A Phase 1 Study to Evaluate the Safety and Tolerability of Autologous Bone Marrow Derived Mesenchymal Stem Cells in Subjects with Multiple System Atrophy
Overview
- Phase
- Phase 1
- Intervention
- CS10BR05
- Conditions
- Multiple System Atrophy
- Sponsor
- Corestemchemon, Inc.
- Enrollment
- 9
- Locations
- 1
- Primary Endpoint
- Adverse events, frequency and rate of vitals sign, laboratory test, physical examination, ECG, etc.
- Status
- Completed
- Last Updated
- last year
Overview
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.
Investigators
Eligibility 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
Arms & Interventions
CS10BR05
The single injection of CS10BR05 Inj. in the carotid artery
Intervention: CS10BR05
Outcomes
Primary Outcomes
Adverse events, frequency and rate of vitals sign, laboratory test, physical examination, ECG, etc.
Time Frame: 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.
Time Frame: 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 Outcomes
- Unified Multiple System Atrophy Rating Scale(UMSARS)(-35days, 28days)