Intraspinal Transplantation of Autologous ADRC in ALS Patients

Phase 1

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Biological: Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients

• Registration Number: NCT03296501
• Lead Sponsor: 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).

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 Timeline

• Study Start: 2015-10-13
• Study First Submitted: 2017-09-21
• Study First Submitted QC: 2017-09-23
• Study First Posted: 2017-09-28
• Status Verified: 2020-04
• Last Update Submitted: 2020-04-14
• Last Update Posted: 2020-04-15
• Primary Completion: 2020-10-31
• Study Completion: 2022-10-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Autologous ADRC injection	Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients	-

Primary Outcome Measures

Name	Time	Method
Functional progression rate	3 months	Delta ALS Functional Rating Scale (ALSFRS-R)/month

Secondary Outcome Measures

Name	Time	Method
Electrophisiological progression rate	3 months	electromyography (EMG)/ Motor unit number index (MUNIX)

Trial Locations

Locations (1)

Medical University of Warsaw; 🇵🇱 Warsaw, Poland