Safety/Efficacy Study for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
A Phase I, single center, prospective, non-randomized, open label, safety/efficacy study of the infusion of autologous bone marrow-derived stem cells, in 6 patients with Amyotrophic Lateral Sclerosis according to established criteria (1), (2) with a moderate to severe diagnosis of ALS according to the World Federation of Neurology El Escorial criteria.
The primary purpose of this study is to evaluate safety of the infusion procedure, as assessed by absence of complications at the site of infusion or the appearance of new neurologic deficit not attributed to the natural progression of the disease.
Secondary outcomes will include a)neurological evidence of trends toward a slowing down of the decline of the forced vital capacity (FVC) (3) and of the functional rating scale (ALS-FRS) scores, as assessed at 3-month intervals, b)evidence of a decline of the maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z (4) scores and c)patient evaluation that the treatment was effective and consider the possibility of a new cell product stem cell infusion.
Subjects who fulfill inclusion/exclusion criteria and sign informed consent will undergo an aspiration of bone marrow from the iliac crest for preparation of the cellular product.
The day of infusion, the investigational product will be injected into the patient's intrathecal space.
After cell infusion patients will be followed at WK 2, MN 1, MN 2, MN 6 and a long-term followup at MN 12 in the clinic and/or office. Electromyographic (EMG) studies, Forced vital capacity (FVC), functional rating scale (FRS) and maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z scores will have been used to assess the status of the disease before (historical record acceptable if done within three months of Screening Visit) and during the 12-month study period after cell infusion.
|Amyotrophic Lateral Sclerosis||Biological: autologous bone marrow-derived stem cells||Phase 1|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
|Official Title:||Phase I, Single Center, Prospective, Non-randomized, Open Label, Safety/Efficacy Study of the Infusion of Autologous Bone Marrow-derived Stem Cells, in Patients With Amyotrophic Lateral Sclerosis|
- Safety [ Time Frame: one year ]Safety of the infusion procedure, as assessed by absence of complications at the site of infusion or the appearance of new neurologic deficit not attributed to the natural progression of the disease.
- Efficacy [ Time Frame: one year ]
Neurological evidence of trends toward a slowing down of the decline of the forced vital capacity and of the functional rating scale scores, as assessed at 3 month intervals.
Evidence of a decline of the maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z scores.
Patient evaluation that the treatment was effective.
|Study Start Date:||March 2010|
|Estimated Study Completion Date:||May 2014|
|Estimated Primary Completion Date:||May 2014 (Final data collection date for primary outcome measure)|
infusion of autologous bone marrow-derived stem cells
Biological: autologous bone marrow-derived stem cells
All patients will receive a unique one-time intrathecal infusion of the cell product suspended in infusion medium.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01082653
|United States, Louisiana|
|TCA Cellular Therapy|
|Covington, Louisiana, United States, 70433|