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

NCT01609283 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: 11-008415
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Number of patients with dose-limiting toxicities

  baseline -2 years after completion of the final infusion

Secondary Outcomes (7)

• Number of patients with adverse events

  baseline -2 years after completion of the final infusion

• Change in serum sedimentation rate

  baseline, 2 years after completion of the final infusion

• Change in C-reactive protein levels

  baseline, 2 years after completion of the final infusion

• Change in complete blood counts

  baseline, 2 years after completion of the final infusion

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

  baseline, 2 years after completion of the final infusion

Study Arms (1)

Autologous Mesenchymal Stem Cells

EXPERIMENTAL

Biological: autologous mesenchymal stem cells

Interventions

autologous mesenchymal stem cells BIOLOGICAL

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

Autologous Mesenchymal Stem Cells

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Related Publications (1)

• 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.

  PMID: 27515308 DERIVED

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Anthony Windebank, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: May 18, 2012
• First Posted: May 31, 2012
• Study Start: May 1, 2012
• Primary Completion: January 31, 2019
• Study Completion: January 31, 2019
• Last Updated: September 3, 2019

Record last verified: 2019-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)