Phase 1 Single Ascending Dose Study

A Randomized, Double-blind, Placebo-controlled Study of the Safety, Tolerability and Pharmacokinetics of Single Ascending Doses of NM-101

Phase 1, double-blind, placebo-controlled, single-centre, single ascending dose escalation study to assess safety and tolerability of a monoclonal antibody, NM-101 in healthy volunteer subject and multiple system atrophy (MSA) patients.

Study Overview

• Status: Recruiting
• Conditions: Healthy Subjects
• Intervention / Treatment: Drug: Placebo; Drug: NM-101

Detailed Description

Phase 1, double-blind, placebo-controlled, single-centre, single ascending dose escalation study to assess safety and tolerability of a monoclonal antibody, NM-101 in healthy volunteer subject and multiple system atrophy (MSA) patients.

• Study Type: Interventional
• Enrollment (Estimated): 36
• Phase: Phase 1

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Recruiting
    • Seoul National University Hospital
    • Contact: Seung Hwan Lee, MD, PhD; Phone Number: 82-2-2072-1441; Email: leejh413@snu.ac.kr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

1. Male Healthy subjects, 18 to 60 years of age and in good health with no clinically significant abnormality identified on the medical history, physical examination or laboratory evaluation at screening
2. Body weight≥ 45kg and body mass index between 18 - 30 kg/m2
3. Normal blood pressure (systolic >90 and <140 mmHg, diastolic >40 and <90mmHg) and pulse rate 40-100bpm at screening visit. Blood pressure and pulse are measured after 3 minutes in supine position
4. Baseline QTc must be <450msec for men and <470msec for women
5. Normal 12-lead electrocardiogram at screening
6. No clinically significant abnormal laboratory test values at screening
7. No clinically significant findings on the clinical neurological and ophthalmic examinations at screening and at baseline
8. Good venous access in both arms
9. Willing to consent to participate in study prior to study specific screening procedures with the understanding that the subject has the right to withdraw from the study at any time without prejudice
10. Female subject who is surgically sterile, is postmenopausal, or agrees to use a highly effective method of birth control (2 methods strongly recommended) during the study and for 6 months after the dosing of NM-101.

Exclusion Criteria:

1. History of serious adverse reaction or hypersensitivity to biological agents including immunoglobulins
2. Presence or history of any allergy requiring acute or chronic treatment; seasonal allergic rhinitis can be permitted unless the subject is taking systemic medication (nasal spray or local treatments permitted). Subjects having clinically significant drug or food allergies are also excluded.
3. History of autoimmune or inflammatory disease
4. Clinically significant (i.e., active) cardiovascular disease (e.g., hypertension, arrhythmia, myocardial infarction, heart failure, long QT syndrome or other conditions causing prolongation of the QT/QT interval corrected with Fridericia's formula [QTcF]) prior to screening.
5. History of cerebral vascular accident or stroke. Subjects having high risk of developing a stroke are also excluded.
6. History or positive test results at screening for human immunodeficiency virus (HIV), hepatitis B, hepatitis C
7. Positive in tuberculosis screening test (Quantiferon test)
8. Active infection within 4 weeks from screening and body temperature >38℃
9. Active immunization within 3 months prior to dosing of NM-101
10. History or clinically significant evidence of cardiovascular, endocrine (e.g. diabetes mellitus), respiratory, renal, hepatic, gastrointestinal, haematological neurologic, psychiatric or other disease
11. Chronic symptoms of pronounced constipation or diarrhoea or conditions associated with total or partial obstruction of the urinary track
12. History or presence of malignancy including solid tumors and hematologic malignancies except for basal cell and squamous cell carcinoma of the skin that had been completely excised and were considered cured with no evidence of disease ≥ 3 years
13. Participation in a clinical study during the previous 24 weeks i.e. from completion of the previous study to the planned first administration of the current study
14. Loss of >500mL blood including blood donation within 12 weeks prior to screening visit
15. Intake more than 24 units of alcohol per week (1 unit=250mL of beer, 100mL of wine or 35mL of spirits)
16. Smokers >5 cigarettes or the equivalent in tobacco daily. Must have been willing to abstain from using tobacco and tobacco-containing products for 72 hours prior to the dosing of NM-101 and during the inpatient monitoring period.
17. Treatment with any prescription medication and/or over-the-counter products (excluding acetaminophen, hormone replacement therapy (HRT) and birth control) within 30 days prior to dosing of NM-101. Routine vitamin therapy is allowed.
18. History of drug or alcohol abuse within the past 5 years, a positive urine drug test and/or a positive alcohol urine test at Screening visit
19. Surgery other than minor cosmetic surgery and minor dental surgery within 3 months from the screening visit
20. History of lumbar surgery for any reasons (e.g., herniated disc) or other contraindications to having al lumbar puncture (LP)
21. Vigorous physical exercise 48 hours prior to screening
22. Women with a positive pregnancy test at screening
23. Women who are breast feeding

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Single Group Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Placebo
Experimental: NM-101; NM-101 iv	Drug: NM-101; NM-101 IV versus placebo; Other Names: Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To determine the pharmacokinetic profile of NM-101 after a single iv infusion	Peak plasma drug concentration (Cmax)	Pre-dose, 0, 0.5, 1, 2, 4, 6, 8, 12hour post-dose at Day 1 and Day 2, 3, 4, 8, 15, 22, 29 and week 6, 8, 12
To determine the pharmacokinetic profile of NM-101 after a single iv infusion	Area under the plasma concentration versus time curve (AUC) at steady-state and until last observation	Pre-dose, 0, 0.5, 1, 2, 4, 6, 8, 12hour post-dose at Day 1 and Day 2, 3, 4, 8, 15, 22, 29 and week 6, 8, 12
To determine the pharmacokinetic profile of NM-101 after a single iv infusion	Concentration at trough	Pre-dose, 0, 0.5, 1, 2, 4, 6, 8, 12hour post-dose at Day 1 and Day 2, 3, 4, 8, 15, 22, 29 and week 6, 8, 12
To determine the pharmacokinetic profile of NM-101 after a single iv infusion	Time to maximum drug concentration	Pre-dose, 0, 0.5, 1, 2, 4, 6, 8, 12hour post-dose at Day 1 and Day 2, 3, 4, 8, 15, 22, 29 and week 6, 8, 12
To determine the pharmacokinetic profile of NM-101 after a single iv infusion	half-life of drug	Pre-dose, 0, 0.5, 1, 2, 4, 6, 8, 12hour post-dose at Day 1 and Day 2, 3, 4, 8, 15, 22, 29 and week 6, 8, 12
To determine the pharmacokinetic profile of NM-101 after a single iv infusion	Volume distribution of drug	Pre-dose, 0, 0.5, 1, 2, 4, 6, 8, 12hour post-dose at Day 1 and Day 2, 3, 4, 8, 15, 22, 29 and week 6, 8, 12
To determine the pharmacokinetic profile of NM-101 after a single iv infusion	Clearance rate of drug	Pre-dose, 0, 0.5, 1, 2, 4, 6, 8, 12hour post-dose at Day 1 and Day 2, 3, 4, 8, 15, 22, 29 and week 6, 8, 12

Collaborators and Investigators

• Sponsor: Neuramedy Co. Ltd.
• Collaborators: Seoul National University Hospital

Study record dates

Study Major Dates

• Study Start (Actual): November 25, 2024
• Primary Completion (Estimated): October 30, 2025
• Study Completion (Estimated): March 30, 2026

Study Registration Dates

• First Submitted: April 3, 2025
• First Submitted That Met QC Criteria: April 10, 2025
• First Posted (Actual): April 18, 2025

Study Record Updates

• Last Update Posted (Actual): April 18, 2025
• Last Update Submitted That Met QC Criteria: April 10, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Keywords: Single Ascending Dose study
• Other Study ID Numbers: NM-101-01; NM-101 (Other Identifier: Neuramedy)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No