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

NCT03214146 | Unknown Phase 1

• 1 other identifier: HYNR-CS-Allo-02
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

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")

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS; Motor Neuron Disease; Neuromuscular Disease; Neurodegenerative Disease; Central Nervous System Disease; HLA-haplo matched allogenic bone marrow derived stem cells

Outcome Measures

Primary Outcomes (1)

• The Incidence of any treatment related serious adverse events(SAE)

  12 months

Secondary Outcomes (3)

• ALS-Functional rating scales(ALS-FRS)

  12 months

• Incidence & Degree of Adverse Events(AE)

  12 months

• PRA test to identify generation status of HLA antibody

  12 months

Study Arms (1)

HYNRCS-Allo inj.

EXPERIMENTAL

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

Interventions

HYNRCS-Allo inj BIOLOGICAL

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.

Also known as: HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell

HYNRCS-Allo inj.

Eligibility Criteria

• Age: 25 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Hanyang University Seoul Hospital: lead
• Corestemchemon, Inc.: collaborator

Study Sites (1)

Hanyang University Seoul Hospital, Cell Therapy Center for Neurologic Disorders

Seoul, Haengdang-dong, Seongdong-gu, 133-792, South Korea

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Motor Neuron Disease; Neuromuscular Diseases; Neurodegenerative Diseases; Central Nervous System Diseases

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Seung Hyun Kim, M.D.,Ph.D.

  Hanyang University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: July 9, 2017
• First Posted: July 11, 2017
• Study Start: February 1, 2017
• Primary Completion: March 25, 2019
• Study Completion: October 1, 2021
• Last Updated: May 16, 2019

Record last verified: 2019-05

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)