SPG302 for ALS

No longer 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a new treatment, SPG302, to determine its safety and tolerability in people with ALS (a disease affecting nerve cells in the brain and spinal cord) and healthy volunteers. The study will examine how the body processes the drug and its effects. Individuals with ALS who are on a stable treatment and can manage daily activities without extra breathing support might be suitable candidates. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

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.

Is there any evidence suggesting that SPG302 is likely to be safe for humans?

Research has shown that SPG302 has potential based on earlier studies. In one study with Alzheimer's patients, SPG302 was well-tolerated and safe. Another report from a program for ALS patients noted that the FDA allowed its use, indicating some trust in its safety for ALS as well.

Although the current trial is in its early stages, these findings offer some reassurance about its safety. However, this is the first time SPG302 is being tested in humans for ALS, and all new treatments carry some risks. Participants should discuss these risks with the trial team to make an informed choice. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for ALS?

SPG302 is unique because it offers a potential new approach for treating ALS, a condition with limited options like riluzole and edaravone. Unlike these existing treatments, SPG302 is designed to be administered daily over a short period and may provide quicker outcomes. Researchers are excited about SPG302 because it targets the disease through a potentially novel mechanism, which could lead to improved symptom management and quality of life for ALS patients.

What evidence suggests that SPG302 might be an effective treatment for ALS?

Research has shown that SPG302 may help treat ALS (Amyotrophic Lateral Sclerosis). In an earlier study, 82% of patients who took SPG302 had symptoms that either remained stable or improved, according to the ALS Functional Rating Scale. This finding is promising because it suggests SPG302 might slow or improve the disease's progression. In this trial, participants with ALS will be randomized to receive either SPG302 or a placebo. SPG302 may work by addressing a major issue in ALS, which is the loss of certain nerve connections. While more research is needed, these early results offer hope for its effectiveness in treating ALS. ^{1 2 4 6 7}

Who Is on the 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

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

Citations

1. prnewswire.com prnewswire.com/il/news-releases/spinogenix-presents-topline-phase-2a-clinical-trial-results-for-spg302-a-first-in-class-als-treatment-302603975.html

Spinogenix Presents Topline Phase 2a Clinical Trial ... 82% of patients treated with SPG302 exhibited a stable or improved rate of decline at end of treatment, as assessed with the ALS Functional ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05882695

Study of SPG302 in Healthy Volunteers and ALS Participants This study is a Phase 1 randomized, double-blind, placebo-controlled, single, and multiple ascending dose study in HV with food effect cohort, ...

3. spinogenix.com spinogenix.com/spinogenix-announces-positive-first-cohort-results-from-phase-2a-trial-evaluating-spg302-for-alzheimers-disease/

Spinogenix Announces Positive First Cohort Results from ... SPG302, a Potential First-in-Class Synaptic Regenerative Therapy, Showed Progress in Cognitive Outcome Measures and a Favorable Safety ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05882695?term=spg302&cond=als&rank=2

Study of SPG302 in Healthy Volunteers and ALS Participants Clinical efficacy measures of SPG302 in participants with ALS, The Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R). 12 mon. Other ...

5. spinogenix.com spinogenix.com/spinogenixs-spg302-the-first-synaptic-regenerative-therapy-to-treat-als-granted-orphan-drug-designation-by-the-european-medicines-agency/

Spinogenix's SPG302, the First Synaptic Regenerative ... “SPG302 has the potential to reverse an unaddressed, yet critical, aspect of ALS pathogenesis: an early and progressive loss of glutamatergic ...

6. spinogenix.com spinogenix.com/spinogenix-announces-fda-authorized-expanded-access-programfor-spg302-the-first-synaptic-regenerative-therapy-to-treat-als/

FDA Authorizes Expanded Access Program (EAP ... FDA Authorizes Expanded Access Program (EAP) to Help Provide Access to SPG302 for 200 ALS Individuals Ineligible for Clinical Study.

7. ctv.veeva.com ctv.veeva.com/study/extension-study-of-participants-from-spg302-als101

Extension Study of Participants From SPG302-ALS-001 This study will evaluate the long-term safety and efficacy of participants enrolled in SPG302-ALS-101 with Amyotrophic Lateral Sclerosis (ALS).