NCT03887273

A Phase 1/2a Open-Label Study to Investigate the Safety of the Transplantation (by Injection) of Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) Into Subjects With Transverse Myelitis (TM)

Status unknown Phase 1/Phase 2 Last updated 17 August 2023

What this trial tests

Phase 1/Phase 2 trial testing Q-Cells in Transverse Myelitis in 9 participants. Status unknown.

Timeline

20 September 2022

Primary endpoint
1 July 2024

1 December 2024

Quick facts

Lead sponsor	Q Therapeutics, Inc.
Phase	Phase 1/Phase 2
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	9
Start date	20 September 2022
Primary completion	1 July 2024
Estimated completion	1 December 2024
Sites	1 location across United States

Drugs / interventions tested

• Q-Cells

Conditions studied

• Transverse Myelitis — all drugs for Transverse Myelitis →

Sponsor

Q Therapeutics, Inc. — full company profile →

Who can join

Adults 18 to 70, any sex, with Transverse Myelitis. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability].
  Time frame: 9-months
  Safety will be measured by the number of therapy related adverse events.

Sponsor's own description

This study is a non-randomized, open-label, partially blinded, sequential cohort, dose-escalation study designed to obtain preliminary data on the safety, tolerability, and early activity of Q-Cells® transplantation in subjects with Transverse Myelitis. 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 of treatment.

Idiopathic Transverse Myelitis is a monophasic disorder characterized predominantly by demyelination. Patients are left with disability from damage to ascending and descending white matter tracts. Q-Cells® are comprised of glial progenitor cells.It is postulated that the Q-Cells® glial progeny (healthy astrocytes and oligodendrocytes) will integrate into the spinal cord lesion site and remyelinate demyelinated axons as well as provide trophic support for damaged axons. 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 planned to enroll up to 9 subjects. Each subject will be followed for 9 months after transplantation of Q-Cells®. Each subject will receive a single time point administration of Q-Cells®: with transplantation foci targeted to posterior columns in the spinal cord (all transplantation foci below C7) on one side.

Study participation consists of Screening, Pre-operative/Treatment, and Post-treatment study periods that will generally last from 9 to 12 months in total. The study data will be assessed for safety and activity until the last subject has completed the 9-month study visit. Following completion of the 9-month follow-up period, subjects who consent will continue to be followed for safety and activity in a separate long-term follow-up protocol.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Stem cell therapeutics and gene therapy for neurologic disorders.
   Chen KS, Koubek EJ, Sakowski SA, Feldman EL. · · 2024 · cited 26× · PMID 39096590 · DOI 10.1016/j.neurot.2024.e00427
2. Challenges and Opportunities of Targeting Astrocytes to Halt Neurodegenerative Disorders.
   Valori CF, Possenti A, Brambilla L, Rossi D. · · 2021 · cited 26× · PMID 34440788 · DOI 10.3390/cells10082019
3. Glial restricted precursor cells in central nervous system disorders: Current applications and future perspectives.
   Martins-Macedo J, Lepore AC, Domingues HS, Salgado AJ, et al · · 2021 · cited 22× · PMID 33052610 · DOI 10.1002/glia.23922