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Safety and Tolerability of CS10BR05 Inj. in Subjects With Multiple System Atrophy

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ClinicalTrials.gov Identifier: NCT03265444
Recruitment Status : Recruiting
First Posted : August 29, 2017
Last Update Posted : April 19, 2018
Sponsor:
Information provided by (Responsible Party):
Corestem, Inc.

Brief Summary:

The purpose of conducting phase 1 trial is to evaluate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cells(CS10BR05) in subjects with Multiple System Atrophy.

Evaluation of DLT by carotid artery(intra-arterial) injection according to dose-escalating in Multiple System Atrophy.


Condition or disease Intervention/treatment Phase
Multiple System Atrophy Biological: CS10BR05 Phase 1

Detailed Description:

Multiple system atrophy is a neurodegenerative disease of the central nervous system which is accompanied by signs of autonomic imbalance (orthostatic hypotension, urinary problems, erectile dysfunction), Parkinson's symptoms (movement decreases, limb tremors) and cerebellar ataxia symptoms (grogginess, pronounced incorrectly). It shows signs similar to Parkinson's disease, however, it doesn't show improvement of symptoms by dopaminergic drugs and occurs at any age.

The clinical trial was designed as a single center, open-label, public phase 1 clinical trials.

If that subject's written consent to participate in this clinical trial will be conducted the required examinations and tests in accordance with the study protocol.

Then, the register for each dose step. For the final 3 subjects in the inclusion / exclusion criteria and be administered a drug test.

And check the adverse events for more than four hours after administration of the investigational drug, and will determine whether the expression of adverse drug reaction(ADR) by the visit after 1 days, 14 days and 28 days.

Except for dose administration, all subject are tested in the same schedule. As a result of evaluating severity of AE divided into subjective, objective in accordance with the CTCAE (Version 4.0) standards.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 9 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1 Study to Evaluate the Safety and Tolerability of Autologous Bone Marrow Derived Mesenchymal Stem Cells in Subjects With Multiple System Atrophy
Actual Study Start Date : April 13, 2018
Estimated Primary Completion Date : June 30, 2018
Estimated Study Completion Date : December 31, 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: CS10BR05
The single injection of CS10BR05 Inj. in the carotid artery
Biological: CS10BR05
The single injection of CS10BR05 Inj. in the carotid artery; Step 1 dose : 3.0x105cells/kg; Step 2 dose : 6.0x105cells/kg; Step 3 dose : 9.0x105cells/kg; The duration of follow up study following the single dose of CS10BR05 is 28 days.




Primary Outcome Measures :
  1. Adverse event rate [ Time Frame: up to 28days ]
    Adverse event assessed by CTCAE v4.03

  2. Safety [ Time Frame: up to 28days ]
    Frequency and rate of vitals sign, laboratory test, physical examination, ECG


Secondary Outcome Measures :
  1. Unified Multiple System Atrophy Rating Scale(UMSARS) [ Time Frame: -35days, 28days ]
    Part I (Historical review), part II (Motor examination scale), part III (Autonomic examination), part IV (Global disability scale) assessment



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Ages Eligible for Study:   30 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and Female patients aged ≥30 years and ≤ 75 years
  • Meet criteria for diagnosis of probable MSA -cerebellar type according to the Gilman Criteria(2008)
  • MRI or PET imaging showed a decrease in the cerebellar atrophy or cerebral metabolism
  • Patients who are less than 4 years from the time of documented MSA diagnosis
  • Patients unified MSA rating scale 30~50
  • Those who have no hematologic abnormalities and who are not suspected of failing bone marrow function
  • Patients who consented to participate in the study in writing by themselves or their legal representatives

Exclusion Criteria:

  • Suspected clear Dementia (K-MMSE < 24)
  • DSM-IV criteria for Dementia
  • Radiologic imaging findings suggest that vascular encephalopathy coexist
  • Other central nervous system diseases except MSA (Parkinsons disease etc.)
  • Patients with Stroke or Brain surgery
  • If there is a coexistence of severe medical illness, or if it is in a severe pyrexia state
  • Serum SGOT / SGPT measures above three times of upper limit of normal or creatine levels were above 1.5 times of upper limit of normal levels were more than 1.5 times normal
  • disease that affects the patient's long-term survival(Tumor, Serious Heart failure)
  • Patients with genetic tests showed that spinocerebellar ataxia 1, 2, 3, 6, 7, 17
  • Patients with unstable vital signs
  • Patients with uncontrolled comorbidities such as moderate to severe infections, bleeding
  • Those who are found to be active to viral infection(HBV, HCV, HIV, CMV, HTLVⅠ/Ⅱ, VDRL)
  • Patients who are hypersensitive to bovine protein or antibiotics such as penicillin and streptomycin
  • Patients with difficult catheter insertion(bleeding disorder, artery hardening narrowness, Patients who are at risk of stroke in cerebral angiography)
  • Patients with cardiovascular disease(for example, hypertension, myocardial infarction etc;)
  • Severe disease uncontrolled (diabetes)
  • Those who are using drug likely to affect bone marrow functions
  • Pregnant women or nursing women
  • Women of childbearing age and male who do not consent to use proper contraception to prevent his partner from being pregnant during participation in the study
  • Now clinical trials treated with other drugs and in clinical trials the previous 4 weeks
  • Subjects who by the investigator to make them ineligible for participation in this clinical study

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): NCT03265444


Contacts
Contact: Ji-min Kim +82-70-4707-3545 jmkim@corestem.com

Locations
Korea, Republic of
Yonsei University College of Medicine Recruiting
Seoul, Korea, Republic of
Contact: Phil Hyu Lee, M.D.    +82 02 2228 1600    phlee@yuhs.ac   
Sponsors and Collaborators
Corestem, Inc.
Investigators
Principal Investigator: Phil Hyu Lee, M.D. Yonsei University

Additional Information:
Responsible Party: Corestem, Inc.
ClinicalTrials.gov Identifier: NCT03265444     History of Changes
Other Study ID Numbers: CS10BR05-MSA101
First Posted: August 29, 2017    Key Record Dates
Last Update Posted: April 19, 2018
Last Verified: August 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 Corestem, Inc.:
MSA

Additional relevant MeSH terms:
Multiple System Atrophy
Shy-Drager Syndrome
Atrophy
Pathological Conditions, Anatomical
Primary Dysautonomias
Autonomic Nervous System Diseases
Nervous System Diseases
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases
Hypotension
Vascular Diseases
Cardiovascular Diseases