Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic La… (NCT01363401) | Clinical Trial Compass
CompletedPhase 1/2
Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis
South Korea72 participantsStarted 2011-02
Plain-language summary
The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow-derived stem cells("HYNR-CS inj"), through intrathecal delivery for the treatment in patients with ALS.
This study consists of 2 steps. First step is a safety study of the intrathecal(IT) injection of "HYNR-CS inj" in 8 patients with ALS. In this phase 1 study, AE, laboratory test, physical examination, vital signs, Electrocardiogram, and Chest X-Ray examination were evaluated in terms of safety.
Second step is to compare the efficacy and safety between test group and control group of total 64 patients with ALS.
Who can participate
Age range25 Years – 75 Years
SexALL
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Inclusion Criteria:
* Patients between 25 and 75 years old
* Patients who have both signs of lower motor neuron(LMN) and upper motor neuron(UMN) degeneration by clinical, electrophysiological or neuropathologic examination
* Patients diagnosed as 'Probable' or 'Definite' ALS according to the World Federation of Neurology El Escorial criteria
* Patients who have taken Rilutek at stable background dose from 3 months ago at least before screening entry
* Patients whose duration of disease is within 5 years from the first diagnosis
* Patients with ALSFRS-R score within 31 to 46 at screening
* Patients who can visit to a hospital by walk personally or by protector's help
* Patients who provide the written consent by oneself or his/her legal representative
Exclusion Criteria:
* Patients who doesn't appropriate to the diagnostic criteria of ALS
* Patients who are diagnosed as primary lateral sclerosis(PLS) or progressive muscular atrophy(PMA)
* Patients suspected of adverse effect after stem cell injection(patients suspected of malignant tumor, risk group of psychogenic shock, patients with serious hypertension)
* Patients with ALSFRS-R score below 30 at screening
* Patients performed ventilator or tracheostomy at screening
* Patients performed gastrostomy at screening
* Patients unable to assess the efficacy of this clinical trial due to unattainable PFT(Pulmonary Functional Test) or patients with suspected 40% or less of FVC at screening
* Patients with finding of myocardial in…
What they're measuring
1
The Difference in the Changes of Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Between Treatment Groups and Control Groups.