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

• Register: ClinicalTrials.gov
• Last refreshed on: 21 January 2013
• Main ID: NCT01363401
• Date of registration: 30/05/2011
• Primary sponsor: Corestem, Inc.
• Public title: Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis
• Scientific title: An Open-label, Phase I/II Trial for Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis
• Date of first enrolment: February 2011
• Target sample size: 71
• Recruitment status: Active, not recruiting
• URL: http://clinicaltrials.gov/show/NCT01363401
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Countries of recruitment

Korea, Republic of

Contacts

• Name: Seung Hyun Kim, M.D., Ph.D.
• Affiliation: Hanyang University

Key inclusion & exclusion 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



Age minimum: 25 Years
Age maximum: 75 Years
Gender: Both

Health Condition(s) or Problem(s) studied

ALS

Amyotrophic Lateral Sclerosis

Intervention(s)

Biological: HYNR-CS inj

Other: Control group

Primary Outcome(s)

Changes in ALSFRS-R score [Time Frame: Week 12, -8, -4, 0, 4, 8, 12, 16]

Secondary Outcome(s)

Change in Appel scale [Time Frame: Week -12, 0, 16]

Change in FVC [Time Frame: Week -12, 0, 16]

Change in SF-36 [Time Frame: Week 0, 16]

Secondary ID(s)

HYNR_CS_ALS201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Hanyang University