Intraspinal Transplantation of Autologous ADRC in ALS Patients

Amyotrophic Lateral Sclerosis Clinical Trial

— ADIPOSTEM  

Official title:

Safety and Efficacy of Intraspinal Transplantation of Autologous ADRC in ALS Patients

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03296501
• Other study ID #: DPO/014/33/09/2015
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: October 13, 2015
• Est. completion date: October 31, 2022

Study information

• Verified date: April 2020
• Source: Mossakowski Medical Research Centre Polish Academy of Sciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 30
• Est. completion date: October 31, 2022
• Est. primary completion date: October 31, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease

Intervention

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

Locations

Country	Name	City	State
Poland	Medical University of Warsaw	Warsaw

Sponsors (2)

Lead Sponsor	Collaborator
Mossakowski Medical Research Centre Polish Academy of Sciences	Medical University of Warsaw

Country where clinical trial is conducted

Poland, 

References & 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 Late — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Respiratory insufficiency estimation	Forced Vital Capacity (FVC)	3 months
Primary	Functional progression rate	Delta ALS Functional Rating Scale (ALSFRS-R)/month	3 months
Secondary	Electrophisiological progression rate	electromyography (EMG)/ Motor unit number index (MUNIX)	3 months