This clinical trial is a single-center, open-label, and phase I clinical trial to Evaluate the Safety of Neuronata-R® Inj. suspended with HypoTHermosol® FRS (HTS-FRS) in Patients with Amyotrophic Lateral Sclerosis.
Neuronata-R is produced by mixing the patient's own cerebrospinal fluid with cultured mesenchymal stem cells the day before administration. Therefore, as autologous cerebrospinal fluid is collected from the patient for drug production at each administration, the patient must endure the occurrence of adverse events (headache, pain, etc.) and pain caused by collection, and problems such as fatigue and time consumption of the medical staff's procedure have continuously emerged. Therefore, a comparative equivalence tests and stability tests were conducted with HypoThermosol® FRS (HTS-FRS) as a suspension agent for Neuronata-R, and it was confirmed that there was no difference in the quality of the finished product depending on the type of additive and Neuronata-R using autologous cerebrospinal fluid as a suspension agent, and it was confirmed that it was safe as an additive through non-clinical trials. The dose determination for the safety evaluation of HypoThermosol® FRS (HTS-FRS), which will be used as a suspension, was determined in consultation with the MFDS to use a 1:1 mixture of autologous CSF and HypoThermosol® FRS (HTS-FRS) as a suspension in the first stage dose, and only HypoThermosol® FRS (HTS-FRS) was used as a suspension in the second stage dose without cerebrospinal fluid. And, the administration period is follow-up for 4 weeks after administration twice every 26 days.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
1.0 ⅹ 10\^6 cells/kg of Neuronata-R mixed with HTS-FRS suspension are administered twice in the cerebrospinal fluid at intervals of 26 days
In the case of a subject who is not taking Riluzole at the time of screening (visit 1), Riluzole should be taken except when discontinued due to Adverse events. The dose can be changed within the authorization according to the medical expert's judgement
Hanyang university hospital
Seoul, South Korea
tolerability Assessment
HTS-FRS DLT expression for each dose step
Time frame: through study period, an average of 8week
Safety Assessment
Adverse Events(including DLT)
Time frame: through study period, an average of 8week
Safety Assessment
Clinical Laboratory Analysis(Change in laboratory test results at the time of visit after IP administration compared to baseline)
Time frame: Baseline and 26day, 8week
Safety Assessment
Cerebrospinal Fluid Analysis(Changes in cerebrospinal fluid results at 26day compared to 0day)
Time frame: 0 Day, 26 Day
Exploratory Assessment
Change in total ALSFRS-R score from before treatment (screening) to after administration
Time frame: baseline and 26day , 8week
Exploratory Assessment
Change rate in ALSFRS-R score from before treatment (screening) to after administration
Time frame: within -8week and 0day, 8week
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