Clinical trial NCT01759797: Intravenous Transplantation of Mesenchymal Stem Cell in Patients With ALS
Official title: Safety of Intravenous Transplantation of Bone Marrow Derived Mesenchymal Stem Cell in Patients With ALS
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS bone marrow mesenchymal stem cell intravenous injection
Lead sponsor: Royan Institute
Sponsor class: OTHER_GOV
First posted: 2013-01-03
Enrollment: 6
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: ALS is a debilitating disease with varied etiology characterized by rapidly progressive weakness, muscle atrophy and fasciculations, muscle spasticity, difficulty speaking (dysarthria), difficulty swallowing (dysphagia), and difficulty breathing (dyspnea). ALS is the most common of the five motor neuron diseases.Riluzole (Rilutek) is the only treatment that has been found to improve survival but only to a modest extent. It lengthens survival by several months, and may have a greater survival benefit for those with a bulbar onset. It also extends the time before a person needs ventilation support.Stem cell transplantation is a new hopeful way to improve the patients conditions and reduce the period of disabilities.

Detailed description: In this study our purpose is to evaluate the safety of intraventricular injection of bone marrow derived mesenchymal stem cell.the patients who are eligible,underwent bone marrow aspiration.after cell separation and preparation,the patients underwent mesenchymal stem cell intraventricular injection by stereotaxis.after injection he will be under observed in ICU to monitor the adverse events(allergic and neurological side effects).patients are followed 1th,3th ,6th and 12 months after injection and each time these parameters are checked:ALS-FRS,EMG-NCV,FVC,side effect check list.

Eligibility criteria: Inclusion Criteria:

* - Age:18-65
* both gender
* duration of disease\<2 years
* FVC\>40% ALS-FRS\>26

Exclusion Criteria:

* - neurological and psychiatric concomitant disease
* concomitant systemic disease
* treatment with corticosteroid,Ig,immunosuppressive during 12 months.

Primary outcomes:
measure: Fever
timeFrame: 48hours
measure: Unconsciousness
timeFrame: 48hours
measure: vomiting
timeFrame: 48hours
Secondary outcomes:
measure: ALS-FRS
timeFrame: 6months
measure: FVC
timeFrame: 6months
measure: EMG-NCV
timeFrame: 6months
Other outcomes:
Locations:
city: Tehran
country: Iran, Islamic Republic of
facility: Royan Institute
Study officials:
affiliation: Head of Royan Institute
name: Hamid Gourabi, PhD
role: STUDY_CHAIR
affiliation: Head of Royan department of degenerative medicine,Head of Royan celltherapy center
name: Nasser Aghdami, MD,PhD
role: STUDY_DIRECTOR
affiliation: Proffessor assistant of Shahed University
name: Seyed Masoud Nabavi, MD
role: STUDY_DIRECTOR
References:
citation: Nabavi SM, Arab L, Jarooghi N, Bolurieh T, Abbasi F, Mardpour S, Azimyian V, Moeininia F, Maroufizadeh S, Sanjari L, Hosseini SE, Aghdami N. Safety, Feasibility of Intravenous and Intrathecal Injection of Autologous Bone Marrow Derived Mesenchymal Stromal Cells in Patients with Amyotrophic Lateral Sclerosis: An Open Label Phase I Clinical Trial. Cell J. 2019 Jan;20(4):592-598. doi: 10.22074/cellj.2019.5370. Epub 2018 Aug 1.