Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy ofmEphA4-Fc

Phase 1

Completed

• Conditions: Neurological - Neurodegenerative diseases; Amyotrophic Lateral Sclerosis; Neurological - Other neurological disorders

• Registration Number: ACTRN12621000514808
• Lead Sponsor: uNerve Pty. Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-05-03
• Study Completion: 2023-05-19
• Last Update Posted: 22 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 28

Inclusion Criteria

Healthy Volunteers:

1. Healthy male or female volunteers aged 18 to 55 years of age (inclusive at the time
of informed consent).
2. Subjects must be in good general health, with no significant medical history, have
no clinically significant abnormalities on physical examination at Screening and/or
before administration of the initial dose of study drug.
3. Subjects must have a minimum body weight of 50 kg and a Body Mass Index
(BMI) between greater than or equal to 18.0 and lesser than or equal to 32.0 kg/m2 at Screening.
4. Subjects must have clinical laboratory values within normal range as specified by
the testing laboratory, unless deemed not clinically significant by the Investigator
or delegate.
5. Subjects who smoke no more than 2 cigarettes or equivalent per week can be
included in the study but must be willing to abstain from smoking during the
confinement period.

ALS Patients;

1. Males and females aged 18 to 70 years of age (inclusive at the time of informed consent).
2. Must have a minimum body weight of 50 kg and a BMI between greater than or equal to 18.0 and less than or equal to 32.0 kg/m2 at Screening.
3. Patients who smoke no more than 2 cigarettes or equivalent per week can be
included in the study but must be willing to abstain from smoking during the
confinement period.
4. Patients with diagnosis of familial or sporadic ALS, defined as meeting the
possible, laboratory-supported probable, probable, or definite diagnosis of ALS
according to modified El Escorial criteria.
5. Onset of muscle weakness within 60 months of study entry.

Exclusion Criteria

Healthy Volunteers:

1. Females of childbearing potential, pregnant (confirmed by serum or urine beta
human chorionic gonadotropin (ßhCG) test at Screening and prior to dosing) or
lactating or planning to become pregnant (self or partner) at any time during the
study, including the follow-up period.
2. Prior or ongoing medical conditions, medical history, physical findings, or
laboratory abnormality that, in the Investigator’s (or delegate’s) opinion, could
adversely affect the safety of the subject.
3. Presence of any underlying physical or psychological medical condition that, in
the opinion of the Investigator, would make it unlikely that the subject will comply
with the protocol or complete the study per protocol.
4. A history of substance abuse or dependency or history of recreational IV drug use
over the last 5 years (by self-declaration).
5. Blood donation or significant blood loss within 60 days prior to the first study
drug administration.

ALS Patients:

1. Patients with other neuromuscular disorders (in addition to their ALS diagnosis),
unless the Investigator determines that such additional disorder will not affect safety or other measures in this study.
2. Patients with frontotemporal dementia (FTD).
3. Patients with evidence of psychiatric illness or impaired cognitive function which,
in the Investigator’s opinion, is likely to prevent them from a full understanding of and/or compliance with the study requirements and procedures.
4. Patients with abnormalities detected during the Screening evaluations which, in the Investigator’s medical judgement, are sufficiently significant to exclude them from participation in the study.
5. Patients with Type I or poorly controlled Type II Diabetes Mellitus (HbA1c greater than 8%).

Study & Design

• Study Type: Interventional
• Study Design: Not specified