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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Corestem, Inc.
ClinicalTrials.gov Identifier:
NCT01363401
First received: May 30, 2011
Last updated: June 8, 2016
Last verified: March 2016
Results First Received: December 8, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Amyotrophic Lateral Sclerosis
ALS
Interventions: Biological: HYNR-CS inj
Other: Control group

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Between March 2011 to May 2013, Of 10 subjects who consented to participate in the Stage 1 study, 2 subjects failed in screening, and 8 subjects were enrolled in the study. Among 71 subjects who consented to participate in the Stage 2 study, 7 subjects failed in screening, and 64 subjects were randomized.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Phase 1 : March 17, 2011 to November 24, 2011 Phase 2 : December 5, 2011 to May 6, 2013 The study consisted of stage 1 for safety evaluation and stage 2 for efficacy and safety evaluation of the study drug, and at stage 1, safety evaluation of the study drug, and at stage 1, safety evaluation for 28 days and then they followed stage 2.

Reporting Groups
  Description
HYNR-CS Inj.

Treatment group with HYNR-CS inj.

intrathecal injection with 1ml/10kg of body weight administer twice at an interval of 26day.

No Treatment No treatment with HYNR-CS inj.

Participant Flow:   Overall Study
    HYNR-CS Inj.   No Treatment
STARTED   41 [1]   31 
COMPLETED   39   27 
NOT COMPLETED   2   4 
[1] Including 8 subjects of Phase 1 safety assessment. (Called "Modified ITT" in this clinical trials)



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
HYNR-CS Inj.

Treatment group with HYNR-CS inj.

intrathecal injection with 1ml/10kg of body weight administer twice at an interval of 26day.

No Treatment

No treatment with HYNR-CS inj.

Take each 50mg 1 hour before a meal or 2 hours after a meal at least at an interval of 12 hours, 28 weeks(12 weeks of run-in phase plus 16 weeks of treatment phase)

Total Total of all reporting groups

Baseline Measures
   HYNR-CS Inj.   No Treatment   Total 
Overall Participants Analyzed 
[Units: Participants]
 41   31   72 
Age 
[Units: Years]
Mean (Standard Deviation)
 52.59  (8.59)   52.65  (8.93)   52.61  (8.61) 
Gender 
[Units: Participants]
     
Female   17   18   35 
Male   24   13   37 
Region of Enrollment 
[Units: Participants]
     
Korea, Republic of   41   31   72 
Riluzole use 
[Units: Participants]
     
on Riluzole   41   31   72 
Not on Riluzole   0   0   0 


  Outcome Measures
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1.  Primary:   The Difference in the Changes of Amyotrophic Lateral Sclerosis Functional Rating Scale – Revised (ALSFRS-R) Between Treatment Groups and Control Groups.   [ Time Frame: 28 weeks (12-week lead-in period + 16-week treatment and follow-up period) ]

2.  Secondary:   Change in Appel Scale   [ Time Frame: week -12,0,16 ]

3.  Secondary:   Change in Forced Vital Capacity (FVC) (Percent of Predicted Normal)   [ Time Frame: week -12,0,16 ]

4.  Secondary:   Change in SF-36 (The Short Form (36) Health Survey is a 36 Item)   [ Time Frame: week 0, 16 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Seung-Hyun Kim, M.D., Ph.D.
Organization: Hanyang University Seoul Hospital
phone: +82-2-2290-8114
e-mail: kimsh1@hanyang.ac.kr


Publications of Results:
Other Publications:

Responsible Party: Corestem, Inc.
ClinicalTrials.gov Identifier: NCT01363401     History of Changes
Other Study ID Numbers: HYNR_CS_ALS201
Study First Received: May 30, 2011
Results First Received: December 8, 2015
Last Updated: June 8, 2016
Health Authority: Korea: Food and Drug Administration