Therapeutic Treatment of Amyotrophic Lateral Sclerosis (UwmWjmscAls)

August 23, 2016 updated by: Wojciech Maksymowicz, University of Warmia and Mazury

Application of Wharton's Jelly-derived Mesenchymal Stem Cells in the Treatment of Amyotrophic Lateral Sclerosis

The goal of this study is to investigate the safety and tolerability of allogeneic Wharton's jelly-derived mesenchymal stem cells administration in the individuals with diagnosed amyotrophic lateral sclerosis.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Detailed Description

Amyotrophic lateral sclerosis (ALS) is one of the progressive neurodegenerative disorders, affecting upper and lower motor neurons in the cerebral cortex, brainstem and spinal cord. Hence, the signs of damage motor neurons are both at the peripheral (eg. atrophy), and central (eg. spasticity) level. There is no effective treatment for ALS and the majority of patients die within 5 years after diagnosis, usually due to the respiratory failure. Numerous studies on murine models revealed that mesenchymal stem cells (MSCs) successfully improve the clinical and pathological features of ALS patients. The goal of this nonrandomized, open label study is to investigate the safety and tolerability of allogeneic Wharton's jelly-derived mesenchymal stem cell transplantation into the individuals with diagnosed amyotrophic lateral sclerosis. This clinical trial is conducted to test the therapeutic (neuroprotective and paracrine) effect of allogeneic Wharton's jelly-derived mesenchymal stem cells (WJ-MSCs). All patients enrolled will have a documented history of ALS disease prior to enrollment. Patients are recruited for a clinical trial no more than 1 year from the disease diagnosis. Then, patients are divided into two groups: Group I - patients receiving intrathecally one application of WJ-MSCs and Group II - patients receiving intrathecally three applications (each administration every two months) of WJ-MSCs. Subsequently, allogeneic Wharton's jelly-derived mesenchymal stem cell transplantation to the cerebrospinal fluid at the site of the spinal cord will be performed. Finally, treatment safety, adverse events and exploratory parameters, including electromyographic (EMG) studies, forced vital capacity (FVC) and functional rating scale (FRS) to establish ALS progression rate will be recorded throughout the duration and in the post-treatment follow up period.

Study Type

Interventional

Enrollment (Anticipated)

30

Phase

  • Phase 1

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years to 61 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Allogeneic WJ-MSCs injection
Intervention: Biological: Cell-based therapy of allogeneic Wharton's jelly-derived mesenchymal stem cells which are transplanted intrathecally (via a standard lumbar puncture) into the ALS subjects.
Other: Biological: Cell-based therapy
Human allogeneic Wharton's jelly-derived mesenchymal stem cell transplantation in ALS patients.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes in the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) between patients before and after stem cell transplantation.
Time Frame: 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
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Warmia and Mazury

Investigators

  • Principal Investigator: Wojciech Maksymowicz, MD, Prof., Faculty of Medical Sciences, University of Warmia and Mazury in Olsztyn, Poland

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2015

Primary Completion (Anticipated)

April 1, 2018

Study Completion (Anticipated)

December 1, 2018

Study Registration Dates

First Submitted

April 22, 2016

First Submitted That Met QC Criteria

August 23, 2016

First Posted (Estimate)

August 29, 2016

Study Record Updates

Last Update Posted (Estimate)

August 29, 2016

Last Update Submitted That Met QC Criteria

August 23, 2016

Last Verified

August 1, 2016

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • UWM/ALS-MSC.2015/001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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