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SUN-093 Prospective Clinical Assessment Study in Children with Achondroplasia: The PROPEL Trial

BACKGROUND: Achondroplasia (ACH) is the most common non-lethal form of skeletal dysplasia, affecting between 1 in 15,000 to 1 in 30,000 live births [Horton et al. 2007; Waller et al. 2008]. Children and adults with ACH have disproportionate short stature, with a final height of approximately 131 cm...

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Autores principales: Savarirayan, Ravi, Kannu, Peter, Dambkowski, Carl L, Rogoff, Daniela, Irving, Melita
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7209755/
http://dx.doi.org/10.1210/jendso/bvaa046.557
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author Savarirayan, Ravi
Kannu, Peter
Dambkowski, Carl L
Rogoff, Daniela
Irving, Melita
author_facet Savarirayan, Ravi
Kannu, Peter
Dambkowski, Carl L
Rogoff, Daniela
Irving, Melita
author_sort Savarirayan, Ravi
collection PubMed
description BACKGROUND: Achondroplasia (ACH) is the most common non-lethal form of skeletal dysplasia, affecting between 1 in 15,000 to 1 in 30,000 live births [Horton et al. 2007; Waller et al. 2008]. Children and adults with ACH have disproportionate short stature, with a final height of approximately 131 cm for males and 124 cm for females. They are prone to significant co-morbidities, including obstructive sleep apnea, chronic otitis media with conductive hearing loss, spinal stenosis and a propensity towards obesity. In some infants, narrowing of the foramen magnum may result in compression of the spinal cord with neurologic sequelae, requiring timely neurosurgical intervention. There are currently no approved therapies for the treatment of ACH in either the United States or the European Union, and management is generally supportive in nature. The PROPEL trial is a prospective, non-interventional study examining baseline growth parameters and health status in children with ACH being assessed for potential enrollment into interventional studies with infigratinib, an oral FGFR1-3 inhibitor in development for achondroplasia. Methods: Children with ACH between the ages of 2.5 and 10 years are eligible for enrollment in this prospective, non-interventional trial to evaluate growth parameters and determine clinical status. Participants will be assessed at baseline, month 3, month 6, and every 6 months thereafter. The primary endpoint is annualized height growth velocity. Secondary endpoints include change from baseline in other growth parameters (including body proportionality); analysis of bone biomarkers (e.g., bone alkaline phosphatase, collagen X fragment); and the occurrence of medical events and surgical procedures. Participants will be enrolled in the study for a minimum of 6 months up to a maximum of 2 years before being considered for enrollment in a QED-sponsored phase 2/3 interventional trial. Current status: The PROPEL study is underway with the first patient enrolled in August 2019. Planned total enrollment is 200 children with ACH.
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spelling pubmed-72097552020-05-13 SUN-093 Prospective Clinical Assessment Study in Children with Achondroplasia: The PROPEL Trial Savarirayan, Ravi Kannu, Peter Dambkowski, Carl L Rogoff, Daniela Irving, Melita J Endocr Soc Pediatric Endocrinology BACKGROUND: Achondroplasia (ACH) is the most common non-lethal form of skeletal dysplasia, affecting between 1 in 15,000 to 1 in 30,000 live births [Horton et al. 2007; Waller et al. 2008]. Children and adults with ACH have disproportionate short stature, with a final height of approximately 131 cm for males and 124 cm for females. They are prone to significant co-morbidities, including obstructive sleep apnea, chronic otitis media with conductive hearing loss, spinal stenosis and a propensity towards obesity. In some infants, narrowing of the foramen magnum may result in compression of the spinal cord with neurologic sequelae, requiring timely neurosurgical intervention. There are currently no approved therapies for the treatment of ACH in either the United States or the European Union, and management is generally supportive in nature. The PROPEL trial is a prospective, non-interventional study examining baseline growth parameters and health status in children with ACH being assessed for potential enrollment into interventional studies with infigratinib, an oral FGFR1-3 inhibitor in development for achondroplasia. Methods: Children with ACH between the ages of 2.5 and 10 years are eligible for enrollment in this prospective, non-interventional trial to evaluate growth parameters and determine clinical status. Participants will be assessed at baseline, month 3, month 6, and every 6 months thereafter. The primary endpoint is annualized height growth velocity. Secondary endpoints include change from baseline in other growth parameters (including body proportionality); analysis of bone biomarkers (e.g., bone alkaline phosphatase, collagen X fragment); and the occurrence of medical events and surgical procedures. Participants will be enrolled in the study for a minimum of 6 months up to a maximum of 2 years before being considered for enrollment in a QED-sponsored phase 2/3 interventional trial. Current status: The PROPEL study is underway with the first patient enrolled in August 2019. Planned total enrollment is 200 children with ACH. Oxford University Press 2020-05-08 /pmc/articles/PMC7209755/ http://dx.doi.org/10.1210/jendso/bvaa046.557 Text en © Endocrine Society 2020. http://creativecommons.org/licenses/by-nc-nd/4.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs licence (http://creativecommons.org/licenses/by-nc-nd/4.0/), which permits non-commercial reproduction and distribution of the work, in any medium, provided the original work is not altered or transformed in any way, and that the work is properly cited. For commercial re-use, please contact journals.permissions@oup.com
spellingShingle Pediatric Endocrinology
Savarirayan, Ravi
Kannu, Peter
Dambkowski, Carl L
Rogoff, Daniela
Irving, Melita
SUN-093 Prospective Clinical Assessment Study in Children with Achondroplasia: The PROPEL Trial
title SUN-093 Prospective Clinical Assessment Study in Children with Achondroplasia: The PROPEL Trial
title_full SUN-093 Prospective Clinical Assessment Study in Children with Achondroplasia: The PROPEL Trial
title_fullStr SUN-093 Prospective Clinical Assessment Study in Children with Achondroplasia: The PROPEL Trial
title_full_unstemmed SUN-093 Prospective Clinical Assessment Study in Children with Achondroplasia: The PROPEL Trial
title_short SUN-093 Prospective Clinical Assessment Study in Children with Achondroplasia: The PROPEL Trial
title_sort sun-093 prospective clinical assessment study in children with achondroplasia: the propel trial
topic Pediatric Endocrinology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7209755/
http://dx.doi.org/10.1210/jendso/bvaa046.557
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