Clinical trial NCT05882695: Study of SPG302 in Healthy Volunteers and ALS Participants
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Amyotrophic Lateral Sclerosis
regenerative
synapse
Lead sponsor: Spinogenix
Sponsor class: INDUSTRY
Collaborators: name: Novotech (Australia) Pty Limited
First posted: 2023-05-31
Enrollment: 112
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: Phase 1 randomized, double-blind, placebo-controlled, single, and multiple ascending dose study in HV and a repeat dose expansion in ALS cohort(s)
Primary purpose: TREATMENT

Brief 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

Detailed 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

Eligibility criteria: 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

Primary outcomes:
measure: Safety and tolerability in healthy volunteers (SAD cohort)
timeFrame: 7 days
measure: Safety and tolerability in healthy volunteers (SAD food effect cohort)
timeFrame: 15 days
measure: Safety and tolerability in healthy volunteers (MAD cohort)
timeFrame: 12 days
measure: Safety and tolerability in participants with ALS
timeFrame: 60 days
Secondary outcomes:
measure: Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD cohort)
timeFrame: 7 days
measure: Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD food effect cohort)
timeFrame: 15 days
measure: Plasma pharmacokinetics of SPG302 in healthy volunteers (MAD cohort)
timeFrame: 12 days
measure: Plasma pharmacokinetics of SPG302 in participants with ALS
timeFrame: 12mon
measure: Clinical outcomes of multiple oral doses of SPG302 in participants with ALS
timeFrame: 12 mon
measure: Clinical efficacy measures of SPG302 in participants with ALS
timeFrame: 12 mon
Other outcomes:
measure: Effect of repeated dosing of SPG302 on electroencephalogram in healthy volunteers (MAD cohort)
timeFrame: 12 mon
measure: Clinical outcomes of multiple oral doses of SPG302 in participants with ALS
timeFrame: 12 mon
measure: Clinical outcomes of multiple oral doses of SPG302 in participants with ALS
timeFrame: 12 mon
measure: Clinical outcomes of multiple oral doses of SPG302 in participants with ALS
timeFrame: 12 mon
measure: Clinical outcomes of multiple oral doses of SPG302 in participants with ALS
timeFrame: 12 mon
measure: Clinical outcomes of multiple oral doses of SPG302 in participants with ALS
timeFrame: 12 mon
measure: Clinical outcomes of multiple oral doses of SPG302 in participants with ALS
timeFrame: 12mon
measure: Effect of SPG302 on proteins and biomarkers in participants with ALS
timeFrame: 12mon
measure: The effect of SPG302 on protein(s) and biomarkers
timeFrame: 12mon
Locations:
city: North Ryde
country: Australia
facility: Macquarie University
state: New South Wales
city: Herston
country: Australia
facility: Royal Brisbane and Women's Hospital
state: Queensland
city: Adelaide
country: Australia
facility: Flinders Medical center
state: South Australia
city: Melbourne
country: Australia
facility: Nucleus Melbourne (healthy volunteers)
state: Victoria
Study officials:
affiliation: Nucleus Network (for healthy volunteers)
name: Ofer M Gonen, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Finders Medical Center (ALS)
name: David Schultz (ALS site), MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Royal Brisbane Hospital (ALS)
name: Robert Henderson (ALS site), MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Macquarie Hospital
name: Dominic Rowe, MD
role: PRINCIPAL_INVESTIGATOR
References: