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

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:

Genetics Home Reference related topics: amyotrophic lateral sclerosis

MedlinePlus related topics: Amyotrophic Lateral Sclerosis

Genetic and Rare Diseases Information Center resources: Amyotrophic Lateral Sclerosis

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 (Adult)
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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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


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:


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

ClinicalTrials.gov processed this record on September 27, 2016