A Long Term Follow-Up Study for Subjects Who Have Received Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) (LTFU Study)
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 9
- 试验地点
- 2
- 主要终点
- Safety Endpoints: Clinical Laboratory assessments: hematology
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •1. The study is intended to follow all subjects who have received Q-Cells® without exception.
研究组 & 干预措施
Cohort 3: 20 microliters of Q cells
20 microliters of Q cells per site
干预措施: Q-Cells® (Drug)
Cohort 1: 10 microliters of Q cells
10 microliters of Q cells per site
干预措施: Q-Cells® (Drug)
Cohort 2: 15 microliters of Q cells
15 microliters of Q cells per site
干预措施: Q-Cells® (Drug)
结局指标
主要结局
Safety Endpoints: Clinical Laboratory assessments: hematology
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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.
次要结局
- 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: Sexual Function(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)
- 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)
研究者
Benjamin Greenberg
Benjamin M. Greenberg, Principal Investigator
University of Texas Southwestern Medical Center