Transverse MyelitisPhase 1/2NCT03887273
Study to Investigate the Safety of the Transplantation of Human Glial Restricted Progenitor Cells Into Subjects With Transverse Myelitis
A Phase 1/2 study testing Q-Cells in people with Transverse Myelitis, run by Q Therapeutics, Inc. at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Transverse Myelitis
- What is being tested?
- Q-Cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Sep 2022to Dec 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- Live within reasonable travel distance to center or have reliable mechanism to travel to the center.
- Have a caregiver willing/able to assist in the transportation and care required by study participation.
- Subject is 18 - 70 years of age (inclusive) on day of Screening Visit.
- Subject is diagnosed with idiopathic TM within the past 120 months in accord with the Transverse Myelitis Consortium Working Group (2002).
- Subject has an MRI with a single focus of T2 hyperintensity with its most rostral extent at or below C8 myotome/dermatome level.
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- Subject has negative NMO IgG (anti-AQP4) test at two separate time points, separated by at least 6 months.
- Subject has brain MRI not consistent with multiple sclerosis or other autoimmune or demyelinating disease.
- Subject is more than 12 months from TM onset.
- Subject has ASIA A, B or C categorization, and is unable to ambulate (Cohort A) or unable to ambulate 100 feet without significant bilateral support. (Cohort B).
- Subject's neurological deficits related to TM have been stable for at least 3 months.
- Subject is medically able to undergo the study procedures and physically able to adhere to the visit schedule at the time of study entry.
- For women of child bearing capacity, negative pregnancy test during the Screening Period and at the Pre-Operative Visit.
- Males and females will agree to practice effective birth control during study participation and up to one year after.
- Current, up to date, COVID vaccination.
- Current, up to date, varicella zoster vaccination.
- Current, seasonally up to date, influenza vaccination.
Who cannot join
- Subject with causes of weakness, sensory loss and/or autonomic dysfunction other than TM have not been practically excluded.
- Subject with significant cognitive impairment, clinical dementia, or major psychiatric illness including psychosis, bipolar disease, major depression, as determined by the DSM-V that will interfere with participation in the trial.
- Subject with a diagnosis of a neurodegenerative disease (e.g., ALS, Parkinson's disease, Alzheimer's disease).
- Subject suffering with medical conditions that impair nerve or muscle function (e.g., notable peripheral neuropathy, metabolic muscle disease) or any disease or condition that would impair the subject's neuromuscular function or impair the adequate assessment of the subject's function (e.g., severe osteoarthritis).
- Subject with a clinically significant history of unstable cardiac, pulmonary, renal, hepatic, endocrine, hematologic, or active malignancy or infectious disease or other medically significant illness that may render them at an unacceptable risk for surgery or that may cause them to be unable to complete the scheduled duration of the trial.
- History of spine surgery or anatomic variation incompatible with route of administration (as determined by neurosurgeon).
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- Severe spinal stenosis or cord compression causing myelopathy.
- Abnormal flow voids on the surface of the spinal cord suggestive of arteriovenous malformation (AVM) or evidence of a vascular cause of a myelopathy (e.g., infarct of spinal artery).
- Any evidence of CNS malignancy or clinically significant CNS lesions as defined by imaging studies of the CNS (MRI of brain and spinal cord).
- Uncontrolled hypertension (Systolic BP>180mmHg and/or Diastolic BP >110mmHg).
- Any poorly controlled medical conditions that, in the opinion of the site investigator and/or surgeon, increase risk of surgery to a medically unacceptable degree.
- Subjects who cannot undergo MRI examination because of any contraindication to the procedure, including the presence of a pacemaker, an implanted defibrillator or certain other implanted electronic or metallic devices, or who have been or might have been exposed to metal fragments, or any reason the subject cannot undergo an MRI routinely for the duration of the trial.
- Subject with clinically significant abnormal clinical laboratory values, as determined by the Investigator at the screening visit (Visit 1).
- Subject who is immune compromised (by therapeutic agent or disease) or who has a condition contraindicated to treatment with immunosuppression agents (e.g., tuberculosis, latent infection) as determined by history or testing. Any subject with an ongoing infection until it has been adequately treated and it is deemed to be resolved.
- Subject with aspartate aminotransferase (AST) or alanine aminotransferase (ALT) value >3.0 times the upper limit of normal at the screening visit (Visit 1).
- Subject with diabetes or HgbA1c > 6.5.
- Subject with a history of alcohol or drug abuse or dependence within 1 year of screening visit (Visit 1), per DSM-V criteria.
- Subject unlikely to comply with study requirements, as determined by Investigator.
- Subject who has been exposed to any other experimental agent (off-label use or investigational) within 60 days of screening visit (Visit 1). Biologic agents may need additional time for washout and will be evaluated by the Sponsor on a case-by-case basis.
- Subject with pre-existing severe or high titer anti-human leukocyte antigen (HLA) class I or class II antibodies directed against the Q-Cells®, as determined by panel reactive antibody (PRA) assay.
- Allergy to study treatment (Q-Cells®) or any of its constituents (e.g., chicken eggs), or allergy to any of the co-administered immunosuppressants or any of their excipients.
- Subject with any medical condition or using concomitant medication that would contraindicate the use of tacrolimus, mycophenolate mofetil, or prednisone as determined by Investigator.
- Subject has undergone stem cell transplantation (including T-cell or bone marrow transplants) at any time prior to study (within or outside the US).
- Subject with prior embolus or evidence of deep vein thrombosis (DVT) by venous ultrasound without adequate treatment.
- Subject has recent (1 year) or recurrent history of gastrointestinal bleeding or peptic ulcer disease or is under active treatment to prevent recurrence.
- Subject with estimated glomerular filtration rate at screening of less than 60 mL/min/1.73m2.
- Subjects with hereditary deficiency of hypoxanthine-guanine phosphoribosyl-transferase (HGPRT) such as Lesch-Nyhan and Kelley-Seegmiller syndrome.
- Vaccination with live virus within 6 weeks of screening.
- History or evidence of optic neuritis.
- Any reason, in the judgment of the investigator, which would make the subject inappropriate for entry into this trial.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Taylor Hinojo, 214-645-3230
Benjamin Greenberg, MD
About this study
Official title: 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)
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.
Read the detailed description
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.
- Conditions
- Transverse Myelitis
- Being tested
- Q-Cells
- Keywords
- TM
- Medical subject headings
- Myelitis, Transverse
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Q-CellsBiological
Also called Glial restricted progenitor cells
Cellular therapeutic comprised of human cells of the glial lineage
| Study group | Role | What they receive |
|---|---|---|
| Q-Cells dose level 1 | Experimental | One time surgical transplantation of Q-Cells dose level 1 unilaterally into spinal cord demyelinated lesion Receives: Biological: Q-Cells |
| Q-Cells dose level 2 | Experimental | One time surgical transplantation of Q-Cells dose level 2 unilaterally into spinal cord demyelinated lesion |
| Q-Cells dose level 3 | Experimental | One time surgical transplantation of Q-Cells dose level 3 unilaterally into spinal cord demyelinated lesion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability].
Safety will be measured by the number of therapy related adverse events.
Measured: 9-months
Secondary outcomes (6)
Scores on American Spinal Injury Association (ASIA) scale testing including motor and sensory evaluations
The patient's grade is based on how much sensation he or she can feel at multiple points on the body, as well as tests of motor function. Sensory grading is 0 (sensation absent), 1 (impaired sensitivity), or 2 (normal sensation) for each of light touch and pin prick sensation at each of 28 locations bilaterally: a maximum of 224 for total normal sensation. Motor function is graded from 0 (total paralysis) to 5 (normal active movement) for each of ten motor functions (e.g. knee extension; 10 total motions bilaterally) for a maximum of 100.
Measured: 9-months
Quantitative muscle strength values from a hand held dynamometer (HHD)
Assess changes in neurological function as measured by muscle strength
Measured: 9-months
Pain Scores on the Visual Analog Scale (VAS)
VAS is a linear score where subjects rank their current pain on a scale of 0 to 10 where 0 is no pain and 10 is the worst pain imaginable.
Measured: 9-months
Score on Quality of Life (QOL) Questionnaire
The National Institutes of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS) QOL questionnaire will be used to document patient reported outcomes in the following areas: Lower extremity function, Urinary/Bladder functions, Bowel functions, and Sexual functions.
Measured: 9-months
Score on Ashworth Spasticity Scale
The Modified Ashworth Scale (MAS) will be used to measure spasticity. During the administration of the MAS the examiner passively moves the joint being tested and rates the perceived level of resistance in the muscle groups opposing the movement. The scale is single-item measures ranging from 0 to 4, where 0 indicates no increase in muscle tone and 4 indicates that the affected part is rigid in flexion or extension.
Measured: 9-months
Latency and conduction velocities from tibial somatosensory evoked potentials (SSEPs)
Somatosensory evoked potentials (SEPs or SSEPs) are a useful, noninvasive means of assessing the somatosensory pathway functioning. Electrodes are placed on the lower extremity, the back, neck and head of a patient. A nonpainful electrical stimulus is applied to the lower extremity and the signal transduction is captured by the electrodes along the back, neck and scalp. The speed of signal transduction and amplitude of the signal is measured. Standardized values are recorded and can be monitored over time. Changes in the latency (conduction velocity) will be tracked from baseline to the 9 month outcome. Transverse Myeltitis decreases the conduction speed (an increase in latency); shorter latency (increased conduction velocity) would be an indication of recovery of function.
Measured: 9-months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2019
- Study start20 Sep 2022
- Primary completionJul 2027 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transverse MyelitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Q Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Benjamin Greenberg, MD, Principal investigator, University of Texas Southwestern Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03887273
- Sponsor's protocol ID
- QTM-101
- Status last verified
- Jul 2026
- First submitted
- 14 Mar 2019
- Last updated
- 21 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT03887273
- Acronym / sponsor ID
- QTM-101
- Conditions
- Transverse Myelitis
- Keywords
- TM
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Q Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 20 Sep 2022 (actual)
- Primary completion
- Jul 2027 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 22 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Q-Cells dose level 1 | Experimental | Biological: Q-CellsOne time surgical transplantation of Q-Cells dose level 1 unilaterally into spinal cord demyelinated lesion |
| Q-Cells dose level 2 | Experimental | –One time surgical transplantation of Q-Cells dose level 2 unilaterally into spinal cord demyelinated lesion |
| Q-Cells dose level 3 | Experimental | –One time surgical transplantation of Q-Cells dose level 3 unilaterally into spinal cord demyelinated lesion |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- 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.
- Live within reasonable travel distance to center or have reliable mechanism to travel to the center.
- Have a caregiver willing/able to assist in the transportation and care required by study participation.
- Subject is 18 - 70 years of age (inclusive) on day of Screening Visit.
- Subject is diagnosed with idiopathic TM within the past 120 months in accord with the Transverse Myelitis Consortium Working Group (2002).
Key exclusion criteria (30 listed)
- Subject with causes of weakness, sensory loss and/or autonomic dysfunction other than TM have not been practically excluded.
- Subject with significant cognitive impairment, clinical dementia, or major psychiatric illness including psychosis, bipolar disease, major depression, as determined by the DSM-V that will interfere with participation in the trial.
- Subject with a diagnosis of a neurodegenerative disease (e.g., ALS, Parkinson's disease, Alzheimer's disease).
- Subject suffering with medical conditions that impair nerve or muscle function (e.g., notable peripheral neuropathy, metabolic muscle disease) or any disease or condition that would impair the subject's neuromuscular function or impair the adequate assessment of the subject's function (e.g., severe osteoarthritis).
- Subject with a clinically significant history of unstable cardiac, pulmonary, renal, hepatic, endocrine, hematologic, or active malignancy or infectious disease or other medically significant illness that may render them at an unacceptable risk for surgery or that may cause them to be unable to complete the scheduled duration of the trial.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Q-CellsAlso: Glial restricted progenitor cellsCellular therapeutic comprised of human cells of the glial lineage | Biological | Q-Cells dose level 1 |
Primary outcome
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.
6 secondary outcomes
Scores on American Spinal Injury Association (ASIA) scale testing including motor and sensory evaluations
Time frame: 9-months
Quantitative muscle strength values from a hand held dynamometer (HHD)
Time frame: 9-months
Pain Scores on the Visual Analog Scale (VAS)
Time frame: 9-months
Score on Quality of Life (QOL) Questionnaire
Time frame: 9-months
Score on Ashworth Spasticity Scale
Time frame: 9-months
Latency and conduction velocities from tibial somatosensory evoked potentials (SSEPs)
Time frame: 9-months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Benjamin Greenberg, MD principal investigator
University of Texas Southwestern Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Q-Cells in people with Transverse Myelitis, run by Q Therapeutics, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 30 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 21 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Taylor Hinojo, MS (214-645-3230, taylor.hinojo@utsouthwestern.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]. (time frame: 9-months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jul 2026.