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A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by Mayo Clinic
Sponsor:
Information provided by (Responsible Party):
Anthony J. Windebank, Mayo Clinic
ClinicalTrials.gov Identifier:
NCT01609283
First received: May 18, 2012
Last updated: February 13, 2014
Last verified: February 2014

May 18, 2012
February 13, 2014
May 2012
December 2014   (final data collection date for primary outcome measure)
Number of patients with dose-limiting toxicities [ Time Frame: baseline -2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
Number of patients with dose-limiting toxicities [ Time Frame: 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT01609283 on ClinicalTrials.gov Archive Site
  • Number of patients with adverse events [ Time Frame: baseline -2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Change in serum sedimentation rate [ Time Frame: baseline, 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Change in C-reactive protein levels [ Time Frame: baseline, 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Change in complete blood counts [ Time Frame: baseline, 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Change in total nucleated cell count in cerebrospinal fluid (CSF) [ Time Frame: baseline, 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Change in protein level in cerebrospinal fluid (CSF) [ Time Frame: baseline, 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Number of patients with presence of cancer cells in their cerebrospinal fluid (CSF) [ Time Frame: baseline -2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Number of patients with adverse events [ Time Frame: 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Change in serum sedimentation rate [ Time Frame: baseline, 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Change in C-reactive protein levels [ Time Frame: baseline, 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Change in complete blood counts [ Time Frame: baseline, 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Change in total nucleated cell count in cerebrospinal fluid (CSF) [ Time Frame: baseline, 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Change in protein level in cerebrospinal fluid (CSF) [ Time Frame: baseline, 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
  • Number of patients with presence of cancer cells in their cerebrospinal fluid (CSF) [ Time Frame: 2 years after completion of the final infusion ] [ Designated as safety issue: Yes ]
Not Provided
Not Provided
 
A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis
A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis

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.

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.

Interventional
Phase 1
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Amyotrophic Lateral Sclerosis
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
Experimental: Autologous Mesenchymal Stem Cells
Intervention: Biological: autologous mesenchymal stem cells
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
25
December 2014
December 2014   (final data collection date for primary outcome measure)

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 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

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
Both
18 Years and older
No
United States
 
NCT01609283
11-008415
Yes
Anthony J. Windebank, Mayo Clinic
Mayo Clinic
Not Provided
Not Provided
Mayo Clinic
February 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP