Ignite Creation Date:
2024-05-06 @ 3:05 PM
Last Modification Date:
2024-10-26 @ 1:42 PM
Study NCT ID:
NCT04514952
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2022-09-26
First Post:
2020-08-12
Brief Title:
Individual Patient Expanded Access IND of Autologous HBadMSCs for the Treatment of Amyotrophic Lateral Sclerosis
Sponsor:
Hope Biosciences Stem Cell Research Foundation
Organization:
Hope Biosciences
Study Overview
Official Title:
Individual Patient Expanded Access IND of Autologous HBadMSCs for the Treatment of Amyotrophic Lateral Sclerosis
Status:
NO_LONGER_AVAILABLE
Status Verified Date:
2022-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The drug for this submission is Hope Biosciences autologous adipose-derived culture-expanded mesenchymal stem cells HB-adMSCs for the treatment of a single patient with Amyotrophic Lateral Sclerosis ALS Stem cells have become a promising tool for the treatment of inflammatory and neurodegenerative conditions including autoimmune diseases traumatic brain injury Parkinsons disease and Alzheimers disease
Detailed Description:
Once the eligibility is confirmed approximately 1-2 weeks after the screening visit the subject will return for the baselinefirst infusion visit Subsequent treatments will occur 2 weeks apart for 18 weeks for a total of 10 infusions Follow-up visits will occur at 22 weeks 26 weeks and 39 weeks End of study visit will occur at 52 weeks
BaselineInfusion 1 Visit
1 A verification of patient consent will be verbally performed and included in the progress note
2 Review of medical history and concomitant medications
3 Physical exam ALS Functional Rating Scale-Revised ALSFRS-R
4 Vital signs Heart Rate Blood Pressure Respirations Temp SpO2
5 Weight measurement
6 Blood samples will be collected for safety and efficacy assessments
Hematology
Chemistry
Coagulation Panel
Proinflammatory Cytokines IL-2 IL-6 TNF-a
C- Reactive Protein CRP
7 The HB-adMSCs will be administered and the patient closely observed
One intravenous infusion of HB-adMSCs 2x108 cells to last 1 hour
- Saline Solution
8 The subject will then be monitored for a minimum of 2hr after infusion as follows
Measure Vital signs at minute 0 of infusion
Measure Vital signs at minute 15 after IV infusion
Measure Vital Signs at minute 30 after IV infusion
Measure Vital signs at minute 60 after IV infusion
Measure Vital signs at minute 120 after IV infusion Vital signs will be recorded more frequently if clinically indicated
9 Adverse event monitoring
10 24 hrs Telephone encounter The subject will be contacted by telephone the following day after the infusion visit to determine if any adverse events have occurred
11 ALS-specific Quality of Life Survey-revised ALSSQOL-R
12 A video recording will be made with the purpose to capture patients overall status gait range of motion assessments etc
One week following infusion PI will perform an assessment to determine patient status and discuss any changes since previous infusion
Infusion Visits Week 261014 and 18
1 Review and update medical history
2 Update concomitant medications list
3 Weight measurement
4 Vital signs Heart Rate Blood Pressure Respirations Temp SpO2
5 Physical examination ALS Functional Rating Scale-Revised ALSFRS-R
6 The HB-adMSCs will be administered and the patient closely observed
One intravenous infusion of HB-adMSCs 2x108 cells to last 1 hour
- Saline Solution
7 Adverse event monitoring
8 24 hrs Telephone encounter The subject will be contacted by telephone the following day after the infusion visit to determine if any adverse events have occurred
Infusion Visits Week 4 8 12 and 16
1 Review and update medical history
2 Update concomitant medications list
3 Weight measurement
4 Vital signs Heart Rate Blood Pressure Respirations Temp SpO2
5 Physical examination and ALS Functional Rating Scale-Revised ALSFRS-R
6 Blood samples will be collected for safety and efficacy assessments
Hematology
Chemistry
Coagulation Panel
Proinflammatory Cytokines IL-2 IL-6 TNF-a
C - Reactive Protein
7 The HB-adMSCs will be administered and the patient closely observed
One intravenous infusion of HB-adMSCs 2x108 cells to last 1 hour
- Saline Solution
8 ALS-specific Quality of Life Survey-revised ALSSQOL-R
9 Adverse event monitoring
10 24 hrs Telephone encounter The subject will be contacted by telephone the following day after the infusion visit to determine if any adverse events have occurred
Follow-Up Visit Week 22
1 You will be asked about your current health and medical history if any changes since last study visit or telephone call
2 You will be asked if there was any change in the medication list you have previously provided Concomitant medication list
3 Your blood pressure heart rate respiration rate temperature oxygen saturation and weight will be measured
4 The doctor will perform a physical examination driven by the signs andor symptoms you experience if any
5 The doctor will perform tests including the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised ALSFRS-R to measure the symptoms of ALS Disease on how you are able to perform
6 You will be asked to complete the Amyotrophic Lateral Sclerosis-Specific Quality of Life-Revised ALSSQOL-R questionnaire to assess the impact of Amyotrophic Lateral Sclerosis on your quality of life
7 You will be reminded to stay on your stable regimen of treatment throughout the study
Follow-Up Visit Week 26
1 You will be asked about your current health and medical history
2 You will be asked about the medicines that you have taken and are currently taking for Amyotrophic Lateral Sclerosis and for other health issues
3 Your blood pressure heart rate respiration rate oxygen saturation temperature and weight will be measured
4 The doctor will perform a complete physical examination
5 The doctor will perform tests including the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised ALSFRS-R to measure the symptoms of ALS Disease on how you are able to perform
6 You will be asked to complete the Amyotrophic Lateral Sclerosis-Specific Quality of Life-Revised ALSSQOL-R questionnaire to assess the impact of Amyotrophic Lateral Sclerosis on your quality of life
7 Laboratory test will be done Your blood will be tested to measure your overall health and efficacy markers TNF-a IL-6 IL-2 and C - Reactive Protein
8 You will be given an order for a Chest X ray PA single view and Magnetic Resonance Imaging to be performed If within 7 days of the End of study visit you have undergone any or both such reports would suffice this requirement
9 An Electrocardiogram ECG will be performed to evaluate cardiac function
10 An Electromyogram EMG will be performed to evaluate neuromuscular function
11 A video documenting of yourself walking and sitting will be performed
Follow-Up Visit Week 39
1 You will be asked about your current health and medical history if any changes since last study visit or telephone call
2 You will be asked if there was any change in the medication list you have previously provided Concomitant medication list
3 Your blood pressure heart rate respiration rate temperature oxygen saturation and weight will be measured
4 The doctor will perform a physical examination driven by the signs andor symptoms you experience if any
5 Laboratory test will be done Your blood will be tested to measure your overall health and efficacy markers TNF-a IL-6 IL-2 and C - Reactive Protein
6 The doctor will perform tests including the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised ALSFRS-R to measure the symptoms of ALS Disease on how you are able to perform
7 You will be asked to complete the Amyotrophic Lateral Sclerosis-Specific Quality of Life-Revised ALSSQOL-R questionnaire to assess the impact of Amyotrophic Lateral Sclerosis on your quality of life
8 You will be reminded to stay on your stable regimen of treatment throughout the study
End of Study Visit Week 52
1 Review and update medical history
2 Review and update concomitant medications list
3 Weight measurement
4 Vital signs Heart Rate Blood Pressure Respirations Temp SpO2
5 Physical examination ALS Functional Rating Scale-Revised ALSFRS-R
6 Blood samples will be collected for safety and efficacy assessments
Hematology
Coagulation Panel
Proinflammatory Cytokines IL-2 IL-6 TNF-a
C - Reactive Protein
7 ALS-specific Quality of Life Survey-revised ALSSQOL-R
8 Chest X-Ray PA single view
9 Electromyogram EMG
10 Magnetic Resonance Imaging MRI
11 Adverse event monitoring
12 A video recording will be made with the purpose to capture patients overall status gait range of motion assessments etc
BaselineInfusion 1 Visit
1 A verification of patient consent will be verbally performed and included in the progress note
2 Review of medical history and concomitant medications
3 Physical exam ALS Functional Rating Scale-Revised ALSFRS-R
4 Vital signs Heart Rate Blood Pressure Respirations Temp SpO2
5 Weight measurement
6 Blood samples will be collected for safety and efficacy assessments
Hematology
Chemistry
Coagulation Panel
Proinflammatory Cytokines IL-2 IL-6 TNF-a
C- Reactive Protein CRP
7 The HB-adMSCs will be administered and the patient closely observed
One intravenous infusion of HB-adMSCs 2x108 cells to last 1 hour
- Saline Solution
8 The subject will then be monitored for a minimum of 2hr after infusion as follows
Measure Vital signs at minute 0 of infusion
Measure Vital signs at minute 15 after IV infusion
Measure Vital Signs at minute 30 after IV infusion
Measure Vital signs at minute 60 after IV infusion
Measure Vital signs at minute 120 after IV infusion Vital signs will be recorded more frequently if clinically indicated
9 Adverse event monitoring
10 24 hrs Telephone encounter The subject will be contacted by telephone the following day after the infusion visit to determine if any adverse events have occurred
11 ALS-specific Quality of Life Survey-revised ALSSQOL-R
12 A video recording will be made with the purpose to capture patients overall status gait range of motion assessments etc
One week following infusion PI will perform an assessment to determine patient status and discuss any changes since previous infusion
Infusion Visits Week 261014 and 18
1 Review and update medical history
2 Update concomitant medications list
3 Weight measurement
4 Vital signs Heart Rate Blood Pressure Respirations Temp SpO2
5 Physical examination ALS Functional Rating Scale-Revised ALSFRS-R
6 The HB-adMSCs will be administered and the patient closely observed
One intravenous infusion of HB-adMSCs 2x108 cells to last 1 hour
- Saline Solution
7 Adverse event monitoring
8 24 hrs Telephone encounter The subject will be contacted by telephone the following day after the infusion visit to determine if any adverse events have occurred
Infusion Visits Week 4 8 12 and 16
1 Review and update medical history
2 Update concomitant medications list
3 Weight measurement
4 Vital signs Heart Rate Blood Pressure Respirations Temp SpO2
5 Physical examination and ALS Functional Rating Scale-Revised ALSFRS-R
6 Blood samples will be collected for safety and efficacy assessments
Hematology
Chemistry
Coagulation Panel
Proinflammatory Cytokines IL-2 IL-6 TNF-a
C - Reactive Protein
7 The HB-adMSCs will be administered and the patient closely observed
One intravenous infusion of HB-adMSCs 2x108 cells to last 1 hour
- Saline Solution
8 ALS-specific Quality of Life Survey-revised ALSSQOL-R
9 Adverse event monitoring
10 24 hrs Telephone encounter The subject will be contacted by telephone the following day after the infusion visit to determine if any adverse events have occurred
Follow-Up Visit Week 22
1 You will be asked about your current health and medical history if any changes since last study visit or telephone call
2 You will be asked if there was any change in the medication list you have previously provided Concomitant medication list
3 Your blood pressure heart rate respiration rate temperature oxygen saturation and weight will be measured
4 The doctor will perform a physical examination driven by the signs andor symptoms you experience if any
5 The doctor will perform tests including the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised ALSFRS-R to measure the symptoms of ALS Disease on how you are able to perform
6 You will be asked to complete the Amyotrophic Lateral Sclerosis-Specific Quality of Life-Revised ALSSQOL-R questionnaire to assess the impact of Amyotrophic Lateral Sclerosis on your quality of life
7 You will be reminded to stay on your stable regimen of treatment throughout the study
Follow-Up Visit Week 26
1 You will be asked about your current health and medical history
2 You will be asked about the medicines that you have taken and are currently taking for Amyotrophic Lateral Sclerosis and for other health issues
3 Your blood pressure heart rate respiration rate oxygen saturation temperature and weight will be measured
4 The doctor will perform a complete physical examination
5 The doctor will perform tests including the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised ALSFRS-R to measure the symptoms of ALS Disease on how you are able to perform
6 You will be asked to complete the Amyotrophic Lateral Sclerosis-Specific Quality of Life-Revised ALSSQOL-R questionnaire to assess the impact of Amyotrophic Lateral Sclerosis on your quality of life
7 Laboratory test will be done Your blood will be tested to measure your overall health and efficacy markers TNF-a IL-6 IL-2 and C - Reactive Protein
8 You will be given an order for a Chest X ray PA single view and Magnetic Resonance Imaging to be performed If within 7 days of the End of study visit you have undergone any or both such reports would suffice this requirement
9 An Electrocardiogram ECG will be performed to evaluate cardiac function
10 An Electromyogram EMG will be performed to evaluate neuromuscular function
11 A video documenting of yourself walking and sitting will be performed
Follow-Up Visit Week 39
1 You will be asked about your current health and medical history if any changes since last study visit or telephone call
2 You will be asked if there was any change in the medication list you have previously provided Concomitant medication list
3 Your blood pressure heart rate respiration rate temperature oxygen saturation and weight will be measured
4 The doctor will perform a physical examination driven by the signs andor symptoms you experience if any
5 Laboratory test will be done Your blood will be tested to measure your overall health and efficacy markers TNF-a IL-6 IL-2 and C - Reactive Protein
6 The doctor will perform tests including the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised ALSFRS-R to measure the symptoms of ALS Disease on how you are able to perform
7 You will be asked to complete the Amyotrophic Lateral Sclerosis-Specific Quality of Life-Revised ALSSQOL-R questionnaire to assess the impact of Amyotrophic Lateral Sclerosis on your quality of life
8 You will be reminded to stay on your stable regimen of treatment throughout the study
End of Study Visit Week 52
1 Review and update medical history
2 Review and update concomitant medications list
3 Weight measurement
4 Vital signs Heart Rate Blood Pressure Respirations Temp SpO2
5 Physical examination ALS Functional Rating Scale-Revised ALSFRS-R
6 Blood samples will be collected for safety and efficacy assessments
Hematology
Coagulation Panel
Proinflammatory Cytokines IL-2 IL-6 TNF-a
C - Reactive Protein
7 ALS-specific Quality of Life Survey-revised ALSSQOL-R
8 Chest X-Ray PA single view
9 Electromyogram EMG
10 Magnetic Resonance Imaging MRI
11 Adverse event monitoring
12 A video recording will be made with the purpose to capture patients overall status gait range of motion assessments etc
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None