A Phase 1, SAD and MAD Study to Evaluate the Safety and Tolerability of FB418

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, SAD and MAD Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral FB418 in Healthy Adult Subjects and Healthy Elderly Subjects

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics and pharmacodynamics of oral doses of FB418 in healthy adult subjects and healthy elderly subjects.

Study Overview

• Status: Recruiting
• Conditions: Amyotrophic Lateral Sclerosis (ALS); Parkinson Disease Psychosis
• Intervention / Treatment: Drug: FB418

Detailed Description

Part A:

Primary:

To assess the safety and tolerability of single ascending oral doses of FB418 in healthy adult subjects and healthy elderly subjects.

Secondary:

To assess the pharmacokinetics (PK) of FB418 and metabolite after single ascending oral doses of FB418 in healthy adult subjects and healthy elderly subjects.

To assess the effect of a high-fat meal on the PK of FB418 and metabolite after a single oral dose of FB418 when administered to healthy adult subjects.

Part B:

Primary:

To assess the safety and tolerability of multiple ascending oral doses FB418 in healthy adult subjects.

Secondary:

To assess the PK of FB418 and metabolite after multiple ascending oral doses of FB418 in healthy adult subjects.

• Study Type: Interventional
• Enrollment (Estimated): 64
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: 1STBIO information team; Phone Number: +82-31-895-4677; Email: info@1stbio.com

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of, 03080
    • Recruiting
    • Seoul National University
    • Contact: Jang Injin, M.D; Email: ijjang@snu.ac.kr

Participation Criteria

Eligibility Criteria

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

Description

Key Inclusion Criteria:

1. Healthy, adult, male 19 55 years of age, inclusive, at the screening.
2. For elderly subjects(Part A Cohort 5 only), healthy, elderly, male or female (of non childbearing potential only) over 60 years of age, inclusive, at screening.
3. In the case of the elderly(Part A Cohort 5 only), they have a history of mild disease, but can participate if the researcher judges that they can stop taking the drug at least 2 weeks before the expected first dose or before the half-life is 5 times or more)
4. The continuous non smoker who have not used nicotine containing products for at least 3 months prior to (the first) dosing and throughout the study are based on subjective self reporting.
5. Body mass index (BMI) ≥ 18 and ≤ 32 kg/m2 at the screening.
6. Medically healthy with no clinically significant medical history, physical examination, simplified neurological examination, laboratory profiles, vital signs or ECGs, as deemed by the PI or designee.
7. In Cohort 5, A female subject must be of non childbearing potential

Key Exclusion Criteria:

1. Is mentally or legally incapacitated or has significant emotional problems at the time of the screening visit or expected during the conduct of the study.
2. History or presence of a clinically significant medical or psychiatric condition or disease in the opinion of the PI or designee.
3. History of any illness that, in the opinion of the PI or designee, might confound the results of the study or poses an additional risk to the subject by their participation in the study.

4. Is at suicidal risk in the opinion of the PI as per the following criteria:

   1. Any suicide attempts within 12 months prior to screening or any suicidal intent, including a plan, within 3 months prior to screening.
   2. C-SSRS answer of "YES" on suicidal ideation within 3 months prior to screening.
5. History or presence of alcoholism or drug abuse within the past 2 years prior to (the first) dosing.
6. History or presence of hypersensitivity or idiosyncratic reaction to the study drug or related compounds.
7. History of seizures (childhood febrile seizures are excepted).
8. Positive urine drug or alcohol results at screening or check in.
9. Has an abnormal screening ECG indicating a second- or third- degree AV block, or one or more of the following: QRS > 120 msec, QTcF > 450 msec for males and > 460 msec for females, PR interval > 220 msec. Any rhythm other than normal sinus rhythm, which is interpreted by the PI or designee to be clinically significant at screening or check-in.
10. Has any medical or surgical condition in which lumbar puncture is contraindicated in the opinion of the PI.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: FB418; Part A - Single ascending oral doses of FB418 in healthy adult subjects and healthy elderly subjects. Part B - Multiple ascending oral doses FB418 in healthy adult subjects.	Drug: FB418; Oral dose; Other Names: FB418 active drug
Placebo Comparator: FB418-Placebo; Part A - Single Ascending Dose matching placebo study Part B - Multiple Ascending Dose matching placebo study	Drug: FB418; Oral dose; Other Names: FB418 active drug

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment Emergent Adverse Events (TEAE)	Number of TEAEs including serious adverse events (SAEs)	Up to 7 ~ 14 days after the last dose

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
PK parameter 1	Maximum observed concentration (Cmax) of FB418 in plasma	Up to 72 hours after the last dose
PK parameter 2	Time to maximum observed concentration (Tmax) of FB418 in plasma	Up to 72 hours after the last dose
PK parameter 3	The area under the concentration-time curve from time zero extrapolated to infinity (AUC0-∞) of FB418 in plasma	Up to 72 hours after the last dose
PK parameter 4	Area under the concentration-time curve from time zero to the time of last quantifiable concentration (AUC[0-last]) of FB418 in plasma	Up to 72 hours after the last dose
PK parameter 5	Apparent terminal elimination half-life (t1/2) of FB418 in plasma	Up to 72 hours after the last dose

Collaborators and Investigators

• Sponsor: 1ST Biotherapeutics, Inc.
• Investigators: Study Director: 1STBIO study manager, 1ST Biotherapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): December 20, 2023
• Primary Completion (Estimated): December 1, 2024
• Study Completion (Estimated): December 1, 2024

Study Registration Dates

• First Submitted: August 7, 2023
• First Submitted That Met QC Criteria: August 13, 2023
• First Posted (Actual): August 16, 2023

Study Record Updates

• Last Update Posted (Estimated): December 2, 2024
• Last Update Submitted That Met QC Criteria: November 27, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Synucleinopathies; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Movement Disorders; Parkinsonian Disorders; Basal Ganglia Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Parkinson Disease; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: FB418_P101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No