Autologous Multipotent Mesenchymal Stromal Cells in the Treatment of Amyotrophic Lateral Sclerosis (AMSC-ALS-001)
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|ClinicalTrials.gov Identifier: NCT03828123|
Recruitment Status : Completed
First Posted : February 4, 2019
Last Update Posted : February 12, 2019
|Condition or disease||Intervention/treatment||Phase|
|Motor Neuron Disease, Amyotrophic Lateral Sclerosis||Biological: Suspension of human autologous MSC 3P in 1.5 ml||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||26 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Prospective, Non-randomized, Open Label Study to Assess the Safety and the Efficacy of Autologous Multipotent Mesenchymal Stromal Cells in the Treatment of Amyotrophic Lateral Sclerosis|
|Actual Study Start Date :||January 2012|
|Actual Primary Completion Date :||August 18, 2017|
|Actual Study Completion Date :||August 18, 2017|
Experimental: Autologous Multipotent MSC
Patients with intrathecal administration of Suspension of human autologous MSC 3P in 1.5 ml
Biological: Suspension of human autologous MSC 3P in 1.5 ml
Intrathecal application of Autologous Multipotent Mesenchymal Stromal Cells 3P suspension
- Safety: Complications related to the medicinal product application - new neurological deficit and occurrence of other adverse events [ Time Frame: 1 year ]
Complications at the site of intrathecal infusion of the medicinal product and no new neurological deficit (meningism, paraplegia, urinary incontinence) not attributed to the natural progression of the ALS disease will be recorded at Visits I, III, IV, V, VI, and IX. Occurrence of other potential adverse events, including headache, respiratory failure, leukocytosis, cervical spine stenosis, cystitis and hyperhydrosis will be evaluated on the severity scale (1=mild, 2=moderate, 3=severe).
Brain and spinal cord MRI will be performed at Visits I and IX to exclude treatment-related tumor formation, pathological contrast enhancement or other structural pathology.
- Efficacy: Inhibition of the disease progression - ALS functional rating scale [ Time Frame: 18 months ]
Inhibition of the disease progression will be recorded by ALS functional rating scale (ALSFRS) at Visits I, III, and VI through X.
Measures (all 4-0):
- cutting food and handling utensils (with or without gastrostomy)
- dressing and hygiene
- turning in bed and adjusting bed clothes
- climbing stairs
ALSFRS = SUM (points for all 10 measures)
minimum score: 0 maximum score: 40 The higher the score the more function is retained.
- Efficacy: Inhibition of the disease progression - Norris scale [ Time Frame: 18 months ]
Inhibition of the disease progression will be recorded by Norris scale at Visits I, III, and VI through X.
Norris scal has has 22 items examining bulbar, respiratory, trunk, arm, leg, and general domains involving reflexes, fasciculation, and muscle atrophy. The scale also measures emotional lability, fatigability and leg rigidity. The Norris scale has a linear decline during the course of ALS.
- Efficacy: Inhibition of the disease progression - Forced vital capacity (FVC) [ Time Frame: 18 months ]FVC (%) will be measured at Visits I, and VI through X.