Back to resultsA Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS)
Primary Purpose
ALS (Amyotrophic Lateral Sclerosis)
Status
Completed
Phase
Phase 1
Locations
Israel
Study Type
Interventional
Intervention
AstroRx
Sponsored by
About this trial
This is an interventional treatment trial for ALS (Amyotrophic Lateral Sclerosis)
Eligibility Criteria
Main Inclusion Criteria:
- El Escorial criteria for probable or definite ALS
- Males and and non pregnant females between 18 and 70 years of age
- Patients with an ALS-FRS-R score of at least 30 with an ALS diagnosis of two years or less
- No history of active psychiatric disorder. Patients receiving antidepressants as a preventive treatment, with no history of active psychiatric disorder may be included.
- Patient has a good understanding of the study and nature of the procedure
- Patient provides written informed consent prior to any study procedure
- Patients should either be on a stable dose of Riluzole and/or Radicava® (if applicable) for at least 30 days, or not be treated with Riluzole and/or Radicava®
- Patient is medically able to tolerate immunosuppression regimen
- Presence of a willing and able caregiver who understands the need to attend all follow-up visits, even if mobility declines
Main Exclusion Criteria:
- Patient has a past infection or a positive test for HBV,HCV or HIV
- Patient is in need of respiratory support
- Patient has a lower than 10/12 in ALS-FRS-R respiratory parameters or below 70% of predicted slow vital capacity (SVC)
- Patient has renal failure
- Patient has impaired hepatic function
- Patient has a Body Mass Index (BMI) of <18.5 or > 30
- Patient suffers from significant cardiac disease, diabetes, autoimmune diseases, chronic severe infection, malignant disease or any other disease or condition that may risk the patient or interfere with the ability to interpret the study results
- Patient has systemic inflammation or active infections
- Patient has been treated previously with any stem cell therapy
- Current use of immunosuppressant medication or use of such medication within 6 weeks of Screening visit (Visit 0)
- Patient has participated in another clinical treatment trial or received other experimental medications outside of a clinical trial within 1 month prior to start of this study
- Any known immunodeficiency syndrome
- Any concomitant disease or condition limiting patient safety to participate
Sites / Locations
- Hadassah Ein Kerem Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
AstroRx
Arm Description
Outcomes
Primary Outcome Measures
Number of participants with treatment emergent adverse events as assessed by CTCAE Version 4.03
Safety and tolerability assessment will be based on treatment emerged adverse events
Secondary Outcome Measures
Change in the ALS functional rating scale
Preliminary efficacy assessment to measure response to treatment or progression of disease. Scale includes 12 questions related to tasks. Each task rated on a five-point scale from 0 = unable to do the task, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0 = worst and 48 = best.
Change in predicted slow vital capacity (%SVC)
Preliminary efficacy assessment to measure respiratory muscle strength
Change in muscle strength grading by JAMAR grip strength
Preliminary efficacy assessment to measure hand grip strength
Change in muscle strength grading by hand held dynamometer (HHD)
Preliminary efficacy assessment to measure muscle strength in limb muscles
Change in Quality of Life questionnaire (ALSAQ-40)
Preliminary efficacy assessment to measure the subjective well-being of patients
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03482050
Brief Title
A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS)
Official Title
A Phase I/IIa, Open Label, Dose-escalating Clinical Study to Evaluate the Safety, Tolerability and Theraputic Effects of Transplantation of Astrocytes Derived From Human Embryonic Stem Cells (hESC), in Patients With Amyotrophic Lateral Sclerosis (ALS)
Study Type
Interventional
2. Study Status
Record Verification Date
January 2021
Overall Recruitment Status
Completed
Study Start Date
April 12, 2018 (Actual)
Primary Completion Date
June 22, 2020 (Actual)
Study Completion Date
June 22, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Kadimastem
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This is a study of transplantation of Astrocytes derived from human embryonic stem cells, in patients with Amyotrophic Lateral Sclerosis (ALS).
There will be no change in the routine ALS treatment of the patients enrolled into the study. Treatment will be administered in addition to the appropriate standard of care treatment.
The study hypothesis is that transplantation of Astrocyte(AstroRx) cells can compensate for the malfunctioning of patients' own astrocytes by restoring physiological capabilities like the reuptake of excessive glutamate, reducing oxidative stress, reducing other toxic compounds, as well as by secreting different neuroprotective factors
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
ALS (Amyotrophic Lateral Sclerosis)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Model Description
Dose-escalating, four subject-groups clinical study; a single treatment administration of AstroRx, administered in an escalating low, medium and high dose or two consecutive administrations of the medium dose separated by an interval. AstroRx will be administered by intrathecal (spinal) injection to subjects with ALS at the early disease stage.
Masking
None (Open Label)
Allocation
N/A
Enrollment
16 (Actual)
8. Arms, Groups, and Interventions
Arm Title
AstroRx
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
AstroRx
Intervention Description
Astrocytes derived from human embryonic stem cells
Primary Outcome Measure Information:
Title
Number of participants with treatment emergent adverse events as assessed by CTCAE Version 4.03
Description
Safety and tolerability assessment will be based on treatment emerged adverse events
Time Frame
11 Months
Secondary Outcome Measure Information:
Title
Change in the ALS functional rating scale
Description
Preliminary efficacy assessment to measure response to treatment or progression of disease. Scale includes 12 questions related to tasks. Each task rated on a five-point scale from 0 = unable to do the task, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0 = worst and 48 = best.
Time Frame
11 Months
Title
Change in predicted slow vital capacity (%SVC)
Description
Preliminary efficacy assessment to measure respiratory muscle strength
Time Frame
11 Months
Title
Change in muscle strength grading by JAMAR grip strength
Description
Preliminary efficacy assessment to measure hand grip strength
Time Frame
11 Months
Title
Change in muscle strength grading by hand held dynamometer (HHD)
Description
Preliminary efficacy assessment to measure muscle strength in limb muscles
Time Frame
11 Months
Title
Change in Quality of Life questionnaire (ALSAQ-40)
Description
Preliminary efficacy assessment to measure the subjective well-being of patients
Time Frame
11 Months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Main Inclusion Criteria:
El Escorial criteria for probable or definite ALS
Males and and non pregnant females between 18 and 70 years of age
Patients with an ALS-FRS-R score of at least 30 with an ALS diagnosis of two years or less
No history of active psychiatric disorder. Patients receiving antidepressants as a preventive treatment, with no history of active psychiatric disorder may be included.
Patient has a good understanding of the study and nature of the procedure
Patient provides written informed consent prior to any study procedure
Patients should either be on a stable dose of Riluzole and/or Radicava® (if applicable) for at least 30 days, or not be treated with Riluzole and/or Radicava®
Patient is medically able to tolerate immunosuppression regimen
Presence of a willing and able caregiver who understands the need to attend all follow-up visits, even if mobility declines
Main Exclusion Criteria:
Patient has a past infection or a positive test for HBV,HCV or HIV
Patient is in need of respiratory support
Patient has a lower than 10/12 in ALS-FRS-R respiratory parameters or below 70% of predicted slow vital capacity (SVC)
Patient has renal failure
Patient has impaired hepatic function
Patient has a Body Mass Index (BMI) of <18.5 or > 30
Patient suffers from significant cardiac disease, diabetes, autoimmune diseases, chronic severe infection, malignant disease or any other disease or condition that may risk the patient or interfere with the ability to interpret the study results
Patient has systemic inflammation or active infections
Patient has been treated previously with any stem cell therapy
Current use of immunosuppressant medication or use of such medication within 6 weeks of Screening visit (Visit 0)
Patient has participated in another clinical treatment trial or received other experimental medications outside of a clinical trial within 1 month prior to start of this study
Any known immunodeficiency syndrome
Any concomitant disease or condition limiting patient safety to participate
Facility Information:
Facility Name
Hadassah Ein Kerem Medical Center
City
Jerusalem
Country
Israel
12. IPD Sharing Statement
Learn more about this trial
A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS)
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