LCTOPC1 Delivery Device for Spinal Cord Injury

(DOSED Trial)

Overseen By Reference Study ID Number: LCTOPC1-SCI-03 https://lineagecell.com/products-pipeline/opc1/

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Lineage Cell Therapeutics, Inc.

Disqualifiers: Penetrating trauma, Multiple lesions, Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

10 Participants Needed

Trial Summary

What is the purpose of this trial?

The DOSED clinical study evaluates the safety and utility of a novel delivery device to deliver LCTOPC1, a cell therapy, to the spinal cord of patients with a spinal cord injury (SCI). LCTOPC1 is designed to replace or support cells that are absent or dysfunctional due to traumatic injury, with a goal to help improve the quality of life and restore or augment functional activity in persons suffering from a traumatic cervical or thoracic injuries.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the treatment LCTOPC1 for spinal cord injury?

The research on spinal cord stimulation and cell-based therapies shows promising results in animal models, suggesting potential benefits for spinal cord injury recovery. For example, studies have shown that cell delivery systems and spinal cord stimulators can promote functional recovery and pain suppression in animal models, which may indicate similar potential for treatments like LCTOPC1. ^{1 2 3 4 5}

Is the LCTOPC1 treatment generally safe for humans?

LCTOPC1, also known as GRNOPC1 and AST-OPC1, has been tested for safety in humans with spinal cord injuries. Studies have shown that it is generally safe, with no serious adverse effects, toxicities, or tumors reported in both human trials and preclinical animal studies. ^{6 7 8 9 10}

How does the LCTOPC1 treatment for spinal cord injury differ from other treatments?

LCTOPC1 is unique because it involves a specialized delivery device for spinal cord injury, which may offer a novel approach compared to traditional methods like cell transplants or electrical stimulation. This treatment could potentially provide more precise and effective delivery of therapeutic agents directly to the injury site. ^{1 2 3 5 11}

Eligibility Criteria

This trial is for adults aged 18-65 with traumatic spinal cord injuries (SCI) at certain levels of the spine, occurring from 21 days to 5 years prior. Participants must have some arm muscle strength and be able to undergo surgery. Women who can get pregnant and men must agree to use contraception post-treatment.

Inclusion Criteria

Informed consent for this protocol must be provided and documented (i.e., signed ICF)

Single injury with sufficient visualization of the spinal cord injury epicenter and lesion margins to enable post-injection safety monitoring

I agree to use birth control for 1 year after getting the LCTOPC1 injection.

Exclusion Criteria

My spinal cord injury was caused by a penetrating injury.

I have had a spinal injury or surgery that didn't properly relieve pressure on my spinal cord.

I do not have any organ damage or diseases that make surgery or immunosuppression too risky.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a one-time injection of LCTOPC1 using a novel delivery device

1 day

1 visit (in-person)

Initial Follow-up

Participants are monitored for adverse events related to the delivery device or injection procedure

4 weeks

Multiple visits (in-person)

Extended Follow-up

Participants are monitored for adverse events related to LCTOPC1 and/or concomitant immunosuppression

3 months

Regular visits (in-person)

Long-term Follow-up

Participants are monitored for long-term safety for up to 10 years following the administration of LCTOPC1

10 years

Treatment Details

Interventions

• LCTOPC1

Trial Overview The DOSED study tests a new device's safety in delivering LCTOPC1 cell therapy directly into the injured spinal cord area. The goal is to replace or support damaged cells, potentially improving function and quality of life after SCI.

Participant Groups

Group I: LCTOPC1 Experimental Treatment 1 Intervention

Open Label Single Injection of LCTOPC1 at a Single Time Point

Who Is Running the Clinical Trial?

Lineage Cell Therapeutics, Inc.

Lead Sponsor

Trials

6

Recruited

90+

Findings from Research

Only 30% of the 40 reviewed clinical trials on acute spinal cord injury provided satisfactory reporting of adverse events, indicating a significant gap in safety information that is crucial for assessing risk and designing future studies.

A staggering 82.5% of trials failed to report laboratory-defined toxicity satisfactorily, highlighting the need for improved standards in safety reporting to ensure comprehensive understanding of treatment risks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Systematic Review of Safety Reporting in Acute Spinal Cord Injury Clinical Trials: Challenges and Recommendations. Aspinall, P., Harrison, L., Scheuren, P., et al.[2023]

References

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

Magnetic field-based delivery of human CD133⁺ cells promotes functional recovery after rat spinal cord injury. [2016]

2. Switzerland pubmed.ncbi.nlm.nih.gov

A Fully Implantable Miniaturized Liquid Crystal Polymer (LCP)-Based Spinal Cord Stimulator for Pain Control. [2022]

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

Intraspinal Delivery of Schwann Cells for Spinal Cord Injury. [2019]

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

Cellular therapeutics delivery to the spinal cord: technical considerations for clinical application. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Protective effects of extract of Ginkgo biloba (EGb 761) on nerve cells after spinal cord injury in rats. [2011]

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

A phase 1/2a dose-escalation study of oligodendrocyte progenitor cells in individuals with subacute cervical spinal cord injury. [2023]

7. England pubmed.ncbi.nlm.nih.gov

Human Embryonic Stem Cell-Derived Oligodendrocyte Progenitor Cells: Preclinical Efficacy and Safety in Cervical Spinal Cord Injury. [2020]

8. England pubmed.ncbi.nlm.nih.gov

Spinal cord injury - assessing tolerability and use of combined rehabilitation and NeuroAiD (SATURN) study - primary results of an exploratory study. [2023]

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

A Systematic Review of Safety Reporting in Acute Spinal Cord Injury Clinical Trials: Challenges and Recommendations. [2023]

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

Ten-year safety of pluripotent stem cell transplantation in acute thoracic spinal cord injury. [2022]

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

Expandable Sendai-Virus-Reprogrammed Human iPSC-Neuronal Precursors: In Vivo Post-Grafting Safety Characterization in Rats and Adult Pig. [2023]