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

NCT02669849 | Terminated Phase 2 Results DMC

• 1 other identifier: VX15-210-101
• Study Type: interventional
• Target: 70
• Locations: 2 countries
• Sites: 39

Brief 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.

Conditions

Cervical Spinal Cord Injury

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (8)

• Spinal Cord Independence Measure (SCIM) III Self-Care Subscore

  At 6 months post-treatment

• Capabilities of Upper Extremity Test (CUE-T) Score

  At 6 months post-treatment

• Graded Redefined Assessment of Strength, Sensibility and Prehension (GRASSP) Quantitative Prehension Score

  At 6 months post-treatment

• Percentage of American Spinal Injury Association Impairment Scale (AIS) Grade Responders

  At 6 months post-treatment

• Percentage of Motor Level Responders

  At 6 months post-treatment

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Drug: Placebo

VX-210

EXPERIMENTAL

Drug: VX-210

Interventions

VX-210 DRUG

VX-210

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 14 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (39)

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Birmingham, Alabama, United States

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Tucson, Arizona, United States

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Fresno, California, United States

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Orange, California, United States

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Sacramento, California, United States

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Jacksonville, Florida, United States

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Tampa, Florida, United States

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Atlanta, Georgia, United States

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Savannah, Georgia, United States

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Iowa City, Iowa, United States

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Louisville, Kentucky, United States

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Baltimore, Maryland, United States

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Boston, Massachusetts, United States

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Ann Arbor, Michigan, United States

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Minneapolis, Minnesota, United States

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Jackson, Mississippi, United States

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St Louis, Missouri, United States

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Albuquerque, New Mexico, United States

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Columbus, Ohio, United States

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Toledo, Ohio, United States

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Oklahoma City, Oklahoma, United States

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Hershey, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Providence, Rhode Island, United States

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Austin, Texas, United States

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Dallas, Texas, United States

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San Antonio, Texas, United States

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Salt Lake City, Utah, United States

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Falls Church, Virginia, United States

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Richmond, Virginia, United States

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Roanoke, Virginia, United States

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Seattle, Washington, United States

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Vancouver, British Colombia, Canada

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Halifax, Nova Scotia, Canada

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Toronto, Ontario, Canada

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Montreal, Quebec, Canada

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Québec, Quebec, Canada

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Saskatoon, Canada

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Results Point of Contact

• Title: Medical Monitor
• Organization: Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com

617-341-6777

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 21, 2016
• First Posted: February 1, 2016
• Study Start: July 1, 2016
• Primary Completion: November 1, 2018
• Study Completion: November 1, 2018
• Last Updated: January 22, 2020
• Results First Posted: January 22, 2020

Record last verified: 2020-01

Locations

CA (6); US (33)