LH-001 vs Placebo in Healthy Participants

NCT06546449 | Suspended Phase 1 DMC FDA Drug

• 2 other identifiers: 2024W00611U01AG068822-01
• Study Type: interventional
• Target: 56
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this clinical trial is to evaluate the safety and tolerability of LH-001 when administered as an oral, single or multiple dose(s) at ascending dose levels in healthy participants.

Conditions

Healthy

Keywords

LH-001

Outcome Measures

Primary Outcomes (5)

• Percentage of participants who experience at least one treatment-emergent AE

  8 days for SAD and 21 days for MAD

• Percentage of participants who discontinue due to an AE

  8 days for SAD and 21 days for MAD

• Percentage of participants who meet the markedly abnormal criteria for vital sign measurements at least once post-dose

  8 days for SAD and 21 days for MAD

• Percentage of participants who meet the markedly abnormal criteria for safety ECG parameters at least once post-dose

  8 days for SAD and 21 days for MAD

• Percentage of participants who meet the markedly abnormal criteria for safety laboratory tests at least once post-dose

  8 days for SAD and 21 days for MAD

Secondary Outcomes (4)

• Maximum concentration (Cmax)

  Day 1 for all cohorts and Day 14 for MAD cohorts

• Time to reach maximum concentration (Tmax)

  Day 1 for all cohorts and Day 14 for MAD cohorts

• Area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration (AUC0-last)

  Day 1 for all cohorts and Day 14 for MAD cohorts

• Time for LH-001 concentration to fall to half of its original value (T½)

  Day 1 for all cohorts and Day 14 for MAD cohorts

Study Arms (9)

SAD Cohort 1

EXPERIMENTAL

Participants will be administered a single 12.5 mg dose of LH-001

Drug: LH-001

SAD Cohort 2

EXPERIMENTAL

Participants will be administered a single 25 mg dose of LH-001

Drug: LH-001

SAD Cohort 3

EXPERIMENTAL

Participants will be administered a single 50 mg dose of LH-001

Drug: LH-001

SAD Cohort 4

EXPERIMENTAL

Participants will be administered a single 100 mg dose of LH-001

Drug: LH-001

MAD Cohort 1

EXPERIMENTAL

Participants will be administered multiple 25 mg doses of LH-001

Drug: LH-001

MAD Cohort 2

EXPERIMENTAL

Participants will be administered multiple 50 mg doses of LH-001

Drug: LH-001

MAD Cohort 3

EXPERIMENTAL

Participants will be administered multiple 100 mg doses of LH-001

Drug: LH-001

SAD Placebo cohorts 1, 2, 3 and 4

PLACEBO COMPARATOR

Participants will be administered a single dose of placebo

Drug: Placebo

MAD Placebo cohorts 1, 2 and 3

PLACEBO COMPARATOR

Participants will be administered multiple doses of placebo

Drug: Placebo

Interventions

LH-001 DRUG

LH-001 will be administered oral

MAD Cohort 1; MAD Cohort 2; MAD Cohort 3; SAD Cohort 1; SAD Cohort 2; SAD Cohort 3; SAD Cohort 4

Placebo DRUG

Placebo will be administered oral

MAD Placebo cohorts 1, 2 and 3; SAD Placebo cohorts 1, 2, 3 and 4

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Healthy males or females aged 18-60 years at the time of consent
• Must provide written informed consent
• Physically and mentally able and willing to participate in the safety and other assessments including staying overnight
• BMI 18-29.9 kg/m2
• Sexually active male participants, sexually active female participants of childbearing potential, and their sexual partners are to adhere to the contraception requirements. These requirements include utilizing highly effective birth control (including hormonal methods, intrauterine devices, and/or barrier methods) from the screening phase through the completion of the last study follow-up. Note, the barrier method may not be used as a standalone method and must be combined with an additional approved method.
• Participants taking non-prescribed medication must cease taking the medication for at least 48 hours prior to dosing of LH-001.

You may not qualify if:

• Taking any prescription medications outlined in the prohibited/conditional drug list (see Section 6.8.1)
• History or presence of gastrointestinal, renal, or hepatic disease or any other condition known to interfere with absorption, distribution, metabolism, or excretion of drugs
• History or presence of major disorder of any other major organ system (cardiovascular, respiratory, central nervous system, or endocrine system)
• History of cancer within 5 years of consent (exceptions are squamous and basal cell carcinomas of the skin)
• Any clinically important illness, medical/surgical procedure, or trauma within 4 weeks prior to dosing.
• History or presence of alcohol or substance abuse
• History of chronic or current use of recreational or illicit drugs
• History of, or treatment for, major psychiatric illness
• History of, or treatment for, seizures or epilepsy
• Pregnant or breast-feeding females
• History of, or treatment for, an autoimmune disease (e.g., Rheumatoid Arthritis, Multiple Sclerosis, Myasthenia Gravis, etc.)
• History of asplenia, hyposplenia, or splenectomy
• History or presence of drug hypersensitivity
• Poor venous access
• Receipt of investigational therapy within 4 months prior to screening

Sponsors & Collaborators

• Chien-Liang Lin: lead
• National Institute on Aging (NIA): collaborator

Study Sites (1)

The Ohio State University

Columbus, Ohio, 43221, United States

Location

Study Officials

• Chien-Liang Glenn Lin, PhD

  Ohio State University

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The investigators and participants will remain blinded throughout the study. At the completion of each cohort, the statistician will be unblinded to analyze data.
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Neuroscience

Study Record Dates

• First Submitted: August 6, 2024
• First Posted: August 9, 2024
• Study Start: May 5, 2025
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: April 21, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)