A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS)

NCT03482050 | Completed Phase 1 DMC

• 1 other identifier: ASTRO-001-IL
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

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

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (5)

• Change in the ALS functional rating scale

  11 Months

• Change in predicted slow vital capacity (%SVC)

  11 Months

• Change in muscle strength grading by JAMAR grip strength

  11 Months

• Change in muscle strength grading by hand held dynamometer (HHD)

  11 Months

• Change in Quality of Life questionnaire (ALSAQ-40)

  11 Months

Study Arms (1)

AstroRx

EXPERIMENTAL

Biological: AstroRx

Interventions

AstroRx BIOLOGICAL

Astrocytes derived from human embryonic stem cells

AstroRx

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Kadimastem: lead

Study Sites (1)

Hadassah Ein Kerem Medical Center

Jerusalem, Israel

Location

Related Publications (1)

• 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.

  PMID: 36788520 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: March 14, 2018
• First Posted: March 29, 2018
• Study Start: April 12, 2018
• Primary Completion: June 22, 2020
• Study Completion: June 22, 2020
• Last Updated: January 15, 2021

Record last verified: 2021-01

Locations

IL (1)