Amyotrophic Lateral SclerosisPhase 1NCT01609283
A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis
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 determine the safety of intraspinal delivery of mesenchymal stem cells (MSCs) to the cerebral spinal fluid of patients with Amyotrophic Lateral Sclerosis (ALS) using a dose-escalation study. 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
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- May 2012to 31 Jan 2019
- 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
- All patients must have clinically-defined ALS as defined by the World Federation of Neurology criteria
- Age greater than 18 years
- If female, must be post-menopausal or had a hysterectomy
- Permanent resident or citizen of the United States
- History of a chronic onset of a progressive motor weakness of greater than one year, but less than two years duration
- Must have vital capacity greater than 65% of predicated for age, gender, and body type
- Able to comply with protocol requirements, including MRI testing
- Can provide written informed consent
Who cannot join
- 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.
- Autoimmunity, including Crohn's disease, rheumatoid arthritis, psoriasis
- Malignancy including melanoma with the exception of localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline). Any other malignancy will not be allowed.
- Active systemic or local infection near the lumbar puncture site
- Other active systemic disease as defined by laboratory abnormalities
- Use of herbal medications or other unapproved drugs
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- Enrolled in an investigational drug trial within 30 days of baseline visit
- Kokmen Short Test of Mental Status score <32
- Beck's Depression Inventory score >18
- Presence of a tracheostomy
- Ventilator dependent
Trial locations and contacts
1 site in 1 country.
About this study
The purpose of this study is to determine determine the safety of intraspinal delivery of mesenchymal stem cells (MSCs) to the cerebral spinal fluid of patients with Amyotrophic Lateral Sclerosis (ALS) using a dose-escalation study.
Read the detailed description
The primary objective of this study is to determine the safety of intrathecal delivery of autologous mesenchymal stem cells (MSCs) to the cerebrospinal fluid (CSF) of patients with ALS using a dose-escalation study. The trial will include 25 adult, non-ventilator-dependent patients with clinically definite amyotrophic lateral sclerosis (ALS). Cells will be isolated from adipose tissue, expanded ex vivo and then, after ~8 weeks, intrathecal (IT) autologous delivery of MSCs will be performed. There will be 5 treatment groups of up to 5 patients each. Groups 1, 2, and 4 will receive a single dose of cells. Groups 3 and 5 will receive 2 doses of cells separated by 1 month. Groups will be completed sequentially so that patients will not be enrolled into the next treatment group until at least 3 patients in the preceding group have completed the treatment and 1 month of additional observation without significant toxicity. All patients will be followed on a regular basis until death or for a minimum of 2 years after completion of the final infusion. Initial clinical follow-up will be weekly with scheduled blood, CSF and magnetic resonance imaging (MRI) evaluations. After 1 month, patients will have clinical evaluations at 3 month intervals, or earlier if indicated by clinical status.
Addendum: Five subjects from the Group 5 dosing level will received additional injections of MSCs in an extension of the original study, if tolerated.
- 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
There will be five treatment groups of up to five patients each. Groups 1, 2 and 4 will receive a single dose of cells. Groups 3 and 5 will receive 2 doses of cells separated by one month. Intrathecal injections into new subjects will be timed so that there is a minimum of one week between subject injections. The cell dose per group is as follows:
- Group 1: single intrathecal dose of 1 x 107 cells
- Group 2: single intrathecal dose of 5 x 107 cells
- Group 3: one intrathecal dose of 5 x 107 cells followed one month later by a second intrathecal dose of 5 x 107 cells
- Group 4: single intrathecal dose of 1 x 108 cells
- Group 5: one intrathecal dose of 1 x 108 cells followed one month later by a second intrathecal dose of 1 x 108 cells
| Study group | Role | What they receive |
|---|---|---|
| Autologous Mesenchymal Stem Cells | Experimental | Not described Receives: Biological: autologous mesenchymal stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with dose-limiting toxicities
Measured: baseline -2 years after completion of the final infusion
Secondary outcomes (7)
Number of patients with adverse events
Measured: baseline -2 years after completion of the final infusion
Change in serum sedimentation rate
Measured: baseline, 2 years after completion of the final infusion
Change in C-reactive protein levels
Measured: baseline, 2 years after completion of the final infusion
Change in complete blood counts
Measured: baseline, 2 years after completion of the final infusion
Change in total nucleated cell count in cerebrospinal fluid (CSF)
Measured: baseline, 2 years after completion of the final infusion
Change in protein level in cerebrospinal fluid (CSF)
Measured: baseline, 2 years after completion of the final infusion
Number of patients with presence of cancer cells in their cerebrospinal fluid (CSF)
Measured: baseline -2 years after completion of the final infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2012 (estimated)
- Study startMay 2012
- Primary completion31 Jan 2019
- Study completion31 Jan 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Anthony J. Windebank, Mayo Clinic
- Investigators
- Anthony Windebank, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01609283
- Sponsor's protocol ID
- 11-008415
- Status last verified
- Aug 2019
- First submitted
- 18 May 2012
- Last updated
- 3 Sep 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Camilleri ET, Gustafson MP, Dudakovic A, Riester SM, Garces CG, Paradise CR, Takai H, Karperien M, Cool S, Sampen HJ, Larson AN, Qu W, Smith J, Dietz AB, van Wijnen AJ. Identification and validation of multiple cell surface markers of clinical-grade adipose-derived mesenchymal stromal cells as novel release criteria for good manufacturing practice-compliant production. Stem Cell Res Ther. 2016 Aug 11;7(1):107. doi: 10.1186/s13287-016-0370-8.
PubMed 27515308
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT01609283
- Acronym / sponsor ID
- 11-008415
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- May 2012
- Primary completion
- 31 Jan 2019 (actual)
- Study completion
- 31 Jan 2019 (actual)
- First posted
- 31 May 2012 (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 determine the safety of intraspinal delivery of mesenchymal stem cells (MSCs) to the cerebral spinal fluid of patients with Amyotrophic Lateral Sclerosis (ALS) using a dose-escalation study.
Detailed description
The primary objective of this study is to determine the safety of intrathecal delivery of autologous mesenchymal stem cells (MSCs) to the cerebrospinal fluid (CSF) of patients with ALS using a dose-escalation study. The trial will include 25 adult, non-ventilator-dependent patients with clinically definite amyotrophic lateral sclerosis (ALS). Cells will be isolated from adipose tissue, expanded ex vivo and then, after ~8 weeks, intrathecal (IT) autologous delivery of MSCs will be performed. There will be 5 treatment groups of up to 5 patients each. Groups 1, 2, and 4 will receive a single dose of cells. Groups 3 and 5 will receive 2 doses of cells separated by 1 month. Groups will be completed sequentially so that patients will not be enrolled into the next treatment group until at least 3 patients in the preceding group have completed the treatment and 1 month of additional observation without significant toxicity. All patients will be followed on a regular basis until death or for a minimum of 2 years after completion of the final infusion. Initial clinical follow-up will be weekly with scheduled blood, CSF and magnetic resonance imaging (MRI) evaluations. After 1 month, patients will have clinical evaluations at 3 month intervals, or earlier if indicated by clinical status.
Addendum: Five subjects from the Group 5 dosing level will received additional injections of MSCs in an extension of the original study, if tolerated.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous Mesenchymal Stem Cells | Experimental | Biological: autologous mesenchymal stem cells |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- All patients must have clinically-defined ALS as defined by the World Federation of Neurology criteria
- Age greater than 18 years
- If female, must be post-menopausal or had a hysterectomy
- Permanent resident or citizen of the United States
- History of a chronic onset of a progressive motor weakness of greater than one year, but less than two years duration
Key exclusion criteria (11 listed)
- 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.
- Autoimmunity, including Crohn's disease, rheumatoid arthritis, psoriasis
- Malignancy including melanoma with the exception of localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline). Any other malignancy will not be allowed.
- Active systemic or local infection near the lumbar puncture site
- Other active systemic disease as defined by laboratory abnormalities
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 cellsThere will be five treatment groups of up to five patients each. Groups 1, 2 and 4 will receive a single dose of cells. Groups 3 and 5 will receive 2 doses of cells separated by one month. Intrathecal injections into new subjects will be timed so that there is a minimum of one week between subject injections. The cell dose per group is as follows: * Group 1: single intrathecal dose of 1 x 107 cells * Group 2: single intrathecal dose of 5 x 107 cells * Group 3: one intrathecal dose of 5 x 107 cells followed one month later by a second intrathecal dose of 5 x 107 cells * Group 4: single intrathecal dose of 1 x 108 cells * Group 5: one intrathecal dose of 1 x 108 cells followed one month later by a second intrathecal dose of 1 x 108 cells | Biological | Autologous Mesenchymal Stem Cells |
Primary outcome
Number of patients with dose-limiting toxicities
Time frame: baseline -2 years after completion of the final infusion
7 secondary outcomes
Number of patients with adverse events
Time frame: baseline -2 years after completion of the final infusion
Change in serum sedimentation rate
Time frame: baseline, 2 years after completion of the final infusion
Change in C-reactive protein levels
Time frame: baseline, 2 years after completion of the final infusion
Change in complete blood counts
Time frame: baseline, 2 years after completion of the final infusion
Change in total nucleated cell count in cerebrospinal fluid (CSF)
Time frame: baseline, 2 years after completion of the final infusion
Change in protein level in cerebrospinal fluid (CSF)
Time frame: baseline, 2 years after completion of the final infusion
Number of patients with presence of cancer cells in their cerebrospinal fluid (CSF)
Time frame: baseline -2 years after completion of the final infusion
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 Sep 2019.
- Derived from this study: PubMed 27515308Camilleri ET, Gustafson MP, Dudakovic A, Riester SM, Garces CG, Paradise CR, Takai H, Karperien M, Cool S, Sampen HJ, Larson AN, Qu W, Smith J, Dietz AB, van Wijnen AJ. Identification and validation of multiple cell surface markers of clinical-grade adipose-derived mesenchymal stromal cells as novel release criteria for good manufacturing practice-compliant production. Stem Cell Res Ther. 2016 Aug 11;7(1):107. doi: 10.1186/s13287-016-0370-8.
- 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.
Anthony 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 8 inclusion and 11 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 Sep 2019. 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?
Number of patients with dose-limiting toxicities (time frame: baseline -2 years after completion of the final infusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01609283, which also lists 1 linked publication.
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 Sep 2019; AzTrials last refreshed its copy on 8 Nov 2026.