NCT03296501

Brief Summary

The goal of our nonrandomized, open label study is to investigate the safety and efficacy of autologous adipose derived mesenchymal regenerative cells (ADRC) transplantation into the individuals with diagnosed amyotrophic lateral sclerosis (ALS). All enrolled patients will have a documented at least 3-months clinical and electrophisiological observation of ALS disease course prior to study enrollment. Each patient will recive 3 injections of ADRC every 3 months: an intraspinal injection followed by 2 subsequent intrathecal infusions. Safety, adverse events and efficacy will be confirmed by clinical, elecrophisiological ( EMG, MUNIX), neuroimmaging and spirometry together with functional (ALSFRS-R) and objective motor assesment (MRC and dynamometer).

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
30

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Oct 2015

Longer than P75 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

October 13, 2015

Completed
1.9 years until next milestone

First Submitted

Initial submission to the registry

September 21, 2017

Completed
7 days until next milestone

First Posted

Study publicly available on registry

September 28, 2017

Completed
3.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2020

Completed
2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2022

Completed
Last Updated

April 15, 2020

Status Verified

April 1, 2020

Enrollment Period

5.1 years

First QC Date

September 21, 2017

Last Update Submit

April 14, 2020

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALSADRCadipose derived regenerative cellsintraspinal transplantationmesenchymal cells

Outcome Measures

Primary Outcomes (1)

  • Functional progression rate

    Delta ALS Functional Rating Scale (ALSFRS-R)/month

    3 months

Secondary Outcomes (1)

  • Electrophisiological progression rate

    3 months

Other Outcomes (1)

  • Respiratory insufficiency estimation

    3 months

Study Arms (1)

Autologous ADRC injection

EXPERIMENTAL
Biological: Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients

Interventions

Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patientsBIOLOGICAL

3 injections of ADRC: 1 intraspinal and 2 intrathecal

Autologous ADRC injection

Eligibility Criteria

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

You may qualify if:

  • clinically definite or probable ALS according to El Escorial criteria
  • life expectancy of more than 1 year
  • INR ≤2 before liposuction
  • lack of treatment with immune-suppressants and/or corticosteroids within min. 20 days prior to recruitment
  • constant riluzole treatment (50 mg/bid) throughout the study period
  • compliance with treatment regimen e.g. will and possibility to attend check-up visits
  • Polish citizens

You may not qualify if:

  • primary haematological disease, including hypercoagulable states
  • Presence of comorbidity that would stand in the way of neurosurgical treatment-
  • previous history of a spinal-cord surgery at the clinically affected level
  • previous/current history of neoplasm or comorbidity that could impact upon patient's survival
  • PEG
  • pregnancy /lactation
  • noninvasive/invasive mechanical ventilation at time of recruitment
  • alcohol abuse, cocaine amphetamine, etc.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Medical University of Warsaw

Warsaw, 02-097, Poland

Location

Related Publications (1)

  • Kuzma-Kozakiewicz M, Marchel A, Kaminska A, Gawel M, Sznajder J, Figiel-Dabrowska A, Nowak A, Maj E, Krzesniak NE, Noszczyk BH, Domanska-Janik K, Sarnowska A. Intraspinal Transplantation of the Adipose Tissue-Derived Regenerative Cells in Amyotrophic Lateral Sclerosis in Accordance with the Current Experts' Recommendations: Choosing Optimal Monitoring Tools. Stem Cells Int. 2018 Aug 12;2018:4392017. doi: 10.1155/2018/4392017. eCollection 2018.

    PMID: 30158984RESULT

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 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
M.D., Ph.D.

Study Record Dates

First Submitted

September 21, 2017

First Posted

September 28, 2017

Study Start

October 13, 2015

Primary Completion

October 31, 2020

Study Completion

October 31, 2022

Last Updated

April 15, 2020

Record last verified: 2020-04

Data Sharing

IPD Sharing
Will not share

Locations

PL(1)