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

Phase 1
Completed
Conditions
Multiple System Atrophy
Interventions
Biological: CS10BR05
Registration Number
NCT03265444
Lead Sponsor
Corestemchemon, 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.

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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
9
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
CS10BR05CS10BR05The single injection of CS10BR05 Inj. in the carotid artery
Primary Outcome Measures
NameTimeMethod
Adverse events, frequency and rate of vitals sign, laboratory test, physical examination, ECG, etc.up to 28days

To evaluate the safety and tolerability of CS10BR05 Inj. in patients with Multiple System Atrophy.

Dose-Limiting Toxicity and Maximum Tolerated Dose of CS10BR05 Inj.up to 28days

To evaluate the safety and tolerability of CS10BR05 Inj. in patients with Multiple System Atrophy including determination of its Dose Limiting Toxicity (DLT) and Maximum Tolerated Dose (MTD).

Secondary Outcome Measures
NameTimeMethod
Unified Multiple System Atrophy Rating Scale(UMSARS)-35days, 28days

Part I (Historical review), part II (Motor examination scale), part III (Autonomic examination), part IV (Global disability scale) assessment

Trial Locations

Locations (1)

Yonsei University College of Medicine

🇰🇷

Seoul, Korea, Republic of

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