Amyotrophic Lateral SclerosisPhase 1NCT01142856
Mesenchymal Stem Cells for Treatment of Amyotrophic Lateral Sclerosis (ALS)
A Phase 1 study testing autologous mesenchymal stem cells in people with Amyotrophic Lateral Sclerosis, run by Mayo Clinic at 1 site in United States.
Trial summary
The purpose of this study is to determine the safety of injecting mesenchymal stem cells through intraspinal delivery for the treatment of ALS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- autologous mesenchymal stem cells
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2010to Apr 2011
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- age greater than 18 years, if female, must be menopausal or had hysterectomy
- resident and citizen of the United States
- history of a chronic onset of a progressive motor weakness
- able to comply with protocol requirements
- can provide written consent
Who cannot join
- does not have renal disease (Creatine > 2.0)
- does not have active systemic disease
- does not have any clinically significant abnormalities on prestudy laboratory evaluation
- does not have any clinically significant medical condition (e.g.,within 6 months of baseline, had a myocardial infarct, angina pectoris, and/or congestive heart failure), that in the opinion of the investigator, would compromise the safety of the patient
- does not have a history of cancer including melanoma with the exception of localized skin cancers (with no evidence of metastasis, significant invasion,or re-occurrence within 3 years of baseline).
- has not used an investigational drug within 30 days of baseline visit
- does not have a tracheostomy
- does not have a Beck's Depression Inventory score >16
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Single Patient Treatment Protocol for Autologous Mesenchymal Stem Cell Intraspinal Therapy in Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study is to determine the safety of injecting mesenchymal stem cells through intraspinal delivery for the treatment of ALS.
Read the detailed description
A single patient safety study for harvesting, expanding ex vivo and injecting autologous mesenchymal stem cells (MSC's) into the subarachnoid space of a patient with amyotrophic lateral sclerosis (ALS). Cells will be isolated from adipose tissue by subcutaneous biopsy and expanded using an FDA-approved protocol. They will then be injected by lumbar puncture into the cerebrospinal fluid. Injection will be completed in the in- patient clinical research unit (CRU). the patient will be followed for two years.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- autologous mesenchymal stem 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 mesenchymal stem cellsBiological
single intrathecal dose of 10x6 cells by lumbar puncture.
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety
Clinical monitoring of possible reaction to intrathecal MSC delivery including blood pressure, temperature and pain score.
Measured: 2 year follow-up
Secondary outcomes (1)
Neurologic disability score
Quantitative, summated, manual muscle testing
Measured: Two year follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2010 (estimated)
- Study startJun 2010
- Primary completionApr 2011
- Study completionApr 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Investigators
- Anthony J. Windebank, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01142856
- Sponsor's protocol ID
- 09-001995
- Status last verified
- May 2012
- First submitted
- 10 Jun 2010
- Last updated
- 3 May 2012
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Patient Treatment Protocol for Autologous Mesenchymal Stem Cell Intraspinal Therapy in Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT01142856
- Acronym / sponsor ID
- 09-001995
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- Jun 2010
- Primary completion
- Apr 2011 (actual)
- Study completion
- Apr 2011 (actual)
- First posted
- 11 Jun 2010 (estimated)
- Results
- Not posted in the source record
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 of injecting mesenchymal stem cells through intraspinal delivery for the treatment of ALS.
Detailed description
A single patient safety study for harvesting, expanding ex vivo and injecting autologous mesenchymal stem cells (MSC's) into the subarachnoid space of a patient with amyotrophic lateral sclerosis (ALS). Cells will be isolated from adipose tissue by subcutaneous biopsy and expanded using an FDA-approved protocol. They will then be injected by lumbar puncture into the cerebrospinal fluid. Injection will be completed in the in- patient clinical research unit (CRU). the patient will be followed for two years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- age greater than 18 years, if female, must be menopausal or had hysterectomy
- resident and citizen of the United States
- history of a chronic onset of a progressive motor weakness
- able to comply with protocol requirements
- can provide written consent
Key exclusion criteria (8 listed)
- does not have renal disease (Creatine > 2.0)
- does not have active systemic disease
- does not have any clinically significant abnormalities on prestudy laboratory evaluation
- does not have any clinically significant medical condition (e.g.,within 6 months of baseline, had a myocardial infarct, angina pectoris, and/or congestive heart failure), that in the opinion of the investigator, would compromise the safety of the patient
- does not have a history of cancer including melanoma with the exception of localized skin cancers (with no evidence of metastasis, significant invasion,or re-occurrence within 3 years of baseline).
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 mesenchymal stem cellssingle intrathecal dose of 10x6 cells by lumbar puncture. | Biological | – |
Primary outcome
Safety
Time frame: 2 year follow-up
Clinical monitoring of possible reaction to intrathecal MSC delivery including blood pressure, temperature and pain score.
1 secondary outcome
Neurologic disability score
Time frame: Two year follow-up
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2012.
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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.
Anthony J. Windebank, MD principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing autologous mesenchymal stem cells in people with Amyotrophic Lateral Sclerosis, run by Mayo Clinic at 1 site 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 5 inclusion and 8 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: finished, as of the sponsor's last update on 3 May 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety (time frame: 2 year follow-up). 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 3 May 2012.