Clinical trial NCT02669849: Study to Assess the Efficacy and Safety of VX-210 in Subjects With Acute Traumatic Cervical Spinal Cord Injury
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Cervical Spinal Cord Injury
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2016-02-01
Enrollment: 70
Sex: ALL
Minimum age (years): 14
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Change in Upper Extremity Motor Score (UEMS)
timeFrame: From baseline at 6 months post-treatment
Secondary outcomes:
measure: Spinal Cord Independence Measure (SCIM) III Self-Care Subscore
timeFrame: At 6 months post-treatment
measure: Capabilities of Upper Extremity Test (CUE-T) Score
timeFrame: At 6 months post-treatment
measure: Graded Redefined Assessment of Strength, Sensibility and Prehension (GRASSP) Quantitative Prehension Score
timeFrame: At 6 months post-treatment
measure: Percentage of American Spinal Injury Association Impairment Scale (AIS) Grade Responders
timeFrame: At 6 months post-treatment
measure: Percentage of Motor Level Responders
timeFrame: At 6 months post-treatment
measure: Time to Reach Maximum Observed Plasma Concentration (Tmax) of VX-210
timeFrame: up to 53 hours post-treatment
measure: Maximum Observed Plasma Concentration (Cmax) of VX-210
timeFrame: up to 53 hours post-treatment
measure: Area Under Plasma Concentration Time Curve (AUC) of VX-210
timeFrame: up to 53 hours post-treatment
Other outcomes:
Locations:
city: Birmingham
country: United States
state: Alabama
city: Tucson
country: United States
state: Arizona
city: Fresno
country: United States
state: California
city: Orange
country: United States
state: California
city: Sacramento
country: United States
state: California
city: Jacksonville
country: United States
state: Florida
city: Tampa
country: United States
state: Florida
city: Atlanta
country: United States
state: Georgia
city: Savannah
country: United States
state: Georgia
city: Iowa City
country: United States
state: Iowa
city: Louisville
country: United States
state: Kentucky
city: Baltimore
country: United States
state: Maryland
city: Boston
country: United States
state: Massachusetts
city: Ann Arbor
country: United States
state: Michigan
city: Minneapolis
country: United States
state: Minnesota
city: Jackson
country: United States
state: Mississippi
city: Saint Louis
country: United States
state: Missouri
city: Albuquerque
country: United States
state: New Mexico
city: Columbus
country: United States
state: Ohio
city: Toledo
country: United States
state: Ohio
city: Oklahoma City
country: United States
state: Oklahoma
city: Hershey
country: United States
state: Pennsylvania
city: Philadelphia
country: United States
state: Pennsylvania
city: Pittsburgh
country: United States
state: Pennsylvania
city: Providence
country: United States
state: Rhode Island
city: Austin
country: United States
state: Texas
city: Dallas
country: United States
state: Texas
city: San Antonio
country: United States
state: Texas
city: Salt Lake City
country: United States
state: Utah
city: Falls Church
country: United States
state: Virginia
city: Richmond
country: United States
state: Virginia
city: Roanoke
country: United States
state: Virginia
city: Seattle
country: United States
state: Washington
city: Vancouver
country: Canada
state: British Colombia
city: Halifax
country: Canada
state: Nova Scotia
city: Toronto
country: Canada
state: Ontario
city: Montreal
country: Canada
state: Quebec
city: Québec
country: Canada
state: Quebec
city: Saskatoon
country: Canada
Study officials:
References: