Amyotrophic Lateral Sclerosis
Conditions
Keywords
ALS, stem cell
Brief summary
Patients with Amyotrophic Lateral Sclerosis (ALS) typically endure a progressive paralysis due to the continued loss of motoneurons that leads them to death in less than 5 years. No treatment has changed its natural history. Intrathecal injection of umbilical cord mesenchymal stem cells can secret trophic factors that keep the motorneurons functional. The investigators have designed a phase I/II clinical trial to check the feasibility of this approach in humans.
Detailed description
A total of 30 diagnosed ALS patients. The patients would got these symptoms such as gait difficulty and tremor, hand incoordination or speech difficulties.
Interventions
PROCEDUREstem cell transplantation
after stem cell prepared, the patients accepted 4 times stem cell transplantations through lumbar puncture, the time is 3-5days between two treatments. The patient would have to be in the bed at least 6 hours and removed the pillow.
Sponsors
General Hospital of Chinese Armed Police Forces
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
20 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Diagnose established following the World Federation of Neurology criteria * More than 6 and less than 36 months of evolution of the disease * Medullar onset of the disease * More than 20 and less than 65 years old * Forced Vital Capacity equal or superior to 50% * Total time of oxygen saturation \<90% inferior to 2% of the sleeping time * Signed informed consent
Exclusion criteria
* Neurological or psychiatric concomitant disease * Need of parenteral or enteral nutrition through percutaneous endoscopic gastrostomy or nasogastric tube * Concomitant systemic disease * Treatment with corticosteroids, immunoglobulins or immunosuppressors during the last 12 months * Inclusion in other clinical trials * Unability to understand the informed consent
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Nerve functional evaluation | within one week before,1month,6months,12months and 24months after transplantation | 1. Medical Research Council scale：To determine the strength of bilateral little finger abductor muscle and anterior tibial. 2. To evaluate the situation of upper motor neuron impairment through the Ellis reflex scale 3. The severity evaluation: NorrisALS score and ALS functional rating scale, ALSFRS 4. Speed of disease progression by the following formula：Progress rate = (40-ALSFRS score) / course 5. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI) and memory (Mini Mental State Examination) |
| Forced vital capacity | within one week before,1month,6months,12months and 24months after transplantation | vital capacity(VC）、forced vital capacity( FVC）、forced expiratory volune(FEV1）、FEV1/FVC、maximal voluntary ventilation(MVV）、peak expiratory flow(PEF） |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Blood test | within one week before,1month,6months,12months and 24months after transplantation | white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers , Lymphocytes classification, cholesterol total、Triglyceride、low density lipoprotein、Glycosylated serum protein glycosylated hemoglobin、Islet function，Na+、K+ |
| Urinal test | within one week before,1month,6months,12months and 24months after transplantation | proteinum、akaryocyte、α1- microglobulin、β2- microglobulin. CSF test:：IgA, IgG quantitation |
| Electrophysiology examination | within one week before,1month,6months,12months and 24months after transplantation | motor evoked potential(MEP)、nerve conduction and electromyologram(EMG) |
Countries
China
Outcome results
None listed