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

Transverse Myelitis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT06163508
• Other study ID #: STU-2023-0803
• Status: Enrolling by invitation
• Start date: December 12, 2023
• Est. completion date: December 2035

Study information

• Verified date: March 2024
• Source: University of Texas Southwestern Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 9
• Est. completion date: December 2035
• Est. primary completion date: December 2035
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Exclusion Criteria:

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

Related Conditions & MeSH terms

• Myelitis
• Myelitis, Transverse
• Transverse Myelitis

Intervention

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

Locations

Country	Name	City	State
United States	The University of Texas Southwestern Medical Center	Dallas	Texas

Sponsors (1)

Lead Sponsor	Collaborator
University of Texas Southwestern Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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)
Primary	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
Primary	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	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
Secondary	Neurological change: American Spinal Injury Association (ASIA) Impairment Scale	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.	through study completion, an average of 10 years
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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