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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04573309
Registration number
NCT04573309
Ethics application status
Date submitted
28/09/2020
Date registered
5/10/2020
Titles & IDs
Public title
Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
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Scientific title
A Phase 2, Open-label Study to Assess Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
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Secondary ID [1]
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2020-001104-41
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Secondary ID [2]
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ALXN1840-WD-204
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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:
Wilson Disease
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Condition category
Condition code
Oral and Gastrointestinal
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Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
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Neurological
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Other neurological disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - ALXN1840
Experimental: ALXN1840 - Participants will be administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39
Treatment: Drugs: ALXN1840
Administered orally as tablets.
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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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Mean Daily Copper Balance: Day 1 Through Day 8
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Assessment method [1]
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Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
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Timepoint [1]
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Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)
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Primary outcome [2]
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Mean Daily Copper Balance: Day 31 Through Day 35
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Assessment method [2]
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Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
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Timepoint [2]
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Accumulation: Day 31 through Day 35 (ALXN1840 30 mg)
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Primary outcome [3]
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Mean Daily Copper Balance: Day 25 Through Day 28
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Assessment method [3]
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Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
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Timepoint [3]
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Accumulation: Day 25 through Day 28 (ALXN1840 15 mg)
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Primary outcome [4]
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Mean Daily Copper Balance: Day 36 Through Day 39
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Assessment method [4]
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Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
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Timepoint [4]
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Accumulation: Day 36 through Day 39 (ALXN1840 30 mg)
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Secondary outcome [1]
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Change From Baseline In Mean Daily Copper Balance
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Assessment method [1]
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Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
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Timepoint [1]
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Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
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Secondary outcome [2]
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Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC)
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Assessment method [2]
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Copper was assessed through measurement of copper intake (in food and drink), and copper output (in feces and urine) as well as plasma total and labile bound copper.
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Timepoint [2]
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Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg; Steady state: Day 25 through Day 28 for ALXN1840 15 mg and Day 36 through Day 39 for ALXN1840 30 mg
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Secondary outcome [3]
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Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State
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Assessment method [3]
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The amount of molybdenum in food, drink, feces and urine is reported in this outcome measure.
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Timepoint [3]
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Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
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Secondary outcome [4]
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Change From Baseline In Total Molybdenum Excretion In Urine And Feces
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Assessment method [4]
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Timepoint [4]
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Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
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Secondary outcome [5]
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Mean Daily Molybdenum Balance At ALXN1840 Steady State
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Assessment method [5]
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Molybdenum balance at steady state was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine). Steady state is defined as molybdenum (out) equal to molybdenum (in).
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Timepoint [5]
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Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
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Secondary outcome [6]
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Accumulation Of Molybdenum As Determined By Molybdenum Balance
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Assessment method [6]
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Molybdenum balance was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine).
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Timepoint [6]
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Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 ((ALXN1840 30 mg)
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Secondary outcome [7]
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Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
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Assessment method [7]
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Timepoint [7]
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Day 1 up to Day 39
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Secondary outcome [8]
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Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
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Assessment method [8]
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Timepoint [8]
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Day 39
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Eligibility
Key inclusion criteria
1. Diagnosis of WD by Leipzig Criteria = 4.
2. Able to reside in the clinical research unit for intensive metabolic monitoring of copper and molybdenum.
3. Participants willing to adhere to copper/molybdenum-controlled diet during the study.
4. Willing and able to follow protocol-specified contraception requirements.
5. Capable of giving signed informed consent.
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Minimum age
18
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
1. Decompensated cirrhosis or model for end stage liver disease score > 13.
2. Modified Nazer score > 7.
3. Clinically significant gastrointestinal bleed within past 3 months.
4. Alanine aminotransferase > 2 × upper limit of normal.
5. Hemoglobin less than lower limit of the reference range for age and sex.
6. Significant medical history (current or past).
7. Previous treatment with zinc within 30 days prior to the Screening Visit.
8. Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance < 30 milliliters/minute.
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Study design
Purpose of the study
Treatment
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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
Single group
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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
7/09/2020
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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
7/06/2022
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Sample size
Target
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Accrual to date
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Final
9
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Connecticut
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Country [2]
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New Zealand
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State/province [2]
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Grafton
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Country [3]
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United Kingdom
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State/province [3]
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London
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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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Ethics approval
Ethics application status
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Summary
Brief summary
This exploratory study will investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD).
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Trial website
https://clinicaltrials.gov/study/NCT04573309
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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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Eugene S. Swenson, MD, PhD
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Address
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Alexion Pharmaceuticals, Inc.
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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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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/09/NCT04573309/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/09/NCT04573309/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT04573309