Individual Patient Expanded Access IND of Autologous HBadMSCs for the Treatment of Amyotrophic Lateral Sclerosis

Studies

Study First Submitted Date 2020-08-12
Study First Posted Date 2020-08-17
Last Update Posted Date 2022-09-26
Verification Month Year September 2022
Verification Date 2022-09-30
Last Update Posted Date 2022-09-26

Detailed Descriptions

Sequence: 20838131
Description Once the eligibility is confirmed, approximately 1-2 weeks after the screening visit, the subject will return for the baseline/first 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. Baseline/Infusion 1 Visit A verification of patient consent will be verbally performed and included in the progress note. Review of medical history, and concomitant medications. Physical exam + ALS Functional Rating Scale-Revised (ALSFRS-R) Vital signs (Heart Rate, Blood Pressure, Respirations, Temp., SpO2) Weight measurement 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) The HB-adMSCs will be administered and the patient closely observed: • One intravenous infusion of HB-adMSCs (2×108 cells) to last 1 hour: – Saline Solution. 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). Adverse event monitoring 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. ALS-specific Quality of Life Survey-revised (ALSSQOL-R) A video recording will be made with the purpose to capture patient’s 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 2,6,10,14 and 18 Review and update medical history Update concomitant medications list Weight measurement Vital signs (Heart Rate, Blood Pressure, Respirations, Temp., SpO2) Physical examination + ALS Functional Rating Scale-Revised (ALSFRS-R) The HB-adMSCs will be administered and the patient closely observed: • One intravenous infusion of HB-adMSCs (2×108 cells) to last 1 hour: – Saline Solution. Adverse event monitoring 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 Review and update medical history Update concomitant medications list Weight measurement Vital signs (Heart Rate, Blood Pressure, Respirations, Temp., SpO2) Physical examination and ALS Functional Rating Scale-Revised (ALSFRS-R) 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 The HB-adMSCs will be administered and the patient closely observed: • One intravenous infusion of HB-adMSCs (2×108 cells) to last 1 hour: – Saline Solution. ALS-specific Quality of Life Survey-revised (ALSSQOL-R) Adverse event monitoring 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 You will be asked about your current health and medical history (if any changes since last study visit or telephone call). You will be asked if there was any change in the medication list you have previously provided (Concomitant medication list). Your blood pressure, heart rate, respiration rate, temperature, oxygen saturation and weight will be measured. The doctor will perform a physical examination driven by the signs and/or symptoms you experience, if any. 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. 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. You will be reminded to stay on your stable regimen of treatment throughout the study Follow-Up Visit Week 26 You will be asked about your current health and medical history. You will be asked about the medicines that you have taken and are currently taking for Amyotrophic Lateral Sclerosis and for other health issues. Your blood pressure, heart rate, respiration rate, oxygen saturation, temperature, and weight will be measured. The doctor will perform a complete physical examination. 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. 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. 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). 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. An Electrocardiogram (ECG) will be performed to evaluate cardiac function. An Electromyogram (EMG) will be performed to evaluate neuromuscular function. A video documenting of yourself walking and sitting will be performed. Follow-Up Visit Week 39 You will be asked about your current health and medical history (if any changes since last study visit or telephone call). You will be asked if there was any change in the medication list you have previously provided (Concomitant medication list). Your blood pressure, heart rate, respiration rate, temperature, oxygen saturation and weight will be measured. The doctor will perform a physical examination driven by the signs and/or symptoms you experience, if any. 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). 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. 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. You will be reminded to stay on your stable regimen of treatment throughout the study End of Study Visit Week 52 Review and update medical history Review and update concomitant medications list Weight measurement Vital signs (Heart Rate, Blood Pressure, Respirations, Temp., SpO2) Physical examination + ALS Functional Rating Scale-Revised (ALSFRS-R) Blood samples will be collected for safety and efficacy assessments: Hematology Coagulation Panel Proinflammatory Cytokines (IL-2, IL-6, TNF-a) C – Reactive Protein ALS-specific Quality of Life Survey-revised (ALSSQOL-R) Chest X-Ray (PA single view) Electromyogram (EMG) Magnetic Resonance Imaging (MRI) Adverse event monitoring A video recording will be made with the purpose to capture patient’s overall status (gait, range of motion assessments, etc.).

Facilities

Sequence: 201176986
Name River Oaks Hospital and Clinics
City Houston
State Texas
Zip 77027
Country United States

Conditions

Sequence: 52469759
Name Amyotrophic Lateral Sclerosis
Downcase Name amyotrophic lateral sclerosis

Id Information

Sequence: 40372690
Id Source org_study_id
Id Value HBALS01

Countries

Sequence: 42808162
Name United States
Removed False

Interventions

Sequence: 52780034
Intervention Type Biological
Name HB-adMSCs
Description Hope Biosciences autologous adipose-derived mesenchymal stem cells

Keywords

Sequence: 80273963 Sequence: 80273964 Sequence: 80273965 Sequence: 80273966 Sequence: 80273967 Sequence: 80273968
Name Neurodegenerative Name MSCs Name Stem cells Name Sclerosis Name ALS Name Amyotrophic
Downcase Name neurodegenerative Downcase Name mscs Downcase Name stem cells Downcase Name sclerosis Downcase Name als Downcase Name amyotrophic

Browse Conditions

Sequence: 194627715 Sequence: 194627716 Sequence: 194627717 Sequence: 194627718 Sequence: 194627719 Sequence: 194627720 Sequence: 194627721 Sequence: 194627722 Sequence: 194627723 Sequence: 194627724 Sequence: 194627725 Sequence: 194627726
Mesh Term Motor Neuron Disease Mesh Term Amyotrophic Lateral Sclerosis Mesh Term Sclerosis Mesh Term Pathologic Processes Mesh Term Neurodegenerative Diseases Mesh Term Nervous System Diseases Mesh Term Neuromuscular Diseases Mesh Term Spinal Cord Diseases Mesh Term Central Nervous System Diseases Mesh Term TDP-43 Proteinopathies Mesh Term Proteostasis Deficiencies Mesh Term Metabolic Diseases
Downcase Mesh Term motor neuron disease Downcase Mesh Term amyotrophic lateral sclerosis Downcase Mesh Term sclerosis Downcase Mesh Term pathologic processes Downcase Mesh Term neurodegenerative diseases Downcase Mesh Term nervous system diseases Downcase Mesh Term neuromuscular diseases Downcase Mesh Term spinal cord diseases Downcase Mesh Term central nervous system diseases Downcase Mesh Term tdp-43 proteinopathies Downcase Mesh Term proteostasis deficiencies Downcase Mesh Term metabolic diseases
Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor

Sponsors

Sequence: 48596760 Sequence: 48596761
Agency Class OTHER Agency Class INDUSTRY
Lead Or Collaborator lead Lead Or Collaborator collaborator
Name Hope Biosciences Stem Cell Research Foundation Name Hope Biosciences

Overall Officials

Sequence: 29441823
Role Principal Investigator
Name Rajiv Thakur, MD
Affiliation River Oaks Hospital and Clinics

Eligibilities

Sequence: 30936685
Gender All
Minimum Age N/A
Maximum Age N/A
Criteria Inclusion Criteria: Documented diagnosis of Amyotrophic Lateral Sclerosis. Capable of giving informed consent (signed, verbal or assent as applicable and as listed in the protocol) Exclusion Criteria: Other acute or chronic medical conditions that, in the opinion of the investigator, may increase the risks associated with study participation or HB-adMSCs administration. Any abnormal, inexplicable laboratory result that, in the opinion of the investigator, may increase the risks associated with study participation or HB-adMSCs administration. Participation in other interventional research studies within the past 30 days. Unwillingness to return for follow-up visits.
Adult True
Child True
Older Adult True

Calculated Values

Sequence: 254229624
Number Of Facilities 1
Registered In Calendar Year 2020
Were Results Reported False
Has Us Facility True
Has Single Facility True

Responsible Parties

Sequence: 29049037
Responsible Party Type Sponsor