Intrathecal Administration of Expanded Wharton's Jelly Mesenchymal Stem Cells in Chronic Traumatic Spinal Cord Injury

Phase 1

Completed

• Conditions: Spinal Cord Injury, Chronic
• Interventions: Drug: XCEL-UMC-BETA; Drug: Placebo

• Registration Number: NCT03003364
• Lead Sponsor: Banc de Sang i Teixits

Brief Summary

This is a phase I/IIa, randomized, double-blind, two-arms, two-dose administration, placebo controlled, two-way crossover clinical trial in which 10 patients from 18 to 65 years of age affected with chronic traumatic spinal cord will enter the study with the objective to assess the safety and to obtain efficacy data in intrathecal administration of expanded Wharton's jelly mesenchymal stem cells.

Detailed Description

This is a phase I/IIa, randomized, double-blind, two-arms, two-dose administration, placebo controlled, clinical trial in which 10 patients from 18 to 65 years of age affected with chronic traumatic spinal cord will enter the study with the objective to assess the safety and to obtain efficacy data in intrathecal administration (L3 level) of expanded Wharton's jelly mesenchymal stem cells. Following the administration patients will remain for 24 h at the hospital and thereafter will be discharged. For the first period, the follow-up is planned at day 7 and at 1, 3 and 6 months. At month 6, the patients will be treated in a crossover way (second period) and will follow the same schedule for the follow-up. First clinical trial evaluation will be performed at 12 month follow-up. From 12 to 18 month after the first infusion, patients will be randomized again to active treatment or placebo (double-blind) in order to assess the safety and efficacy of a second dose at 12 month follow-up. Thereafter, patients will be followed up at 24 and 36 months as part of a long-term follow-up.

Study Timeline

• Study First Submitted: 2016-12-14
• Study First Submitted QC: 2016-12-21
• Study Start: 2016-12-27
• Study First Posted: 2016-12-28
• Primary Completion: 2019-07-25
• Status Verified: 2020-02
• Study Completion: 2020-02-11
• Last Update Submitted: 2020-02-13
• Last Update Posted: 2020-02-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

1. Single spinal cord injury lesion caused by trauma
2. Affected cord segments between T2 and T11, confirmed by magnetic resonance
3. Complete paraplegia (ASIA A)
4. Chronic disease state (between 12 months and 5 years after the injury)
5. Patients from 18 to 65 years of age, both sexes
6. Life expectancy > 2 years
7. Confidence that the patient will attend the follow-up visits.
8. Given informed consent in writing
9. Patient is able to understand the study and its procedures

Exclusion Criteria

1. Mechanic ventilation
2. Lesion affecting multiple levels
3. Lesion length superior to 3 spinal cord segments, assessed by magnetic resonance
4. Penetrating trauma affecting the spinal cord
5. Positive serology to HIV, HBV, HCV and or syphilis
6. Pregnant woman or without proper anticonceptive measures according to the investigator, or breath feeding
7. Use of metal implants that complicates the MRI interpretation
8. Planned spinal surgery within subsequent 24 month after entering the trial
9. Intrathecal medication or immunosuppressive drugs the previous 60 days.
10. Neurodegenerative diseases
11. Significant abnormal laboratory tests that contraindicates patient's participation in the study.
12. Neoplasia within the previous 5 years, or without complete remission
13. Patient with difficulty for communicating
14. Participation in another clinical trial or treated with an investigational medicinal product the previous 60 days
15. Contraindication for lumbar punction
16. Other pathologic conditions or circumstances that could complicate the participation of the patient in the study according to medical criteria
17. The patient does not accept to be followed-up for a period that could exceed the clinical trial length

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
XCEL-UMC-BETA/placebo	XCEL-UMC-BETA	Ex vivo cultured human mesenchymal stem cells from Wharton jelly, in a blinded syringe (initial treatment) / Placebo (month 6)
Placebo/XCEL-UMC-BETA	Placebo	Placebo in a blinded syringe (initial treatment) / XCEL-UMC-BETA (month 6)
XCEL-UMC-BETA/placebo	Placebo	Ex vivo cultured human mesenchymal stem cells from Wharton jelly, in a blinded syringe (initial treatment) / Placebo (month 6)
Placebo/XCEL-UMC-BETA	XCEL-UMC-BETA	Placebo in a blinded syringe (initial treatment) / XCEL-UMC-BETA (month 6)

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent adverse events	12 months	Adverse events

Secondary Outcome Measures

Name	Time	Method
Motor electrophysiology assessment	6 month	Evoked potentials
Electrical nerve stimulation on pain perception	6 month	Pain threshold perception
Mictional dysfunction	6 month	Urodynamic testing
Immunology	1 months	Antibodies anti-HLA in peripherical blood and in cerebrospinal fluid
Neuropathic pain	6 months	Numerical scale (0 to 10)
Size injury	12 months	Magnetic Resonance Imaging
Anal sphincter integrity	6 month	Anorectal manometry test
Extent and severity of a patient's spinal cord injury	6 months	ASIA Impairment Scale
Functionality	6 months	SCIM III scale
Quality of life (individual overall perception of quality of life, individual overall perception of their health, Physical health, Psychological, Social relationships, Environment)	6 months	WHOQOL BREF questionnaire
Somatosensory electrophysiology assessment	6 month	Evoked potentials
Spasticity	6 months	Modified Ashworth scale
Urinary disorder	6 months	Qualiveen questionnaire
Presence of allogeneic cells	1 month	Chimerism in cerebrospinal fluid

Trial Locations

Locations (1)

Hospital de Neurorehabilitació Institut Guttmann; 🇪🇸 Badalona, Barcelona, Spain