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

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ClinicalTrials.gov Identifier: NCT03268603
Recruitment Status : Recruiting
First Posted : August 31, 2017
Last Update Posted : September 19, 2017
Sponsor:
Collaborator:
Information provided by (Responsible Party):

Study Description
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.


Condition or disease Intervention/treatment Phase
ALS Amyotrophic Lateral Sclerosis Drug: Autologous Adipose-derived Mesenchymal Stromal Cells Phase 2

Detailed Description:
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 Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study of Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis
Anticipated Study Start Date : September 2017
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019


Arms and Interventions

Arm Intervention/treatment
Experimental: Mesenchymal Stromal Cells
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 1 x 10^8 intrathecal aaMSCs at the first injection (Visit 4). Subsequent doses may be reduced to 5 x 10^7, based on Dose Modification Rules. One further dose reduction to 1 x 10^7 may occur in later injections.
Drug: Autologous Adipose-derived Mesenchymal Stromal Cells
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.


Outcome Measures

Primary Outcome Measures :
  1. Number of Adverse Events [ Time Frame: approximately 2 years ]
    Number of adverse events will be recorded from the time of enrollment until the end of the follow-up period or, in the case of early withdrawal, to the time of study withdrawal.


Secondary Outcome Measures :
  1. Change in slope of ALS Functional Rating Scale - Revised (ALSFRS-R) [ Time Frame: baseline, approximately 1 year ]
    The ALSFRS-R includes 12 questions. Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 48=best.


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations

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): NCT03268603


Contacts
Contact: Michelle Turner RSTALSRESEARCH@mayo.edu
Contact: Carol Denny RSTALSRESEARCH@mayo.edu

Locations
United States, Minnesota
Mayo Clinic in Rochester Recruiting
Rochester, Minnesota, United States, 55905
Contact: Michelle Turner       RSTALSRESEARCH@mayo.edu   
Contact: Carol Denny       RSTALSRESEARCH@mayo.edu   
Sponsors and Collaborators
Mayo Clinic
State of Minnesota Regenerative Medicine Minnesota
Investigators
Principal Investigator: Nathan P Staff, MD, PhD Mayo Clinic
Principal Investigator: Anthony J Windebank, MD Mayo Clinic
More Information

Responsible Party: Nathan P. Staff, Associate Professor of Neurology, Mayo Clinic
ClinicalTrials.gov Identifier: NCT03268603     History of Changes
Other Study ID Numbers: 15-008008
UL1TR000135 ( U.S. NIH Grant/Contract )
First Posted: August 31, 2017    Key Record Dates
Last Update Posted: September 19, 2017
Last Verified: September 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases