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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03447249
Registration number
NCT03447249
Ethics application status
Date submitted
21/02/2018
Date registered
27/02/2018
Date last updated
13/03/2020
Titles & IDs
Public title
A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
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Scientific title
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
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Secondary ID [1]
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2017-004132-11
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Secondary ID [2]
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VX17-659-102
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Cystic fibrosis
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Respiratory
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Other respiratory disorders / diseases
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Oral and Gastrointestinal
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Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
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Inflammatory and Immune System
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Connective tissue diseases
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Inflammatory and Immune System
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Other inflammatory or immune system disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - VX-659/TEZ/IVA
Treatment: Drugs - IVA
Treatment: Drugs - Placebo
Placebo comparator: Placebo - Participants who received placebo matched to VX-659/TEZ/IVA for 24 weeks in the TC treatment period.
Experimental: VX-659/TEZ/IVA TC - Participants who received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as fixed-dose combination (FDC) tablets in the morning and IVA 150 mg as mono tablet in the evening for 24 weeks in the TC treatment period.
Treatment: Drugs: VX-659/TEZ/IVA
Participants received VX-659/TEZ/IVA orally once daily in the morning.
Treatment: Drugs: IVA
Participants received IVA orally once daily in the evening.
Treatment: Drugs: Placebo
Participants received placebo matched VX-659/TEZ/IVA orally once daily in the morning and placebo matched to IVA orally once daily in the evening.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
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Assessment method [1]
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
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Timepoint [1]
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From Baseline at Week 4
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Secondary outcome [1]
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Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
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Assessment method [1]
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
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Timepoint [1]
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From Baseline through Week 24
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Secondary outcome [2]
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Number of Pulmonary Exacerbations (PEx)
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Assessment method [2]
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Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
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Timepoint [2]
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From Baseline through Week 24
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Secondary outcome [3]
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Absolute Change in Sweat Chloride (SwCl)
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Assessment method [3]
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Sweat samples were collected using an approved collection device.
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Timepoint [3]
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From Baseline through Week 24
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Secondary outcome [4]
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Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score
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Assessment method [4]
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The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
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Timepoint [4]
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From Baseline through Week 24
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Secondary outcome [5]
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Absolute Change in Body Mass Index (BMI)
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Assessment method [5]
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BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2).
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Timepoint [5]
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From Baseline at Week 24
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Secondary outcome [6]
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Absolute Change in Sweat Chloride
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Assessment method [6]
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Sweat samples were collected using an approved collection device.
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Timepoint [6]
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From Baseline at Week 4
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Secondary outcome [7]
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Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score
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Assessment method [7]
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The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
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Timepoint [7]
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From Baseline at Week 4
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Secondary outcome [8]
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Time-to-first Pulmonary Exacerbation (PEx)
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Assessment method [8]
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Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
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Timepoint [8]
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From Baseline through Week 24
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Secondary outcome [9]
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Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline
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Assessment method [9]
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BMI was defined as weight in kg divided by height in m\^2. z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI.
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Timepoint [9]
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From Baseline at Week 24
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Secondary outcome [10]
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Absolute Change in Body Weight
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Assessment method [10]
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Timepoint [10]
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From Baseline at Week 24
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Secondary outcome [11]
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Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
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Assessment method [11]
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Timepoint [11]
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From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to 28 weeks)
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Secondary outcome [12]
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Observed Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, and IVA
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Assessment method [12]
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Timepoint [12]
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Pre-dose on Week 4, 8, 12, and 16
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Eligibility
Key inclusion criteria
Key
* Heterozygous for F508del and an MF mutation (as defined in the protocol)
* Forced expiratory volume in 1 second (FEV1) value =40% and =90% of predicted mean for age, sex, and height
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Minimum age
12
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Clinically significant cirrhosis with or without portal hypertension
* Lung infection with organisms associated with a more rapid decline in pulmonary status
* Solid organ or hematological transplantation
Other protocol defined Inclusion/Exclusion criteria may apply
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
7/03/2018
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
5/02/2019
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Sample size
Target
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Accrual to date
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Final
385
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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The Alfred Hospital - Melbourne
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Royal Adelaide Hospital - Adelaide
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Prince Charles Hospital - Chermside
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Royal Brisbane & Women's Hospital - Herston
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Institute for Respiratory Health Inc./ Sir Charles Gairdner Hospital - Nedlands
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John Hunter Hospital & Hunter Medical Research Institute - New Lambton Heights
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Recruitment hospital [7]
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Sydney Children's Hospital, Randwick - Randwick
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Recruitment hospital [8]
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Princess Margaret Hospital for Children - Subiaco
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Recruitment postcode(s) [1]
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- Melbourne
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- Adelaide
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- Chermside
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- Herston
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- Nedlands
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- New Lambton Heights
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- Randwick
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Recruitment postcode(s) [8]
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- Subiaco
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Recruitment outside Australia
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Vertex Pharmaceuticals Incorporated
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).
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Trial website
https://clinicaltrials.gov/study/NCT03447249
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/49/NCT03447249/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/49/NCT03447249/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT03447249
Download to PDF