Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis

NCT01363401 | Completed Phase 1 Results

• 1 other identifier: HYNR_CS_ALS201
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow-derived stem cells("HYNR-CS inj"), through intrathecal delivery for the treatment in patients with ALS. This study consists of 2 steps. First step is a safety study of the intrathecal(IT) injection of "HYNR-CS inj" in 8 patients with ALS. In this phase 1 study, AE, laboratory test, physical examination, vital signs, Electrocardiogram, and Chest X-Ray examination were evaluated in terms of safety. Second step is to compare the efficacy and safety between test group and control group of total 64 patients with ALS.

Conditions

Amyotrophic Lateral Sclerosis; ALS

Keywords

Amyotrophic lateral sclerosis; ALS; Motor neuron disease; MND; HYNR-CS inj; Autologous bone marrow derived stem cell; Intrathecal injection

Outcome Measures

Primary Outcomes (1)

• The Difference in the Changes of Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Between Treatment Groups and Control Groups.

  ALSFRS-R is ordinal rating scale questionnaire (rating 0-4 for each question, 4 is most functional, 0-48 total) of 12 functional activities. The most functional total score is 48. ALSFRS-R was evaluated at baseline and week 28.(The first injection was performed at 0 week) ALSFRS-R total score variation baseline(Visit 5) and week 16(Visit 9)

  baseline(Visit 5) and week 16(Visit 9)

Secondary Outcomes (3)

• Change in Appel Scale

  baseline(Visit 5) and week 16(Visit 9)

• Change in Forced Vital Capacity (FVC) (Percent of Predicted Normal)

  baseline(Visit 5) and week 16(Visit 9)

• Change in SF-36 (The Short Form (36) Health Survey is a 36 Item)

  baseline(Visit 5) and week 16(Visit 9)

Study Arms (2)

Test group

EXPERIMENTAL

Treatment group with HYNR-CS inj.

Biological: HYNR-CS inj

Control group

EXPERIMENTAL

No treatment with HYNR-CS inj.

Other: Control group

Interventions

HYNR-CS inj BIOLOGICAL

Intrathecal injection with 1ml/10kg of body weight at an interval of 26 days.

Test group

Control group OTHER

No treatment of HYNR-CS inj

Control group

Eligibility Criteria

• Age: 25 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients between 25 and 75 years old
• Patients who have both signs of lower motor neuron(LMN) and upper motor neuron(UMN) degeneration by clinical, electrophysiological or neuropathologic examination
• Patients diagnosed as 'Probable' or 'Definite' ALS according to the World Federation of Neurology El Escorial criteria
• Patients who have taken Rilutek at stable background dose from 3 months ago at least before screening entry
• Patients whose duration of disease is within 5 years from the first diagnosis
• Patients with ALSFRS-R score within 31 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

You may not qualify if:

• Patients who doesn't appropriate to the diagnostic criteria of ALS
• Patients who are diagnosed as primary lateral sclerosis(PLS) or progressive muscular atrophy(PMA)
• 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 30 at screening
• Patients performed ventilator or tracheostomy at screening
• Patients performed gastrostomy at screening
• Patients unable to assess the efficacy of this clinical trial due to unattainable PFT(Pulmonary Functional Test) or patients with suspected 40% or less of FVC at screening
• Patients with finding of myocardial infarction or angina pectoris according to ECG, patients who have been performed Stenting or Bypass operation at screening
• Patients who have taken any other drug for clinical trial within the past 3 months at screening entry
• Patients with epilepsy
• Patients with severe renal dysfunction(serum creatinine≥2.0mg/dl)
• Patients with severe liver dysfunction(ALT, AST, bilirubin≥upper limit of normal X 2)
• 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 virus infection at screening

Sponsors & Collaborators

• Corestemchemon, Inc.: lead

Study Sites (1)

Hanyang University Hospital

Seoul, 133-792, South Korea

Location

Related Publications (5)

• Kim H, Kim HY, Choi MR, Hwang S, Nam KH, Kim HC, Han JS, Kim KS, Yoon HS, Kim SH. Dose-dependent efficacy of ALS-human mesenchymal stem cells transplantation into cisterna magna in SOD1-G93A ALS mice. Neurosci Lett. 2010 Jan 14;468(3):190-4. doi: 10.1016/j.neulet.2009.10.074. Epub 2009 Oct 29.

  PMID: 19879334 BACKGROUND

• Choi MR, Kim HY, Park JY, Lee TY, Baik CS, Chai YG, Jung KH, Park KS, Roh W, Kim KS, Kim SH. Selection of optimal passage of bone marrow-derived mesenchymal stem cells for stem cell therapy in patients with amyotrophic lateral sclerosis. Neurosci Lett. 2010 Mar 19;472(2):94-8. doi: 10.1016/j.neulet.2010.01.054. Epub 2010 Feb 1.

  PMID: 20117176 BACKGROUND

• Kim HY, Kim H, Oh KW, Oh SI, Koh SH, Baik W, Noh MY, Kim KS, Kim SH. Biological markers of mesenchymal stromal cells as predictors of response to autologous stem cell transplantation in patients with amyotrophic lateral sclerosis: an investigator-initiated trial and in vivo study. Stem Cells. 2014 Oct;32(10):2724-31. doi: 10.1002/stem.1770.

  PMID: 24966156 BACKGROUND

• Oh KW, Moon C, Kim HY, Oh SI, Park J, Lee JH, Chang IY, Kim KS, Kim SH. Phase I trial of repeated intrathecal autologous bone marrow-derived mesenchymal stromal cells in amyotrophic lateral sclerosis. Stem Cells Transl Med. 2015 Jun;4(6):590-7. doi: 10.5966/sctm.2014-0212. Epub 2015 May 1.

  PMID: 25934946 RESULT

• Oh KW, Noh MY, Kwon MS, Kim HY, Oh SI, Park J, Kim HJ, Ki CS, Kim SH. Repeated Intrathecal Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis. Ann Neurol. 2018 Sep;84(3):361-373. doi: 10.1002/ana.25302. Epub 2018 Aug 31.

  PMID: 30048006 RESULT

Related Links

• Corestem Inc.

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Motor Neuron Disease

Interventions

Control Groups

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Epidemiologic Research Design; Epidemiologic Methods; Investigative Techniques; Research Design; Methods

Results Point of Contact

• Title: Seung-Hyun Kim, M.D., Ph.D.
• Organization: Hanyang University Seoul Hospital

kimsh1@hanyang.ac.kr

+82-2-2290-8114

Study Officials

• Seung Hyun Kim, M.D., Ph.D.

  Hanyang University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 30, 2011
• First Posted: June 1, 2011
• Study Start: February 1, 2011
• Primary Completion: May 1, 2013
• Study Completion: August 1, 2013
• Last Updated: March 17, 2022
• Results First Posted: July 20, 2016

Record last verified: 2018-09

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)