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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03432520
Registration number
NCT03432520
Ethics application status
Date submitted
24/01/2018
Date registered
14/02/2018
Date last updated
29/07/2024
Titles & IDs
Public title
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
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Scientific title
A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia A
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Secondary ID [1]
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SPK-8011/8016-LTFU
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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:
Hemophilia A
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Condition category
Condition code
Blood
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Clotting 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
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
Treatment: Other - SPK-8011
Treatment: Other: SPK-8011
Observational long-term safety follow-up study of subjects previously treated with SPK-8011 in any Spark-sponsored SPK-8011 study
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Intervention code [1]
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Treatment: Other
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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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Incidence of FVIII Inhibitors
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Assessment method [1]
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Incidence of FVIII Inhibitors
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Timepoint [1]
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4 years
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Primary outcome [2]
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Incidence of all adverse events
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Assessment method [2]
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Incidence of all adverse events
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Timepoint [2]
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4 years
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Primary outcome [3]
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Incidence(s) of new or exacerbation of adverse events of interest
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Assessment method [3]
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Such as: malignancies, neurologic, rheumatologic or other autoimmune, or hematologic disorders
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Timepoint [3]
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4 years
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Primary outcome [4]
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Annual bleeding rate
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Assessment method [4]
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Annual bleeding rate
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Timepoint [4]
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4 years
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Primary outcome [5]
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FVIII activity levels
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Assessment method [5]
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FVIII activity levels
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Timepoint [5]
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4 years
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Primary outcome [6]
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Total FVIII consumption
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Assessment method [6]
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Total FVIII consumption
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Timepoint [6]
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4 years
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Primary outcome [7]
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Number of FVIII infusions
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Assessment method [7]
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Number of FVIII infusions
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Timepoint [7]
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4 years
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Secondary outcome [1]
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Joint Health Assessment
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Assessment method [1]
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Assessment of joint health for six index joints using a modified Hemophilia Joint Health Score (HJHS). The HJHS is a physical examination assessment tool, that measures joint health in the domain of body structure and function (i.e. impairment), of the joints most commonly affected by bleeding in hemophilia: the knees, ankles, and elbows
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Timepoint [1]
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4 years
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Secondary outcome [2]
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Target Joint Assessment
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Assessment method [2]
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Investigator assessment of bleeding in target joints (e.g., hip, elbow, wrist, shoulder, knee, and ankle)
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Timepoint [2]
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4 years
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Secondary outcome [3]
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Haem-A-QoL
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Assessment method [3]
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Quality-of-life (QoL) assessment
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Timepoint [3]
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4 years
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Secondary outcome [4]
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EQ-5D-5L
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Assessment method [4]
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Quality-of-life (QoL) assessment
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Timepoint [4]
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4 years
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Secondary outcome [5]
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Activities Assessments
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Assessment method [5]
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Hemophilia Activities List
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Timepoint [5]
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4 years
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Secondary outcome [6]
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Health Economics Assessment
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Assessment method [6]
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Collection of information including number of hospitalizations, emergency room visits, physician visits and work/school days missed
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Timepoint [6]
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4 years
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Eligibility
Key inclusion criteria
Inclusion Criteria
Participants are eligible to be included in the study only if all of the following criteria apply:
1. Have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored gene therapy study; and
2. Understand the purpose and risks of the study and provide signed and dated informed consent before undergoing any study-specific procedures.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
Exclusion Criteria
Participants are excluded from the study if any of the following criteria apply:
1.Unable or unwilling to comply with the schedule of visits and study assessments as described in this study protocol.
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Study design
Purpose
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Duration
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Selection
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Timing
Prospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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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
14/08/2018
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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
1/12/2032
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Actual
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Sample size
Target
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Accrual to date
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Final
29
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Royal Prince Alfred Hospital Department of Cell & Molecular Therapies - Sydney
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Recruitment hospital [2]
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The Alfred Hospital & Monash Medical Centre - Melbourne
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Recruitment postcode(s) [1]
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2050 - Sydney
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Recruitment postcode(s) [2]
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- Melbourne
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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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Massachusetts
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Country [2]
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United States of America
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State/province [2]
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Mississippi
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Country [3]
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United States of America
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State/province [3]
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Missouri
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Country [4]
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United States of America
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State/province [4]
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Oregon
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Country [5]
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United States of America
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State/province [5]
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Pennsylvania
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Country [6]
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United States of America
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State/province [6]
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Virginia
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Country [7]
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Canada
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State/province [7]
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Ontario
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Spark Therapeutics, 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 long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 and SPK-8016 in males with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored SPK-8011 or SPK-8016 study.
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Trial website
https://clinicaltrials.gov/study/NCT03432520
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Trial related presentations / publications
George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med. 2021 Nov 18;385(21):1961-1973. doi: 10.1056/NEJMoa2104205.
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Public notes
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Contacts
Principal investigator
Name
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Clinical Trial Director
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Address
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Spark Therapeutics, 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
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT03432520
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