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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new method of delivering a cell therapy called LCTOPC1 to individuals with spinal cord injuries. The goal is to determine if this method is safe and can enhance quality of life by restoring function in those with traumatic spinal injuries. It targets individuals with complete or partial loss of movement or sensation in specific spinal areas, who have either recently sustained the injury or have not shown improvement over time. This trial may suit someone with a spinal cord injury affecting movement or sensation between the neck and mid-back who can undergo surgery. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this innovative therapy.

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 prior data suggests that this delivery device is safe for spinal cord injury?

Research has shown that LCTOPC1, a cell therapy for spinal cord injuries, is generally safe for humans. One study found that patients could safely receive LCTOPC1 soon after a spinal cord injury, indicating that the treatment and its administration were well-tolerated. Another long-term study found that using LCTOPC1, derived from special stem cells, did not cause serious problems over ten years. In some cases, patients even showed improvements in sensation and movement. These findings suggest that LCTOPC1 is a promising and safe option for those with spinal cord injuries. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike standard treatments for spinal cord injury, which mostly focus on managing symptoms and rehabilitation, LCTOPC1 is a cell-based therapy that aims to repair damaged neural tissue. Researchers are excited about LCTOPC1 because it uses specialized cells, called oligodendrocyte progenitor cells, to potentially regenerate the protective myelin sheath around neurons, promoting healing at a cellular level. This approach could offer a groundbreaking way to restore function and improve recovery for patients, which current therapies cannot achieve.

What evidence suggests that this delivery device is effective for spinal cord injury?

Research has shown that LCTOPC1, a cell therapy for spinal cord injury, delivers promising results from early studies. In one study, 96% of participants regained some nerve function after receiving LCTOPC1. This treatment aims to replace or support damaged cells in the spinal cord, potentially improving movement and quality of life for people with spinal cord injuries. The therapy has been safely administered to humans, indicating it is a promising option for further research. ^{1 3 6 7 8}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35901693/

A phase 1/2a dose-escalation study of oligodendrocyte ... At 1-year follow-up, 21/22 (96%) of the intention-to-treat group recovered one or more levels of neurological function on at least one side of ...

2. thejns.org thejns.org/spine/view/journals/j-neurosurg-spine/37/6/article-p812.xml

A phase 1/2a dose-escalation study of oligodendrocyte ... LCTOPC1 can be safely administered to participants in the subacute period after cervical SCI. The injection procedure, low-dose temporary immunosuppression ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06841770?term=stem%20cells%20cervical%20spinal%20cord%20injury&viewType=Table&rank=5

NCT06841770 | A Study to Evaluate the Safety of ... 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 ...

4. nature.com nature.com/articles/s41392-023-01477-6

Spinal cord injury: molecular mechanisms and therapeutic ... The challenges of SCI repair include its complex pathological mechanisms and the difficulties of neural regeneration in the central nervous system.

5. withpower.com withpower.com/trial/lctopc1-delivery-device-for-spinal-cord-injury-0bdfb

LCTOPC1 Delivery Device for Spinal Cord Injury 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 ...

6. thejns.org thejns.org/spine/view/journals/j-neurosurg-spine/37/3/article-p321.xml

Ten-year safety of pluripotent stem cell transplantation in ... The purpose of this study was to evaluate the safety of oligodendrocyte progenitor cells (LCTOPC1) derived from human pluripotent stem cells administered ...

7. clinicaltrials.ucsd.edu clinicaltrials.ucsd.edu/trial/NCT06841770

Delivery Device for Administering LCTOPC1 in Participants ... 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 ...

8. sciencedirect.com sciencedirect.com/science/article/pii/S2772529425000268

Stem cell therapies for spinal cord injury in humans Up to 27 months of follow-up data showed the safety of the transplantation protocol in all four participants. Also, one-level sensory and motor improvement in ...