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

NCT ID: NCT03482050

Last Updated: 2021-01-15

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-04-12
• Study Completion Date: 2020-06-22

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)

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

AstroRx

Group Type: EXPERIMENTAL

AstroRx

Intervention Type: BIOLOGICAL

Astrocytes derived from human embryonic stem cells

Interventions

AstroRx

Astrocytes derived from human embryonic stem cells

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

1. El Escorial criteria for probable or definite ALS

2. Males and and non pregnant females between 18 and 70 years of age

3. Patients with an ALS-FRS-R score of at least 30 with an ALS diagnosis of two years or less

4. No history of active psychiatric disorder. Patients receiving antidepressants as a preventive treatment, with no history of active psychiatric disorder may be included.

5. Patient has a good understanding of the study and nature of the procedure

6. Patient provides written informed consent prior to any study procedure

7. 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®

8. Patient is medically able to tolerate immunosuppression regimen

9. Presence of a willing and able caregiver who understands the need to attend all follow-up visits, even if mobility declines

Exclusion Criteria

1. Patient has a past infection or a positive test for HBV,HCV or HIV

2. Patient is in need of respiratory support

3. Patient has a lower than 10/12 in ALS-FRS-R respiratory parameters or below 70% of predicted slow vital capacity (SVC)

4. Patient has renal failure

5. Patient has impaired hepatic function

6. Patient has a Body Mass Index (BMI) of <18.5 or > 30

7. 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

8. Patient has systemic inflammation or active infections

9. Patient has been treated previously with any stem cell therapy

10. Current use of immunosuppressant medication or use of such medication within 6 weeks of Screening visit (Visit 0)

11. 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

12. Any known immunodeficiency syndrome

13. Any concomitant disease or condition limiting patient safety to participate

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Kadimastem

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Hadassah Ein Kerem Medical Center

Jerusalem, , Israel

Countries

Israel

References

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.

Reference Type: DERIVED

PMID: 36788520 (View on PubMed)

Other Identifiers

ASTRO-001-IL

Identifier Type: -

Identifier Source: org_study_id