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

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
General Hospital of Chinese Armed Police Forces
ClinicalTrials.gov Identifier:
NCT01494480
First received: December 7, 2011
Last updated: June 18, 2012
Last verified: June 2012
History of Changes
  Purpose

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.


Condition Intervention Phase
Amyotrophic Lateral Sclerosis
 Procedure: stem cell transplantation
 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: The Clinical Study on the Use of Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by General Hospital of Chinese Armed Police Forces:

Primary Outcome Measures:
  • Nerve functional evaluation [ Time Frame: within one week before,1month,6months,12months and 24months after transplantation ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: within one week before,1month,6months,12months and 24months after transplantation ] [ Designated as safety issue: Yes ]
    vital capacity(VC）、forced vital capacity( FVC）、forced expiratory volune(FEV1）、FEV1/FVC、maximal voluntary ventilation(MVV）、peak expiratory flow(PEF）


Secondary Outcome Measures:
  • Blood test [ Time Frame: within one week before,1month,6months,12months and 24months after transplantation ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: within one week before,1month,6months,12months and 24months after transplantation ] [ Designated as safety issue: Yes ]
    proteinum、akaryocyte、α1- microglobulin、β2- microglobulin. CSF test:：IgA, IgG quantitation

  • Electrophysiology examination [ Time Frame: within one week before,1month,6months,12months and 24months after transplantation ] [ Designated as safety issue: Yes ]
    motor evoked potential(MEP)、nerve conduction and electromyologram(EMG)


Estimated Enrollment: 30
Study Start Date: March 2012
Estimated Study Completion Date: April 2015
Estimated Primary Completion Date: January 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: the stem cell treatment of ALS

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.

  Eligibility

Ages Eligible for Study:   20 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01494480

Locations
China
Yihua An
Beijing, China
Sponsors and Collaborators
General Hospital of Chinese Armed Police Forces
Investigators
Study Director: YiHua An Chinese People's Armed Police Force
  More Information

No publications provided

Responsible Party: General Hospital of Chinese Armed Police Forces
ClinicalTrials.gov Identifier: NCT01494480     History of Changes
Other Study ID Numbers: 20111207ALS
Study First Received: December 7, 2011
Last Updated: June 18, 2012
Health Authority: China: Food and Drug Administration

Keywords provided by General Hospital of Chinese Armed Police Forces:
ALS
stem cell

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Sclerosis
Motor Neuron Disease
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
 Neurodegenerative Diseases
TDP-43 Proteinopathies
Neuromuscular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on May 22, 2013