Clinical trial NCT04495582: Long-term Follow-up of Phase 1 Clinical Trial of CS10BR05(CS10BR05-MSA101)
Official title: Observational Study of Long-term Safety and Efficacy of Subjects Who Participated in CS10BR05 Inj. Phase 1 Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Multiple System Atrophy
Keywords: Mesenchymal stem cell
Cell therapy
Multiple system atrophy
Neuroprotection
CS10BR05
Long-term Safety Study
Lead sponsor: Corestemchemon, Inc.
Sponsor class: INDUSTRY
First posted: 2020-08-03
Enrollment: 8
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The subjects who participated in the phase 1 clinical trial(NCT03265444, Protocol No.: CS10BR05-MSA101) will be enrolled in this study, so the number of enrolled subjects will depend on the number of actual enrolled subjects for the phase 1 clinical trial.

Brief summary: The study purpose is conducting follow-up surveillance for the incidence of adverse events and efficacy of subjects participated in phase 1 trial to evaluate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cells (CS10BR05) in subjects with Multiple System Atrophy until 60 months from administering investigational product (IP).

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 and erectile dysfunction), Parkinson's symptoms (movement decreases and limb tremors) and cerebellar ataxia symptoms (grogginess and incorrect pronunciation). 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 purpose is conducting follow-up surveillance for the incidence of adverse events and efficacy of subjects participated in phase 1 trial to evaluate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cells (CS10BR05) in subjects with Multiple System Atrophy until 60 months from administering investigational product (IP).

Because it is the following observational study for subjects participated in phase 1 trial, there is no additional administration of the investigational product in the study.

If the subjects who participated in phase 1 trial voluntarily consent in writing to take part in this observational study, the subjects visit the institution according to the protocol that designed the follow-up visits every 3 months (Visit 1, Visit 2) until 6 months from administration of investigational product and since then 6 months later (Visit 3), every 12 months (Visit 4\~7) to observe the incidence of adverse events and efficacy.

As a result of evaluating severity of AE divided into subjective, objective in accordance with the CTCAE (Version 4.0) standards.

Eligibility criteria: Inclusion Criteria:

* Subjects who participated in the phase 1 clinical trial(Protocol No.: CS10BR05- MSA101) at 3 months after administering investigational product
* Subjects who give a written, signed and dated informed consent spontaneously

Exclusion Criteria:

* Subjects who decided as inappropriate cases to participate in the observational study by investigator

Primary outcomes:
measure: Safety assessment(evaluation)
timeFrame: 5 years
measure: Efficacy assessment(evaluation)
timeFrame: 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Yonsei University College of Medicine
Study officials:
affiliation: Yonsei University
name: Phil Hyu Lee, MD
role: PRINCIPAL_INVESTIGATOR
References: