Clinical trial NCT01377870: Evaluation of Autologous Mesenchymal Stem Cell Transplantation (Effects and Side Effects) in Multiple Sclerosis
Official title: Effect and Side Effect of Mesenchymal Stem Cell in Multiple Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Multiple Sclerosis
Keywords: multiple sclerosis mesenchymal stem cell intravenous injection
Lead sponsor: Royan Institute
Sponsor class: OTHER_GOV
First posted: 2011-06-21
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Multiple sclerosis is a multifocal inflammatory disease of the central nervous system which affects young individuals and causes paralysis of the limbs, sensation, visual and sphincter problems. The disease is caused by an autoimmune mechanism, ie the immune system produces antibodies and cells which attack the self myelin antigens, causing therefore demyelination. The disease is clinically evident with relapses of neurological disability due to the dysfunction of the areas (plaques of multiple sclerosis) in which damage of myelin occurs. Disability can accumulate with time and the disease enters a progressive phase due to damage of the axons and irreversible neurodegeneration. Although, effective immunotherapies exist which downregulate the autoimmune anti-myelin reactivity and reduce the rate of relapses of MS (like Copaxone and interferons), there is no effective means today to stop the progression of disability and induce rebuilding of the destroyed myelin.Adult bone marrow derived stromal cells (MSC) were shown to induce similar (to the neuronal stem cells) immunomodulatory and neuroregenerative effects and were shown in our laboratory to induce neuroprotection in the animal model of chronic experimental autoimmune encephalomyelitis (EAE). These bone marrow derived MSCs offer practical advantages for clinical therapeutic applications, since they can be obtained from the adult bone marrow and therefore the patient can be the donor for himself, without any danger for rejection of the cells. In addition, MSCs carry a safer profile and are less prone to malignant transformation.

Our center will perform a clinical trial with intra venous transplantation of bone marrow derived mesenchymal stem cell.our purpose is to evaluate the safety and feasibility of cell transplantation after 1year following up.

Detailed description: In the clinical trial 30 patients with multiple sclerosis who are drug resistance will take apart.Based on inclusion and exclusion criteria patients are chosen.Bone marrow aspiration will be done for all of them under local anesthesia.Patients are randomly divided in 2 groups:case and control. Then mesenchymal stem cells which are separated and prepared will be transplanted by intravenous injection to the patients in case group and the cells which obtain from patients in control group are frozen and inject after 6 months. Patients will be followed by Evaluation of EDSS MSFC RAO Test brain and cervical MRI and quality of life questionnaire after 1th 3th 6th and 12th months after transplantation.all these tests will be done before transplantation as basic evaluation.

Eligibility criteria: Inclusion Criteria:

* Both gender
* Age: 18-55years
* Duration of disease: 2-10 years
* Relapsing remitting with drug resistance
* EDSS: 3-6.5
* Resistance to immunomodulatory and cytotoxic drugs:

  * At least 1-2 sever relapse during 1 year drug treatment
  * At least increase 1 point of EDSS during 1 year drug treatment
* Secondary progressive or relapsing multiple sclerosis
* Primary progressive MS with relapse or GAD positive enhancement
* Secondary progressive MS with relapse
* Secondary progressive MS without relapse:progression of disease with 1 point increase of EDSS during last 18 months

Exclusion Criteria:

* Pregnancy positive test
* Under treatment with cytotoxic drugs at the same time or during last 3 months
* Under treatment with immunomodulatory drugs at the same time or during last month
* Relapse of disease 30 days or less than 30 days before transplantation
* Primary progressive MS with out relapse or GAD positive enhancement

Primary outcomes:
measure: MRI metrics changes
timeFrame: 6 months
measure: Brain atrophy
timeFrame: 12 months
measure: number of sever relapses
timeFrame: 6 months
measure: EDSS
timeFrame: 6 months
measure: MSFC
timeFrame: 6 months
Secondary outcomes:
measure: quality of life
timeFrame: 6 months
measure: RAO Test
timeFrame: 6 months
measure: intravenous cell transplantation
timeFrame: 6 months
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 cell therapy center of Royan Institute
name: Nasser Aghdami, MD,PhD
role: STUDY_DIRECTOR
affiliation: scientist and clinician
name: Masoud Nabavi, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Department of regenerative medicine,Royan Institute
name: Leila Arab, MD
role: PRINCIPAL_INVESTIGATOR
References: