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History of Changes for Study: NCT01609283
A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis
Latest version (submitted August 29, 2019) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 May 29, 2012 None (earliest Version on record)
2 June 27, 2013 Contacts/Locations, Outcome Measures and Study Status
3 February 13, 2014 Study Status and Eligibility
4 August 7, 2014 Contacts/Locations and Study Status
5 June 3, 2015 Study Status
6 December 29, 2015 Recruitment Status, Study Status, Contacts/Locations and Study Design
7 May 12, 2016 Study Status
8 May 15, 2017 Study Status, Oversight, IPDSharing, Contacts/Locations and Study Description
9 January 10, 2018 Study Status
10 January 19, 2019 Study Status
11 August 29, 2019 Recruitment Status, Study Status and References
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Study NCT01609283
Submitted Date:  May 29, 2012 (v1)

Open or close this module Study Identification
Unique Protocol ID: 11-008415
Brief Title: A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis
Official Title: A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis
Secondary IDs:
Open or close this module Study Status
Record Verification: May 2012
Overall Status: Recruiting
Study Start: May 2012
Primary Completion: May 2014 [Anticipated]
Study Completion: May 2014 [Anticipated]
First Submitted: May 18, 2012
First Submitted that
Met QC Criteria:
May 29, 2012
First Posted: May 31, 2012 [Estimate]
Last Update Submitted that
Met QC Criteria:
May 29, 2012
Last Update Posted: May 31, 2012 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: Mayo Clinic
Responsible Party: Principal Investigator
Investigator: Anthony J. Windebank
Official Title: Principal Investigator
Affiliation: Mayo Clinic
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring: Yes
Open or close this module Study Description
Brief 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.
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.
Open or close this module Conditions
Conditions: Amyotrophic Lateral Sclerosis
Keywords:
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 1
Interventional Study Model: Single Group Assignment
Number of Arms: 1
Masking: None (Open Label)
Allocation: N/A
Enrollment: 25 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: Autologous Mesenchymal Stem Cells Biological: autologous mesenchymal stem cells

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
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Number of patients with dose-limiting toxicities
[ Time Frame: 2 years after completion of the final infusion ]

Secondary Outcome Measures:
1. Number of patients with adverse events
[ Time Frame: 2 years after completion of the final infusion ]

2. Change in serum sedimentation rate
[ Time Frame: baseline, 2 years after completion of the final infusion ]

3. Change in C-reactive protein levels
[ Time Frame: baseline, 2 years after completion of the final infusion ]

4. Change in complete blood counts
[ Time Frame: baseline, 2 years after completion of the final infusion ]

5. Change in total nucleated cell count in cerebrospinal fluid (CSF)
[ Time Frame: baseline, 2 years after completion of the final infusion ]

6. Change in protein level in cerebrospinal fluid (CSF)
[ Time Frame: baseline, 2 years after completion of the final infusion ]

7. Number of patients with presence of cancer cells in their cerebrospinal fluid (CSF)
[ Time Frame: 2 years after completion of the final infusion ]

Open or close this module Eligibility
Minimum Age: 18 Years
Maximum Age:
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

Inclusion Criteria:

  • 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 less than two years duration
  • 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.
  • 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
  • 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
Open or close this module Contacts/Locations
Locations: United States, Minnesota
Mayo Clinic
[Recruiting]
Rochester, Minnesota, United States, 55905
Contact:Contact: Anthony J. Windebank, MD 507-284-2511 rstalsresearch@mayo.edu
Contact:Contact: Nathan P. Staff, MD, PhD 507-284-2511 rstalsresearch@mayo.edu
Contact:Principal Investigator: Anthony J. Windebank, MD
Contact:Principal Investigator: Nathan P. Staff, MD, PhD
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Links:
Available IPD/Information:

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