Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis (ALS)

NCT03268603 | Active Not Recruiting Phase 2 Results FDA Drug

• 1 other identifier: 15-008008
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 3

Brief Summary

The purpose of this study is to determine the safety and efficacy of intrathecal treatment delivered to the cerebrospinal fluid (CSF) of mesenchymal stem cells in ALS patients every 3 months for a total of 4 injections over 12 months. Mesenchymal stem cells (MSCs) are a type of stem cell that can be grown into a number of different kinds of cells. In this study, MSCs will be taken from the subject's body fat and grown. CSF is the fluid surrounding the spine. The use of mesenchymal stem cells is considered investigational, which means it has not been approved by the Food and Drug Administration (FDA) for routine clinical use. However, the FDA has allowed the use of mesenchymal stem cells in this research study.

Conditions

ALS; Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (2)

• Adverse Events

  The number of adverse events experienced by subjects

  12-months

• Unexpected Severe Adverse Events

  The number of unexpected severe adverse events experienced by subjects

  12-months

Secondary Outcomes (1)

• Change in Slope of ALS Functional Rating Scale - Revised (ALSFRS-R)

  baseline, approximately 1 year

Study Arms (1)

Mesenchymal Stromal Cells

EXPERIMENTAL

Autologous Adipose-derived Mesenchymal Stromal Cells (aaMSCs) will be administered intrathecally at a single dose in a volume of 5-10 mL, all patients will receive 5 x 10\^7 intrathecal aaMSCs at the first injection (Visit 4). Subsequent doses may be reduced to 1 x 10\^7 or increased to 1 x 10\^8, based on Dose Modification Rules.

Drug: Autologous Adipose-derived Mesenchymal Stromal Cells

Interventions

Autologous Adipose-derived Mesenchymal Stromal Cells DRUG

The investigational product consists of autologous adipose-derived Mesenchymal Stromal Cells (MSCs), suspended in 5-10 mL Lactated Ringer's. The MSC are provided in a sterile syringe labelled with appropriate patient and product identifiers ready for intrathecal injection.

Mesenchymal Stromal Cells

Eligibility Criteria

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

You may qualify if:

• All patients will have ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by the World Federation of Neurology criteria for the diagnosis of ALS.
• Examination and neurophysiological testing confirm a pure motor syndrome compatible with the diagnosis of ALS. All other possible causes of weakness have been excluded by extensive investigations.
• Age greater than 18 years, if female, must be post-menopausal, had a hysterectomy, or agree to two forms of birth control.
• Permanent resident or citizen of the United States.
• Geographic accessibility to the study site and willingness and ability to comply with follow-up.
• History of a chronic onset of a progressive motor weakness of less than two years duration.
• Subjects must be taking a stable dose of riluzole for at least 30 days prior to enrolment or not be on riluzole, and not have been on it for at least 30 days prior to enrolment (riluzole-naïve subjects are permitted in the study).
• Subjects must be taking a stable dose of oral Radicava® (edaravone) for at least 30 days prior to enrolment or not be on oral Radicava® (edaravone), and not have been on it for at least 30 days prior to enrolment (edaravone-naïve subjects are permitted in the study).
• Able to comply with protocol requirements, including MRI testing.
• Can provide written informed consent.

You may not qualify if:

• Use of Radicava® (edaravone) within 30 days of screening or intent to use Radicava® at any time during the course of the study including the follow up period.
• Any clinically significant medical condition (e.g., within six months of baseline, had myocardial infarction, angina pectoris, and/or congestive heart failure) that, in the opinion of the investigator, would compromise the safety of patient.
• Pulmonary Slow Vital Capacity (SVC) less than 65% of predicted for age, gender, and body type.
• Autoimmunity, including Crohn's disease or rheumatoid arthritis
• Current use of immunosuppressant medication or use of such medication within 4 weeks of Screening visit (Visit 1).
• Malignancy 5 years prior to enrollment, including melanoma,with the exception of localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline).
• Active systemic or local infection near the lumbar puncture site.
• Inability to lie flat for the duration of intrathecal cell transplantation, or inability to tolerate study procedures for any other reason.
• Other active systemic disease as defined by laboratory abnormalities delineated in Appendix IV.
• Use of herbal medications, nutritional supplements or other unapproved drugs or investigational medicinal products being used or studied for the treatment of ALS.
• Unwilling to forgo initiating the use of any new supplements during participation in the study.
• Enrolled in an investigational drug trial within 30 days of baseline visit
• Prior stem cell therapy for a neurological disease
• Kokmen Short Test of Mental Status score \<32
• Presence of a tracheostomy

Sponsors & Collaborators

• Mayo Clinic: lead
• State of Minnesota Regenerative Medicine Minnesota: collaborator

Study Sites (3)

Mayo Clinic

Scottsdale, Arizona, 85259, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

• Mayo Clinic Clinical Trials

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

Results Point of Contact

• Title: Nathan Staff, M.D., Ph.D.
• Organization: Mayo Clinic

Staff.Nathan@mayo.edu

507-293-1283

Study Officials

• Nathan P Staff, MD, PhD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

• Anthony J Windebank, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Neurology

Study Record Dates

• First Submitted: August 30, 2017
• First Posted: August 31, 2017
• Study Start: October 10, 2017
• Primary Completion: January 31, 2024
• Study Completion (Estimated): December 31, 2026
• Last Updated: March 9, 2026
• Results First Posted: April 2, 2025

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)