Treatment of Multiple Sclerosis and Neuromyelitis Optica With Regulatory Dendritic Cell: Clinical Trial Phase 1 B

First in human study to assess the tolerability and safety profile of treatment with dendritic cell in patients with multiple sclerosis or neuromyelitis optica.

Study Overview

• Status: Completed
• Conditions: Multiple Sclerosis; Neuromyelitis Optica
• Intervention / Treatment: Biological: Tolerogenic Dendritic cells loaded with myelin peptides

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 1

Contacts and Locations

Study Locations

• Spain
  • Barcelona, Spain, 08036
    • Hospital Clinic of Barcelona

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• patients with Multiple sclerosis or neuromyelitis optica
• diagnosed more than a year before inclusion
• Expanded Disability Status Scale between 3.0 and 8.5
• all subtypes of multiple sclerosis or Neuromyelitis optica
• Multiple Sclerosis patients who have previously been offered therapeutic alternatives available in indications and either decline or that after receiving treatment for at least 6 months have had an outbreak or an increase of at least 1 point on the Expanded Disability Status Scale (EDSS) (non-responders) or who have not tolerated treatment
• Patients with Neuromyelitis optica (NMO) in stable immunomodulatory treatment in the past 6 months or without treatment because they are not candidates to receive it

Exclusion Criteria:

• Corticosteroid treatment in the last 30 days
• Presence of an outbreak in the last month
• Inability to perform brain Magnetic resonance imaging (with paramagnetic contrast)
• Serious systemic diseases, including Hepatitis B virus, Hepatitis C Virus, and Human Immunodeficiency Virus. Uncontrolled hypertension, insulin-dependent diabetes mellitus, heart disease or kidney failure or severe respiratory
• Personal history of cancer or family history of known hereditary cancer
• patient participating in other experimental study in the last 3 months
• women childbearing-aged that do not use effective contraceptive methods
• pregnant or breastfeeding women

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Tolerogenic dendritic cells; Somatic-cell therapy medicines: tolerogenic dendritic cells loaded with myelin peptides. Patients will receive intravenous administration every two weeks (week 0 , 2 and 4 ) representing a total of three administrations per patient. The dose escalation will occur as expected in the absence of limiting toxicity in the previous dosage level.	Biological: Tolerogenic Dendritic cells loaded with myelin peptides; Somatic-cell therapy medicines application

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
number of patients with adverse events	after 12 weeks of follow up

Secondary Outcome Measures

Outcome Measure	Time Frame
Multiple Sclerosis Functional Composite scale	after 12 weeks of follow up
Multiple Sclerosis Spasticity Scale	after 12 weeks of follow up
Expanded Disability Status Scale	after 12 weeks of follow up
SF36 Health Status questionnaire	after 12 weeks of follow up
EuroQol5D	after 12 weeks of follow up
Changes in immunological profile	after 12 weeks of follow up
number of disease outbreaks	after 12 weeks of follow up

Collaborators and Investigators

• Sponsor: Sara Varea
• Investigators: Study Chair: Pablo Villoslada, MD PhD, Institut d'Investigacions Biomèdiques August Pi i Sunyer

Study record dates

Study Major Dates

• Study Start: September 1, 2015
• Primary Completion (Actual): July 10, 2019
• Study Completion (Actual): July 10, 2019

Study Registration Dates

• First Submitted: October 29, 2014
• First Submitted That Met QC Criteria: November 3, 2014
• First Posted (Estimate): November 5, 2014

Study Record Updates

• Last Update Posted (Actual): February 27, 2020
• Last Update Submitted That Met QC Criteria: February 26, 2020
• Last Verified: February 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Eye Diseases; Optic Nerve Diseases; Cranial Nerve Diseases; Myelitis, Transverse; Optic Neuritis; Multiple Sclerosis; Sclerosis; Neuromyelitis Optica
• Other Study ID Numbers: TolDec-EM-NMO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No