Over years, the disease can worsen and shift to a progressive form that may also include relapses. The Food and Drug Administration has approved more than a dozen drugs for the treatment of relapsing forms of MS. These drugs vary in efficacy, safety and cost. For many people with severe forms of relapsing MS, first- and second-line drugs fail to adequately control the disease.
Previous studies have suggested that AHSCT may be an effective and durable treatment for these individuals, but it has never been formally compared head-to-head with the available third-line drugs, which are highly effective but can have harsh side effects.
AHSCT also carries the risks of serious side effects, and even death. Given these risks and benefits, investigators aim to determine whether AHSCT is an appropriate treatment option for people with severe forms of relapsing MS who would otherwise receive one of the best available third-line biologic drugs.
Leading the trial is Jeffrey A. Cohen, M. Participants will be randomly assigned to receive either AHSCT or one of the best available high-efficacy biologic drugs, and then will be followed for 6 years. The neurologists who periodically examine the participants and assess their level of disability will not know which type of treatment they were assigned.
The researchers also will examine the mechanisms of action of the two treatment strategies and will compare the newly developing immune systems of participants who receive AHSCT with the immunologic features of participants who receive the best available biologic drugs.
The ClinicalTrials. Additional information about the study, including how to enroll, is available at www. NIAID conducts and supports research — at NIH, throughout the United States, and worldwide — to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses.
Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www. Any concomitant medical disease or condition limiting the safety to participate:. Try the modernized ClinicalTrials.
Learn more about the modernization effort. Hide glossary Glossary Study record managers: refer to the Data Element Definitions if submitting registration or results information. Search for terms. Save this study. Warning You have reached the maximum number of saved studies Listing a study does not mean it has been evaluated by the U. Federal Government. Read our disclaimer for details. Recruitment status was: Active, not recruiting First Posted : May 5, Last Update Posted : March 10, Study Description.
This is a first-in-human trial of spinal derived stem cells transplanted into the spinal cord of patients with Amyotrophic Lateral Sclerosis ALS. The goal of the study is to see if the cells and the procedure to transplant them are safe. Detailed Description:. Resource links provided by the National Library of Medicine MedlinePlus Genetics related topics: Amyotrophic lateral sclerosis Juvenile primary lateral sclerosis.
MedlinePlus related topics: Amyotrophic Lateral Sclerosis. FDA Resources. Arms and Interventions. Outcome Measures. Primary Outcome Measures : The primary objective of this study is to determine the safety of human spinal cord-derived neural stem cell transplantation for the treatment of amyotrophic lateral sclerosis. Eligibility Criteria. Information from the National Library of Medicine Choosing to participate in a study is an important personal decision. Age 18 years or older. Females must have a negative serum pregnancy test and practice an acceptable method of contraception or be of non-childbearing potential post-menopausal for at least 2 years or surgically sterile [hysterectomy, oophorectomy or surgical sterilization].
Geographic accessibility to the study center and the ability to travel to the clinic for study visits. Presence of a willing and able caregiver. Medically able to undergo lumbar or cervical laminectomy as determined by the Investigator, surgeon and anesthesiologist.
Medically able to tolerate immunosuppression regimen consisting of basiliximab, tacrolimus, mycophenolate mofetil, and methylprednisolone as determined by the site Investigator.
Agrees to the visit schedule as outlined in the informed consent. Exclusion Criteria: Etiology of paraplegia or weakness is due to causes other than ALS such as spinal ischemia, traumatic spinal injury, traumatic brain injury, multiple sclerosis, cerebral stroke, cerebral palsy, or infection. Any known immunodeficiency syndrome. Receipt of any investigational drug,device or biologic within 30 days of surgery. Any concomitant medical disease or condition limiting the safety to participate: Coagulopathy Active uncontrolled infection Hypotension requiring vasopressor therapy Previous spinal surgery at the site of planned transplantation except for anterior cervical dissection fusion ACDF Skin breakdown over the site of surgery Malignancy except for non-melanoma skin cancer Primary or secondary immune deficiency Spinal stenosis.
Presence of any of the following conditions: Current drug abuse or alcoholism Unstable medical conditions Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening Positive blood test for hepatitis B or C.
Any condition that the site PI feels may interfere with participation in the study. Any condition that the surgeon feels may pose complications for the surgery.
Known hypersensitivity to basiliximab, tacrolimus, mycophenolate mofetil, or methylprednisolone. Inability to provide informed consent as determined by screening protocol. Inadequate family or caregiver support as determined by the site PI. Contacts and Locations. Information from the National Library of Medicine To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials. More Information. Additional Information: Related Info. Lumbar intraspinal injection of neural stem cells in patients with amyotrophic lateral sclerosis: results of a phase I trial in 12 patients.
Stem Cells. Intraspinal stem cell transplantation in amyotrophic lateral sclerosis: a phase I safety trial, technical note, and lumbar safety outcomes. Translational stem cell therapy for amyotrophic lateral sclerosis. Nat Rev Neurol. Stem cell technology for the study and treatment of motor neuron diseases.
Regen Med. Erratum in: Regen Med. Glass JD. The promise and the reality of stem-cell therapies for neurodegenerative diseases.
Epub Dec
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