A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS)
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|ClinicalTrials.gov Identifier: NCT03482050|
Recruitment Status : Recruiting
First Posted : March 29, 2018
Last Update Posted : August 8, 2018
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
|Condition or disease||Intervention/treatment||Phase|
|ALS (Amyotrophic Lateral Sclerosis)||Biological: AstroRx||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||21 participants|
|Intervention Model:||Sequential Assignment|
|Intervention 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)|
|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)|
|Actual Study Start Date :||April 12, 2018|
|Estimated Primary Completion Date :||August 2020|
|Estimated Study Completion Date :||August 2020|
Astrocytes derived from human embryonic stem cells
- Number of participants with treatment emergent adverse events as assessed by CTCAE Version 4.03 [ Time Frame: 11 Months ]Safety and tolerability assessment will be based on treatment emerged adverse events
- 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.
- Change in predicted slow vital capacity (%SVC) [ Time Frame: 11 Months ]Preliminary efficacy assessment to measure respiratory muscle strength
- Change in muscle strength grading by JAMAR grip strength [ Time Frame: 11 Months ]Preliminary efficacy assessment to measure hand grip strength
- Change in muscle strength grading by hand held dynamometer (HHD) [ Time Frame: 11 Months ]Preliminary efficacy assessment to measure muscle strength in limb muscles
- Change in Quality of Life questionnaire (ALSAQ-40) [ Time Frame: 11 Months ]Preliminary efficacy assessment to measure the subjective well-being of patients
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03482050
|Contact: Yael Feinsod||+972-2-6779372||HCRC_ALS@hadassah.org.il|
|Hadassah Ein Kerem Medical Center||Recruiting|
|Contact: Yael Feinsod|
|Principal Investigator: Marc Gotkine, MD|