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Trial record 11 of 29 for:    msc | Amyotrophic Lateral Sclerosis

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 : April 11, 2019
Sponsor:
Collaborator:
State of Minnesota Regenerative Medicine Minnesota
Information provided by (Responsible Party):
Nathan P. Staff, Mayo Clinic

Tracking Information
First Submitted Date  ICMJE August 30, 2017
First Posted Date  ICMJE August 31, 2017
Last Update Posted Date April 11, 2019
Actual Study Start Date  ICMJE October 10, 2017
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 30, 2017)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03268603 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 30, 2017)
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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis (ALS)
Official Title  ICMJE A Phase II Study of Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis
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.

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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • ALS
  • Amyotrophic Lateral Sclerosis
Intervention  ICMJE 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.
Study Arms  ICMJE 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.
Intervention: Drug: Autologous Adipose-derived Mesenchymal Stromal Cells
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 30, 2017)
60
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2019
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Michelle M Turner 507-284-2676 RSTALSRESEARCH@mayo.edu
Contact: Carol Denny 507-284-2676 RSTALSRESEARCH@mayo.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03268603
Other Study ID Numbers  ICMJE 15-008008
UL1TR000135 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Nathan P. Staff, Mayo Clinic
Study Sponsor  ICMJE Mayo Clinic
Collaborators  ICMJE State of Minnesota Regenerative Medicine Minnesota
Investigators  ICMJE
Principal Investigator: Nathan P Staff, MD, PhD Mayo Clinic
Principal Investigator: Anthony J Windebank, MD Mayo Clinic
PRS Account Mayo Clinic
Verification Date April 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP