Study to Assess the Efficacy and Safety of VX-210 in Subjects With Acute Traumatic Cervical Spinal Cord Injury

Cervical Spinal Cord Injury Clinical Trial

Official title:

A Phase 2b/3, Double-blind, Randomized, Placebo Controlled, Multicenter Study to Assess the Efficacy and Safety of VX-210 in Subjects With Acute Traumatic Cervical Spinal Cord Injury

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02669849
• Other study ID #: VX15-210-101
• Status: Terminated
• Phase: Phase 2/Phase 3
• Start date: July 2016
• Est. completion date: November 2018

Study information

• Verified date: January 2020
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to determine the efficacy and safety of VX-210 in subjects with Acute Traumatic Cervical Spinal Cord Injury. Secondary objectives include the specific evaluation of the effects of VX-210 on neurological recovery and daily function after spinal cord injury.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 70
• Est. completion date: November 2018
• Est. primary completion date: November 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years to 75 Years

Eligibility

Inclusion Criteria:

• Acute traumatic cervical SCI, motor level of C4, C5, C6, or C7 on each side:

• Screening UEMS score must be =16 points on each side.

• AIS A subjects with a C4 motor level on both sides must have at least 1 point of motor activity between C5 and T1 on at least 1 side.

• AIS B subjects with a C4 motor level on both sides must have at least 1 point of motor activity between C5 and C7 on at least 1 side.

• American Spinal Injury Association Impairment Scale (AIS) grade A or AIS grade B.

• Scheduled and planned to undergo a spinal decompression/stabilization surgery that commences within 72 hours after the initial injury.

• Computed tomography (CT) scan or magnetic resonance imaging (MRI) is consistent with the subject's neurological deficit.

Exclusion Criteria:

• Participation in any other clinical study for acute SCI without approval by the sponsor.

• Inability to undergo decompression/stabilization surgery that commences within 72 hours after injury.

• One or more upper extremity muscle groups untestable during screening ISNCSCI examination.

• Acute SCI from gunshot or penetrating/stab wound; non-traumatic SCI (e.g., transverse myelitis, acute disc herniation); brachial plexus injury; complete spinal cord transection; or multifocal SCI.

• Females who are breastfeeding or have a positive serum pregnancy test.

• Body mass index (BMI) of =40 kg/m^2 at screening.

• History of an adverse reaction to a fibrin sealant or its components.

• Unconsciousness or other mental impairment that precludes reliable International Standards for the Neurological Classification of Spinal Cord Injury (ISNCSCI) examination.

• Known immunodeficiency, including human immunodeficiency virus, or use of immunosuppressive or cancer chemotherapeutic drugs.

• Any significant medical or psychiatric comorbidities that would significantly increase the risk of study enrollment and/or significantly interfere with study outcomes or assessments, in the judgment of the investigator.

Other protocol defined Inclusion/Exclusion criteria may apply.

Related Conditions & MeSH terms

• Cervical Spinal Cord Injury
• Spinal Cord Injuries
• Wounds and Injuries

Intervention

Drug:
• VX-210

• Placebo

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Upper Extremity Motor Score (UEMS)	UEMS focuses selectively on the hand and arm control most relevant to individuals with a cervical spinal cord injury. UEMS ranges from 0 to 50, where a higher score indicates a better movement of hand and arm.	From baseline at 6 months post-treatment
Secondary	Spinal Cord Independence Measure (SCIM) III Self-Care Subscore	SCIM self-care subscore measures self-care abilities (feeding, dressing, grooming, bathing), respiration and sphincter management and mobility. The score ranges from 0-20, where a higher score represents a better outcome.	At 6 months post-treatment
Secondary	Capabilities of Upper Extremity Test (CUE-T) Score	CUE-T measures a participant's ability to perform specific functional movements/tasks with the arms and hands (for example: grasping a pencil, pushing or lifting a weight). CUE-T score ranges from 0-128, where a higher score indicates an improvement in participant's ability.	At 6 months post-treatment
Secondary	Graded Redefined Assessment of Strength, Sensibility and Prehension (GRASSP) Quantitative Prehension Score	GRASSP measures participant's ability to perform specific functional tasks with the arms, hands, and fingers. GRASSP quantitative prehension score ranges from 0-60, where a higher score indicates a better performance.	At 6 months post-treatment
Secondary	Percentage of American Spinal Injury Association Impairment Scale (AIS) Grade Responders	AIS ranks impairment according to body-wide motor/sensory results: Grade A: Complete (no sensory or motor function is preserved in the sacral segments S4 to 5); Grade B: Sensory Incomplete (sensory but not motor function is preserved below the neurological level and includes the sacral segments S4 to 5); Grade C: Motor Incomplete (motor function is preserved at the most caudal sacral segments); Grade D: Motor Incomplete (motor incomplete status as defined above, with at least half or more of key muscle functions below the single neurological level of injury having a muscle grade >=3; Grade E: Normal (sensation and motor function as tested are graded as normal in all segments). An AIS responder was defined as a subject with improvement by =2 AIS grades (i.e., baseline AIS Grade A changed to Grade C, D, or E; baseline AIS Grade B changed to D or E at 6 months after treatment).	At 6 months post-treatment
Secondary	Percentage of Motor Level Responders	The motor level score for the right or left side assesses contraction strength of 10 key muscles in the upper and lower extremities on each side of the body; each muscle receives a score from 0 (total paralysis) to 5 ([normal] active movement). A motor level responder was defined as a subject with improvement by =2 motor levels on either side of the body (i.e., baseline level C4 changed to C6, C7, C8 on the left; or baseline level C5 changed to C7, C8 on the right).	At 6 months post-treatment
Secondary	Time to Reach Maximum Observed Plasma Concentration (Tmax) of VX-210		up to 53 hours post-treatment
Secondary	Maximum Observed Plasma Concentration (Cmax) of VX-210		up to 53 hours post-treatment
Secondary	Area Under Plasma Concentration Time Curve (AUC) of VX-210		up to 53 hours post-treatment