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A Phase I/II, Open Label Study to Evaluate Safety, Tolerability and Therapeutic Effects of Transplantation of Autologous Cultured Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in ALS Patients |
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Study Focus:
This study will evaluate the safety, tolerability and therapeutic effects of autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF), in patients with ALS. |
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Disease:
Amyotrophic Lateral Sclerosis (ALS), Sporadic ALS |
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Study Category:
Stem cell trial |
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Study Status:
Active, no longer recruiting |
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Phase:
Phase II |
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Type:
Interventional Trial
with active agents/drugs only |
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Funding Source:
Brainstorm Cell Therapeutics Ltd; Ticker BCLI |
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Study Chair(s)/Principal Investigator(s):
Dimitrios Karussis, MD, PhD (Hadassah Medical Organization, Jerusalem, Israel) |
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Clinicaltrials.gov ID:
NCT01051882 |
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Study ID:
MSC-NTF-001-HMO-CTIL |
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NEALS Affiliated?
No |
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Study Summary:
This study is a single center trial to be conducted at the Department of Neurology & Laboratory of Neuroimmunology, at the Hadassah Hebrew University Medical Center, Jerusalem in Israel. All patients enrolled will have a documented history of ALS disease prior to study enrolment. Patients diagnosed as early stage ALS disease with duration of less than 6 months and patients diagnosed with progressive stage ALS disease with duration of 6-12 months. Overall, 24 patients will be recruited and allocated based on their ALS disease severity to 2 treatment groups: Group A - 12 patients of early ALS disease stage and Group B - 12 patients of progressive ALS disease.
Eligible patients will be enrolled into the study and will be observed for every 2 weeks during a "run in period" of 3 months for determination of the progression rate of the disease. During the "run in period" after about 6 weeks following enrollment, patients of both study groups will undergo a Bone Marrow Aspiration procedure and MSC-NTF cells will be produced from the bone marrow aspirate based on Brainstorm Cell Therapeutics Ltd proprietary method. On the last "run in period" visit, patients of both study groups will undergo the treatment and MSC-NTF will be transplanted by IM or IT injection to the early and progressive ALS patients respectively.
After the MSC-NTF transplantation patients will be observed on a monthly basis for a post treatment follow up period of 6 months.
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Participant Duration:
9 months |
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# of Subjects:
24 |
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Enrollment Start Date:
06/01/2011 |
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Enrollment End Date:
03/01/2013 |
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Posting Last Modified Date:
11/01/2011 |
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Date Study Added to alscortium.org:
11/07/2011 |
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More information:
http://www.brainstorm-cell.com/ |
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| Eligibility Criteria |
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Gender:
Male & Female |
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Minimum Age:
20 |
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Maximum Age:
75 |
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Min Vital Capacity (% predicted normal):
60% |
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Time since Symptom Onset:
Less than 24 months |
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Time since Diagnosis:
Less than 24 months |
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Can participants use Riluzole?
Yes |
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Coordinating Center
Contact Information |
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| Dimitrios Karussis, MD, PhD |
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| karus@cc.huji.ac.il |
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| (+972-2-) 6776939 |
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| Hadassah University Hospital |
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Ein Kerem POB 12000
Jerusalem 91120
UNITED STATES |
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Other Eligibility Criteria:
Exclusion Criteria
-Familial ALS |
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Site Contact Information (Please click to show/hide) |
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Hadassah University Hospital
Dimitrios Karussis, MD, PhD
karus@cc.huji.ac.il
(+972-2-) 6776939
Ein Kerem POB 12000
Jerusalem 91120
ISRAEL |
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Familial ALS- ALS cases are inherited from 2 or more family members, affects 5-10% of ALS cases. Sporadic ALS- disease occurs at random with no clearly associated risk factors; most common form of ALS, affects approximately 90-95% ALS cases. Interventional trial- study where exposure, for example a drug, is assigned; used to determine the effectiveness of a treatment or intervention. With drugs and a placebo- One group gets the active treatment, the other gets the placebo. Everything else is held the same between the two groups, so that any difference in their outcome can be attributed to the active treatment. Observational study- study in which patients are observed and where no treatment is given; researcher has no control over the experiment because they are done in a more natural setting than an interventional trial. Trial Phase- objectives are different for each phase of a trial. - Phase I, drug is given to a small number of people to see if it is safe.
- Phase II begins to look at effectiveness of the drug.
- Phase III is considered the definitive test of whether a drug is effective, held at multiple research sites and much larger than the previous phases.
Vital Capacity- the maximum amount of air that can be expelled from the lungs after a maximum drawing of air into the lungs, measured by a spirometer and then scored as a percentage. |
