Phase1 First in Human Ascending Dose Study to Evaluate the Safety and Tolerability of FC-12738 in Health Adults

Phase 1

Completed

• Conditions: Safety Issues; Tolerance
• Interventions: Drug: FC-12738; Drug: Saline

• Registration Number: NCT05978908
• Lead Sponsor: Neurodegenerative Disease Research Inc

Brief Summary

A Phase I, Randomized, Double-Blind, Placebo-Controlled, First-in-human, Single-Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of retro enversio (RT) thymopentin in Healthy Adult Participants and Patients with Amyotrophic Lateral Sclerosis (ALS)

Detailed Description

This is a first-in-human, randomized, double-blind, placebo-controlled, single-ascending-dose (SAD) study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of retro enversio thymopentin (FC-12738) in healthy adult participants and patients with ALS. The clinical trial will be conducted in two parts, initially a SAD study in 3-sequential cohorts of healthy adult participants (PartA), and then follows another SAD study in 4-6-sequential cohorts of patients with ALS (Part B). Dose escalation in the SAD cohorts will be determined after having reviewed of the interim safety data as well as all available PK and/or PD data from the preceding dose level(s).

Study Timeline

• Study First Submitted: 2023-07-31
• Study First Submitted QC: 2023-07-31
• Study First Posted: 2023-08-07
• Study Start: 2024-02-20
• Primary Completion: 2024-06-03
• Study Completion: 2024-06-28
• Status Verified: 2025-02
• Last Update Submitted: 2025-05-21
• Last Update Posted: 2025-05-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
SAD study in Healthy adult participants	FC-12738	A total of 24 healthy adult participants will be enrolled, and then sequentially allocate to 3 planned dose cohorts (A1-A3): 4 mg, 8 mg and 16 mg, respectively.
Placebo	Saline	Placebo

Primary Outcome Measures

Name	Time	Method
Characterize the safety/tolerability of single-ascending-dose study of FC-12738 in healthy adult participants	0.5 hour, 1 hour, 2 hour, 4 hour, 8 hour, 24 hour, 48 hour, post-dose and follow-up visit on Day 8.	AEs (Adverse Events), AEs of special interest (AESI), treatment-emergent AE (TEAE) and serious adverse events(SAE);Serious Adverse Event(s) AE's will be identified from the time of signed ICF (consent form), throughout the entire study until completion of the follow-up visit.
Heart rate measure	at screening, Day-1, pre-dose and 0.5 hour, 1 hour, 2 hour, 4 hour, 8 hour, 24 hour, 48 hour, post-dose and follow-up visit on Day 8.	Heart rate will be measured by counting beats per minute
Measure blood pressure with a blood pressue cuff	at screening, Day-1, pre-dose and 0.5 hour, 1 hour, 2 hour, 4 hour, 8 hour, 24 hour, 48 hour, post-dose and follow-up visit on Day 8.	blood pressure (BP) systolic and diastolic will be measured in millimeters of mercury (mmHg)
Physical exam signs by counting respiratory rate	at screening, Day-1, pre-dose and 0.5 hour, 1 hour, 2 hour, 4 hour, 8 hour, 24 hour, 48 hour, post-dose and follow-up visit on Day 8.	respiratory rate (RR) will be measured in breaths per minute
Physical exam by temperature taken by a thermometer	at screening, Day-1, pre-dose and 0.5 hour, 1 hour, 2 hour, 4 hour, 8 hour, 24 hour, 48 hour, post-dose and follow-up visit on Day 8.	The temperature will be measured in degrees centegrade or Fahrenheit 12-lead ECGs parameters; Laboratory data (including fasting serum biochemistry, hematology, coagulation, and urinalysis); Assessment of injection site reactions; and Neurological examination；
Physical exam by 12 lead electrocardiogram	at screening, Day-1, pre-dose and 0.5 hour, 1 hour, 2 hour, 4 hour, 8 hour, 24 hour, 48 hour, post-dose and follow-up visit on Day 8.	12-lead ECGs parameters will measure amplitude of the electrical wave in milimeters
Laboratory data will be measured by blood analysis	at screening, Day-1, pre-dose and 0.5 hour, 1 hour, 2 hour, 4 hour, 8 hour, 24 hour, 48 hour, post-dose and follow-up visit on Day 8.	Levels of red blood cells will be measured by in number per microliter and white blood cells will be provided as a percent of total white count measured in number per microlliter.
Laboratory data will be measured by serum biochemistry and urinalysis	at screening, Day-1, pre-dose and 0.5 hour, 1 hour, 2 hour, 4 hour, 8 hour, 24 hour, 48 hour, post-dose and follow-up visit on Day 8.	Serum biochemistry values and urine components will be measured in miligrams per deciliter
Injection site reactions in normal participants	at screening, Day-1, pre-dose and 0.5 hour, 1 hour, 2 hour, 4 hour, 8 hour, 24 hour, 48 hour, post-dose and follow-up visit on Day 8.	Assessment of injection site reactions will be by observation (color) and palpation for soreness or thickening.

Secondary Outcome Measures

Name	Time	Method
Determine the pharmacokinetic (PK) and pharmacodynamic (PD) profiles of single-ascending-dose study of FC-12738 in healthy adult participants	48 hours	Plasma PK parameters for each dose level will be calculated from the concentration of study drug measured in predose and postdose plasma samples. The PD parameters for each dose level will be calculated over time in predose and postdose plasma samples. The following parameters will be assessed during the trial: The area under the plasma concentration-time curve from time 0 extrapolated to infinity and from time 0 to the last measurable non-zero concentration. The area under the plasma concentration-time curve from time zero to 24 hours, the maximum observed concentration, the time to reach maximal value (Tmax). If the maximum value occurs at more than one time point, Tmax is defined as the first time point with this value. Apparent terminal elimination half-life of study drug, and apparent total plasma clearance.; Urine PK parameters Cumulative urinary excretion from time zero to time t, calculated as the sum of the amounts excreted over each time.
To determine the pharmacokinetic (PK) profiles in urine of single-ascending-dose study of FC-12738 in healthy adult participants	48 hours	Urine PK parameters Cumulative urinary excretion from time zero to time t, calculated as the sum of the amounts of drug excreted over each time in micrograms per deciliter.

Trial Locations

Locations (1)

Hassman Research Inc; 🇺🇸 Berlin, New Jersey, United States