A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis
Study type: Interventional What is this?
Describes the nature of a clinical study. Types include:
- Observational study — observes people and measures outcomes without affecting results.
- Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
- Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
Study phase: I What is this?
During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.
- Rochester, Minnesota: 11-008415
NCT ID: NCT01609283
Sponsor Protocol Number: 11-008415
About this study
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.
Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.
See eligibility 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
- 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
Participating Mayo Clinic locations
Study statuses change often. Please contact us for help.