Dose Escalation Study of AST-OPC1 in Spinal Cord Injury

• ClinicalTrials.gov Identifier: NCT02302157
• Recruitment Status: Completed
• First Posted: November 26, 2014
• Last Update Posted: July 3, 2019
• Sponsor: Asterias Biotherapeutics, Inc.
• Information provided by (Responsible Party): Asterias Biotherapeutics, Inc.

Study Description

Brief Summary:

The purpose of this study is to evaluate the safety of cross sequential escalating doses of AST-OPC1 administered among 5 cohorts at a single time-point between 21 and 42 days post injury, inclusively, to subjects with subacute cervical spinal cord injuries (SCI).

Condition or disease	Intervention/treatment	Phase
Cervical Spinal Cord Injury; Spine Injury; Spinal Cord Trauma	Biological: AST-OPC1	Phase 1; Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 25 participants
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase 1/2a Dose Escalation Study of AST-OPC1 in Subjects With Subacute Cervical Spinal Cord Injury
• Study Start Date: March 2015
• Actual Primary Completion Date: December 2018
• Actual Study Completion Date: December 2018

Arms and Interventions

Arm	Intervention/treatment
Experimental: AST-OPC1; Open label, dose escalation, cross-sequential cohort of subjects who receive an injection or two injections of AST-OPC1 at a single time-point	Biological: AST-OPC1; One injection of 2 million or 10 million AST-OPC1 cells, or 2 injections of 10 million AST-OPC1 cells for a total of 20 million cells; cohort dependent

Outcome Measures

Primary Outcome Measures :

1. Number of adverse events within 1 year (365 days) that are related to AST-OPC1 injection [ Time Frame: One Year ]

Secondary Outcome Measures :

1. Neurological function as measured by upper extremity motor scores and motor level on International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) examinations at 30, 60, 90, 180, 270, and 365 days after injection of AST-OPC1 [ Time Frame: One Year ]

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 69 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Major Inclusion Criteria:

• Sensorimotor complete, traumatic SCI (ASIA Impairment Scale A) for cohorts 1,2,3
• Sensorimotor incomplete, traumatic SCI (ASIA Impairment Scale B) for cohorts 4,5
• Last fully preserved single neurological level (SNL) from C-4 to C-7
• From 18 through 69 years of age at time of injury
• Single spinal cord lesion on a post-stabilization magnetic resonance imaging (MRI) scan, with sufficient visualization of the spinal cord injury epicenter and lesion margins to enable post-injection safety monitoring
• Informed consent for this protocol and the companion long term follow-up protocol must be provided and documented (i.e., signed informed consent forms) no later than 37 days following injury
• Able to participate in an elective surgical procedure to inject AST-OPC1 21-42 days following SCI

Major Exclusion Criteria:

• SCI due to penetrating trauma
• Traumatic anatomical transection or laceration of the spinal cord based on prior surgery or MRI
• Any concomitant injury that interferes with the performance, interpretation or validity of neurological examinations
• Inability to communicate effectively with neurological examiner such that the validity of patient data could be compromised
• Significant organ damage or systemic disease that would create an unacceptable risk for surgery or immunosuppression
• History of any malignancy (except non-melanoma skin cancers)
• Pregnant or nursing women
• Body mass index (BMI) > 35 or weight > 300 lbs.
• Active participation in another experimental procedure/intervention

Contacts and Locations

Locations

United States, California

Univ. of California at San Diego

La Jolla, California, United States, 92093

Rancho Los Amigos/USC

Los Angeles, California, United States

Stanford University/Santa Clara Valley Medical Center

San Jose, California, United States, 95128

United States, Georgia

Shepherd Center

Atlanta, Georgia, United States, 30309

United States, Illinois

Rush University Medical Center

Chicago, Illinois, United States, 60612

United States, Indiana

Indiana University

Indianapolis, Indiana, United States, 46202

United States, Missouri

Washington University

Saint Louis, Missouri, United States, 63110

United States, Pennsylvania

Thomas Jefferson University/Magee Rehabilitation

Philadelphia, Pennsylvania, United States, 19107

United States, Wisconsin

Medical College of Wisconsin

Milwaukee, Wisconsin, United States, 53226

Sponsors and Collaborators

Asterias Biotherapeutics, Inc.

Investigators

• Study Director: Edward D Wirth III, MD, PhD; Asterias Biotherapeutics

More Information

• Responsible Party: Asterias Biotherapeutics, Inc.
• ClinicalTrials.gov Identifier: NCT02302157 History of Changes
• Other Study ID Numbers: AST-OPC1-01
• First Posted: November 26, 2014
• Last Update Posted: July 3, 2019
• Last Verified: July 2019

Keywords provided by Asterias Biotherapeutics, Inc.:

Cervical spinal cord injury; Neural cell; Trauma spinal cord; stemcell transplant; Cervical spine injury; Spine injury; oligodendrocyte progenitor cell; Subacute spinal cord injury; AST-OPC1; SCiStar; OPC1; Paralysis; Quadriplegia; Tetraplegia

Additional relevant MeSH terms:

Spinal Cord Injuries; Spinal Injuries; Wounds and Injuries; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Trauma, Nervous System; Back Injuries