NCT02917681

Brief Summary

The study aims to evaluate primarily safety of two injections of autologous mesenchymal stem cells in Amyotrophic Lateral Sclerosis patients. Secondary outcomes of efficacy will also be evaluated

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
28

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Sep 2016

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2016

Completed
25 days until next milestone

First Submitted

Initial submission to the registry

September 26, 2016

Completed
2 days until next milestone

First Posted

Study publicly available on registry

September 28, 2016

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2019

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2019

Completed
Last Updated

September 28, 2016

Status Verified

September 1, 2016

Enrollment Period

2.4 years

First QC Date

September 26, 2016

Last Update Submit

September 27, 2016

Conditions

AMYOTROPHIC LATERAL SCLEROSIS

Keywords

ALSmesenchymal stem cellsintrathecal

Outcome Measures

Primary Outcomes (1)

  • Safety

    Clinical and laboratory monitoring of possible reactions to intrathecal MSC delivery

    10 months

Secondary Outcomes (6)

  • Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)

    10 months

  • Change in Handheld dynamometry

    10 months

  • Change in Electrical Impedance Myography

    10 months

  • Change in Forced Vital Capacity (FVC)

    10 months

  • Change in Isometric Strength

    10 months

  • +1 more secondary outcomes

Study Arms (1)

MSC injection

EXPERIMENTAL

Patients will be followed for 3 months before bone marrow aspiration (BMA). Patients will receive 2 intrathecal MSC injections, 1 and 2 months after BMA. The patients will be followed for 6 months after the interventions.

Other: Two intrathecal MSC injections

Interventions

Two intrathecal MSC injectionsOTHER
MSC injection

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age between 18 and 70
  • Possible, probable or definite ALS following El Escorial Revised Criteria (Brooks, 2000)
  • ALSFRS-r ≥30 at enrollment
  • Forced Vital Capacity ≥65% of the height and weight standard
  • No-pregnancy agreement
  • Regional accessibility to the study site
  • Capability to give away agreed consensus
  • Patients will be followed at Academic Institutions at their hometown

You may not qualify if:

  • Previous cellular therapy
  • Incapacity to lay still during bone marrow aspirate or intrathecal MSC injections
  • Personal history of auto-Immune, myeloproliferative or myelodysplastic diseases, leukemia, lymphoma, whole-body irradiation, hip fracture, severe scoliosis or incapacity to undergo any of the study's proposed procedures
  • Any other disease that may interfere with the study
  • Any other neurological diseases
  • Aspartate or alanine aminotransferases elevated \>3x normality upper limit
  • Serum creatinine \>2x normality upper limit
  • Hepatitis B and C, HIV, HTLV I and II and syphilis
  • Immunosuppressant drug use within 6 weeks from the study's screening
  • Pregnancy or breast-feeding
  • Acquired or inherited Immunodeficiency
  • Participation in other clinical trials
  • Non-invasive ventilation, tracheostomy or diaphragm pacing
  • Substance abuse within one year and other unstable mental health diseases according to researcher's judgement
  • Gastrostomy or any alternative feeding means
  • +1 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Sao Paulo School of Medicine Clinics Hospital

São Paulo, São Paulo, 05403000, Brazil

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Gerson Chadi, MD, PhD

    Full Professor

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Fabrício Castro Borba, MD

CONTACT

551130617460contato@projetoelabrasil.com.br

Jéssica Ruivo Maximino, PhD

CONTACT

551130617460contato@projetoelabrasil.com.br

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

September 26, 2016

First Posted

September 28, 2016

Study Start

September 1, 2016

Primary Completion

February 1, 2019

Study Completion

August 1, 2019

Last Updated

September 28, 2016

Record last verified: 2016-09

Data Sharing

IPD Sharing
Will not share

Locations

BR(1)