Amyotrophic Lateral SclerosisPhase 1NCT05882695
Study of SPG302 in Healthy Volunteers and ALS Participants
A Phase 1 study testing SPG302 in people with Amyotrophic Lateral Sclerosis, run by Spinogenix at 4 sites in Australia.
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 Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- SPG302
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 88 participants
- Where
- 4 sitesAustralia
- Study dates
- 3 Jul 2023to 27 Jun 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 47Show fewer
- 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
Trial locations and contacts
4 sites in 1 country.
Australia4 sites
Flinders Medical center
Adelaide, South Australia, 5042
Royal Brisbane and Women's Hospital
Herston, Queensland, 4029
Nucleus Melbourne (healthy volunteers)
Melbourne, Victoria, 3004
Macquarie University
North Ryde, New South Wales, 2109
About this study
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
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
Read the 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
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- SPG302, Placebo
- Keywords
- Amyotrophic Lateral Sclerosis, regenerative, synapse
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SPG302Drug
synthetic small molecule
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: 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). Receives: 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). Receives: 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. Receives: 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. Receives: 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). Receives: Drug: SPG302 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability in healthy volunteers (SAD cohort)
- Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Measured: 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)
Measured: 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)
Measured: 12 days
Safety and tolerability in participants with ALS
- Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Measured: 60 days
Secondary outcomes (6)
Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD cohort)
PK parameters of SPG302 on concentrations in plasma
Measured: 7 days
Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD food effect cohort)
Effects of food on SPG302 PK profile
Measured: 15 days
Plasma pharmacokinetics of SPG302 in healthy volunteers (MAD cohort)
PK parameters of SPG302 on concentrations in plasma
Measured: 12 days
Plasma pharmacokinetics of SPG302 in participants with ALS
PK parameters of SPG302 on concentrations in plasma
Measured: 12mon
Clinical outcomes of multiple oral doses of SPG302 in participants with ALS
Spirometry
Measured: 12 mon
Clinical efficacy measures of SPG302 in participants with ALS
The Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R).
Measured: 12 mon
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2023
- Study start3 Jul 2023
- Primary completion27 Jun 2025
- Study completion27 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- 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)
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 88 participants (actual)
Sponsor and investigators
- Lead sponsor
- Spinogenix
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Novotech (Australia) Pty Limited
- Responsible party
- Sponsor
- Investigators
- Ofer M Gonen, MD, Principal investigator, Nucleus Network (for healthy volunteers)David Schultz (ALS site), MD, Principal investigator, Finders Medical Center (ALS)Robert Henderson (ALS site), MD, Principal investigator, Royal Brisbane Hospital (ALS)Dominic Rowe, MD, Principal investigator, Macquarie Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05882695
- Sponsor's protocol ID
- SPG302-ALS-001
- Status last verified
- Jun 2025
- First submitted
- 22 May 2023
- Last updated
- 29 Jun 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05882695
- Acronym / sponsor ID
- SPG302-ALS-001
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Amyotrophic Lateral Sclerosis, regenerative, synapse
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 88 participants (actual)
- Lead sponsor
- SpinogenixIndustry (pharma, biotech, devices)
- Collaborators
- Novotech (Australia) Pty Limited
- Study start
- 3 Jul 2023 (actual)
- Primary completion
- 27 Jun 2025 (actual)
- Study completion
- 27 Jun 2025 (actual)
- First posted
- 31 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 7
- 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)
| Arm | Type | Receives |
|---|---|---|
| Experimental Part 1: Active SPG302 to be administered to healthy volunteers (SAD) | Experimental | Drug: SPG3028 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 |
| Placebo Comparator Part 1: Placebo comparator to be administered to healthy volunteers (SAD) | Placebo comparator | Drug: Placebo8 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 |
| Experimental Part 2: Active SPG302 to be administered to healthy volunteers (MAD) | Experimental | Drug: SPG3028 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). |
| Placebo Comparator Part 2: Placebo comparator to be administered to healthy volunteers (MAD) | Placebo comparator | Drug: Placebo8 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). |
| Experimental Part 3: Active SPG302 to be administered to participants with ALS | Experimental | Drug: SPG302Participants 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. |
| Placebo Comparator Part 3: Placebo comparator to be administered to participants with ALS | Placebo comparator | Drug: PlaceboParticipants 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. |
| Experimental Part 3: Open Label Extension - Active SPG302 administered to participants with ALS | Experimental | Drug: SPG302Participants 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). |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (47 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SPG302synthetic small molecule | Drug | 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 |
| PlaceboPlacebo | Drug | 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 |
Primary outcome
Safety and tolerability in healthy volunteers (SAD cohort)
Time frame: 7 days
• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Safety and tolerability in healthy volunteers (SAD food effect cohort)
Time frame: 15 days
• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Safety and tolerability in healthy volunteers (MAD cohort)
Time frame: 12 days
• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Safety and tolerability in participants with ALS
Time frame: 60 days
• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
6 secondary outcomes
Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD cohort)
Time frame: 7 days
Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD food effect cohort)
Time frame: 15 days
Plasma pharmacokinetics of SPG302 in healthy volunteers (MAD cohort)
Time frame: 12 days
Plasma pharmacokinetics of SPG302 in participants with ALS
Time frame: 12mon
Clinical outcomes of multiple oral doses of SPG302 in participants with ALS
Time frame: 12 mon
Clinical efficacy measures of SPG302 in participants with ALS
Time frame: 12 mon
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ofer M Gonen, MD principal investigator
Nucleus Network (for healthy volunteers)
David Schultz (ALS site), MD principal investigator
Finders Medical Center (ALS)
Robert Henderson (ALS site), MD principal investigator
Royal Brisbane Hospital (ALS)
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SPG302 in people with Amyotrophic Lateral Sclerosis, run by Spinogenix at 4 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 47 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability in healthy volunteers (SAD cohort) (time frame: 7 days). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jun 2025.