Clinical trial NCT02987413: Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients
Official title: Safety and Efficacy of Mesenchymal Stem Cells Escalated Application in Amyotrophic Lateral Sclerosis Patients: Study Design of a Phase I Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Motor Neuron Disease
Keywords: Amyotrophic Lateral Sclerosis
Lead sponsor: Hospital e Maternidade Dr. Christóvão da Gama
Sponsor class: OTHER
Collaborators: name: IEP São Lucas - Instituto de Ensino e Pesquisa
name: Clinica Jordy Sinapse
name: TECHLIFE - Centro de Tecnologia Celular
First posted: 2016-12-09
Enrollment: 3
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that selectively affects motor neurons in the brain and spinal cord, leading to bulbar, respiratory, and limb weakness. There is no effective treatment, and the disease usually progresses to death within 2 to 4 years. The therapeutic plasticity of mesenchymal stem cells (MSCs) may be an attractive therapy to this complex disease, turning MSCs strong candidates for cellular therapy in ALS.

Design-A phase 1 open-safety clinical trial. 4 patients will be selected according to a restricted inclusion and exclusion criteria and after 2 escalated infusions of MSCs, there will be a follow up period of one year Methods - Primary endpoint: safety of mesenchymal autologous stem cells infusions escalated in two intrathecal administrations in patients with ALS defined as severe adverse events (SAe). Secondary endpoints: clinical response, laboratorial and magnetic resonance imaging of patients submitted to cellular escalating doses applied in the study. Quality of life, according to El Escorial criteria, ALSFR scale and functional scales.

Conclusion: This study is a primary step before a large randomized double-blind clinical trial for ALS. It is expected to confirm the safety of escalated MSCs therapy in ALS patients, initial data of efficacy in addition to improved quality of life.


Eligibility criteria: Inclusion Criteria:

* Women and males over 18-year-old.
* Diagnosis of ALS in agreement with the criteria of "EL SCORE"
* Less than 24 months of evolution of the disease (from the beginning of the symptoms).
* Good understanding of the protocol and aptitude to grant the informed consent
* Infertile women (post-menopause or hysterectomized)
* Brazilian citizen and permanent resident.

Exclusion Criteria:

* Any significant medical condition (congestive heart failure, angina, respiratory failure, and others)
* Any auto-immune disease
* Any malignant diseases
* Systemic infection
* Mental illness
* Depressive state

Primary outcomes:
measure: Serious Adverse Events related to the treatment
timeFrame: 12 months
Secondary outcomes:
measure: Revised ALS Functional Rating Scale (ALSFRS-R)
timeFrame: 12 months
Other outcomes:
Locations:
city: Santo Andre
country: Brazil
facility: Hospital e Maternidade Dr Christovao da Gama
state: Sao Paulo
city: Sao Paulo
country: Brazil
facility: Instituto de Ensino e Pesquisas - IEP-São Lucas
state: SP
Study officials:
affiliation: Hospital e Maternidade Dr Christovao da Gama
name: Leandro B Agati, PhD
role: STUDY_DIRECTOR
affiliation: Instituto de Ensino e Pesquisas - IEP-São Lucas
name: Eliseo J Sekiya, MD, PhD
role: STUDY_CHAIR
affiliation: Instituto de Ensino e Pesquisas - IEP-São Lucas
name: Adelson A Silva, MD
role: STUDY_CHAIR
affiliation: TECHLIFE - Centro de Tecnologia Celular
name: Andresa Forte, MSc
role: STUDY_CHAIR
affiliation: Clínica Jordy Sinapse
name: Sergio S Jordy, MD
role: STUDY_CHAIR
References: