Dose Escalation Study of AST-OPC1 in Spinal Cord Injury

NCT02302157 | Completed Phase 1 Results DMC

• 1 other identifier: AST-OPC1-01
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 9

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).

Conditions

Cervical Spinal Cord Injury; Spine Injury; Spinal Cord Trauma

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Number of Adverse Events Within 1 Year (365 Days) That Are Related to AST-OPC1 Injection

  Numbers of adverse events within 1 year (365 days) that are related to AST-OPC1 injection

  One Year

Secondary Outcomes (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 as Secondary End Point - 365 Days After Injection of AST-OPC1

  One Year

Study Arms (1)

AST-OPC1

EXPERIMENTAL

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

Interventions

AST-OPC1 BIOLOGICAL

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

AST-OPC1

Eligibility Criteria

• Age: 18 Years - 69 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Lineage Cell Therapeutics, Inc.: lead

Study Sites (9)

Univ. of California at San Diego

La Jolla, California, 92093, United States

Location

Rancho Los Amigos/USC

Los Angeles, California, United States

Location

Stanford University/Santa Clara Valley Medical Center

San Jose, California, 95128, United States

Location

Shepherd Center

Atlanta, Georgia, 30309, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Indiana University

Indianapolis, Indiana, 46202, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Thomas Jefferson University/Magee Rehabilitation

Philadelphia, Pennsylvania, 19107, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (2)

• Fessler RG, Ehsanian R, Liu CY, Steinberg GK, Jones L, Lebkowski JS, Wirth ED, McKenna SL. A phase 1/2a dose-escalation study of oligodendrocyte progenitor cells in individuals with subacute cervical spinal cord injury. J Neurosurg Spine. 2022 Jul 8;37(6):812-820. doi: 10.3171/2022.5.SPINE22167. Print 2022 Dec 1.

  PMID: 35901693 DERIVED

• McKenna SL, Ehsanian R, Liu CY, Steinberg GK, Jones L, Lebkowski JS, Wirth E, Fessler RG. Ten-year safety of pluripotent stem cell transplantation in acute thoracic spinal cord injury. J Neurosurg Spine. 2022 Apr 1;37(3):321-330. doi: 10.3171/2021.12.SPINE21622. Print 2022 Sep 1.

  PMID: 35364569 DERIVED

MeSH Terms

Conditions

Spinal Injuries; Spinal Cord Injuries; Paralysis; Quadriplegia

Condition Hierarchy (Ancestors)

Back Injuries; Wounds and Injuries; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Trauma, Nervous System; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Limitations and Caveats

Open label study

Results Point of Contact

• Title: Avi Ben Shabat, MD
• Organization: Lineage

avi@cellcure.co.il

+972-509105205

Study Officials

• Edward D Wirth III, MD, PhD

  Asterias Biotherapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2014
• First Posted: November 26, 2014
• Study Start: March 1, 2015
• Primary Completion: December 1, 2018
• Study Completion: December 1, 2018
• Last Updated: July 14, 2021
• Results First Posted: July 13, 2021

Record last verified: 2021-06

Locations

US (9)