Autologous Bone Marrow Mesenchymal Stem Cells in the Treatment of Patients With Amyotrophic Lateral Sclerosis

NCT02881489 | Unknown Phase 1

• 1 other identifier: UWM/ALS-MSC.2015/002
• Study Type: interventional
• Target: 30
• Locations: 0 countries
• Sites: N/A

Brief Summary

The goal of this study is to investigate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cells administration in the individuals with diagnosed amyotrophic lateral sclerosis.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Changes in the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) between patients before and after stem cell transplantation.

  ALSFRS is ordinal rating scale questionnaire (rating 0-4 for each question, 4 is most functional, 0-48 total) of 12 functional activities. The most functional total score is 48. The First time the ALSFRS questionnaire has been dane after enrolment of the patients and then after 6 months of observation and stem cell injections the ALSFRS has been dane every 2 months up to 1,5 year of follow-up period.

  From day of enrolment until the date of first stem cell injection (6 months - first time ALSFRS) + and then every 2 months up to 1,5 year of the trial

Study Arms (1)

Autologous BM-MSCs injection

EXPERIMENTAL

Intervention: Biological: Cell-based therapy of autologous bone marrow-derived mesenchymal stem cells which are transplanted intrathecally (via a standard lumbar puncture) into the ALS subjects.

Other: Biological: Cell-based therapy

Interventions

Biological: Cell-based therapy OTHER

Human autologous bone marrow-derived mesenchymal stem cell transplantation in ALS patients.

Autologous BM-MSCs injection

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• diagnosis of the ALS disease before the cell transplantation (diagnose established following the El Escorial criteria for definite ALS)
• good understanding of the protocol and willingness to consent
• signed informed consent
• disease duration: up to 2 years
• FVC \> 50% / pulmonologist certificate about respiratory function of the patient

You may not qualify if:

• cancer,
• autoimmune diseases
• renal failure,
• subject is a respiratory dependent.
• subject unwilling or unable to comply with the requirements of the protocol
• pregnancy, breastfeeding

Sponsors & Collaborators

• University of Warmia and Mazury: lead

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Wojciech Maksymowicz, MD, Prof.

  Faculty of Medical Sciences, University of Warmia and Mazury in Olsztyn, Poland

  PRINCIPAL INVESTIGATOR

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: MD, PhD, Professor

Study Record Dates

• First Submitted: April 22, 2016
• First Posted: August 29, 2016
• Study Start: November 1, 2015
• Primary Completion: April 1, 2018
• Study Completion: December 1, 2018
• Last Updated: August 29, 2016

Record last verified: 2016-08