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Safety/Efficacy Study of 2nd Cycle Treatment After 6 Months of 1st Cycle HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell Treatment in ALS

This study is currently recruiting participants.
Verified July 2017 by Seung Hyun Kim, Hanyang University Seoul Hospital
Sponsor:
ClinicalTrials.gov Identifier:
NCT03214146
First Posted: July 11, 2017
Last Update Posted: July 13, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Corestem, Inc.
Information provided by (Responsible Party):
Seung Hyun Kim, Hanyang University Seoul Hospital
  Purpose

The purpose of this study is to evaluate the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNRCS-Allo-ALS-02 inj"), through intrathecal delivery for the repeated treatment after 6 months of first treatment in patients with amyotrophic lateral sclerosis(ALS).

This study is an open label, single-dose study to assess the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNRCS-Allo-ALS-02 inj")


Condition Intervention Phase
Amyotrophic Lateral Sclerosis Biological: HYNRCS-Allo inj Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Phase 1 Trial for Safety and Efficacy Study of 2nd Cycle Treatment After 6 Months of 1st Cycle HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell("HYNR-CS-Allo Inj") Treatment in Amyotrophic Lateral Sclerosis(ALS)

Resource links provided by NLM:


Further study details as provided by Seung Hyun Kim, Hanyang University Seoul Hospital:

Primary Outcome Measures:
  • The Incidence of any treatment related serious adverse events(SAE) [ Time Frame: 12 months ]

Secondary Outcome Measures:
  • ALS-Functional rating scales(ALS-FRS) [ Time Frame: 12 months ]
  • Incidence & Degree of Adverse Events(AE) [ Time Frame: 12 months ]
  • PRA test to identify generation status of HLA antibody [ Time Frame: 12 months ]

Estimated Enrollment: 6
Actual Study Start Date: February 1, 2017
Estimated Study Completion Date: December 31, 2018
Estimated Primary Completion Date: November 1, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: HYNRCS-Allo inj.

2 cycles of HYNRCS-Allo inj. with 6 months interval through intrathecal injection.

*1 cycle of HYNRCS-Allo inj. is 2 times administration with 28 days interval by intrathecal.

Biological: HYNRCS-Allo inj
The patients enrolled in the trial will be successively allocated into single cohort for HYNRCS-Allo inj., 1.0 X 10^6 cells/kg according to the protocol design.
Other Name: HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell

Detailed Description:

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease characterized by motor neuron loss. Despite of many trials for disease-modifying, no treatment has so far changed natural course of disease.

The investigators had performed the pre-clinical and clinical studies using autologous bone marrow-derived stem cells in ALS. In the investigators' results of clinical trial, intrathecal injection of autologous bone marrow-derived stem cells is safe and could slow down disease progression and might be used as a disease modifying strategy in patients with ALS.

In the new field, like cell therapy, it is an important issue whether a bone marrow derived mesenchymal stem cells can be used as an allograft. Many investigators had showed that the immunoprivileged and immunosuppressive properties of mesenchymal stem cells result from the absence of major histocompatibility class II antigens and the secretion of T helper type 2 cytokines.

One potential advantage of allogenic bone marrow derived cells could be avoiding the need for procedural delay before treatment. And it is also hypothesized that the function of autologous bone marrow derived cells could be impaired in patients with co-morbidities or advanced age.

This study is to evaluate safety and efficacy of repeated treatment after 6 months of first HYNRCS-Allo-ALS-02 inj(HLA-haplo matched Allogenic bone marrow-derived stem cells) treatment in patients with ALS.

The patients enrolled in the trial will be successively allocated into single cohort for HYNRCS-Allo-ALS-02 inj., 1.0 X 10^6 cells/kg, according to the protocol design. Only a maximum of six patients will be given a particular dosage.

The scheduled assessments and visits will be carried out over three periods: run-in period, treatment period, and follow-up period.

The run-in period includes the screening visit where a written informed consent is obtained and the screening period where patients are assessed for eligibility. It will be completed within 56 days prior to enrollment. The patients meeting inclusion criteria will start the treatment period.

During the treatment period, subjects will be administered HYNRCS-Allo-ALS-02 inj. 2 times(first treatment) by intrathecal administration with 28 days interval and there will be repeated treatment after 6 months of the first treatment.

The Follow-up period starts once subjects complete the treatment period and will continue until the final follow-up visit.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   25 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients between 25 and 80 years old
  • Patients diagnosed as 'Possible with lab-supported' or 'Possible' or 'Probable' or 'Definite' ALS according to the World Federation of Neurology El Escorial criteria
  • Patients whose duration of disease is within 5 years from the first diagnosis
  • Patients with ALSFRS-R score within 21 to 46 at screening
  • Patients who can visit to a hospital by walk personally or by protector's help
  • Patients who provide the written consent by oneself or his/her legal representative
  • Patients who has HLA-haplo matched Bone marrow donor

Exclusion Criteria:

  • Patients who doesn't appropriate to the diagnostic criteria of ALS
  • Patients who doesn't have HLA-haplo-matched bone marrow donor
  • Patients suspected of adverse effect after stem cell injection(patients suspected of malignant tumor, risk group of psychogenic shock, patients with serious hypertension)
  • Patients with ALSFRS-R score below 21 at screening
  • Patients performed Tracheostomy at screening
  • Patients with suspected 20% or less of Forced vital capacity(FVC) at screening
  • Patients who doesn't agree with written consent form by oneself of his/her legal representative
  • Patients who have taken any other drug for clinical trial within the past 3 months at screening entry
  • Patients with epilepsy
  • Patients with severe medical disease
  • Pregnant woman, lactating woman, female patients who has a pregnancy planning or who doesn't agree with adoption of contraception methods proper medically, male patients who doesn't agree with adoption of contraception methods proper to his partner during participating this study
  • Patients with hemorrhagic tendency at screening
  • Patients with a known history of hypersensitivity/allergy to penicillin and streptomycin
  • Patients with severe psychotic diseases (such as alzheimer, schizophrenia excepts slight cognitive dysfunction and secondary emotional disorder)
  Contacts and Locations
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): NCT03214146


Contacts
Contact: Seung Hyun Kim, M.D.,Ph.D. +82-2-2290-8371 kimsh1@hanyang.ac.kr
Contact: Ki Wook Oh, M.D.,Ph.D. +82-2-2290-9367 kiwook.oh@gmail.com

Locations
Korea, Republic of
Hanyang University Seoul Hospital, Cell Therapy Center for Neurologic Disorders Recruiting
Seoul, Haengdang-dong, Seongdong-gu, Korea, Republic of, 133-792
Contact: Seung Hyun Kim, M.D., Ph.D    82-2-2290-9367    kimsh1@hanyang.ac.kr   
Contact: Ki Wook Oh, M.D.    82-2-2290-9365    kiwook.oh@gmail.com   
Principal Investigator: Seung Hyun Klm, M.D., Ph.D         
Sponsors and Collaborators
Hanyang University Seoul Hospital
Corestem, Inc.
Investigators
Principal Investigator: Seung Hyun Kim, M.D.,Ph.D. Hanyang University
  More Information

Responsible Party: Seung Hyun Kim, Principal Investigator, Hanyang University Seoul Hospital
ClinicalTrials.gov Identifier: NCT03214146     History of Changes
Other Study ID Numbers: HYNR-CS-Allo-02
First Submitted: July 9, 2017
First Posted: July 11, 2017
Last Update Posted: July 13, 2017
Last Verified: July 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Seung Hyun Kim, Hanyang University Seoul Hospital:
ALS
Motor Neuron Disease
Neuromuscular Disease
Neurodegenerative Disease
Central Nervous System Disease
HLA-haplo matched allogenic bone marrow derived stem cells

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