Study Purpose: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.
Amyotrophic Lateral Sclerosis
Type of Intervention:
autologous mesenchymal stem cells
Study Chair(s)/Principal Investigator(s):
Anthony Windebank, MD, Mayo Clinic
Coordinating Center Contact Information
Rochester, Minnesota, 55905 United States
Full Study Summary:
Addendum: Five subjects from the Group 5 dosing level will received additional injections of MSCs in an extension of the original study, if tolerated.
Follow up: minimum of 2 years after completion of the final infusion.
Estimated Study Start Date:
04 / 30 / 2012
Estimated Study Completion Date:
01 / 31 / 2019
Posting Last Modified Date:
09 / 03 / 2019
Date Study Added to neals.org:
05 / 31 / 2012
- 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
- 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