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

Study Overview
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Brief Summary
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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.

Official Title
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A Long Term Follow-Up Study for Subjects Who Have Received Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) (LTFU Study)

Conditions
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Transverse Myelitis

Intervention / Treatment
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* Drug: Q-Cells®


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Exclusion Criteria: 1. The study is intended to follow all subjects who have received Q-Cells® without exception.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Cohort 1: 10 microliters of Q cells<br>10 microliters of Q cells per site | Drug: Q-Cells®<br>* Long term observational study of patients who received 1 of 3 different amounts of Q-Cells®.<br>|
| Cohort 2: 15 microliters of Q cells<br>15 microliters of Q cells per site | Drug: Q-Cells®<br>* Long term observational study of patients who received 1 of 3 different amounts of Q-Cells®.<br>|
| Cohort 3: 20 microliters of Q cells<br>20 microliters of Q cells per site | Drug: Q-Cells®<br>* Long term observational study of patients who received 1 of 3 different amounts of Q-Cells®.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety Endpoints: physical exams | The physical examination findings will be presented in data listings. | 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 Endpoints: Electrocardiogram (ECG) | ECG measures as well as change from baseline 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 | 
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Exploratory Endpoints: Visual Analog pain Scale (VAS) | Changes in pain will be assessed using VAS, a self-assessment where patients rank their current pain from 0-10. Analysis of activities is considered exploratory and observational data from each patient will be recorded due to sample size. Descriptive statistics (N, mean, standard deviation, median, minimum and maximum values) and change from baseline will be summarized overall and by original treatment cohort and visit. | through study completion, an average of 10 years | 
| Neurological change: American Spinal Injury Association (ASIA) Impairment Scale | Neurological changes in muscle strength and sensation via the ASIA exam. Analysis of activities is considered exploratory and observational data from each patient will be recorded due to sample size. Descriptive statistics (N, mean, standard deviation, median, minimum and maximum values) and change from baseline will be summarized overall and by original treatment cohort and visit. | 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) | NIH PROMIS scale for Lower Extremity Function is a self-assessment where patients score their lower extremity function. Higher scores in this scale are related to better outcomes. Minimum value: 95 Maximum value: 19 Analysis of activities is considered exploratory and observational data from each patient will be recorded due to sample size. Descriptive statistics (N, mean, standard deviation, median, minimum and maximum values) and change from baseline will be summarized overall and by original treatment cohort and visit. | 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) | NIH PROMIS scale for NBSS is a self-assessment where patients score their bladder symptoms. Lower scores in this scale are related to better outcomes. Minimum value: 28 Maximum value: 0 Analysis of activities is considered exploratory and observational data from each patient will be recorded due to sample size. Descriptive statistics (N, mean, standard deviation, median, minimum and maximum values) and change from baseline will be summarized overall and by original treatment cohort and visit. | 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 | NIH PROMIS scale for Neurogenic Bowel Dysfunction is a self-assessment where patients score their bowel symptoms. Lower scores in this scale are related to better outcomes. Minimum value: 47 Maximum value: 0 Analysis of activities is considered exploratory and observational data from each patient will be recorded due to sample size. Descriptive statistics (N, mean, standard deviation, median, minimum and maximum values) and change from baseline will be summarized overall and by original treatment cohort and visit. | 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: Erectile Function | NIH PROMIS scale for Erectile Function is a self-assessment where patients score their sexual function. Higher scores in this scale are related to better outcomes. Minimum value: 5 Maximum value: 53 Analysis of activities is considered exploratory and observational data from each patient will be recorded due to sample size. Descriptive statistics (N, mean, standard deviation, median, minimum and maximum values) and change from baseline will be summarized overall and by original treatment cohort and visit. | 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: Sexual Function | NIH PROMIS scale for Sexual Function is a self-assessment where patients score their sexual function. Lower scores in this scale are related to better outcomes. Minimum value: 0 Maximum value: 263 Analysis of activities is considered exploratory and observational data from each patient will be recorded due to sample size. Descriptive statistics (N, mean, standard deviation, median, minimum and maximum values) and change from baseline will be summarized overall and by original treatment cohort and visit. | through study completion, an average of 10 years |