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

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

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.

Study Overview

• Status: Unknown
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Procedure: stem cell transplantation

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 Type: Interventional
• Enrollment (Anticipated): 30
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Yihua An

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Procedure: 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.; Other Names: the stem cell treatment of ALS

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Nerve functional evaluation	Medical Research Council scale：To determine the strength of bilateral little finger abductor muscle and anterior tibial.; To evaluate the situation of upper motor neuron impairment through the Ellis reflex scale; The severity evaluation: NorrisALS score and ALS functional rating scale, ALSFRS; Speed of disease progression by the following formula：Progress rate = (40-ALSFRS score) / course; Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI) and memory (Mini Mental State Examination)	within one week before,1month,6months,12months and 24months after transplantation
Forced vital capacity	vital capacity(VC）、forced vital capacity( FVC）、forced expiratory volune(FEV1）、FEV1/FVC、maximal voluntary ventilation(MVV）、peak expiratory flow(PEF）	within one week before,1month,6months,12months and 24months after transplantation

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Blood test	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+	within one week before,1month,6months,12months and 24months after transplantation
Urinal test	proteinum、akaryocyte、α1- microglobulin、β2- microglobulin. CSF test:：IgA, IgG quantitation	within one week before,1month,6months,12months and 24months after transplantation
Electrophysiology examination	motor evoked potential(MEP)、nerve conduction and electromyologram(EMG)	within one week before,1month,6months,12months and 24months after transplantation

Collaborators and Investigators

• Sponsor: General Hospital of Chinese Armed Police Forces
• Investigators: Study Director: YiHua An, Chinese People's Armed Police Force

Study record dates

Study Major Dates

• Study Start: March 1, 2012
• Primary Completion (Anticipated): January 1, 2013
• Study Completion (Anticipated): April 1, 2015

Study Registration Dates

• First Submitted: December 7, 2011
• First Submitted That Met QC Criteria: December 15, 2011
• First Posted (Estimate): December 19, 2011

Study Record Updates

• Last Update Posted (Estimate): June 20, 2012
• Last Update Submitted That Met QC Criteria: June 18, 2012
• Last Verified: June 1, 2012

More Information

Terms related to this study

• Keywords: ALS; stem cell
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: 20111207ALS