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

March 25, 2024 updated by: Benjamin Greenberg, University of Texas Southwestern Medical Center

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

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.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

9

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Texas
      • Dallas, Texas, United States, 75390
        • The University of Texas Southwestern Medical Center
        • Principal Investigator:
          • Benjamin Greenberg, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety: Vital signs: Systolic Blood Pressure
Time Frame: 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
through study completion, an average of 10 years
Safety Endpoints: Vital signs: Diastolic Blood Pressure
Time Frame: 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
through study completion, an average of 10 years
Safety Endpoints: Vital signs: Pulse
Time Frame: 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
through study completion, an average of 10 years
Safety Endpoints: Vital signs: Temperature
Time Frame: 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
through study completion, an average of 10 years
Safety Endpoints: Clinical Laboratory assessments: chemistry
Time Frame: 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.
through study completion, an average of 10 years
Safety Endpoints: Clinical Laboratory assessments: hematology
Time Frame: 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.
through study completion, an average of 10 years
Number of Adverse Events
Time Frame: 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.
through study completion, an average of 10 years
Safety as measured by change in Electrocardiogram (ECG) measures from baseline at 10 years
Time Frame: 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.
through study completion, an average of 10 years (Baseline, 10 years)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Exploratory Endpoints: Visual Analog pain Scale (VAS)
Time Frame: 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.
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)
Time Frame: 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.
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)
Time Frame: 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.
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
Time Frame: 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.
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
Time Frame: 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.
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
Time Frame: 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.
through study completion, an average of 10 years
Neurological change: American Spinal Injury Association (ASIA) Impairment Scale
Time Frame: 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.
through study completion, an average of 10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Texas Southwestern Medical Center

Investigators

  • Principal Investigator: Benjamin Greenberg, MD, University of Texas Southwestern Medial Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 12, 2023

Primary Completion (Estimated)

December 1, 2035

Study Completion (Estimated)

December 1, 2035

Study Registration Dates

First Submitted

November 14, 2023

First Submitted That Met QC Criteria

December 6, 2023

First Posted (Actual)

December 11, 2023

Study Record Updates

Last Update Posted (Actual)

March 29, 2024

Last Update Submitted That Met QC Criteria

March 25, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • STU-2023-0803

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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