ALSPhase 2NCT03268603
Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing Autologous Adipose-derived Mesenchymal Stromal Cells in people with ALS and Amyotrophic Lateral Sclerosis, run by Mayo Clinic at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- ALS, Amyotrophic Lateral Sclerosis
- What is being tested?
- Autologous Adipose-derived Mesenchymal Stromal Cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 75 participants
- Where
- 3 sitesUnited States
- Study dates
- 10 Oct 2017to 31 Dec 2026
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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).
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- 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)
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Mayo Clinic
Jacksonville, Florida, 32224
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
Mayo Clinic
Scottsdale, Arizona, 85259
About this study
Official title: 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.
Read the 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 10-100 million 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 was initially performed at Mayo Clinic in Rochester and subsequently expanded to the two other Mayo Clinic sites in Arizona and Florida. All biopsies and stem cell injections take place at Mayo Clinic Rochester, regardless of where the subject initially enrolls into the study.
- Conditions
- ALS, Amyotrophic Lateral Sclerosis
- Being tested
- Autologous Adipose-derived Mesenchymal Stromal Cells
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Adipose-derived Mesenchymal Stromal CellsDrug
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 group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Autologous Adipose-derived Mesenchymal Stromal Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
The number of adverse events experienced by subjects
Measured: 12-months
Unexpected Severe Adverse Events
The number of unexpected severe adverse events experienced by subjects
Measured: 12-months
Secondary outcomes (1)
Change in Slope of ALS Functional Rating Scale - Revised (ALSFRS-R)
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. The change in slope is the rate at which a patient's functional ability declines over time, as measured by the change in the ALFSFRS score over 1 year. A steeper slope signifies a faster decline in function. A negative change in slope indicates a decline in function and positive change in slope indicates an improvement in function.
Measured: baseline, approximately 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2017
- Study start10 Oct 2017
- Primary completion31 Jan 2024
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for ALSStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 75 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- State of Minnesota Regenerative Medicine Minnesota
- Responsible party
- Principal investigator, Nathan P. Staff, Mayo Clinic
- Investigators
- Nathan P Staff, MD, PhD, Principal investigator, Mayo ClinicAnthony J Windebank, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03268603
- Sponsor's protocol ID
- 15-008008
- Status last verified
- Feb 2026
- First submitted
- 30 Aug 2017
- Last updated
- 9 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT03268603
- Acronym / sponsor ID
- 15-008008
- Conditions
- ALS, Amyotrophic Lateral Sclerosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 75 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Collaborators
- State of Minnesota Regenerative Medicine Minnesota
- Study start
- 10 Oct 2017 (actual)
- Primary completion
- 31 Jan 2024 (actual)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 31 Aug 2017 (actual)
- Results
- Posted 2 Apr 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 10-100 million 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 was initially performed at Mayo Clinic in Rochester and subsequently expanded to the two other Mayo Clinic sites in Arizona and Florida. All biopsies and stem cell injections take place at Mayo Clinic Rochester, regardless of where the subject initially enrolls into the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Mesenchymal Stromal Cells | Experimental | Drug: Autologous Adipose-derived Mesenchymal Stromal CellsAutologous 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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.
Key exclusion criteria (19 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous Adipose-derived Mesenchymal Stromal CellsThe 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. | Drug | Mesenchymal Stromal Cells |
Primary outcome
Adverse Events
Time frame: 12-months
The number of adverse events experienced by subjects
Unexpected Severe Adverse Events
Time frame: 12-months
The number of unexpected severe adverse events experienced by subjects
1 secondary outcome
Change in Slope of ALS Functional Rating Scale - Revised (ALSFRS-R)
Time frame: baseline, approximately 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nathan P Staff, MD, PhD principal investigator
Mayo Clinic
Anthony J Windebank, MD principal investigator
Mayo Clinic
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Autologous Adipose-derived Mesenchymal Stromal Cells in people with ALS and Amyotrophic Lateral Sclerosis, run by Mayo Clinic at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 9 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (time frame: 12-months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Mar 2026.