Status:
ACTIVE_NOT_RECRUITING
Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis (ALS)
Lead Sponsor:
Mayo Clinic
Collaborating Sponsors:
State of Minnesota Regenerative Medicine Minnesota
Conditions:
ALS
Amyotrophic Lateral Sclerosis
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
The purpose of this study is to determine the safety and efficacy of intrathecal treatment delivered to the cerebrospinal fluid (CSF) of mesenchymal stem cells in ALS patients every 3 months for a tot...
Detailed Description
The Goal of the Proposed Study is to perform an open label, 60 subject, Phase II multi-site clinical trial to investigate the safety and efficacy of intrathecal treatment of aaMSCs in ALS. Patients wi...
Eligibility Criteria
Inclusion
- All patients will have ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by the World Federation of Neurology criteria for the diagnosis of ALS.
- Examination and neurophysiological testing confirm a pure motor syndrome compatible with the diagnosis of ALS. All other possible causes of weakness have been excluded by extensive investigations.
- Age greater than 18 years, if female, must be post-menopausal, had a hysterectomy, or agree to two forms of birth control.
- Permanent resident or citizen of the United States.
- Geographic accessibility to the study site and willingness and ability to comply with follow-up.
- History of a chronic onset of a progressive motor weakness of less than two years duration.
- Subjects must be taking a stable dose of riluzole for at least 30 days prior to enrolment or not be on riluzole, and not have been on it for at least 30 days prior to enrolment (riluzole-naïve subjects are permitted in the study).
- Subjects must be taking a stable dose of oral Radicava® (edaravone) for at least 30 days prior to enrolment or not be on oral Radicava® (edaravone), and not have been on it for at least 30 days prior to enrolment (edaravone-naïve subjects are permitted in the study).
- Able to comply with protocol requirements, including MRI testing.
- Can provide written informed consent.
Exclusion
- Use of Radicava® (edaravone) within 30 days of screening or intent to use Radicava® at any time during the course of the study including the follow up period.
- 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.
- Pulmonary Slow Vital Capacity (SVC) less than 65% of predicted for age, gender, and body type.
- Autoimmunity, including Crohn's disease or rheumatoid arthritis
- Current use of immunosuppressant medication or use of such medication within 4 weeks of Screening visit (Visit 1).
- Malignancy 5 years prior to enrollment, including melanoma,with the exception of localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline).
- Active systemic or local infection near the lumbar puncture site.
- Inability to lie flat for the duration of intrathecal cell transplantation, or inability to tolerate study procedures for any other reason.
- Other active systemic disease as defined by laboratory abnormalities delineated in Appendix IV.
- Use of herbal medications, nutritional supplements or other unapproved drugs or investigational medicinal products being used or studied for the treatment of ALS.
- Unwilling to forgo initiating the use of any new supplements during participation in the study.
- Enrolled in an investigational drug trial within 30 days of baseline visit
- Prior stem cell therapy for a neurological disease
- Kokmen Short Test of Mental Status score \<32
- Presence of a tracheostomy
- Ventilator dependent
- Pregnancy
- Men or women of childbearing potential who are unwilling to employ adequate contraception
- Chronic low back pain requiring invasive procedures (i.e. epidural injections or lumbar spine surgery)
Key Trial Info
Start Date :
October 10 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2025
Estimated Enrollment :
75 Patients enrolled
Trial Details
Trial ID
NCT03268603
Start Date
October 10 2017
End Date
December 1 2025
Last Update
April 2 2025
Active Locations (3)
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1
Mayo Clinic
Scottsdale, Arizona, United States, 85259
2
Mayo Clinic
Jacksonville, Florida, United States, 32224
3
Mayo Clinic in Rochester
Rochester, Minnesota, United States, 55905