Safety of Autologous Human Schwann Cells (ahSC) in Subjects With Subacute SCI
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01739023|
Recruitment Status : Completed
First Posted : November 30, 2012
Last Update Posted : March 5, 2019
The purpose of this study is to assess the safety of autologous human Schwann cells (ahSC) transplantation in subjects with subacute SCI.
For humans with subacute SCI, we hypothesize that axons might show improved function if myelin repair is induced with the implantation of ahSC. In addition spinal cord cavitation may be reduced, and neural sprouting and plasticity may be enhanced via neurotrophic effects.
|Condition or disease||Intervention/treatment||Phase|
|Spinal Cord Injury Paraplegia||Biological: Autologous Human Schwann Cells||Phase 1|
Expanded Access : An investigational treatment associated with this study is no longer available outside the clinical trial. More info ...
Schwann cells are excellent candidates for transplantation into humans with SCI. Large numbers of ahSC can be derived for autologous implantation after a minor surgery for peripheral nerve harvesting, and purification and expansion of the cells in culture. Autologous cells offer important safety advantages that include no need for immune suppression, minimal risk of disease transfer, and a low risk of tumorigenicity.
Since 1990, scientists at the Miami Project to Cure Paralysis have generated extensive preclinical data suggesting Schwann cell transplantations are successful in rodents with SCI. The most recent work has focused on contusive injury models that are relevant to human injury. They have also been largely responsible for developing an efficient method for procuring large, essentially pure populations of human Schwann cells from adult peripheral nerve.
The rationale for implantation of ahSC in people with acute SCI is based on the evidence that Schwann cells are neuroprotective and are capable of myelinating axons. Using mitogen expanded human Schwann cells in SCID mice and athymic female nude rats demonstrated that human Schwann cells can survive and are capable of enhancing axonal regeneration and forming myelin after transplantation in animals with sciatic nerve transection or thoracic spinal cord transection. The proposed clinical trial will advance knowledge about the safety and feasibility of a cell-based treatment strategy for human SCI.
This Phase 1 clinical trial will employ an open label, unblinded, nonrandomized and non-placebo controlled dose-escalation design to evaluate the safety of transplantation of ahSC transplantation in subjects with subacute SCI.
A sural nerve harvest will occur within 30 days post-injury. Standard-of-care medical treatment and rehabilitation will proceed while the cells are being processed in a cGMP facility. No later than 72 days post-injury, the ahSC product will be administered via a single injection into the cavity of the spinal cord lesion.
Safety and efficacy assessments will be performed at week 1 and 2 post-transplantation and 2, 6, and 12 months post-transplantation.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||The Safety of Autologous Human Schwann Cells (ahSC) in Subjects With Subacute Spinal Cord Injury (SCI)|
|Study Start Date :||November 2012|
|Actual Primary Completion Date :||August 2016|
|Actual Study Completion Date :||August 2016|
|Experimental: Autologous Human Schwann Cells||
Biological: Autologous Human Schwann Cells
Schwann cells harvested from the sural nerve of the participant will be autologously transplanted into the epicenter of the participant's spinal cord injury.
- International Standards of Neurological Classification for Spinal Cord Injury [ Time Frame: Change from Baseline at 12 months ]
- MRI of spinal cord [ Time Frame: Change from Baseline at 12 months ]
- Neuropathic pain [ Time Frame: Change from Baseline at 12 months ]
- Spinal Cord Independence Measure III [ Time Frame: Change from Baseline at 12 months ]
- Functional Independence Measure [ Time Frame: Change from Baseline at 12 months ]
- Motor Evoked Potentials [ Time Frame: Change from Baseline at 12 months ]
- Somatosensory Evoked Potentials [ Time Frame: Change from Baseline at 12 months ]
- Autonomic - Head-up Tilt [ Time Frame: Change from Baseline at 12 months ]
- Autonomic - Sympathetic Skin Response [ Time Frame: Change from Baseline at 12 months ]
- ISCI Basic Bowel Dataset [ Time Frame: Change from Baseline at 12 months ]
- ISCI Basic Lower Urinary Tract Dataset [ Time Frame: Change from Baseline at 12 months ]
- SF-12 [ Time Frame: Change from Baseline at 12 months ]
- Patient Global Impression of Change [ Time Frame: Change from Baseline at 12 months ]
- Modified Ashworth Scale [ Time Frame: Change from Baseline at 12 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01739023
|United States, Florida|
|University of Miami Miller School of Medicine|
|Miami, Florida, United States, 33136|
|Study Director:||Dalton Dietrich, PhD||University of Miami|
|Principal Investigator:||Allan Levi, MD, PhD||University of Miami|
|Principal Investigator:||James Guest, MD, PhD||University of Miami|