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Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04474197
Recruitment Status : Completed
First Posted : July 16, 2020
Results First Posted : May 26, 2022
Last Update Posted : May 26, 2022
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 Alpha1-Antitrypsin Deficiency
Interventions Drug: VX-864
Drug: Placebo
Enrollment 44
Recruitment Details  
Pre-assignment Details This study was conducted in participants 18 through 80 of years of age, inclusive with the PiZZ genotype.
Arm/Group Title Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
Hide Arm/Group Description Participants received placebo matched to VX-864 in the treatment period for 28 days. Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days. Participants received VX-864 300 mg q12h in the treatment period for 28 days. Participants received VX-864 500 mg q12h in the treatment period for 28 days.
Period Title: Overall Study
Started 7 10 9 18
Completed 7 9 9 18
Not Completed 0 1 0 0
Reason Not Completed
Withdrawal of consent (not due to adverse event)             0             1             0             0
Arm/Group Title Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg Total
Hide Arm/Group Description Participants received placebo matched to VX-864 in the treatment period for 28 days. Participants received VX-864 100 mg q12h in the treatment period for 28 days. Participants received VX-864 300 mg q12h in the treatment period for 28 days. Participants received VX-864 500 mg q12h in the treatment period for 28 days. Total of all reporting groups
Overall Number of Baseline Participants 7 10 9 18 44
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 7 participants 10 participants 9 participants 18 participants 44 participants
63.4  (10.5) 55.1  (5.3) 53.2  (16.2) 57.4  (9.9) 57.0  (10.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 10 participants 9 participants 18 participants 44 participants
Female
5
  71.4%
6
  60.0%
6
  66.7%
14
  77.8%
31
  70.5%
Male
2
  28.6%
4
  40.0%
3
  33.3%
4
  22.2%
13
  29.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 10 participants 9 participants 18 participants 44 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
1
   5.6%
1
   2.3%
Not Hispanic or Latino
7
 100.0%
8
  80.0%
8
  88.9%
15
  83.3%
38
  86.4%
Unknown or Not Reported
0
   0.0%
2
  20.0%
1
  11.1%
2
  11.1%
5
  11.4%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 10 participants 9 participants 18 participants 44 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
7
 100.0%
10
 100.0%
9
 100.0%
18
 100.0%
44
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Plasma Functional Alpha-1 Antitrypsin (AAT) Levels  
Mean (Standard Deviation)
Unit of measure:  Micromole per liter
Number Analyzed 7 participants 10 participants 9 participants 18 participants 44 participants
4.7  (1.3) 4.0  (0.7) 3.8  (0.9) 4.1  (0.6) 4.1  (0.9)
1.Primary Outcome
Title Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
Hide Description [Not Specified]
Time Frame From Baseline at Day 28
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set (FAS) included all randomized participants who received at least 1 dose of study drug.
Arm/Group Title Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
Hide Arm/Group Description:
Participants received placebo matched to VX-864 in the treatment period for 28 days.
Participants received VX-864 100 mg q12h in the treatment period for 28 days.
Participants received VX-864 300 mg dose q12h in the treatment period for 28 days.
Participants received VX-864 500 mg dose q12h in the treatment period for 28 days.
Overall Number of Participants Analyzed 7 10 9 18
Least Squares Mean (Standard Error)
Unit of Measure: micromole per liter
-0.1  (0.3) 2.3  (0.3) 2.3  (0.2) 2.1  (0.2)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, VX-864 100 mg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
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 2.3
Confidence Interval (2-Sided) 95%
1.5 to 3.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, VX-864 300 mg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Mixed-effects Model for Repeated Measure
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value 2.3
Confidence Interval (2-Sided) 95%
1.6 to 3.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, VX-864 500 mg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Mixed-effects Model for Repeated Measure
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value 2.2
Confidence Interval (2-Sided) 95%
1.5 to 2.9
Estimation Comments [Not Specified]
2.Primary Outcome
Title Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Hide Description [Not Specified]
Time Frame Day 1 up to Week 8
Hide Outcome Measure Data
Hide Analysis Population Description
The safety set included all participants who received at least 1 dose of study drug.
Arm/Group Title Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
Hide Arm/Group Description:
Participants received placebo matched to VX-864 in the treatment period for 28 days.
Participants received VX-864 100 mg q12h in the treatment period for 28 days.
Participants received VX-864 300 mg q12h in the treatment period for 28 days.
Participants received VX-864 500 mg q12h in the treatment period for 28 days.
Overall Number of Participants Analyzed 7 10 9 18
Measure Type: Number
Unit of Measure: participants
Participants With AEs 4 7 7 17
Participants With SAEs 0 0 0 2
3.Secondary Outcome
Title Change in Plasma Antigenic AAT Levels
Hide Description [Not Specified]
Time Frame From Baseline at Day 28
Hide Outcome Measure Data
Hide Analysis Population Description
FAS.
Arm/Group Title Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
Hide Arm/Group Description:
Participants received placebo matched to VX-864 in the treatment period for 28 days.
Participants received VX-864 100 mg q12h in the treatment period for 28 days.
Participants received VX-864 300 mg q12h in the treatment period for 28 days.
Participants received VX-864 500 mg q12h in the treatment period for 28 days.
Overall Number of Participants Analyzed 7 10 9 18
Least Squares Mean (Standard Error)
Unit of Measure: micromole per liter
-0.1  (0.4) 3.4  (0.4) 2.9  (0.4) 2.6  (0.2)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, VX-864 100 mg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Mixed-effects Model for Repeated Measure
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value 3.5
Confidence Interval (2-Sided) 95%
2.4 to 4.6
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, VX-864 300 mg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Mixed-effects Model for Repeated Measure
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value 3.0
Confidence Interval (2-Sided) 95%
1.9 to 4.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, VX-864 500 mg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Mixed-effects Model for Repeated Measure
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value 2.7
Confidence Interval (2-Sided) 95%
1.8 to 3.7
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Observed Pre-dose Plasma Concentration (Ctrough) of VX-864
Hide Description [Not Specified]
Time Frame Pre-dose at Day 7, Day 14, Day 21 and Day 28
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic (PK) set included participants who received at least 1 dose of study drug. Here "Number analyzed" signifies those participants who were evaluable for this endpoint at specified time points.
Arm/Group Title VX-864 100 mg VX-864 300 mg VX-864 500 mg
Hide Arm/Group Description:
Participants received VX-864 100 mg q12h in the treatment period for 28 days.
Participants received VX-864 300 mg q12h in the treatment period for 28 days.
Participants received VX-864 500 mg q12h in the treatment period for 28 days.
Overall Number of Participants Analyzed 10 9 18
Mean (Standard Deviation)
Unit of Measure: microgram per milliliter
Day 7 Number Analyzed 9 participants 8 participants 16 participants
1.13  (0.743) 1.62  (0.665) 2.74  (2.89)
Day 14 Number Analyzed 8 participants 9 participants 18 participants
0.852  (0.340) 1.47  (0.847) 4.35  (5.36)
Day 21 Number Analyzed 8 participants 8 participants 18 participants
0.868  (0.503) 1.21  (0.770) 1.79  (0.922)
Day 28 Number Analyzed 9 participants 8 participants 18 participants
0.797  (0.327) 1.29  (0.646) 2.55  (2.40)
Time Frame Day 1 up to Week 8
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
Hide Arm/Group Description Participants received placebo matched to VX-864 in the treatment period for 28 days. Participants received VX-864 100 mg q12h in the treatment period for 28 days. Participants received VX-864 300 mg q12h in the treatment period for 28 days. Participants received VX-864 500 mg q12h in the treatment period for 28 days.
All-Cause Mortality
Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/7 (0.00%)   0/10 (0.00%)   0/9 (0.00%)   0/18 (0.00%) 
Hide Serious Adverse Events
Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/7 (0.00%)   0/10 (0.00%)   0/9 (0.00%)   2/18 (11.11%) 
General disorders         
Chest pain  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Respiratory, thoracic and mediastinal disorders         
Pneumothorax spontaneous  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
1
Term from vocabulary, Meddra 24.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo VX-864 100 mg VX-864 300 mg VX-864 500 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   4/7 (57.14%)   7/10 (70.00%)   7/9 (77.78%)   17/18 (94.44%) 
Blood and lymphatic system disorders         
Anaemia  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Eye disorders         
Eye allergy  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Macular degeneration  1  1/7 (14.29%)  0/10 (0.00%)  0/9 (0.00%)  0/18 (0.00%) 
Gastrointestinal disorders         
Abdominal discomfort  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  0/18 (0.00%) 
Abdominal pain  1  0/7 (0.00%)  1/10 (10.00%)  1/9 (11.11%)  1/18 (5.56%) 
Abdominal pain upper  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Constipation  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  2/18 (11.11%) 
Diarrhoea  1  0/7 (0.00%)  1/10 (10.00%)  1/9 (11.11%)  4/18 (22.22%) 
Flatulence  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Gingival pain  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Nausea  1  0/7 (0.00%)  0/10 (0.00%)  2/9 (22.22%)  4/18 (22.22%) 
Toothache  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  0/18 (0.00%) 
Vomiting  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
General disorders         
Asthenia  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Chills  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Fatigue  1  0/7 (0.00%)  2/10 (20.00%)  0/9 (0.00%)  3/18 (16.67%) 
Feeling abnormal  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  0/18 (0.00%) 
Feeling cold  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  1/18 (5.56%) 
Malaise  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Pyrexia  1  0/7 (0.00%)  2/10 (20.00%)  1/9 (11.11%)  0/18 (0.00%) 
Infections and infestations         
Infective exacerbation of bronchiectasis  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Skin infection  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Upper respiratory tract infection  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Urinary tract infection  1  1/7 (14.29%)  1/10 (10.00%)  0/9 (0.00%)  1/18 (5.56%) 
Injury, poisoning and procedural complications         
Back injury  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Muscle strain  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Post procedural contusion  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Post procedural haemorrhage  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Procedural pain  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Skin laceration  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Vaccination complication  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  1/18 (5.56%) 
Investigations         
Aspartate aminotransferase increased  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  0/18 (0.00%) 
Blood bicarbonate decreased  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Blood cholesterol increased  1  1/7 (14.29%)  0/10 (0.00%)  1/9 (11.11%)  1/18 (5.56%) 
Blood potassium decreased  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Blood pressure diastolic increased  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Blood triglycerides increased  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  1/18 (5.56%) 
Haemoglobin increased  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  1/18 (5.56%) 
Low density lipoprotein increased  1  1/7 (14.29%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Lymphocyte count decreased  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Oxygen saturation decreased  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  0/18 (0.00%) 
Protein total decreased  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Red blood cell count increased  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
White blood cells urine positive  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Musculoskeletal and connective tissue disorders         
Back pain  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Muscle spasms  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Myalgia  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Pain in extremity  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Nervous system disorders         
Carpal tunnel syndrome  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Dizziness  1  1/7 (14.29%)  1/10 (10.00%)  2/9 (22.22%)  1/18 (5.56%) 
Dysaesthesia  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Headache  1  0/7 (0.00%)  1/10 (10.00%)  2/9 (22.22%)  2/18 (11.11%) 
Tremor  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Psychiatric disorders         
Insomnia  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Poor quality sleep  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Reproductive system and breast disorders         
Heavy menstrual bleeding  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Vaginal haemorrhage  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  0/18 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Chronic obstructive pulmonary disease  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Cough  1  0/7 (0.00%)  2/10 (20.00%)  0/9 (0.00%)  0/18 (0.00%) 
Dyspnoea  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  0/18 (0.00%) 
Epistaxis  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Nasal congestion  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  0/18 (0.00%) 
Respiration abnormal  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  0/18 (0.00%) 
Sinus congestion  1  0/7 (0.00%)  0/10 (0.00%)  1/9 (11.11%)  0/18 (0.00%) 
Sneezing  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Sputum increased  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  0/18 (0.00%) 
Tachypnoea  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  0/18 (0.00%) 
Skin and subcutaneous tissue disorders         
Photosensitivity reaction  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Polymorphic light eruption  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  1/18 (5.56%) 
Rash  1  0/7 (0.00%)  1/10 (10.00%)  0/9 (0.00%)  0/18 (0.00%) 
Urticaria  1  0/7 (0.00%)  0/10 (0.00%)  0/9 (0.00%)  2/18 (11.11%) 
1
Term from vocabulary, MedDRA 24.0
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
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT04474197    
Other Study ID Numbers: VX19-864-101
2019-004881-16 ( EudraCT Number )
First Submitted: July 13, 2020
First Posted: July 16, 2020
Results First Submitted: May 3, 2022
Results First Posted: May 26, 2022
Last Update Posted: May 26, 2022