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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AstroRx | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AstroRx | Biological | Astrocytes derived from human embryonic stem cells |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 11 Months |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 11 Months |
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Main Inclusion Criteria:
Main Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hadassah Ein Kerem Medical Center | Jerusalem | Israel |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36788520 | Derived | Gotkine M, Caraco Y, Lerner Y, Blotnick S, Wanounou M, Slutsky SG, Chebath J, Kuperstein G, Estrin E, Ben-Hur T, Hasson A, Molakandov K, Sonnenfeld T, Stark Y, Revel A, Revel M, Izrael M. Safety and efficacy of first-in-man intrathecal injection of human astrocytes (AstroRx(R)) in ALS patients: phase I/IIa clinical trial results. J Transl Med. 2023 Feb 14;21(1):122. doi: 10.1186/s12967-023-03903-3. |
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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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.
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| Change in predicted slow vital capacity (%SVC) | Preliminary efficacy assessment to measure respiratory muscle strength | 11 Months |
| Change in muscle strength grading by JAMAR grip strength | Preliminary efficacy assessment to measure hand grip strength | 11 Months |
| Change in muscle strength grading by hand held dynamometer (HHD) | Preliminary efficacy assessment to measure muscle strength in limb muscles | 11 Months |
| Change in Quality of Life questionnaire (ALSAQ-40) | Preliminary efficacy assessment to measure the subjective well-being of patients | 11 Months |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |