SPG302 for ALS

Recruiting at 5 trial locations

Overseen By Public queries for healthy volunteers

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Spinogenix

Must be taking: Standard care

Must not be taking: Prescriptions, over-the-counter

Disqualifiers: Cardiac disease, Malignancy, Infection, others

Compensation: Varies

Number of Visits: 1

112 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

Yes, participants must stop taking any prescriptions, over-the-counter, or herbal medications at least 7 days before the trial starts.

How does the drug SPG302 differ from other treatments for ALS?

The research does not provide specific information about SPG302, so it's unclear how it differs from other ALS treatments. ^{1 2 3 4 5}

What is the purpose of this trial?

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

Research Team

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

Principal Investigator

Macquarie Hospital

Eligibility Criteria

This trial is for healthy adults aged 18-55 with no significant medical history, normal lab values, and a BMI between 18-32. Participants must use contraception and agree to the study's terms. It also includes those diagnosed with ALS.

Inclusion Criteria

BMI 18-32 (inclusive)

Clinical laboratory values within normal range or < 1.2 times ULN

Able and willing to provide written informed consent

Exclusion Criteria

HIV, hepatitis B and hepatitis C positive

Other investigational products within 30 days

Pregnant or breastfeeding

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Single Ascending Dose (SAD)

Single dose administration in healthy volunteers with a food effect cohort

7-15 days

Multiple visits for dosing and safety evaluation

Multiple Ascending Dose (MAD)

Multiple dose administration over 5 days in healthy volunteers

12 days

Daily visits for dosing and monitoring

ALS Treatment

Once daily dosing over 28-day cycles in participants with ALS

28 days per cycle

Daily visits for dosing and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

30 days

1 follow-up safety visit

Open-label Extension

Participants may opt into continuation of treatment long-term

Up to 12 cycles

Treatment Details

Interventions

• SPG302

Trial Overview The study tests SPG302 against a placebo to assess its safety and effects in the body (pharmacokinetics) and how it influences disease processes (pharmacodynamics) in both healthy individuals and ALS patients.

Participant Groups

Group I: Experimental Part 3: Open Label Extension - Active SPG302 administered to participants with ALS Experimental Treatment 1 Intervention

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).

Group II: Experimental Part 3: Active SPG302 to be administered to participants with ALS Experimental Treatment 1 Intervention

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.

Group III: Experimental Part 2: Active SPG302 to be administered to healthy volunteers (MAD) Experimental Treatment 1 Intervention

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).

Group IV: Experimental Part 1: Active SPG302 to be administered to healthy volunteers (SAD) Experimental Treatment 1 Intervention

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

Group V: Placebo Comparator Part 2: Placebo comparator to be administered to healthy volunteers (MAD) Placebo Group 1 Intervention

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).

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

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

Group VII: Placebo Comparator Part 3: Placebo comparator to be administered to participants with ALS Placebo Group 1 Intervention

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.

Who Is Running the Clinical Trial?

Spinogenix

Lead Sponsor

Trials

6

Recruited

220+

Novotech (Australia) Pty Limited

Industry Sponsor

Trials

76

Recruited

7,800+

Dr. John Moller

Novotech (Australia) Pty Limited

Chief Executive Officer

MD and MBA from the University of Oxford

Dr. Judith Ng-Cashin

Novotech (Australia) Pty Limited

Chief Medical Officer since 2023

MD

Findings from Research

Genetic research has identified numerous mutations linked to amyotrophic lateral sclerosis (ALS), which can help explain the disease's varied clinical presentations and improve clinical trial outcomes.

Advanced genetic sequencing techniques are paving the way for better patient stratification in clinical trials, allowing for more personalized treatment approaches and improved understanding of ALS's underlying mechanisms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Genetic heterogeneity of amyotrophic lateral sclerosis: implications for clinical practice and research. Su, XW., Broach, JR., Connor, JR., et al.[2015]

References

1. United States pubmed.ncbi.nlm.nih.gov

Whole-exome sequencing identifies a missense mutation in hnRNPA1 in a family with flail arm ALS. [2019]

2. England pubmed.ncbi.nlm.nih.gov

Heterozygous S44L missense change of the spastin gene in amyotrophic lateral sclerosis. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Early-onset ALS with long-term survival associated with spastin gene mutation. [2019]

4. England pubmed.ncbi.nlm.nih.gov

The first ALS2 missense mutation associated with JPLS reveals new aspects of alsin biological function. [2021]

5. United States pubmed.ncbi.nlm.nih.gov

Genetic heterogeneity of amyotrophic lateral sclerosis: implications for clinical practice and research. [2015]