Study to Investigate the Safety of the Transplantation of Human Glial Restricted Progenitor Cells Into Subjects With Transverse Myelitis

NCT03887273 | Unknown Phase 1 DMC FDA Drug

• 1 other identifier: QTM-101
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Transverse Myelitis

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability].

  Safety will be measured by the number of therapy related adverse events.

  9-months

Secondary Outcomes (6)

• Scores on American Spinal Injury Association (ASIA) scale testing including motor and sensory evaluations

  9-months

• Quantitative muscle strength values from a hand held dynamometer (HHD)

  9-months

• Pain Scores on the Visual Analog Scale (VAS)

  9-months

• Score on Quality of Life (QOL) Questionnaire

  9-months

• Score on Ashworth Spasticity Scale

  9-months

Study Arms (3)

Q-Cells dose level 1

EXPERIMENTAL

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

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

Biological: Q-Cells

Q-Cells dose level 3

EXPERIMENTAL

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

Biological: Q-Cells

Interventions

Q-Cells BIOLOGICAL

cellular therapeutic comprised of human cells of the glial lineage

Also known as: glial restricted progenitor cells

Q-Cells dose level 1; Q-Cells dose level 2; Q-Cells dose level 3

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 a MRI with a single focus of T2 hyperintensity that is 4 to 10 cm in length if no post contrast enhancement seen, or a single focus T1 post contrast enhancing lesion of 4 to 10 cm, with its most rostral extent at or below C8 myotome/dermatome level.
• 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 categorization.
• 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.

You may not qualify if:

• 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.
• 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).
• 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 history of thrombotic or embolic events.
• 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.

Sponsors & Collaborators

• Q Therapeutics, Inc.: lead

Study Sites (1)

UT Southwestern

Dallas, Texas, 75390, United States

RECRUITING

MeSH Terms

Conditions

Myelitis, Transverse

Condition Hierarchy (Ancestors)

Myelitis; Central Nervous System Infections; Infections; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Central Nervous System Diseases; Spinal Cord Diseases; Demyelinating Diseases; Neurodegenerative Diseases; Neuroinflammatory Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Taylor Hinojo

CONTACT

214-645-3230; taylor.hinojo@utsouthwestern.edu

Erev King

CONTACT

18282304040; erevbking@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 14, 2019
• First Posted: March 22, 2019
• Study Start: September 20, 2022
• Primary Completion: July 1, 2024
• Study Completion: December 1, 2024
• Last Updated: August 21, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)