A Phase II Study of Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis

Study Purpose:

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.

Disease:

Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Stem Cell

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Nathan P Staff, MD, PhD, Mayo Clinic
Anthony J Windebank, MD, Mayo Clinic

Clinicaltrials.gov ID (11 digit #):

NCT03268603

Neals Affiliated?

Coordinating Center Contact Information

Mayo Clinic in Rochester
Michelle Turner / .(JavaScript must be enabled to view this email address)
Carol Denny / .(JavaScript must be enabled to view this email address) Rochester, Minnesota 55905 United States

Full Study Summary:

The Goal of the Proposed Study is to perform an open label, 60 subject, Phase II multi-site clinical trial to investigate the safety and efficacy of intrathecal treatment of aaMSCs in ALS. Patients will be treated with 1 x 10^8 aaMSCs every 3 months for a total of 4 intrathecal injections over 12 months. Reduced dose treatments will be allowed based on specific adverse events. Multiple biomarkers will be tracked throughout the clinical trial and correlated with response to treatment. This study will initially be performed at Mayo Clinic in Rochester. Subsequently, the study will expand to the two other Mayo Clinic sites in Arizona and Florida as the manufacturing capabilities are finalized.

Study Sponsor:

Mayo Clinic

Participant Duration:

Estimated Enrollment:

Estimated Study Start Date:

10/10/2017

Estimated Study Completion Date:

12/31/2022

Posting Last Modified Date:

05/12/2020

Date Study Added to alsconsortium.org:

10/31/2017

Eligibility Criteria

Gender:
Female, Male
Minimum Age:
18
Maximum Age:
N/A
Min Vital Capacity (% predicted normal):
65

Time since Symptom Onset:

Time since Diagnosis:

Can participants use Riluzole?
Yes

Inclusion Criteria:

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).
Able to comply with protocol requirements, including MRI testing.
Can provide written informed consent.

Exclusion Criteria:

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 or other unapproved drugs
Enrolled in an investigational drug trial within 30 days of baseline visit
Prior stem cell therapy of any kind
Kokmen Short Test of Mental Status score <32
Presence of a tracheostomy
Ventilator dependent
Pregnancy
Men or women of childbearing potential who are unwilling to employ adequate contraception
Site Contact Information

Mayo Clinic
Tom Osgood, CCRP / .(JavaScript must be enabled to view this email address) / 480-301-4992
Alex Evans / .(JavaScript must be enabled to view this email address) / 480-301-4750
Scottsdale, Arizona 85259
United States

Mayo Clinic
Lisa Thuro / .(JavaScript must be enabled to view this email address) / 904-953-8630
Adrienne Graham / .(JavaScript must be enabled to view this email address) / 904-953-3726
Jacksonville, Florida 32224
United States

Mayo Clinic
Michelle Turner / .(JavaScript must be enabled to view this email address) / Carol Denny / .(JavaScript must be enabled to view this email address) / Rochester, Minnesota 55905
United States