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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03124368
Registration number
NCT03124368
Ethics application status
Date submitted
12/04/2017
Date registered
21/04/2017
Date last updated
4/11/2021
Titles & IDs
Public title
A Proof-of-Mechanism Study to Determine the Effect of Danicopan on C3 Levels in Participants With C3G or IC-MPGN
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Scientific title
A Phase 2a Proof-of-Mechanism, Open-Label Study to Determine the Effect of ACH-0144471 on C3 Levels in Participants With Low C3 Levels Due to Either C3 Glomerulopathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
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Secondary ID [1]
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2016-003525-42
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Secondary ID [2]
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ACH471-201
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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:
C3 Glomerulonephritis
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Dense Deposit Disease
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C3 Glomerulopathy
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Immune Complex Mediated Membranoproliferative Glomerulonephritis
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Membranoproliferative Glomerulonephritis Types I, II, and III
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Condition category
Condition code
Renal and Urogenital
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Kidney disease
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Inflammatory and Immune System
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Autoimmune diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Danicopan
Experimental: Group 1: Danicopan 100 mg TID (Sentinel) - All participants received 100 milligrams (mg) of danicopan three times per day (TID) during the Treatment Period.
Experimental: Group 2: Danicopan up to 200 mg TID - All participants received not more than 200 mg of danicopan TID depending on the available safety, pharmacokinetic, and pharmacodynamic data from Group 1 (Sentinel) during the Treatment Period.
Treatment: Drugs: Danicopan
Participants received study drug for 14 days (Treatment Period), followed by a taper over the next 7 days (Taper Period).
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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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Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15
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Assessment method [1]
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Serum C3 levels were measured by conventional Roche immunoturbidimetric assay method.
Change from Baseline = Serum C3 levels on Day 15 - Baseline Serum C3 levels
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Timepoint [1]
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Baseline, Day 15
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Primary outcome [2]
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Change From Baseline In Plasma Intact C3 Level On Day 15
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Assessment method [2]
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Plasma Intact C3 level were measured by a novel multiplex assay method. Change from Baseline = Plasma Intact C3 levels on Day 15 - Baseline Plasma Intact C3 levels
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Timepoint [2]
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Baseline, Day 15
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Secondary outcome [1]
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Change From Baseline In Total Complement Classical Pathway (CP) Activity On Day 14
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Assessment method [1]
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CP activity was measured in serum by the DiaSorin Complement Activation Enzyme (CAE) functional immunoassay method, which measures terminal complement complex formation following activation. Results are expressed in CAE units which are calculated relative to previously established CAE activity of a positive control serum.
Change from Baseline = Total Complement CP Activity on Day 14 - Baseline Total Complement CP Activity
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Timepoint [1]
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Baseline, Day 14
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Secondary outcome [2]
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Change From Baseline In Total Complement Alternative Pathway (AP) Functional Activity (AP Wieslab) On Day 15
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Assessment method [2]
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AP functional activity was measured in serum by the Wieslab functional immunoassay method, which measures terminal complement complex (TCC) formation following AP-specific activation. Results are expressed as percent TCC production relative to a positive control serum.
Change from Baseline = Total Complement AP Functional Activity on Day 15 - Baseline Total Complement AP Functional Activity
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Timepoint [2]
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Baseline, Day 15
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Secondary outcome [3]
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Time To Achieving Peak Serum C3 Levels
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Assessment method [3]
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Serial serum samples were collected on Days 1, 7, and 14.
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Timepoint [3]
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From The First Day Of Dosing through Day 14
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Secondary outcome [4]
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Number Of Participants With Serious Adverse Events (SAEs), Grade 3 And Grade 4 Treatment-emergent Adverse Events (TEAEs), And Adverse Events (AEs) Leading To Discontinuation
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Assessment method [4]
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An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of SAEs and other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
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Timepoint [4]
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Up to Day 49
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Secondary outcome [5]
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Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve From Time Of Administration To 8 Hours Postdose (AUC0-8)
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Assessment method [5]
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Serial blood samples were collected at 0, 1, 1.5, 2, 2.5, 3, 4, 6, and 8 hours post-dose on Days 1 and 7.
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Timepoint [5]
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Days 1 and 7
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Secondary outcome [6]
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PK: Maximum Plasma Concentration (Cmax)
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Assessment method [6]
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Serial blood samples were collected at 0, 1, 1.5, 2, 2.5, 3, 4, 6, and 8 hours post-dose on Days 1 and 7.
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Timepoint [6]
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Days 1 and 7
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Secondary outcome [7]
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PK: Time To Maximum Concentration (Tmax)
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Assessment method [7]
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Serial blood samples were collected at 0, 1, 1.5, 2, 2.5, 3, 4, 6, and 8 hours post-dose on Days 1 and 7.
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Timepoint [7]
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Days 1 and 7
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Secondary outcome [8]
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Change From Baseline In Bb Fragment Of Complement Factor B (Bb) At Day 15
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Assessment method [8]
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Plasma Bb was measured by enzyme-linked immunosorbent assay (ELISA).
Change from Baseline = Complement Bb on Day 15 - Baseline
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Timepoint [8]
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Baseline, Day 15
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Secondary outcome [9]
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Change From Baseline In Soluble Terminal Complement Complex (sC5b-9) At Day 15
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Assessment method [9]
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Plasma sC5b-9 was measured by ELISA.
Change from Baseline = sC5b-9 on Day 15 - Baseline sC5b-9
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Timepoint [9]
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Baseline, Day 15
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Eligibility
Key inclusion criteria
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- Must have had clinical diagnosis of C3G (C3 glomerulonephritis or dense deposit
disease, the 2 types of C3G) or idiopathic IC-MPGN by renal biopsy for at least 3
months prior to dosing, with the pathologic diagnosis verified by a review of the
renal biopsy by the study central pathologist
- C3 must have been <50% of the lower limit of normal
- C4 complement protein (C4) must have been >90% of the lower limit of normal
- Must have been willing to comply with study-specific vaccination requirements for
Haemophilus influenzae, Streptococcus pneumoniae, and Neisseria meningitidis strains
A, C, W, and Y
- Negative pregnancy test for females prior to dosing and throughout the study
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Minimum age
16
Years
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Maximum age
65
Years
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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
- History of a major organ transplant (for example, heart, lung, kidney, liver) or
hematopoietic stem cell/marrow transplant. Individuals receiving renal replacement
therapy were also excluded
- Evidence of monoclonal gammopathy of unclear significance, infections, malignancy,
autoimmune diseases, or other conditions to which C3G or IC-MPGN may have been
secondary
- Estimated glomerular filtration rate (using Modification of Diet in Renal Disease
equation) <45 milliliters/minute/1.73 square meters at the time of Screening or at any
time over the preceding 4 weeks
- Receipt of eculizumab at any dose or interval within the past 75 days prior to dosing
- Use of tacrolimus or cyclosporine within 2 weeks of the first dose of danicopan
- History of febrile illness, a body temperature >38°Celsius, or other evidence of a
clinically significant active infection, within 14 days prior to study drug
administration
- History of meningococcal infection, or a first-degree relative or household contact
with a history of meningococcal infection
- Females who were pregnant, nursing, or planning to become pregnant during the study or
within 90 days of study drug administration or participants with a female partner who
was pregnant, nursing, or planning to become pregnant during the study or within 90
days of study drug administration
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Study design
Purpose of the study
Basic Science
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Allocation to intervention
Non-randomised 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
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Other
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Other design features
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Phase
Phase 2
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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
9/08/2017
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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
9/01/2019
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Sample size
Target
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Accrual to date
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Final
6
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Clinical Trial Site - Melbourne
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Recruitment postcode(s) [1]
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- Melbourne
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Recruitment outside Australia
Country [1]
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Belgium
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State/province [1]
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Antwerpen
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Country [2]
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Netherlands
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State/province [2]
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Leiden
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Alexion Pharmaceuticals, Inc.
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Address
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Country
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Other collaborator category [1]
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Commercial sector/Industry
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Name [1]
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Achillion, a wholly owned subsidiary of Alexion
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Address [1]
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Country [1]
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Ethics approval
Ethics application status
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Summary
Brief summary
The primary objective of this study was to determine whether ACH-0144471 (also known as
danicopan and ALXN2040) increases blood C3 complement protein (C3) levels in participants
with low C3 levels due to either C3G or IC-MPGN.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03124368
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03124368
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