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

• Conditions: Transverse Myelitis
• Interventions: Drug: Q-Cells®

• Registration Number: NCT06163508
• Lead Sponsor: University of Texas Southwestern Medical Center

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-11-14
• Study First Submitted QC: 2023-12-06
• Study First Posted: 2023-12-11
• Study Start: 2023-12-12
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-25
• Last Update Posted: 2024-03-29
• Primary Completion: 2035-12
• Study Completion: 2035-12

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

1. Subjects must have been administered Q-Cells® as part of Protocol QTM-101 or other treatment Protocol.
2. 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.

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Exclusion Criteria

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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Cohort 2: 15 microliters of Q cells	Q-Cells®	15 microliters of Q cells per site
Cohort 3: 20 microliters of Q cells	Q-Cells®	20 microliters of Q cells per site
Cohort 1: 10 microliters of Q cells	Q-Cells®	10 microliters of Q cells per site

Primary Outcome Measures

Name	Time	Method
Safety Endpoints: Clinical Laboratory assessments: hematology	through study completion, an average of 10 years	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.
Safety Endpoints: Vital signs: Diastolic Blood Pressure	through study completion, an average of 10 years	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
Number of Adverse Events	through study completion, an average of 10 years	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.
Safety Endpoints: Vital signs: Pulse	through study completion, an average of 10 years	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
Safety Endpoints: Clinical Laboratory assessments: chemistry	through study completion, an average of 10 years	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.
Safety: Vital signs: Systolic Blood Pressure	through study completion, an average of 10 years	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
Safety Endpoints: Vital signs: Temperature	through study completion, an average of 10 years	Temperature: Criteria for Clinically Relevant Vital Signs Abnormalities: \>38.5°C and an increase from pre-dosing of at least 1°C
Safety as measured by change in Electrocardiogram (ECG) measures from baseline at 10 years	through study completion, an average of 10 years (Baseline, 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.

Secondary Outcome Measures

Name	Time	Method
Exploratory Endpoints: Visual Analog pain Scale (VAS)	through study completion, an average of 10 years	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.
Neurological change: American Spinal Injury Association (ASIA) Impairment Scale	through study completion, an average of 10 years	Neurological changes in muscle strength and sensation via the ASIA exam.; ASIA exam yields overall scores ranked alphabetically A through E where A is the most impaired and E is normal.; 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.
Neurological change: National Institute of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS) Quality of Life Questionnaire: Sexual Function	through study completion, an average of 10 years	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.
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	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.
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	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.
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	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.
Neurological change: National Institute of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS) Quality of Life Questionnaire: Erectile Function	through study completion, an average of 10 years	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.

Trial Locations

Locations (1)

The University of Texas Southwestern Medical Center; 🇺🇸 Dallas, Texas, United States