- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03482050
A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS)
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)
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
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
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-
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Jerusalem, Israel
- Hadassah Ein Kerem Medical Center
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: AstroRx
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Astrocytes derived from human embryonic stem cells
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of participants with treatment emergent adverse events as assessed by CTCAE Version 4.03
Time Frame: 11 Months
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Safety and tolerability assessment will be based on treatment emerged adverse events
|
11 Months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in the ALS functional rating scale
Time Frame: 11 Months
|
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.
|
11 Months
|
Change in predicted slow vital capacity (%SVC)
Time Frame: 11 Months
|
Preliminary efficacy assessment to measure respiratory muscle strength
|
11 Months
|
Change in muscle strength grading by JAMAR grip strength
Time Frame: 11 Months
|
Preliminary efficacy assessment to measure hand grip strength
|
11 Months
|
Change in muscle strength grading by hand held dynamometer (HHD)
Time Frame: 11 Months
|
Preliminary efficacy assessment to measure muscle strength in limb muscles
|
11 Months
|
Change in Quality of Life questionnaire (ALSAQ-40)
Time Frame: 11 Months
|
Preliminary efficacy assessment to measure the subjective well-being of patients
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11 Months
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- ASTRO-001-IL
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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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