Phase 2 N=72 Randomized Treatment

Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis · ALS

Bottom Line

View on ClinicalTrials.gov: NCT01363401 ↗

Enrolled (actual)

Serious AEs

8.3%

Results posted

Jul 2016

Primary outcome: Primary: The Difference in the Changes of Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Between Treatment Groups and Control Groups. — -1.36; -4.67 score on a scale

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: HYNR-CS inj (Biological); Control group (Other)
Age: Adult, Older Adult · 25+ yrs
Sex: All
Sponsor: Corestemchemon, Inc.
Primary completion: May 2013

Outcome Measures

Outcome	Result	p-value
PRIMARY The Difference in the Changes of Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Between Treatment Groups and Control Groups.	-1.36; -4.67	—
SECONDARY Change in Appel Scale	8.97; 17.96	—
SECONDARY Change in Forced Vital Capacity (FVC) (Percent of Predicted Normal)	-10.51; -10.75	—
SECONDARY Change in SF-36 (The Short Form (36) Health Survey is a 36 Item)	-8.59; -11.83	—

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.

Eligibility Criteria

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 infarction or angina pectoris according to ECG, patients who have been performed Stenting or Bypass operation at screening
Patients who have taken any other drug for clinical trial within the past 3 months at screening entry
Patients with epilepsy
Patients with severe renal dysfunction(serum creatinine≥2.0mg/dl)
Patients with severe liver dysfunction(ALT, AST, bilirubin≥upper limit of normal X 2)
Pregnant woman, lactating woman, female patients who has a pregnancy planning or who doesn't agree with adoption of contraception methods proper medically, male patients who doesn't agree with adoption of contraception methods proper to his partner during participating this study
Patients with hemorrhagic tendency at screening
Patients with virus infection at screening
Patients with a known history of hypersensitivity/allergy to penicillin and streptomycin
Patients with previous stem cell therapy
Patients diagnosed with cancer
Patients who have taken any drug thag can effect to bone marrow function
Patients with any other neurological disease except ALS
Patients with psychotic diseases

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01363401). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.