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Catalog
2023 ACMG Annual Clinical Genetics Meeting Digital ...
Objectives and Design of the Acorn Study: A Non-In ...
Objectives and Design of the Acorn Study: A Non-Interventional Study Evaluating Long-term Safety in Achondroplasia Patients Treated with Vosoritide
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Pdf Summary
The Acorn Study is a non-interventional study designed to evaluate the long-term safety of vosoritide in achondroplasia patients. The study will collect real-world data from patients across Europe and provide insights into the safety, effectiveness, and use of vosoritide in the context of other interventions. The study design includes two cohorts: Cohort 1 consists of incident users who have recently initiated treatment with vosoritide, and Cohort 2 consists of prevalent users who have previously been treated with vosoritide in clinical trials or expanded access programs. Data elements of interest include patient demographics, medical history, physical examinations, height and weight measurements, vital signs, safety events, and administration details of vosoritide and concomitant medications. The primary endpoint of the study is the exposure-adjusted incidence rate of new bone-related safety events during the course of the study. Data analysis will be conducted separately for the two cohorts and may be compared with external comparator populations. The study is registered on the EU post-authorization study register and recruitment is expected to start in February 2023. The results of the study will be published in peer-reviewed scientific journals and presented at conferences. The Acorn Study aims to provide valuable long-term data on the safety and effectiveness of vosoritide treatment in achondroplasia patients and contribute to the understanding of this rare genetic disorder.
Asset Subtitle
Submitter Only - Barbara Utterback, MS, MBA; Co-Author - Jeanne Pimenta, PhD; Co-Author - Shelda Cohen, PhD; Co-Author - Swati Mukherjee; Co-Author - Fiona Fettes, PhD; Co-Author - Kala Jayaram, MD; Presenting Author - Ekkehart Lausch, MD;
Meta Tag
Bone/Joint Abnormalities
Musculoskeletal system
Therapy
Co-Author
Jeanne Pimenta, PhD
Co-Author
Shelda Cohen, PhD
Co-Author
Swati Mukherjee
Co-Author
Fiona Fettes, PhD
Co-Author
Kala Jayaram, MD
Presenting Author
Ekkehart Lausch, MD
Submitter Only
Barbara Utterback, MS, MBA
Keywords
Acorn Study
vosoritide
achondroplasia patients
long-term safety
real-world data
effectiveness
interventions
patient demographics
medical history
physical examinations
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