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Trial record 5 of 677 for:    amyotrophic lateral sclerosis

Transplantation of Autologous Peripheral Blood Mononuclear Cells for Amyotrophic Lateral Sclerosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03085706
Recruitment Status : Completed
First Posted : March 21, 2017
Last Update Posted : February 9, 2018
Sponsor:
Information provided by (Responsible Party):
Liu Jing, The First Affiliated Hospital of Dalian Medical University

Brief Summary:
To assess the safety of peripheral blood mononuclear cell transplantation into the subarachnoid space for the treatment of amyotrophic lateral sclerosis.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Biological: PBMC autotransplantation Not Applicable

Detailed Description:

Amyotrophic lateral sclerosis (ALS) is a rapidly evolving, fatal neurodegenerative disease resulting from the degeneration of cortical, bulbar and spinal motor neurons. The disease progresses inexorably to death, usually because by failure of respiratory function, with a median duration of 3 years.

Recent clinical trials using various types of stem cells, including mesenchymal stromal cells, neural stem cells, and peripheral blood mononuclear cells (PBMCs), represent promising strategies for stem cell-based treatment in ALS. It has been demonstrated that the inflammation and neuronal death were reduced in ALS patients after bone marrow transplantation. In addition, the incidence of immune response was decreased by autologous transplantation of bone marrow cells in ALS patients. PBMCs are multi-potent stem cells that are very attractive for a cell therapy approach in ALS because of their plasticity and ability to provide the host tissue with growth factors or modulate the host immune system. PBMCs were used clinically and few adverse effects were attributed to their administration. Early clinical investigations indicated that the transplantation of autologous PBMCs into the dura is feasible in ALS patients; however, one study was limited to three patients and the other recruited eight patients. There are still many questions regarding the intrathecal transplantation of PBMCs for ALS. Therefore, a retrospective study was performed to assess further the safety and efficacy of the procedure and to test the impact of a cell therapy approach in ALS patients.

Statistical analysis Data, expressed as the mean ± SD, were analyzed using SPSS version 17.0 for Windows (SPSS Inc., Chicago, IL, USA). Statistical analyses were performed by paired sample t-test. A value of P < 0.05 was considered statistically significant.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
Intervention Model: Single Group Assignment
Intervention Model Description: 14 patients conducted at the First Affiliated Hospital of Dalian Medical University, China were eligible if they had definite or probable sporadic amyotrophic lateral sclerosis (ALS).
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Transplantation of Autologous Peripheral Blood Mononuclear Cells in the Subarachnoid Space for Amyotrophic Lateral Sclerosis: a Safety Analysis of 14 Patients
Actual Study Start Date : October 2010
Actual Primary Completion Date : December 2012
Actual Study Completion Date : June 2013


Arm Intervention/treatment
Experimental: PBMC autotransplantation
Fourteen amyotrophic lateral sclerosis (ALS) patients are received peripheral blood mononuclear cell (PBMC) autotransplantation.
Biological: PBMC autotransplantation
Fourteen amyotrophic lateral sclerosis (ALS) patients are received peripheral blood mononuclear cell (PBMC) autotransplantation.




Primary Outcome Measures :
  1. adverse events of autologous peripheral blood mononuclear cell mobilization [ Time Frame: 1 week after operation ]
    To assess the safety of autologous peripheral blood mononuclear cell transplantation


Secondary Outcome Measures :
  1. Functional independence measurement(FIM) [ Time Frame: changes of preoperation and week 1, week 2, week 4, week 12 after operation ]
    To assess the self-care ability of daily living

  2. Berg Balance Scale [ Time Frame: changes of preoperation and week 1, week 2, week 4, week 12 after operation ]
    To assess the trunk balance capability and limb movement function

  3. Dysarthria Assessment Scale [ Time Frame: changes of preoperation and week 1, week 2, week 4, week 12 after operation ]
    To assess the progression of bulbar paralysis



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Ages Eligible for Study:   31 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • all subjects had a verifiable diagnosis of ALS for 0.5 to 2 years based on a diagnosis using the Revised Criteria of the World Federation of Neurology. The grades of diagnosis were clinically definite ALS or clinically probable ALS;
  • ALS was mild-to-moderate based on the ALS Functional Rating Scale-Revised. Electrophysiological features showed compound muscle action potential (CMAP) amplitude of motor nerve normal or mild declining;
  • serum creatine kinase was normal or mild upper, less than 500 U/L.

Exclusion Criteria:

  • use of any other investigational agent within 30 days before treatment;
  • severe cardiac, pulmonary, hepatic or/and hematic disease;
  • human immunodeficiency virus positivity or signs and symptoms consistent with human immunodeficiency virus infection;
  • pregnant or nursing women;
  • history of cancer with less than 5 years documentation of a disease-free state;
  • history of anaphylactic reaction or hypersensitivity to granulocyte colony- stimulating factor (G-CSF);
  • alcohol or drug abuse in recent 1 year;
  • cannot understand or obey the rules of treatment;
  • blood donor in recent 30 days.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03085706


Locations
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China, Liaoning
The First Affiliated Hospital of Dalian Medical University
Dalian, Liaoning, China, 116011
Sponsors and Collaborators
The First Affiliated Hospital of Dalian Medical University
Investigators
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Principal Investigator: Jing Liu, Ph.D. The First Affiliated Hospital of Dalian Medical University

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Responsible Party: Liu Jing, Chief Physician, The First Affiliated Hospital of Dalian Medical University
ClinicalTrials.gov Identifier: NCT03085706     History of Changes
Other Study ID Numbers: LCKY2016-58
First Posted: March 21, 2017    Key Record Dates
Last Update Posted: February 9, 2018
Last Verified: February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases