Study of SPG302 in Healthy Volunteers and ALS Participants

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

A Phase 1/2a, Randomized, Double Blind, Placebo Controlled, Single and Multiple Dose Escalation Study in Healthy Volunteers and an Expansion Cohort in Adult Participants With Amyotrophic Lateral Sclerosis (ALS) to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of SPG302

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05882695
• Other study ID #: SPG302-ALS-001
• Status: Recruiting
• Phase: Phase 1
• Start date: July 3, 2023
• Est. completion date: December 2025

Study information

• Verified date: June 2024
• Source: Spinogenix
• Contact: Public queries for healthy volunteers
• Phone: +61 1800 243 733
• Email: melbourne[@]nucleusnetwork.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The first-in-human Phase 1 study described herein will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of SPG302 in healthy volunteers and ALS participants

Description:

This study is a Phase 1 randomized, double-blind, placebo-controlled, single, and multiple ascending dose study in HV with food effect cohort, and a repeat dose expansion cohort(s) in participants with ALS.

The study consists of 3 parts, as follows:

• Part 1: SAD in HV with up to 6 cohorts including a food effect cohort.

• Part 2: MAD over 5 days in HV with up to 5 cohorts

• Part 3: ALS cohorts with once daily (QD) dosing over 28 day cycles

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 112
• Est. completion date: December 2025
• Est. primary completion date: May 2025
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

Inclusion Criteria:

• Age 18-55

• Must be in good health with no significant medical history

• Clinical laboratory values within normal range or < 1.2 times ULN

• BMI 18-32 (inclusive)

• Contraceptive use by men or women consistent with local regulations

• Able and willing to provide written informed consent

Exclusion Criteria:

• Any physical or psychological condition that prohibits study completion

• Known cardiac disease

• Active or history of malignancy in the past 5 years

• Serious infection within 1 month of screening

• Acute illness within 30 days of Day 1

• Surgery, bone fracture, or major musculoskeletal injury in the past 3 months

• History of suicidal behavior or suicidal ideation

• Active cigarette smokers and users of nicotine-containing products

• HIV, hepatitis B and hepatitis C positive

• SBP >140 or <90

• DBP >90 or <40

• HR <40 or >100

• QTcF >450ms, cardiac arrhythmia, or clinically significant abnormal ECG

• Prescriptions, over-the-counter, or herbal medication within 7 days

• Vaccines within 14 days

• Other investigational products within 30 days

• Blood donation within 30 days

• Plasma donation within 7 days

• Pregnant or breastfeeding

• Otherwise unfit, on metabolic-altering lifestyle/diet, positive urine drug screen or intake of alcohol or caffeine-containing products

ALS Cohort Inclusion Criteria:

• Age 18-80

• ALS TRICALS risk score

• Stable dose of standard of care treatment

• Contraception use by men or women consistent with local regulations

• Able and willing to provide written informed consent

ALS Cohort Exclusion Criteria:

• Underlying physical or psychological condition prohibiting study completion

• Known cardiac disease

• Active or history of malignancy in the past 5 years

• Serious infection within 1 month of screening

• Acute illness within 30 days of Day 1

• History of suicidal behavior or suicidal ideation

• Active cigarette smokers and users of nicotine-containing products

• Neurodegenerative disease

• External respiratory support or supplemental oxygen requirement

• HIV, hepatitis B and hepatitis C positive

• SBP >140 or <90

• DBP >90 or <40

• HR <40 or >100

• QTcF >450ms, cardiac arrhythmia, or clinically significant abnormal ECG

• Vaccines within 14 days

• Other investigational products within 30 days

• Blood donation within 30 days

• Plasma donation within 7 days

• Pregnant or breastfeeding

• Otherwise unfit

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• SPG302
synthetic small molecule
• Placebo
Placebo

Locations

Country	Name	City	State
Australia	Flinders Medical center	Adelaide	South Australia
Australia	Royal Brisbane and Women's Hospital	Herston	Queensland
Australia	Nucleus Melbourne (healthy volunteers)	Melbourne	Victoria
Australia	Macquarie University	North Ryde	New South Wales
United States	Mass General Brigham	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Spinogenix	Novotech (Australia) Pty Limited

Countries where clinical trial is conducted

United States,  Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Effect of repeated dosing of SPG302 on electroencephalogram in healthy volunteers (MAD cohort)	Change from baseline in EEG parameters	12 mon
Other	Clinical outcomes of multiple oral doses of SPG302 in participants with ALS	Number of respiratory complications	12 mon
Other	Clinical outcomes of multiple oral doses of SPG302 in participants with ALS	Spirometry	12 mon
Other	Clinical outcomes of multiple oral doses of SPG302 in participants with ALS	TMS	12 mon
Other	Clinical outcomes of multiple oral doses of SPG302 in participants with ALS	EEG	12 mon
Other	Clinical outcomes of multiple oral doses of SPG302 in participants with ALS	Change in Nocturnal Pulse Oximetry	12 mon
Other	Clinical outcomes of multiple oral doses of SPG302 in participants with ALS	Edinburgh Cognitive and Behavioural ALS Screen (ECAS)	12mon
Other	Effect of SPG302 on proteins and biomarkers in participants with ALS	Multiple protein and immunological biomarkers	12mon
Other	The effect of SPG302 on protein(s) and biomarkers	Change from baseline in the analysis of Columbia-Suicide Severity Rating Scale (C-SSRS)	12mon
Primary	Safety and tolerability in healthy volunteers (SAD cohort)	• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)	7 days
Primary	Safety and tolerability in healthy volunteers (SAD food effect cohort)	• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)	15 days
Primary	Safety and tolerability in healthy volunteers (MAD cohort)	• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)	12 days
Primary	Safety and tolerability in participants with ALS	• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)	60 days
Secondary	Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD cohort)	PK parameters of SPG302 on concentrations in plasma	7 days
Secondary	Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD food effect cohort)	Effects of food on SPG302 PK profile	15 days
Secondary	Plasma pharmacokinetics of SPG302 in healthy volunteers (MAD cohort)	PK parameters of SPG302 on concentrations in plasma	12 days
Secondary	Plasma pharmacokinetics of SPG302 in participants with ALS	PK parameters of SPG302 on concentrations in plasma	12mon
Secondary	Clinical outcomes of multiple oral doses of SPG302 in participants with ALS	Spirometry	12 mon
Secondary	Clinical efficacy measures of SPG302 in participants with ALS	The Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R).	12 mon