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

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ClinicalTrials.gov Identifier: NCT02669849
Recruitment Status : Terminated
First Posted : February 1, 2016
Results First Posted : January 22, 2020
Last Update Posted : January 22, 2020
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Cervical Spinal Cord Injury
Interventions Drug: VX-210
Drug: Placebo
Enrollment 70
Recruitment Details Participants were randomized to receive a single 9-mg dose of VX-210 or a placebo (buffer solution). A single 3-mg dose of VX-210 in fibrin sealant was initially included but was subsequently removed during the study through a protocol amendment. The 3-mg arm was not analyzed for primary and secondary efficacy and pharmacokinetic analysis.
Pre-assignment Details A total of 70 participants were randomized, out of which 67 participants received the study drug.
Arm/Group Title Placebo VX-210 3 mg VX-210 9 mg
Hide Arm/Group Description Participants who received placebo matched to VX-210 as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury. Participants who received VX-210 3 milligram (mg) as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury. Participants who received VX-210 9 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Period Title: Overall Study
Started 29 6 32
Completed 11 4 8
Not Completed 18 2 24
Reason Not Completed
Withdrawal of Consent (not due to AE)             0             0             3
Lost to Follow-up             1             0             3
Death             1             1             2
Study terminated by sponsor             16             1             14
Other             0             0             2
Arm/Group Title Placebo VX-210 3 mg VX-210 9 mg Total
Hide Arm/Group Description Participants who received placebo matched to VX-210 as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury. Participants who received VX-210 3 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury. Participants who received VX-210 9 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury. Total of all reporting groups
Overall Number of Baseline Participants 29 6 32 67
Hide Baseline Analysis Population Description
Overall number of Baseline participants includes all randomized participants who received study drug.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 29 participants 6 participants 32 participants 67 participants
43.8  (17.4) 39.5  (24.4) 43.4  (17.3) 43.2  (17.8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants 6 participants 32 participants 67 participants
Female
8
  27.6%
2
  33.3%
6
  18.8%
16
  23.9%
Male
21
  72.4%
4
  66.7%
26
  81.3%
51
  76.1%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants 6 participants 32 participants 67 participants
Hispanic or Latino
1
   3.4%
1
  16.7%
1
   3.1%
3
   4.5%
Not Hispanic or Latino
28
  96.6%
5
  83.3%
31
  96.9%
64
  95.5%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants 6 participants 32 participants 67 participants
American Indian or Alaska Native
1
   3.4%
0
   0.0%
2
   6.3%
3
   4.5%
Asian
2
   6.9%
0
   0.0%
3
   9.4%
5
   7.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
6
  20.7%
0
   0.0%
5
  15.6%
11
  16.4%
White
19
  65.5%
3
  50.0%
21
  65.6%
43
  64.2%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
1
   3.4%
3
  50.0%
1
   3.1%
5
   7.5%
Upper Extremity Motor Score (UEMS)   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 29 participants 6 participants 32 participants 67 participants
13.00  (8.96) 10.17  (6.59) 14.03  (8.33) 13.24  (8.43)
[1]
Measure Description: 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 highest score indicates the positive movement of hand and arm.
1.Primary Outcome
Title Change in Upper Extremity Motor Score (UEMS)
Hide Description 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.
Time Frame From baseline at 6 months post-treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The "Overall Number of Participants Analyzed" included all randomized subjects who received study drug. As per the amended protocol, the 3 mg arm was no longer planned to be assessed for any primary or secondary outcome measure.
Arm/Group Title Placebo VX-210 9 mg
Hide Arm/Group Description:
Participants who received placebo matched to VX-210 as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Participants who received VX-210 9 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Overall Number of Participants Analyzed 29 32
Mean (Standard Deviation)
Unit of Measure: units on a scale
8.90  (9.48) 8.69  (7.35)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, VX-210 9 mg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7519
Comments [Not Specified]
Method Mixed-effects model for repeated measure
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value -0.69
Confidence Interval (2-Sided) 95%
-5.08 to 3.69
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Spinal Cord Independence Measure (SCIM) III Self-Care Subscore
Hide Description 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.
Time Frame At 6 months post-treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The "Overall Number of Participants Analyzed" included all randomized subjects who received study drug. As per the amended protocol, the 3 mg arm was no longer planned to be assessed for any primary or secondary outcome measure.
Arm/Group Title Placebo VX-210 9 mg
Hide Arm/Group Description:
Participants who received placebo matched to VX-210 as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Participants who received VX-210 9 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Overall Number of Participants Analyzed 29 32
Mean (Standard Deviation)
Unit of Measure: units on a scale
6.2  (7.0) 5.9  (6.6)
3.Secondary Outcome
Title Capabilities of Upper Extremity Test (CUE-T) Score
Hide Description 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.
Time Frame At 6 months post-treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The "Overall Number of Participants Analyzed" included all randomized subjects who received study drug. As per the amended protocol, the 3 mg arm was no longer planned to be assessed for any primary or secondary outcome measure.
Arm/Group Title Placebo VX-210 9 mg
Hide Arm/Group Description:
Participants who received placebo matched to VX-210 as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Participants who received VX-210 9 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Overall Number of Participants Analyzed 29 32
Mean (Standard Deviation)
Unit of Measure: units on a scale
39.6  (38.4) 42.8  (41.1)
4.Secondary Outcome
Title Graded Redefined Assessment of Strength, Sensibility and Prehension (GRASSP) Quantitative Prehension Score
Hide Description 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.
Time Frame At 6 months post-treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The "Overall Number of Participants Analyzed" included all randomized subjects who received study drug. As per the amended protocol, the 3 mg arm was no longer planned to be assessed for any primary or secondary outcome measure.
Arm/Group Title Placebo VX-210 9 mg
Hide Arm/Group Description:
Participants who received placebo matched to VX-210 as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Participants who received VX-210 9 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Overall Number of Participants Analyzed 29 32
Mean (Standard Deviation)
Unit of Measure: units on a scale
18.9  (19.4) 20.1  (21.6)
5.Secondary Outcome
Title Percentage of American Spinal Injury Association Impairment Scale (AIS) Grade Responders
Hide Description 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).
Time Frame At 6 months post-treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The "Overall Number of Participants Analyzed" included all randomized subjects who received study drug. As per the amended protocol, the 3 mg arm was no longer planned to be assessed for any primary or secondary outcome measure.
Arm/Group Title Placebo VX-210 9 mg
Hide Arm/Group Description:
Participants who received placebo matched to VX-210 as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Participants who received VX-210 9 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Overall Number of Participants Analyzed 29 32
Measure Type: Number
Unit of Measure: percentage of participants
30.0 26.7
6.Secondary Outcome
Title Percentage of Motor Level Responders
Hide Description 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).
Time Frame At 6 months post-treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The "Overall Number of Participants Analyzed" included all randomized subjects who received study drug. As per the amended protocol, the 3 mg arm was no longer planned to be assessed for any primary or secondary outcome measure.
Arm/Group Title Placebo VX-210 9 mg
Hide Arm/Group Description:
Participants who received placebo matched to VX-210 as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Participants who received VX-210 9 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Overall Number of Participants Analyzed 29 32
Measure Type: Number
Unit of Measure: percentage of participants
35.0 33.3
7.Secondary Outcome
Title Time to Reach Maximum Observed Plasma Concentration (Tmax) of VX-210
Hide Description [Not Specified]
Time Frame up to 53 hours post-treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set included all participants for which PK data was collected. As per the amended protocol, the 3 mg arm was no longer planned to be assessed for any primary or secondary outcome measure.
Arm/Group Title VX-210 9 mg
Hide Arm/Group Description:
Participants who received VX-210 9 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Overall Number of Participants Analyzed 28
Median (Full Range)
Unit of Measure: hours (h)
4.59
(2.58 to 52.38)
8.Secondary Outcome
Title Maximum Observed Plasma Concentration (Cmax) of VX-210
Hide Description [Not Specified]
Time Frame up to 53 hours post-treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set included all participants for which PK data was collected. As per the amended protocol, the 3 mg arm was no longer planned to be assessed for any primary or secondary outcome measure.
Arm/Group Title VX-210 9 mg
Hide Arm/Group Description:
Participants who received VX-210 9 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Overall Number of Participants Analyzed 28
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: nanogram per milliliter (ng/mL)
2.856
(200.35%)
9.Secondary Outcome
Title Area Under Plasma Concentration Time Curve (AUC) of VX-210
Hide Description [Not Specified]
Time Frame up to 53 hours post-treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set included all participants for which PK data was collected. As per the amended protocol, the 3 mg arm was no longer planned to be assessed for any primary or secondary outcome measure.
Arm/Group Title VX-210 9 mg
Hide Arm/Group Description:
Participants who received VX-210 9 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
Overall Number of Participants Analyzed 28
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: h*ng/mL
44.683
(284.95%)
Time Frame Up to 28 months
Adverse Event Reporting Description Death reported under placebo arm was not treatment-emergent.
 
Arm/Group Title Placebo VX-210 3 mg VX-210 9 mg
Hide Arm/Group Description Participants who received placebo matched to VX-210 as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury. Participants who received VX-210 3 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury. Participants who received VX-210 9 mg as a single dose in fibrin sealant topically to the dural surface of the spinal cord within 72 hours after the initial injury.
All-Cause Mortality
Placebo VX-210 3 mg VX-210 9 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/29 (3.45%)   1/6 (16.67%)   2/32 (6.25%) 
Hide Serious Adverse Events
Placebo VX-210 3 mg VX-210 9 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   18/29 (62.07%)   6/6 (100.00%)   16/32 (50.00%) 
Blood and lymphatic system disorders       
Anaemia  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Cardiac disorders       
Bradycardia  1  2/29 (6.90%)  1/6 (16.67%)  0/32 (0.00%) 
Cardiac arrest  1  2/29 (6.90%)  0/6 (0.00%)  0/32 (0.00%) 
Cardiac failure  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
Sinus bradycardia  1  1/29 (3.45%)  1/6 (16.67%)  0/32 (0.00%) 
Ear and labyrinth disorders       
Deafness neurosensory  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Gastrointestinal disorders       
Rectal haemorrhage  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
General disorders       
Pyrexia  1  1/29 (3.45%)  0/6 (0.00%)  1/32 (3.13%) 
Immune system disorders       
Anaphylactic reaction  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
Infections and infestations       
Acute sinusitis  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
Cellulitis  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
Clostridium difficile colitis  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Device related sepsis  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Empyema  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Haemophilus infection  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Infected skin ulcer  1  1/29 (3.45%)  0/6 (0.00%)  1/32 (3.13%) 
Lower respiratory tract infection bacterial  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Osteomyelitis  1  2/29 (6.90%)  0/6 (0.00%)  2/32 (6.25%) 
Pneumonia  1  4/29 (13.79%)  3/6 (50.00%)  4/32 (12.50%) 
Pneumonia staphylococcal  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Sepsis  1  3/29 (10.34%)  0/6 (0.00%)  4/32 (12.50%) 
Septic shock  1  1/29 (3.45%)  0/6 (0.00%)  1/32 (3.13%) 
Staphylococcal bacteraemia  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
Urinary tract infection  1  4/29 (13.79%)  0/6 (0.00%)  2/32 (6.25%) 
Urosepsis  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Vaginal infection  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
Viral infection  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Wound sepsis  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Injury, poisoning and procedural complications       
Autonomic dysreflexia  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
Pneumothorax traumatic  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Postoperative respiratory failure  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Seroma  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Wound dehiscence  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Nervous system disorders       
Loss of consciousness  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Seizure  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
Spinal cord compression  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Syncope  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
Psychiatric disorders       
Delirium  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Mental disorder  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Renal and urinary disorders       
Calculus bladder  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Haematuria  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
Renal failure  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Ureterolithiasis  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Respiratory, thoracic and mediastinal disorders       
Acute respiratory distress syndrome  1  1/29 (3.45%)  0/6 (0.00%)  1/32 (3.13%) 
Acute respiratory failure  1  2/29 (6.90%)  2/6 (33.33%)  2/32 (6.25%) 
Atelectasis  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Bronchial secretion retention  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Bronchopleural fistula  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Chronic respiratory failure  1  0/29 (0.00%)  0/6 (0.00%)  2/32 (6.25%) 
Hypoxia  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
Pleural effusion  1  1/29 (3.45%)  1/6 (16.67%)  1/32 (3.13%) 
Pneumonia aspiration  1  2/29 (6.90%)  0/6 (0.00%)  2/32 (6.25%) 
Pneumothorax spontaneous  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Pulmonary embolism  1  2/29 (6.90%)  2/6 (33.33%)  1/32 (3.13%) 
Respiratory distress  1  1/29 (3.45%)  0/6 (0.00%)  1/32 (3.13%) 
Respiratory failure  1  3/29 (10.34%)  2/6 (33.33%)  3/32 (9.38%) 
Skin and subcutaneous tissue disorders       
Decubitus ulcer  1  2/29 (6.90%)  1/6 (16.67%)  3/32 (9.38%) 
Vascular disorders       
Deep vein thrombosis  1  1/29 (3.45%)  1/6 (16.67%)  1/32 (3.13%) 
Hypotension  1  0/29 (0.00%)  0/6 (0.00%)  1/32 (3.13%) 
Neurogenic shock  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Subclavian vein thrombosis  1  1/29 (3.45%)  0/6 (0.00%)  0/32 (0.00%) 
1
Term from vocabulary, Meddra 21.1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo VX-210 3 mg VX-210 9 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   28/29 (96.55%)   6/6 (100.00%)   32/32 (100.00%) 
Blood and lymphatic system disorders       
Anaemia  1  8/29 (27.59%)  2/6 (33.33%)  6/32 (18.75%) 
Hypercoagulation  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Leukocytosis  1  0/29 (0.00%)  1/6 (16.67%)  1/32 (3.13%) 
Thrombocytosis  1  1/29 (3.45%)  1/6 (16.67%)  1/32 (3.13%) 
Cardiac disorders       
Bradycardia  1  6/29 (20.69%)  0/6 (0.00%)  4/32 (12.50%) 
Sinus bradycardia  1  0/29 (0.00%)  0/6 (0.00%)  2/32 (6.25%) 
Sinus tachycardia  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Tachycardia  1  3/29 (10.34%)  1/6 (16.67%)  0/32 (0.00%) 
Ventricular tachycardia  1  0/29 (0.00%)  0/6 (0.00%)  2/32 (6.25%) 
Ear and labyrinth disorders       
Tinnitus  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Endocrine disorders       
Hypothyroidism  1  0/29 (0.00%)  0/6 (0.00%)  2/32 (6.25%) 
Eye disorders       
Dry eye  1  0/29 (0.00%)  1/6 (16.67%)  2/32 (6.25%) 
Gastrointestinal disorders       
Abdominal discomfort  1  1/29 (3.45%)  0/6 (0.00%)  2/32 (6.25%) 
Anorectal discomfort  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Colitis  1  1/29 (3.45%)  1/6 (16.67%)  0/32 (0.00%) 
Constipation  1  4/29 (13.79%)  0/6 (0.00%)  7/32 (21.88%) 
Diarrhoea  1  3/29 (10.34%)  1/6 (16.67%)  5/32 (15.63%) 
Dry mouth  1  1/29 (3.45%)  1/6 (16.67%)  0/32 (0.00%) 
Dysphagia  1  1/29 (3.45%)  1/6 (16.67%)  6/32 (18.75%) 
Flatulence  1  0/29 (0.00%)  0/6 (0.00%)  2/32 (6.25%) 
Ileus  1  4/29 (13.79%)  0/6 (0.00%)  3/32 (9.38%) 
Melaena  1  1/29 (3.45%)  1/6 (16.67%)  0/32 (0.00%) 
Nausea  1  7/29 (24.14%)  3/6 (50.00%)  5/32 (15.63%) 
Neurogenic bowel  1  3/29 (10.34%)  0/6 (0.00%)  4/32 (12.50%) 
Odynophagia  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Oral pain  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Pancreatitis  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Vomiting  1  4/29 (13.79%)  0/6 (0.00%)  2/32 (6.25%) 
General disorders       
Chest pain  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Chills  1  1/29 (3.45%)  1/6 (16.67%)  0/32 (0.00%) 
Generalised oedema  1  0/29 (0.00%)  0/6 (0.00%)  3/32 (9.38%) 
Impaired healing  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Oedema peripheral  1  2/29 (6.90%)  1/6 (16.67%)  0/32 (0.00%) 
Pain  1  2/29 (6.90%)  0/6 (0.00%)  2/32 (6.25%) 
Pyrexia  1  13/29 (44.83%)  3/6 (50.00%)  16/32 (50.00%) 
Immune system disorders       
Drug hypersensitivity  1  2/29 (6.90%)  0/6 (0.00%)  0/32 (0.00%) 
Infections and infestations       
Clostridium difficile infection  1  0/29 (0.00%)  1/6 (16.67%)  2/32 (6.25%) 
Conjunctivitis  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Fungal skin infection  1  3/29 (10.34%)  1/6 (16.67%)  0/32 (0.00%) 
Infected skin ulcer  1  2/29 (6.90%)  0/6 (0.00%)  0/32 (0.00%) 
Influenza  1  1/29 (3.45%)  0/6 (0.00%)  2/32 (6.25%) 
Oral candidiasis  1  2/29 (6.90%)  1/6 (16.67%)  5/32 (15.63%) 
Pneumonia  1  7/29 (24.14%)  2/6 (33.33%)  11/32 (34.38%) 
Staphylococcal infection  1  0/29 (0.00%)  0/6 (0.00%)  2/32 (6.25%) 
Urinary tract infection  1  18/29 (62.07%)  4/6 (66.67%)  18/32 (56.25%) 
Vulvovaginal mycotic infection  1  1/29 (3.45%)  1/6 (16.67%)  0/32 (0.00%) 
Injury, poisoning and procedural complications       
Autonomic dysreflexia  1  3/29 (10.34%)  0/6 (0.00%)  3/32 (9.38%) 
Limb injury  1  2/29 (6.90%)  0/6 (0.00%)  1/32 (3.13%) 
Muscle rupture  1  0/29 (0.00%)  0/6 (0.00%)  2/32 (6.25%) 
Post procedural haemorrhage  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Postoperative respiratory failure  1  2/29 (6.90%)  0/6 (0.00%)  0/32 (0.00%) 
Procedural pain  1  1/29 (3.45%)  1/6 (16.67%)  0/32 (0.00%) 
Weaning failure  1  0/29 (0.00%)  0/6 (0.00%)  2/32 (6.25%) 
Wound  1  0/29 (0.00%)  1/6 (16.67%)  1/32 (3.13%) 
Investigations       
Aspartate aminotransferase increased  1  2/29 (6.90%)  0/6 (0.00%)  1/32 (3.13%) 
Blood creatine phosphokinase increased  1  2/29 (6.90%)  0/6 (0.00%)  0/32 (0.00%) 
Blood magnesium decreased  1  2/29 (6.90%)  0/6 (0.00%)  2/32 (6.25%) 
Blood phosphorus decreased  1  1/29 (3.45%)  0/6 (0.00%)  2/32 (6.25%) 
Electrocardiogram abnormal  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Haemophilus test positive  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Hepatic enzyme increased  1  1/29 (3.45%)  0/6 (0.00%)  2/32 (6.25%) 
International normalised ratio increased  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Liver function test abnormal  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Neutrophil count increased  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Prothrombin time prolonged  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
White blood cell count increased  1  0/29 (0.00%)  2/6 (33.33%)  0/32 (0.00%) 
Metabolism and nutrition disorders       
Electrolyte imbalance  1  2/29 (6.90%)  0/6 (0.00%)  0/32 (0.00%) 
Fluid overload  1  2/29 (6.90%)  0/6 (0.00%)  1/32 (3.13%) 
Hypercalcaemia  1  1/29 (3.45%)  1/6 (16.67%)  0/32 (0.00%) 
Hyperglycaemia  1  1/29 (3.45%)  1/6 (16.67%)  2/32 (6.25%) 
Hypernatraemia  1  2/29 (6.90%)  1/6 (16.67%)  3/32 (9.38%) 
Hypoalbuminaemia  1  2/29 (6.90%)  0/6 (0.00%)  0/32 (0.00%) 
Hypocalcaemia  1  2/29 (6.90%)  0/6 (0.00%)  1/32 (3.13%) 
Hypoglycaemia  1  0/29 (0.00%)  0/6 (0.00%)  2/32 (6.25%) 
Hypokalaemia  1  4/29 (13.79%)  0/6 (0.00%)  5/32 (15.63%) 
Hypomagnesaemia  1  4/29 (13.79%)  1/6 (16.67%)  1/32 (3.13%) 
Hyponatraemia  1  2/29 (6.90%)  1/6 (16.67%)  5/32 (15.63%) 
Hypophosphataemia  1  4/29 (13.79%)  0/6 (0.00%)  2/32 (6.25%) 
Malnutrition  1  4/29 (13.79%)  0/6 (0.00%)  3/32 (9.38%) 
Musculoskeletal and connective tissue disorders       
Arthralgia  1  0/29 (0.00%)  1/6 (16.67%)  1/32 (3.13%) 
Muscle spasms  1  9/29 (31.03%)  3/6 (50.00%)  14/32 (43.75%) 
Musculoskeletal discomfort  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Musculoskeletal pain  1  5/29 (17.24%)  1/6 (16.67%)  5/32 (15.63%) 
Neck pain  1  4/29 (13.79%)  0/6 (0.00%)  4/32 (12.50%) 
Pain in extremity  1  2/29 (6.90%)  1/6 (16.67%)  0/32 (0.00%) 
Pain in jaw  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Rotator cuff syndrome  1  0/29 (0.00%)  0/6 (0.00%)  2/32 (6.25%) 
Nervous system disorders       
Dizziness  1  0/29 (0.00%)  1/6 (16.67%)  1/32 (3.13%) 
Head discomfort  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Headache  1  3/29 (10.34%)  1/6 (16.67%)  4/32 (12.50%) 
Muscle spasticity  1  2/29 (6.90%)  1/6 (16.67%)  4/32 (12.50%) 
Neuralgia  1  7/29 (24.14%)  2/6 (33.33%)  10/32 (31.25%) 
Nystagmus  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Syncope  1  1/29 (3.45%)  1/6 (16.67%)  0/32 (0.00%) 
Unresponsive to stimuli  1  2/29 (6.90%)  0/6 (0.00%)  0/32 (0.00%) 
Vocal cord paralysis  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Psychiatric disorders       
Adjustment disorder  1  1/29 (3.45%)  1/6 (16.67%)  1/32 (3.13%) 
Agitation  1  1/29 (3.45%)  0/6 (0.00%)  2/32 (6.25%) 
Anxiety  1  6/29 (20.69%)  1/6 (16.67%)  9/32 (28.13%) 
Delirium  1  3/29 (10.34%)  0/6 (0.00%)  2/32 (6.25%) 
Depression  1  7/29 (24.14%)  1/6 (16.67%)  6/32 (18.75%) 
Hallucination  1  2/29 (6.90%)  0/6 (0.00%)  1/32 (3.13%) 
Insomnia  1  11/29 (37.93%)  2/6 (33.33%)  9/32 (28.13%) 
Suicidal ideation  1  0/29 (0.00%)  0/6 (0.00%)  2/32 (6.25%) 
Renal and urinary disorders       
Acute kidney injury  1  1/29 (3.45%)  1/6 (16.67%)  1/32 (3.13%) 
Neurogenic bladder  1  5/29 (17.24%)  0/6 (0.00%)  8/32 (25.00%) 
Urinary retention  1  3/29 (10.34%)  1/6 (16.67%)  1/32 (3.13%) 
Reproductive system and breast disorders       
Vaginal discharge  1  1/29 (3.45%)  1/6 (16.67%)  0/32 (0.00%) 
Respiratory, thoracic and mediastinal disorders       
Apnoea  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Bronchial secretion retention  1  1/29 (3.45%)  0/6 (0.00%)  2/32 (6.25%) 
Dyspnoea  1  3/29 (10.34%)  0/6 (0.00%)  2/32 (6.25%) 
Hypoxia  1  6/29 (20.69%)  0/6 (0.00%)  3/32 (9.38%) 
Nasal congestion  1  2/29 (6.90%)  0/6 (0.00%)  2/32 (6.25%) 
Oropharyngeal pain  1  1/29 (3.45%)  0/6 (0.00%)  2/32 (6.25%) 
Pleural effusion  1  3/29 (10.34%)  1/6 (16.67%)  1/32 (3.13%) 
Pneumonia aspiration  1  0/29 (0.00%)  1/6 (16.67%)  2/32 (6.25%) 
Pneumothorax spontaneous  1  2/29 (6.90%)  1/6 (16.67%)  0/32 (0.00%) 
Productive cough  1  2/29 (6.90%)  0/6 (0.00%)  1/32 (3.13%) 
Pulmonary embolism  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Pulmonary haemorrhage  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Wheezing  1  1/29 (3.45%)  1/6 (16.67%)  1/32 (3.13%) 
Skin and subcutaneous tissue disorders       
Decubitus ulcer  1  11/29 (37.93%)  3/6 (50.00%)  15/32 (46.88%) 
Dry skin  1  2/29 (6.90%)  0/6 (0.00%)  0/32 (0.00%) 
Hyperhidrosis  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Pruritus  1  3/29 (10.34%)  3/6 (50.00%)  1/32 (3.13%) 
Rash  1  4/29 (13.79%)  0/6 (0.00%)  3/32 (9.38%) 
Vascular disorders       
Deep vein thrombosis  1  5/29 (17.24%)  0/6 (0.00%)  1/32 (3.13%) 
Haematoma  1  0/29 (0.00%)  1/6 (16.67%)  0/32 (0.00%) 
Hypotension  1  10/29 (34.48%)  0/6 (0.00%)  11/32 (34.38%) 
Orthostatic hypotension  1  2/29 (6.90%)  3/6 (50.00%)  5/32 (15.63%) 
Thrombophlebitis superficial  1  0/29 (0.00%)  1/6 (16.67%)  2/32 (6.25%) 
1
Term from vocabulary, MedDRA 21.1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Monitor
Organization: Vertex Pharmaceuticals Incorporated
Phone: 617-341-6777
EMail: medicalinfo@vrtx.com
Layout table for additonal information
Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02669849    
Other Study ID Numbers: VX15-210-101
First Submitted: January 21, 2016
First Posted: February 1, 2016
Results First Submitted: November 29, 2019
Results First Posted: January 22, 2020
Last Update Posted: January 22, 2020