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Expanded Access Protocol: Repeated Administration of Nurown® (Autologous MSC-NTF Cells) for the Treatment of ALS

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ClinicalTrials.gov Identifier: NCT04681118
Expanded Access Status : Available
First Posted : December 23, 2020
Last Update Posted : December 23, 2020
Sponsor:
Information provided by (Responsible Party):
Brainstorm-Cell Therapeutics

Brief Summary:
Expanded Access for treatment with investigational product MSC-NTF cells(NurOwn®) for participants who completed all scheduled treatments and follow-up assessments in BCT-002-US study

Condition or disease Intervention/treatment
Amyotrophic Lateral Sclerosis Biological: NurOwn (MSC-NTF cells)

Detailed Description:

This is an Intermediate-size patient population Expanded Access Protocol (EAP) to provide NurOwn® (MSC-NTF cells) for eligible ALS participants who completed the BCT-002-US phase 3 ALS clinical trial and have received all three IT treatments.

The participants will undergo up to three intrathecal (IT) treatments with NurOwn® (MSC-NTF cells).

The first treatment will be at Visit 3, approximately 5-6 weeks after the BMA (Visit 2), with the subsequent treatments at Visit 4 and Visit 5.

Following the third and last treatment, the participant will be followed for three additional monthly visits (in-person, if feasible, or by telephone call or telemedicine in consideration of the ongoing COVID-19 pandemic) through week 28, during which the ALSFRS-R and safety assessments will be collected.

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population, Treatment IND/Protocol
Official Title: Intermediate-size Patient Population Expanded Access Protocol: Repeated Administration of Nurown® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors) for the Treatment of Amyotrophic Lateral Sclerosis (ALS)



Intervention Details:
  • Biological: NurOwn (MSC-NTF cells)
    Intrathecal administration

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 63 Years   (Adult)
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

1. Participation in the BCT-002-US study, successful completion of all scheduled treatments and follow-up assessments.

2 Able to provide to the Investigator written informed consent regarding the investigational drug, or, as applicable, on whose behalf a legally authorized representative of the participant has provided such consent.

3. Able to safely undergo a bone marrow aspiration.

Exclusion Criteria:

  1. Inability to lie flat for the duration of intrathecal cell transplantation and/or bone marrow biopsy, or inability to tolerate treatment procedures for any other reason (lying flat with BiPAP or NIV are not exclusionary).
  2. History of clinically significant autoimmune disease (excluding thyroid disease) myelodysplastic or myeloproliferative disorder, leukemia or lymphoma, whole body irradiation, hip fracture, or severe scoliosis.
  3. Any unstable clinically significant medical condition other than ALS (e.g., within six months of baseline, had myocardial infarction, angina pectoris, and/or congestive heart failure), treatment with anticoagulants that, in the opinion of the Investigator, would compromise the safety of the participant.
  4. Any history of malignancy within the previous 5 years, with the exception of non- melanoma localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline).
  5. Current use of immunosuppressant medication or use of such medication within 4 weeks of the pre-treatment visit.
  6. Any history of acquired or inherited immune deficiency syndrome.
  7. Tracheostomy and/or mechanical ventilation. Feeding tube use, BiPAP or NIV are not exclusionary.
  8. Pregnant women or women currently breastfeeding or unwilling to use effective birth control methods during the treatment (if of childbearing age).
  9. Positive test result for Hepatitis B virus (HBV; surface antigen (HBsAg) and IgM antibodies to core antigen (IgM anti-HBc)), Hepatitis C virus (HCV), Human Immune deficiency virus (HIV) 1 and 2.

Information from the National Library of Medicine

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): NCT04681118


Contacts
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Contact: Mary Kay Turner 201-488-0460 ext 105 mkt@brainstorm-cell.com
Contact: Yael Gothelf, Ph.D. 646-666-3188 ext 111 ygothelf@brainstorm-cell.com

Locations
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United States, California
University of California Irvine Alpha Stem Cell Clinic Available
Irvine, California, United States, 92697
Principal Investigator: Namita A Goyal         
Cedars-Sinai Medical Center Available
Los Angeles, California, United States, 90048
Principal Investigator: Matthew H Burford, MD         
California Pacific Medical Center Available
San Francisco, California, United States, 94115
Principal Investigator: Jonathan Katz, MD         
United States, Massachusetts
Massachusetts General Hospital Available
Boston, Massachusetts, United States, 02115
Principal Investigator: James D Berry, MD         
UMass Medical School Available
Worcester, Massachusetts, United States, 01655
Principal Investigator: Robert H Brown, D.Phil., M.D         
United States, Minnesota
Mayo Clinic Available
Rochester, Minnesota, United States, 55905
Principal Investigator: Anthony Windebank, MD         
Sponsors and Collaborators
Brainstorm-Cell Therapeutics
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Responsible Party: Brainstorm-Cell Therapeutics
ClinicalTrials.gov Identifier: NCT04681118    
Other Study ID Numbers: BCT-003-US
First Posted: December 23, 2020    Key Record Dates
Last Update Posted: December 23, 2020
Last Verified: December 2020
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases