Q-Cells Transplantation for Transverse Myelitis

Overseen By Erev King

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Q Therapeutics, Inc.

Must not be taking: Antidepressants, Immunosuppressants

Disqualifiers: Cognitive impairment, Neurodegenerative disease, Diabetes, others

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

Compensation: Varies

Number of Visits: 1

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial studies a new treatment called Q-Cells® for individuals with Transverse Myelitis, a condition where inflammation damages the spinal cord. The goal is to determine if Q-Cells®, which are special cells that can help repair nerve damage, are safe and can improve symptoms. Participants will receive one of three different doses of Q-Cells® to identify the most effective dose. The trial seeks individuals diagnosed with idiopathic Transverse Myelitis who have stable symptoms beyond one year and have not undergone previous stem cell treatments. As a Phase 1 trial, this research focuses on understanding how Q-Cells® work in people, offering participants the opportunity to be among the first to receive this innovative treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, it mentions that certain medications may be contraindicated with the study treatment, so it's best to discuss your specific medications with the study team.

Is there any evidence suggesting that Q-Cells® transplantation is likely to be safe for humans?

Research shows that Q-Cells® have been studied for safety when transplanted into the spinal cords of patients with Transverse Myelitis. In earlier studies, these cells, which aim to repair the spinal cord, showed promise in terms of safety. Patients who received Q-Cells® generally tolerated the treatment well, and no major safety concerns emerged.

These studies remain in the early stages, and researchers continue to gather information about the safety and effects of Q-Cells®. While the treatment is safe enough for testing in people, more research is needed to fully understand its safety. Overall, current evidence supports the safety of Q-Cells® for further testing in clinical trials. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for Transverse Myelitis?

Unlike the standard treatments for transverse myelitis, which often focus on managing inflammation and symptoms through medications like corticosteroids and plasma exchange, Q-Cells® takes a regenerative approach. Researchers are excited about Q-Cells® because it involves transplanting stem cells directly into the spinal cord's damaged areas. This method aims to repair the myelin sheath, potentially restoring nerve function. By targeting the root of the problem rather than just alleviating symptoms, Q-Cells® could offer a more transformative solution for patients.

What evidence suggests that Q-Cells® transplantation might be an effective treatment for Transverse Myelitis?

Research has shown that Q-Cells® might help treat Transverse Myelitis by repairing damaged parts of the spinal cord. Q-Cells® are special cells that can become healthy astrocytes and oligodendrocytes, which support and protect nerves. These cells might help rebuild the protective layer around nerves, called myelin, often damaged in Transverse Myelitis. Early findings suggest that Q-Cells® could also improve the function of damaged nerves. Although research remains in the early stages, Q-Cells® offer hope for repairing spinal cord injuries caused by this condition. Participants in this trial will receive one of three different dose levels of Q-Cells® to evaluate their effectiveness and safety. ^{1 2 4 5 6}

Are You a Good Fit for This Trial?

This trial is for adults aged 18-70 with idiopathic Transverse Myelitis diagnosed within the last 10 years. Participants must be stable, able to travel to the center, and have a caregiver. They should not have other autoimmune diseases or significant medical conditions that could interfere with the study.

Inclusion Criteria

Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to collect and use protected health information (PHI) in accordance with national and local subject privacy regulations

I was diagnosed with idiopathic transverse myelitis within the last 10 years.

I have someone to help me with travel and care for my study needs.

Exclusion Criteria

My scans show possible blood vessel issues in my spinal cord.

I have a genetic condition like Lesch-Nyhan or Kelley-Seegmiller syndrome.

You are allergic to the study treatment or any of its parts, like chicken eggs, or to any of the other medications given with it.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-operative/Treatment

Participants receive a single time point administration of Q-Cells® with transplantation foci targeted to posterior columns in the spinal cord

1 day

1 visit (in-person)

Post-treatment

Participants are monitored for safety and activity following the transplantation

9 months

Regular visits (in-person)

Long-term follow-up

Subjects who consent will continue to be followed for safety and activity in a separate long-term follow-up protocol

What Are the Treatments Tested in This Trial?

Interventions

• Q-Cells®

Trial Overview The trial tests Q-Cells transplantation into spinal cord lesions in Transverse Myelitis patients. It's an open-label study assessing safety and potential benefits of these glial progenitor cells in repairing nerve damage over a period of up to 12 months post-transplantation.

How Is the Trial Designed?

Group I: Q-Cells dose level 3 Experimental Treatment 1 Intervention

One time surgical transplantation of Q-Cells dose level 3 unilaterally into spinal cord demyelinated lesion

Group II: Q-Cells dose level 2 Experimental Treatment 1 Intervention

One time surgical transplantation of Q-Cells dose level 2 unilaterally into spinal cord demyelinated lesion

Group III: Q-Cells dose level 1 Experimental Treatment 1 Intervention

One time surgical transplantation of Q-Cells dose level 1 unilaterally into spinal cord demyelinated lesion

Who Is Running the Clinical Trial?

Q Therapeutics, Inc.

Lead Sponsor

Trials

2

Recruited

40+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03887273

NCT03887273 | Study to Investigate the Safety of ... For each of the dose levels, transplantation of Q-Cells® unilaterally into spinal cord demyelinated lesions will be evaluated. Subjects will be blinded to side ...

2. wearesrna.org wearesrna.org/resources/update-on-the-q-cells-study/

Update on the Q-Cells Study | SRNA At the 2024 RNDS, Dr. Benjamin Greenberg gave an update on the current state of stem cell therapy trial for transverse myelitis.

3. cdek.pharmacy.purdue.edu cdek.pharmacy.purdue.edu/trial/NCT03887273/

Trial | NCT03887273 Therefore, Q-Cells® have the potential to repair damage that has occurred and could be clinically useful for patients with disability caused by TM. The study is ...

4. withpower.com withpower.com/trial/phase-2-myelitis-2-2021-c8e23

Q-Cells Transplantation for Transverse Myelitis This trial tests the safety and early effects of transplanting Q-Cells® into the spinal cords of patients with Transverse Myelitis. These special cells may ...

5. firstwordpharma.com firstwordpharma.com/story/4409862

FDA Allows Q-Cells for First-In-Human Transverse Myelitis ... Q-Cells may be suitable to treat a range of CNS disorders, including demyelinating conditions such as multiple sclerosis (MS), transverse ...

6. neurology.org neurology.org/doi/10.1212/WNL.92.15_supplement.P1.2-020

Q-Cells®) into Subjects with Transverse Myelitis (TM) (P1.2 ... The primary objective of the study is to evaluate the safety of Q-Cells transplantation into the posterior columns of the spinal cord in patients with ...