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Intraspinal Transplantation of Autologous ADRC in ALS Patients (ADIPOSTEM)

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ClinicalTrials.gov Identifier: NCT03296501
Recruitment Status : Recruiting
First Posted : September 28, 2017
Last Update Posted : October 5, 2017
Sponsor:
Collaborator:
Medical University of Warsaw
Information provided by (Responsible Party):
Anna Sarnowska, Mossakowski Medical Research Centre Polish Academy of Sciences

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).

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Biological: Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients Phase 1

Detailed Description:

Amyotrophic Lateral Sclerosis (ALS) is an incurable disease of unknown etiology that in a short time leads to significant impairment of motor functions and death. The frequency of ALS is 4-8/100 000. Mostly it affects people between 40 and 70 years old, but it can occur at a younger age. Since the symptom onset is most frequently in the fifth or sixth decade, ALS is a huge economic burden for the society. There are many studies conducted to treat the disease and prevent it, but currently the medicine offers only one drug that can slow the appearance of disease symptoms but could not stop the progression. Yet, improvements in medical management, including nutrition and breathing, regularly increase patient survival - 50% of affected patients live at least 3 or more years after diagnosis; 20% live 5 years or more; and up to 10% will survive more than 10 years. The stem-cell-based therapies could be therefore a new waited strategy for ALS clinical treatment.

Since the clinical course of ALS may vary substantially between patients, we are planning to qualify them with a primarily established clinical course based on a detailed anamnesis and clinical assessment. 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. In order to select a group of 30 ALS patients for the ADRC treatment, approximately 50 ALS patients will be examined. All patients enrolled 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).


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy of Intraspinal Transplantation of Autologous ADRC in ALS Patients
Actual Study Start Date : October 13, 2015
Estimated Primary Completion Date : October 31, 2018
Estimated Study Completion Date : October 31, 2019


Arm Intervention/treatment
Experimental: Autologous ADRC injection Biological: Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients
3 injections of ADRC: 1 intraspinal and 2 intrathecal




Primary Outcome Measures :
  1. Functional progression rate [ Time Frame: 3 months ]
    Delta ALS Functional Rating Scale (ALSFRS-R)/month


Secondary Outcome Measures :
  1. Electrophisiological progression rate [ Time Frame: 3 months ]
    electromyography (EMG)/ Motor unit number index (MUNIX)


Other Outcome Measures:
  1. Respiratory insufficiency estimation [ Time Frame: 3 months ]
    Forced Vital Capacity (FVC)



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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03296501


Contacts
Contact: Magdalena Kuzma- Kozakiewicz, Prof. 0048225992875 mkuzma@wum.edu.pl
Contact: Andrzej Marchel, Prof. 0048225992575 amar@amwaw.edu.pl

Locations
Poland
Medical University of Warsaw Recruiting
Warsaw, Poland, 02-097
Contact: Magdalena Kuzma-Kozakiewicz, Prof.    0048225992875    mkuzma@wum.edu.pl   
Contact: Anna Sarnowska, Assoc. Prof.    0048226086598    asarnowska@imdik.pan.pl   
Sponsors and Collaborators
Mossakowski Medical Research Centre Polish Academy of Sciences
Medical University of Warsaw

Responsible Party: Anna Sarnowska, M.D., Ph.D., Mossakowski Medical Research Centre Polish Academy of Sciences
ClinicalTrials.gov Identifier: NCT03296501     History of Changes
Other Study ID Numbers: DPO/014/33/09/2015
First Posted: September 28, 2017    Key Record Dates
Last Update Posted: October 5, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Anna Sarnowska, Mossakowski Medical Research Centre Polish Academy of Sciences:
ALS
ADRC
adipose derived regenerative cells
intraspinal transplantation
mesenchymal cells

Additional relevant MeSH terms:
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases