NCT03482050CompletedPhase 1
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)
Kadimastem1 site in 1 country16 target enrollmentStarted: April 12, 2018Last updated:
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Completed
- Sponsor
- Enrollment
- 16
- Locations
- 1
- Primary Endpoint
- Number of participants with treatment emergent adverse events as assessed by CTCAE Version 4.03
Study Overview
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
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 70 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion 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
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
Outcomes
Primary Outcomes
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
Secondary Outcomes
- Change in muscle strength grading by JAMAR grip strength(11 Months)
- Change in Quality of Life questionnaire (ALSAQ-40)(11 Months)
- Change in predicted slow vital capacity (%SVC)(11 Months)
- Change in the ALS functional rating scale(11 Months)
- Change in muscle strength grading by hand held dynamometer (HHD)(11 Months)
Investigators
Study Sites (1)
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