A Long Term Follow-Up Study for Subjects Who Have Received Q-Cells

NCT06163508 | Enrolling By Invitation FDA Drug

• 1 other identifier: STU-2023-0803
• Study Type: observational
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

This study is an observational study designed to obtain information on the long-term safety, tolerability, and continued activity of Q-Cells®. The study will follow the participants who previously received Q-Cells® for 10 years. The goal of this observational study is to learn about the long term effects of Q-Cells® in people with transverse myelitis. The main objectives the study is to evaluate the safety of patients who have received Q-Cells®. The secondary goal of the study is to get data about the long-term activity of Q-Cells® over a period of 10 years. Patients will complete exams, lab tests, imaging, and questionnaires to monitor their safety.

Conditions

Transverse Myelitis

Outcome Measures

Primary Outcomes (8)

• Number of Adverse Events

  Safety will be assessed by the number of Adverse events.These will be recorded via physical examination findings and will be presented in data listings. Clinically significant changes on physical exam in the areas of: general appearance, head, ears, eyes, nose, throat, neck, chest and lungs, cardiovascular, abdomen, neurological, thyroid, musculoskeletal, lymph nodes, extremities and skin, and operative site will be recorded as Adverse Events.

  through study completion, an average of 10 years

• Safety: Vital signs: Systolic Blood Pressure

  Systolic Blood Pressure: Criteria for Clinically Relevant Vital Signs Abnormalities: \>180 mmHg or an increase from pre-dosing of more than 40 mmHg, or \<90 mmHg or a decrease from pre-dosing of more than 30 mmHg

  through study completion, an average of 10 years

• Safety Endpoints: Vital signs: Diastolic Blood Pressure

  Diastolic Blood Pressure: Criteria for Clinically Relevant Vital Signs Abnormalities: \>105 mmHg or an increase from pre-dosing of more than 30 mmHg, or \<50 mmHg or a decrease from pre-dosing of more than 20 mmHg

  through study completion, an average of 10 years

• Safety Endpoints: Vital signs: Pulse

  Pulse: Criteria for Clinically Relevant Vital Signs Abnormalities: \>120 beats per minute or an increase from pre dosing of more than 20 beats per minute, or \<50 beats per minute or a decrease from pre dosing of more than 20 beats per minute

  through study completion, an average of 10 years

• Safety Endpoints: Vital signs: Temperature

  Temperature: Criteria for Clinically Relevant Vital Signs Abnormalities: \>38.5°C and an increase from pre-dosing of at least 1°C

  through study completion, an average of 10 years

• Safety as measured by change in Electrocardiogram (ECG) measures from baseline at 10 years

  Number of participants with abnormal ECG readings will be summarized using descriptive statistics by original treatment cohort and visit. ECG findings that are determined to be potentially clinically significant will be summarized.

  through study completion, an average of 10 years (Baseline, 10 years)

• Safety Endpoints: Clinical Laboratory assessments: chemistry

  Descriptive statistics for raw values as well as change from baseline (entry into this protocol No. QLTFU-101) by original treatment group will be presented for each visit. The number and percentage of subjects with potentially clinically significant laboratory results will be tabulated by original treatment cohort and overall.

  through study completion, an average of 10 years

• Safety Endpoints: Clinical Laboratory assessments: hematology

  Descriptive statistics for raw values as well as change from baseline (entry into this protocol No. QLTFU-101) by original treatment group will be presented for each visit. The number and percentage of subjects with potentially clinically significant laboratory results will be tabulated by original treatment cohort and overall.

  through study completion, an average of 10 years

Secondary Outcomes (7)

• Exploratory Endpoints: Visual Analog pain Scale (VAS)

  through study completion, an average of 10 years

• Neurological change: American Spinal Injury Association (ASIA) Impairment Scale

  through study completion, an average of 10 years

• Neurological change: National Institute of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS) Quality of Life Questionnaire: Lower Extremity Function (mobility)

  through study completion, an average of 10 years

• Neurological change: National Institute of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS) Quality of Life Questionnaire: Neurogenic Bladder Symptom Score (NBSS)

  through study completion, an average of 10 years

• Neurological change: National Institute of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS) Quality of Life Questionnaire: Neurogenic Bowel Dysfunction

  through study completion, an average of 10 years

Study Arms (3)

Cohort 1: 10 microliters of Q cells

10 microliters of Q cells per site

Drug: Q-Cells®

Cohort 2: 15 microliters of Q cells

15 microliters of Q cells per site

Drug: Q-Cells®

Cohort 3: 20 microliters of Q cells

20 microliters of Q cells per site

Drug: Q-Cells®

Interventions

Q-Cells® DRUG

Long term observational study of patients who received 1 of 3 different amounts of Q-Cells®.

Cohort 1: 10 microliters of Q cells; Cohort 2: 15 microliters of Q cells; Cohort 3: 20 microliters of Q cells

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Subjects must have been administered Q-Cells® as part of Protocol QTM-101 or other treatment Protocol.

You may qualify if:

• Subjects must have been administered Q-Cells® as part of Protocol QTM-101 or other treatment Protocol.
• Subjects must have the 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.

You may not qualify if:

• \. The study is intended to follow all subjects who have received Q-Cells® without exception.

Sponsors & Collaborators

• University of Texas Southwestern Medical Center: lead

Study Sites (1)

The University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

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

Study Officials

• Benjamin Greenberg, MD

  University of Texas Southwestern Medial Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Benjamin M. Greenberg, Principal Investigator

Study Record Dates

• First Submitted: November 14, 2023
• First Posted: December 11, 2023
• Study Start: December 12, 2023
• Primary Completion (Estimated): December 1, 2035
• Study Completion (Estimated): December 1, 2035
• Last Updated: April 21, 2026

Record last verified: 2026-04

Locations

US (1)