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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

autologous mesenchymal stem cells

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Mayo Clinic

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Autologous Mesenchymal Stem Cells

Arm Description

Outcomes

Primary Outcome Measures

Number of patients with dose-limiting toxicities

Secondary Outcome Measures

Number of patients with adverse events

Change in serum sedimentation rate

Change in C-reactive protein levels

Change in complete blood counts

Change in total nucleated cell count in cerebrospinal fluid (CSF)

Change in protein level in cerebrospinal fluid (CSF)

Number of patients with presence of cancer cells in their cerebrospinal fluid (CSF)

Full Information

NCT ID

NCT01609283

First Posted

May 18, 2012

Last Updated

August 29, 2019

Sponsor

Mayo Clinic

1. Study Identification

Unique Protocol Identification Number

NCT01609283

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2019

Overall Recruitment Status

Completed

Study Start Date

May 2012 (undefined)

Primary Completion Date

January 31, 2019 (Actual)

Study Completion Date

January 31, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Mayo Clinic

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. 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. Addendum: Five subjects from the Group 5 dosing level will received additional injections of MSCs in an extension of the original study, if tolerated.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Amyotrophic Lateral Sclerosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

27 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Autologous Mesenchymal Stem Cells

Arm Type

Experimental

Intervention Type

Biological

Intervention Name(s)

autologous mesenchymal stem cells

Intervention Description

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

Primary Outcome Measure Information:

Title

Number of patients with dose-limiting toxicities

Time Frame

baseline -2 years after completion of the final infusion

Secondary Outcome Measure Information:

Title

Number of patients with adverse events

Time Frame

baseline -2 years after completion of the final infusion

Title

Change in serum sedimentation rate

Time Frame