Study of SPG302 in Healthy Volunteers and ALS Participants

NCT05882695 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: SPG302-ALS-001
• Study Type: interventional
• Target: 88
• Locations: 1 country
• Sites: 4

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

Conditions

Amyotrophic Lateral Sclerosis

Keywords

Amyotrophic Lateral Sclerosis; regenerative; synapse

Outcome Measures

Primary Outcomes (4)

• 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

• 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

• 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

• 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 Outcomes (6)

• Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD cohort)

  7 days

• Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD food effect cohort)

  15 days

• Plasma pharmacokinetics of SPG302 in healthy volunteers (MAD cohort)

  12 days

• Plasma pharmacokinetics of SPG302 in participants with ALS

  12mon

• Clinical outcomes of multiple oral doses of SPG302 in participants with ALS

  12 mon

Other Outcomes (9)

• Effect of repeated dosing of SPG302 on electroencephalogram in healthy volunteers (MAD cohort)

  12 mon

• Clinical outcomes of multiple oral doses of SPG302 in participants with ALS

  12 mon

• Clinical outcomes of multiple oral doses of SPG302 in participants with ALS

  12 mon

Study Arms (7)

Experimental Part 1: Active SPG302 to be administered to healthy volunteers (SAD)

EXPERIMENTAL

8 participants will be randomized in a 3:1 ratio to active or placebo. Study intervention will be administered orally once. Randomization to each SAD cohort will be done in a staggered manner; initially 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed and after a safety evaluation period after the dose without clinically significant adverse events (AEs) and investigator approval, then, 6 additional participants will be randomized and dosed (5 active and 1 placebo) at the discretion of the Investigator according to the randomization schedule

Drug: SPG302

Placebo Comparator Part 1: Placebo comparator to be administered to healthy volunteers (SAD)

PLACEBO COMPARATOR

8 participants will be randomized in a 3:1 ratio to active or placebo. Study intervention will be administered orally once. Randomization to each SAD cohort will be done in a staggered manner; initially 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed and after a safety evaluation period after the dose without clinically significant adverse events (AEs) and investigator approval, then, 6 additional participants will be randomized and dosed (5 active and 1 placebo) at the discretion of the Investigator according to the randomization schedule

Drug: Placebo

Experimental Part 2: Active SPG302 to be administered to healthy volunteers (MAD)

EXPERIMENTAL

8 participants will be randomized in a 3:1 ratio to active or placebo. Participants will receive study intervention QD over 5 days and will be discharged on Day 6. A follow-up safety visit will be conducted on Day 12 (±3 days).

Drug: SPG302

Placebo Comparator Part 2: Placebo comparator to be administered to healthy volunteers (MAD)

PLACEBO COMPARATOR

8 participants will be randomized in a 3:1 ratio to active or placebo. Participants will receive study intervention QD over 5 days and will be discharged on Day 6. A follow-up safety visit will be conducted on Day 12 (±3 days).

Drug: Placebo

Experimental Part 3: Active SPG302 to be administered to participants with ALS

EXPERIMENTAL

Participants with ALS will be randomized to receive SPG302 or placebo at a 3:1 ratio. Study intervention will be administered QD over 28 days. A follow-up safety visit will be conducted 30 days after last dose (±7 days). Participants who complete Part 3 may be offered to participate in an open-label extension.

Drug: SPG302

Placebo Comparator Part 3: Placebo comparator to be administered to participants with ALS

PLACEBO COMPARATOR

Participants with ALS will be randomized to receive SPG302 or placebo at a 3:1 ratio. Study intervention will be administered QD over 28 days. A follow-up safety visit will be conducted 30 days after last dose (±7 days). Participants who complete Part 3 may be offered to participate in an open-label extension.

Drug: Placebo

Experimental Part 3: Open Label Extension - Active SPG302 administered to participants with ALS

EXPERIMENTAL

Participants with ALS will be randomized to receive SPG302 or placebo at a 3:1 ratio. Study intervention will be administered QD over 28 days for up to 3 cycles in the USA and up to 12 cycles in Australia. A follow-up safety visit will be conducted 30 days after last dose (±7 days).

Drug: SPG302

Interventions

SPG302 DRUG

synthetic small molecule

Experimental Part 1: Active SPG302 to be administered to healthy volunteers (SAD); Experimental Part 2: Active SPG302 to be administered to healthy volunteers (MAD); Experimental Part 3: Active SPG302 to be administered to participants with ALS; Experimental Part 3: Open Label Extension - Active SPG302 administered to participants with ALS

Placebo DRUG

Placebo

Placebo Comparator Part 1: Placebo comparator to be administered to healthy volunteers (SAD); Placebo Comparator Part 2: Placebo comparator to be administered to healthy volunteers (MAD); Placebo Comparator Part 3: Placebo comparator to be administered to participants with ALS

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Spinogenix: lead
• Novotech (Australia) Pty Limited: collaborator

Study Sites (4)

Macquarie University

North Ryde, New South Wales, 2109, Australia

Location

Royal Brisbane and Women's Hospital

Herston, Queensland, 4029, Australia

Location

Flinders Medical center

Adelaide, South Australia, 5042, Australia

Location

Nucleus Melbourne (healthy volunteers)

Melbourne, Victoria, 3004, Australia

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Ofer M Gonen, MD

  Nucleus Network (for healthy volunteers)

  PRINCIPAL INVESTIGATOR

• David Schultz (ALS site), MD

  Finders Medical Center (ALS)

  PRINCIPAL INVESTIGATOR

• Robert Henderson (ALS site), MD

  Royal Brisbane Hospital (ALS)

  PRINCIPAL INVESTIGATOR

• Dominic Rowe, MD

  Macquarie Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double blinded
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Phase 1 randomized, double-blind, placebo-controlled, single, and multiple ascending dose study in HV and a repeat dose expansion in ALS cohort(s)

Study Record Dates

• First Submitted: May 22, 2023
• First Posted: May 31, 2023
• Study Start: July 3, 2023
• Primary Completion: June 27, 2025
• Study Completion: June 27, 2025
• Last Updated: June 29, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

AU (4)