The Clinical Trial on the Use of Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis

Phase 2

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Procedure: stem cell transplantation

• Registration Number: NCT01494480
• Lead Sponsor: General Hospital of Chinese Armed Police Forces

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.

Study Timeline

• Study First Submitted: 2011-12-07
• Study First Submitted QC: 2011-12-15
• Study First Posted: 2011-12-19
• Study Start: 2012-03
• Status Verified: 2012-06
• Last Update Submitted: 2012-06-18
• Last Update Posted: 2012-06-20
• Primary Completion: 2013-01
• Study Completion: 2015-04

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
stem cell transplantation	stem 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.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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)

Trial Locations

Locations (1)

Yihua An; 🇨🇳 Beijing, China