- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03268603
Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis (ALS)
A Phase II Study of Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis
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.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
-
Arizona
-
Scottsdale, Arizona, United States, 85259
- Mayo Clinic
-
-
Florida
-
Jacksonville, Florida, United States, 32224
- Mayo Clinic
-
-
Minnesota
-
Rochester, Minnesota, United States, 55905
- Mayo Clinic in Rochester
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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:
- 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
- Ventilator dependent
- Pregnancy
- Men or women of childbearing potential who are unwilling to employ adequate contraception
- Chronic low back pain requiring invasive procedures (i.e. epidural injections or lumbar spine surgery)
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / 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 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.
|
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
approximately 2 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
baseline, approximately 1 year
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Nathan P Staff, MD, PhD, Mayo Clinic
- Principal Investigator: Anthony J Windebank, MD, Mayo Clinic
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 15-008008
- UL1TR000135 (U.S. NIH Grant/Contract)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Amyotrophic Lateral Sclerosis
-
Washington University School of MedicineMassachusetts General HospitalSuspendedAmyotrophic Lateral Sclerosis, Familial | Amyotrophic Lateral Sclerosis, SporadicUnited States
-
University of Sao Paulo General HospitalPontifícia Universidade Católica do ParanáUnknownAMYOTROPHIC LATERAL SCLEROSISBrazil
-
Neuromed IRCCSRecruitingAmyotrophic Lateral Sclerosis (ALS)Italy
-
Humanitas Mirasole SpAKU Leuven; UMC Utrecht; University of Sheffield; Istituto Superiore di Sanità; University... and other collaboratorsActive, not recruitingAmyotrophic Lateral Sclerosis (ALS)United Kingdom, Germany, France, Netherlands, Belgium, Ireland, Italy
-
The Methodist Hospital Research InstituteMassachusetts General Hospital; The Center for Clinical and Translational Sciences... and other collaboratorsActive, not recruiting
-
CytokineticsCompletedAmyotrophic Lateral Sclerosis (ALS)United States, Netherlands, Canada, Belgium, United Kingdom, France, Germany, Ireland, Italy, Portugal, Spain
-
Columbia UniversityALS AssociationTerminatedAmyotrophic Lateral Sclerosis (ALS)United States
-
El Instituto Nacional de Neurologia y Neurocirugia...CompletedAmyotrophic Lateral Sclerosis | Amyotrophic Lateral Sclerosis, SporadicMexico
-
University Hospital, GenevaCompletedAmyotrophic Lateral Sclerosis 11Switzerland
-
Fondazione Don Carlo Gnocchi OnlusFondazione Salvatore MaugeriCompleted
Clinical Trials on Autologous Adipose-derived Mesenchymal Stromal Cells
-
Mayo ClinicRegenerative Medicine MinnesotaTerminatedDiabetes Mellitus, Type 2 | Diabetes Mellitus, Type 1 | Diabetic Kidney Disease | Diabetic Nephropathies | Chronic Kidney Disease | Kidney Failure | Diabetic Nephropathy Type 2 | Kidney InsufficiencyUnited States
-
American CryoStem CorporationTerminatedMultiple SclerosisCayman Islands
-
Mayo ClinicRecruitingOsteoarthritis, HipUnited States
-
Jacob L. Sellon, M.D.CompletedOsteoarthritis, KneeUnited States
-
Rigshospitalet, DenmarkEnrolling by invitationMesenchymal Stem Cells | Radiation Toxicity | Hyposalivation | Dry Mouth | Mesenchymal Stromal Cells | Xerostomia Due to Radiotherapy | Long Term Adverse EffectsDenmark
-
Dr. Himanshu Bansal FoundationCompleted
-
Mayo ClinicTerminatedDegenerative Disc DiseaseUnited States
-
Rigshospitalet, DenmarkRecruiting
-
Rigshospitalet, DenmarkCompletedChronic Ischemic Heart DiseaseDenmark
-
Mohamad BydonCompletedParalysis | Spinal Cord InjuriesUnited States