately every 24 weeks, up to 96 weeks |
| Change in positive affect/well-being as assessed by the Quality of Life in Neurological Disorders (Neuro-QOL) Positive Affect/Well-being Subscale | Quality of Life in Neurological Disorders (Neuro-QOL) Positive Affect/Well-being Subscale. Higher scores indicate more of the domain; distribution of T-Score has a mean of 50, standard deviation of 10. | Approximately every 24 weeks, up to 96 weeks |
| Change in self-reported sleep disturbance as assessed by the Quality of Life in Neurological Disorders (Neuro-QOL) Sleep Disturbance Subscale | Quality of Life in Neurological Disorders (Neuro-QOL) Sleep Disturbance Subscale. Higher scores indicate more of the domain; distribution of T-Score has a mean of 50, standard deviation of 10. | Approximately every 24 weeks, up to 96 weeks |
| Change in self-reported ability to participate in social roles Quality of Life in Neurological Disorders (Neuro-QOL) Ability to Participate in Social Roles Subscale | Quality of Life in Neurological Disorders (Neuro-QOL) Ability to Participate in Social Roles Subscale. Higher scores indicate more of the domain; distribution of T-Score has a mean of 50, standard deviation of 10. | Approximately every 24 weeks, up to 96 weeks |
| Change in self-reported stigma as assessed by the Quality of Life in Neurological Disorders (Neuro-QOL) Stigma Subscale | Quality of Life in Neurological Disorders (Neuro-QOL) Stigma Subscale. Higher scores indicate more of the domain; distribution of T-Score has a mean of 50, standard deviation of 10. | Approximately every 24 weeks, up to 96 weeks |
| Adverse events | Proportion of individuals in each arm experiencing adverse events | From randomization to week 96 |
| Serious adverse events | Proportion of individuals in each arm experiencing serious adverse events | From randomization to week 96 |
| Mount Sinai School of Medicine | Not yet recruiting | New York | New York | 10029 | United States |
|
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |