Intrathecal Autologous ADRC Treatment of Autoimmune Refractory Epilepsy

NCT03676569 | Completed Phase 1 DMC

• 1 other identifier: 17/2014 IMiD
• Study Type: interventional
• Target: 6
• Locations: 0 countries
• Sites: N/A

Brief Summary

Refractory epilepsies caused by an autoimmune mechanisms lead in children to progressive neurodegeneration. Immunomodulation therapy is effective only in a half of such cases. New approaches are undertaken. It was found that ADRC (adipose derived regenerative cells) isolated from adipose tissue consist mesenchymal stem cells that act as tissue repair cells. The purpose of this experimental study is to evaluate the possibility and safety of the use of multipotent mesenchymal adipose derived regenerative cells (ADRC) in patients diagnosed with an autoimmune determined refractory epilepsy. Study protocol: Intrathecal infusions of autologous ADRC obtained after liposuction followed by isolation by Cytori system will be performed. Procedure will be repeated 3 times every 3 months in each patient. Neurological status, brain MRI, cognitive function and antiepileptic effect will be supervised during 24 months.

Conditions

Refractory Epilepsy

Keywords

autoimmune epilepsy; refractory epilepsy; ADRC; mezenchymal stem cells; anti-neuronal antibody; Rasmussen encephality; adipose derived regenerative stem cells

Outcome Measures

Primary Outcomes (1)

• Rate of epileptic seizures

  recording of epileptic seizures frequency, EEG

  3 months

Secondary Outcomes (2)

• Intelligence test

  3 months

• School progress

  3 months

Other Outcomes (2)

• Radiological assesment

  12 months

• The level of antineural antibodies

  3 months

Study Arms (1)

Experimental group

EXPERIMENTAL

Autologous ADRC transplantation in autoimmune refractory epilepsy

Biological: ADRC transplantation in autoimmune refractory epilepsy

Interventions

ADRC transplantation in autoimmune refractory epilepsy BIOLOGICAL

Experimental group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Drug-resistant epilepsy confirmed in anamnesis and diagnostic tests (EEG, MR)
• Children between 3 and 18 years old
• Presence of antineuronal antibody in serum or CSF
• Rasmussen encephalitis (proven cellular immunity pathogenesis)
• Probable autoimmune pathogenesis (autoimmune diseases in family, febrile infection associated refractory epilepsy)
• Willing and able to provide written informed consent (patient or legal guardian)

You may not qualify if:

• Refractory epilepsy with proven genetic or metabolic ethiology
• Neoplastic disease
• Active inflammatory process at the time of recruitment
• Coagulation disorder
• Status epilepticus
• Participation in another clinical trial
• Lack of willingness and ability to provide written informed consent

Sponsors & Collaborators

• Mossakowski Medical Research Centre Polish Academy of Sciences: lead
• Institute of Mother and Child, Warsaw, Poland: collaborator

Related Publications (1)

• Szczepanik E, Mierzewska H, Antczak-Marach D, Figiel-Dabrowska A, Terczynska I, Tryfon J, Krzesniak N, Noszczyk BH, Sawicka E, Domanska-Janik K, Sarnowska A. Intrathecal Infusion of Autologous Adipose-Derived Regenerative Cells in Autoimmune Refractory Epilepsy: Evaluation of Safety and Efficacy. Stem Cells Int. 2020 Jan 3;2020:7104243. doi: 10.1155/2020/7104243. eCollection 2020.

  PMID: 32190059 RESULT

MeSH Terms

Conditions

Drug Resistant Epilepsy

Condition Hierarchy (Ancestors)

Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Dorota Antczak-Marach, M.D.

  Institute of Mother and Child

  PRINCIPAL INVESTIGATOR

• Ewa Sawicka, Prof.

  Institute of Mother and Child

  PRINCIPAL INVESTIGATOR

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.; Head of Translative Platform for Regenerative Medicine MRC Mossakowski PAS

Model Details: Experimental clinical treatment

Study Record Dates

• First Submitted: September 14, 2018
• First Posted: September 18, 2018
• Study Start: November 15, 2015
• Primary Completion: April 14, 2019
• Study Completion: April 14, 2019
• Last Updated: April 15, 2020

Record last verified: 2020-04

Data Sharing

• IPD Sharing: Will not share