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2. Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies

Author

Savarirayan, Ravi, De Bergua, Josep Maria, Arundel, Paul, McDevitt, Helen, Cormier-Daire, Valerie, Saraff, Vrinda, Skae, Mars, Delgado, Borja, Leiva-Gea, Antonio, Santos-Simarro, Fernando, Salles, Jean Pierre, Nicolino, Marc, Rossi, Massimiliano, Kannu, Peter, Bober, Michael B, Phillips, John, Saal, Howard, Harmatz, Paul, Burren, Christine, Gotway, Garrett, Cho, Terry, Muslimova, Elena, Weng, Richard, Rogoff, Daniela, Hoover-Fong, Julie, and Irving, Melita

Subjects
Pediatric, Clinical Research, Clinical Trials and Supportive Activities, 6.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, achondroplasia, clinical trial, fibroblast growth factor receptor 3, infigratinib, Clinical Sciences
Abstract

BackgroundAchondroplasia is the most common short-limbed skeletal dysplasia resulting from gain-of-function pathogenic variants in fibroblast growth factor receptor 3 (FGFR3) gene, a negative regulator of endochondral bone formation. Most treatment options are symptomatic, targeting medical complications. Infigratinib is an orally bioavailable, FGFR1-3 selective tyrosine kinase inhibitor being investigated as a direct therapeutic strategy to counteract FGFR3 overactivity in achondroplasia.ObjectivesThe main objective of PROPEL is to collect baseline data of children with achondroplasia being considered for future enrollment in interventional studies sponsored by QED Therapeutics. The objectives of PROPEL 2 are to obtain preliminary evidence of safety and efficacy of oral infigratinib in children with achondroplasia, to identify the infigratinib dose to be explored in future studies, and to characterize the pharmacokinetic (PK) profile of infigratinib and major metabolites.DesignPROPEL (NCT04035811) is a prospective, noninterventional clinical study designed to characterize the natural history and collect baseline data of children with achondroplasia over 6-24 months. PROPEL 2 (NCT04265651), a prospective, phase II, open-label study of infigratinib in children with achondroplasia, consists of a dose-escalation, dose-finding, and dose-expansion phase to confirm the selected dose, and a PK substudy.Methods and analysisChildren aged 3-11 years with achondroplasia who completed ⩾6 months in PROPEL are eligible for PROPEL 2. Primary endpoints include treatment-emergent adverse events and change from baseline in annualized height velocity. Four cohorts at ascending dose levels are planned for dose escalation. The selected dose will be confirmed in the dose-expansion phase.EthicsPROPEL and PROPEL 2 are being conducted in accordance with the International Conference on Harmonization Good Clinical Practice guidelines, principles of the Declaration of Helsinki, and relevant human clinical research and data privacy regulations. Protocols have been approved by local health authorities, ethics committees, and institutions as applicable. Parents/legally authorized representatives are required to provide signed informed consent; signed informed assent by the child is also required, where applicable.DiscussionPROPEL and PROPEL 2 will provide preliminary evidence of the safety and efficacy of infigratinib as precision treatment of children with achondroplasia and will inform the design of future studies of FGFR-targeted agents in achondroplasia.RegistrationClinicalTrials.gov: NCT04035811; NCT04265651.

Published
2022

3. THU165 PROPEL, PROPEL 2 And PROPEL OLE Studies Of Infigratinib In Children With Achondroplasia: Design And Status Of 3 Ongoing Trials

Author

Savarirayan, Ravi, primary, De Bergua, Josep Maria, additional, Arundel, Paul, additional, McDevitt, Helen, additional, Cormier-Daire, Valerie, additional, Saraff, Vrinda, additional, Skae, Mars, additional, Delgado, Borja, additional, Leiva-Gea, Antonio, additional, Salcedo, Maria, additional, Salles, Jean-Pierre, additional, Nicolino, Marc P, additional, Rossi, Massimiliano, additional, Kannu, Peter, additional, Bober, Michael B, additional, Phillips, John, additional, Saal, Howard, additional, Harmatz, Paul, additional, Burren, Christine, additional, Candler, Toby, additional, Cho, Terry, additional, Muslimova, Elena, additional, Weng, Richard, additional, Rogoff, Daniela, additional, Hoover-Fong, Julie, additional, and Irving, Melita, additional

Published
2023
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4. OR27-03 Oral Infigratinib Treatment Is Well Tolerated And Significantly Increases Height Velocity In Children With Achondroplasia: Month 6 Results From The PROPEL 2 Dose-finding Study

Author

Savarirayan, Ravi, primary, De Bergua, Josep Maria, additional, Arundel, Paul, additional, Salles, Jean Pierre, additional, Saraff, Vrinda, additional, Delgado, Borja, additional, Leiva-Gea, Antonio, additional, McDevitt, Helen, additional, Nicolino, Marc P, additional, Rossi, Massimiliano, additional, Salcedo, Maria, additional, Cormier-Daire, Valerie, additional, Skae, Mars, additional, Kannu, Peter, additional, Bober, Michael B, additional, Phillips, John, additional, Saal, Howard, additional, Harmatz, Paul, additional, Burren, Christine Pamela, additional, Candler, Toby, additional, Cho, Terry, additional, Muslimova, Elena, additional, Weng, Richard, additional, Raj, Supriya, additional, Hoover-Fong, Julie, additional, Irving, Melita, additional, and Rogoff, Daniela, additional

Published
2023
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5. THU181 Evaluation Of Bone Mineral Density In A Cohort Of Children With ACH Participating In The PROPEL 2 Study Of Infigratinib

Author

Savarirayan, Ravi, primary, De Bergua, Josep Maria, additional, Arundel, Paul, additional, Salles, Jean Pierre, additional, Leiva-Gea, Antonio, additional, Irving, Melita, additional, Saraff, Vrinda, additional, McDevitt, Helen, additional, Salcedo, Maria, additional, Nicolino, Marc P, additional, Cormier-Daire, Valerie, additional, Kannu, Peter, additional, Skae, Mars, additional, Bober, Michael B, additional, Phillips III, John, additional, Candler, Toby, additional, Harmatz, Paul, additional, Saal, Howard, additional, Hoover-Fong, Julie, additional, Muslimova, Elena, additional, Cho, Terry, additional, Weng, Richard, additional, and Rogoff, Daniela, additional

Published
2023
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7. Early life determinants of infant bone health

Author

McDevitt, Helen

Subjects
618.92, RJ Pediatrics, RG Gynecology and obstetrics
Abstract

This thesis consists of a series of studies utilising quantitative ultrasound (Sunlight Omnisense 7000P) to assess bone health of infants. Preterm infants are at risk of osteopenia of prematurity (OP) which can result in fractures in the short term and may have an impact on growth in infancy and childhood. OP has a multifactorial aetiology including factors such as poor mineral intake and immobility. There is an increasing number of ex-preterm survivors therefore morbidity becomes more important. There is also increasing evidence from epidemiological studies that growth in infancy can have an effect on adult diseases such as osteoporosis. The first study was a cross-sectional study of bone quantitative ultrasound measurements in 110 term and preterm infants shortly after birth. Speed of sound (SOS) was measured at the tibial and the radius. This validated the technique showing reproducible measurements with low inter and intra-observer error, and also showed no benefit to measuring multiple sites. Preterm infants were found to have a significantly lower SOS than term infants. There was a positive correlation between tibial SOS and gestation, with birthweight being a less significant factor than gestation. The second study followed 18 preterm infants longitudinally from birth to hospital discharge or term corrected gestational age (CGA). SOS fell significantly with time in all infants. The most preterm infants had the greatest fall in SOS. SOS at the end of the study period was negatively associated with peak serum alkaline phosphatase and severity of illness score. SOS was significantly lower in the infants who required total parenteral nutrition for longer than 3 weeks. These results show that the neonatal course has a significant impact on SOS trajectory. When preterm infants were followed up in the out-patient clinic over the first two years of life the SOS measurements taken as the next part of this study showed a catch up phenomenon. In the majority of infants, but not all infants, SOS moved into the normal range by 6 months CGA. In the subgroup of infants followed longitudinally those with the lowest SOS at hospital discharge/term corrected age had the greatest increase in SOS over time. An interventional study of passive exercise was performed to explore its role in influencing the bone health of preterm infants. Thirty one infants born at less than 33 weeks gestation were randomised to receive range of motion flexion and extension exercises once daily for 5 days each week starting ‘early’(n=15) or ‘late’ (when on 100kcal/kg/day enteral feeds, n=16) and continuing until term corrected gestational age (CGA) or discharge from hospital. Tibial SOS declined significantly from birth to end of physical activity in both ‘early’ and ‘late’ groups, and this was similar to the decrease seen in a group of historical controls from the earlier longitudinal study. Weight gain and head growth did not show a significant difference between groups or between study infants and controls. No infant was reported to have sustained a fracture, and length of hospital stay was not significantly different between groups. There was no significant increase in sepsis rate, retinopathy of prematurity or chronic lung disease in study infants but numbers were small. On longer term follow-up the intervention was not associated with any adverse effects. To investigate the possibility that the maternal environment plays an important role in influencing infants’ bone health we also studied SOS changes in 188 pregnant women and their offspring. Most women had SOS in the normal range antenatally, and there was no significant change in SOS across pregnancy in the group as a whole. There was a significant negative correlation with SOS SDS and BMI in early pregnancy. Women who smoked cigarettes had lower SOS throughout pregnancy and so did their infants. Serum bone biochemistry was measured in the women antenatally and after delivery, and umbilical cord blood was also taken where possible. Vitamin D deficiency was found to be common at the end of pregnancy. Women of Asian origin had significantly lower vitamin D levels at all stages of pregnancy. There was no significant relationship between maternal and infant SOS, or between maternal vitamin D status and infant SOS. The work of this thesis establishes quantitative ultrasound as a useful technique in the assessment of infant bone health. It is a radiation free tool which provides precise and reproducible measurements in both term and preterm infants. In agreement with a small number of other studies we found that preterm infants have a lower speed of sound at birth compared to term infants; speed of sound increases with increasing gestation while in utero. By including infants who were both appropriately grown and small for gestational age we found maturity to be a more important factor in bone strength than birthweight. Despite the apparent self limiting nature of osteopenia of prematurity an intervention to improve neonatal bone health is still desirable, to prevent fractures. Our results do not substantiate conclusions from previous studies that physical activity alone can improve neonatal bone health. .Findings are however limited by the small sample size. Further studies are needed which investigate alternative exercise regimens, taking into account mineral and nutrient supply. Vitamin D deficiency, smoking and obesity may adversely affect bone health of women and their offspring. In the west of Scotland vitamin D deficiency is common in pregnancy: women of south asian origin are at particularly high risk, and should be supplemented with Vitamin D.

Published
2010

9. RF26 | PMON326 Medical History of Children Enrolled in PROPEL: A Prospective Clinical Assessment Study in Children with Achondroplasia

Author

Irving, Melita, primary, De Bergua, Josep Maria, additional, Rogoff, Daniela, additional, Savarirayan, Ravi, additional, Arundel, Paul, additional, Pierre Salles, Jean, additional, Leiva-Gea, Antonio, additional, Saraff, Vrinda, additional, McDevitt, Helen, additional, Santos-Simarro, Fernando, additional, Nicolino, Marc, additional, Cormier-Daire, Valerie, additional, Kannu, Peter, additional, Skae, Mars, additional, Bober, Michael B, additional, Phillips III, John, additional, Burren, Christine, additional, Harmatz, Paul, additional, Saal, Howard, additional, Hoover-Fong, Julie, additional, Weng, Richard, additional, Muslimova, Elena, additional, and Cho, Terry, additional

Published
2022
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10. PSAT105 Evaluation of Body Mass Index and Metabolic Parameters in Children with Achondroplasia Participating in the PROPEL Study

Author

Rogoff, Daniela, primary, Bergua, Josep Maria De, additional, Savarirayan, Ravi, additional, Arundel, Paul, additional, Salles, Jean Pierre, additional, Leiva-Gea, Antonio, additional, Irving, Melita, additional, Saraff, Vrinda, additional, McDevitt, Helen, additional, Santos-Simarro, Fernando, additional, Nicolino, Marc, additional, Cormier-Daire, Valerie, additional, Kannu, Peter, additional, Skae, Mars, additional, Bober, Michael B, additional, Phillips III, John, additional, Burren, Christine, additional, Harmatz, Paul, additional, Saal, Howard, additional, Hoover-Fong, Julie, additional, Muslimova, Elena, additional, Cho, Terry, additional, and Weng, Richard, additional

Published
2022
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11. PSAT106 Infigratinib in Children with Achondroplasia: Design of the PROPEL, PROPEL2 and PROPEL OLE Studies

Author

Muslimova, Elena, primary, De Bergua, Josep Maria, additional, Savarirayan, Ravi, additional, Arundel, Paul, additional, McDevitt, Helen, additional, Cormier-Daire, Valerie, additional, Saraff, Vrinda, additional, Skae, Mars, additional, Delgado, Borja, additional, Leiva-Gea, Antonio, additional, Santos-Simarro, Fernando, additional, Pierre Salles, Jean, additional, Nicolino, Marc, additional, Rossi, Massimiliano, additional, Kannu, Peter, additional, Bober, Michael B, additional, Phillips III, John, additional, Saal, Howard, additional, Harmatz, Paul, additional, Burren, Christine, additional, Gotway, Garrett, additional, Cho, Terry, additional, Weng, Richard, additional, Rogoff, Daniela, additional, Hoover-Fong, Julie, additional, and Irving, Melita, additional

Published
2022
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14. The European registry for rare bone and mineral conditions (EuRR-Bone): results of a survey on osteogenesis imperfecta and fibrous dysplasia McCune-Albright syndrome

Author

Zurita, Ana Luisa Priego, primary, Danila, Oana O., additional, Allgrove, Jeremy, additional, Brandi, Maria Luisa, additional, Bryce, Jillian, additional, Chapurlat, Roland, additional, Dzivite-Krisane, Iveta, additional, Grasemann, Corinna, additional, Högler, Wolfgang, additional, Javaid, M. Kassim, additional, McDevitt, Helen, additional, Montero-Lopez, Rodrigo, additional, Mortier, Geert, additional, Oheim, Ralf, additional, Riancho, Jose A., additional, de Sanctis, Luisa, additional, Verrijn Stuart, Annemarie A., additional, Tessaris, Daniele, additional, Ahmed, S. Faisal, additional, and Appelman-Dijkstra, Natasha, additional

Published
2022
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15. sj-docx-1-tab-10.1177_1759720X221084848 ��� Supplemental material for Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies

Author

Savarirayan, Ravi, De Bergua, Josep Maria, Arundel, Paul, McDevitt, Helen, Cormier-Daire, Valerie, Saraff, Vrinda, Skae, Mars, Delgado, Borja, Leiva-Gea, Antonio, Santos-Simarro, Fernando, Salles, Jean Pierre, Nicolino, Marc, Rossi, Massimiliano, Kannu, Peter, Bober, Michael B., Phillips, John, Saal, Howard, Harmatz, Paul, Burren, Christine, Gotway, Garrett, Cho, Terry, Muslimova, Elena, Weng, Richard, Rogoff, Daniela, Hoover-Fong, Julie, and Irving, Melita

Subjects
FOS: Clinical medicine, 110604 Sports Medicine, FOS: Health sciences, 111599 Pharmacology and Pharmaceutical Sciences not elsewhere classified, 110314 Orthopaedics
Abstract

Supplemental material, sj-docx-1-tab-10.1177_1759720X221084848 for Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies by Ravi Savarirayan, Josep Maria De Bergua, Paul Arundel, Helen McDevitt, Valerie Cormier-Daire, Vrinda Saraff, Mars Skae, Borja Delgado, Antonio Leiva-Gea, Fernando Santos-Simarro, Jean Pierre Salles, Marc Nicolino, Massimiliano Rossi, Peter Kannu, Michael B. Bober, John Phillips, Howard Saal, Paul Harmatz, Christine Burren, Garrett Gotway, Terry Cho, Elena Muslimova, Richard Weng, Daniela Rogoff, Julie Hoover-Fong and Melita Irving in Therapeutic Advances in Musculoskeletal Disease

Published
2022
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16. Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies

Author

Savarirayan, Ravi, primary, De Bergua, Josep Maria, additional, Arundel, Paul, additional, McDevitt, Helen, additional, Cormier-Daire, Valerie, additional, Saraff, Vrinda, additional, Skae, Mars, additional, Delgado, Borja, additional, Leiva-Gea, Antonio, additional, Santos-Simarro, Fernando, additional, Salles, Jean Pierre, additional, Nicolino, Marc, additional, Rossi, Massimiliano, additional, Kannu, Peter, additional, Bober, Michael B., additional, Phillips, John, additional, Saal, Howard, additional, Harmatz, Paul, additional, Burren, Christine, additional, Gotway, Garrett, additional, Cho, Terry, additional, Muslimova, Elena, additional, Weng, Richard, additional, Rogoff, Daniela, additional, Hoover-Fong, Julie, additional, and Irving, Melita, additional

Published
2022
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20. Nutritional rickets under 16 years: UK surveillance results

Author

Julies, Priscilla, primary, Lynn, Richard M, additional, Pall, Karina, additional, Leoni, Marina, additional, Calder, Alistair, additional, Mughal, Zulf, additional, Shaw, Nick, additional, McDonnell, Ciara, additional, McDevitt, Helen, additional, and Blair, Mitch, additional

Published
2020
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33. Does vitamin D supplementation prevent acute lower respiratory tract infections in children?

Author

Ali, Salma Rashid and McDevitt, Helen

Subjects
*RESPIRATORY infections, *PHYSIOLOGICAL effects of vitamin D, *AUSCULTATION, *RESPIRATORY disease diagnosis, *MEDICAL databases, *X-ray imaging
Abstract

The article presents a case study of a five-year-old boy with a history of cough, fever and increased breathing, who has vitamin D deficiency. Auscultation inferred right-sided basal crepitation, and X-ray examination of chest revealed a right lower lobe pneumonia. Biomedical databases EMBASE and Medline were searched, which identified 35 articles related to lower respiratory tract infection. Further studies inferred association between low serum vitamin D and respiratory tract disorder.

Published
2015
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36. Standardised practices in the networked management of congenital hyperinsulinism: a UK national collaborative consensus.

Author

Shaikh MG, Lucas-Herald AK, Dastamani A, Salomon Estebanez M, Senniappan S, Abid N, Ahmad S, Alexander S, Avatapalle B, Awan N, Blair H, Boyle R, Chesover A, Cochrane B, Craigie R, Cunjamalay A, Dearman S, De Coppi P, Erlandson-Parry K, Flanagan SE, Gilbert C, Gilligan N, Hall C, Houghton J, Kapoor R, McDevitt H, Mohamed Z, Morgan K, Nicholson J, Nikiforovski A, O'Shea E, Shah P, Wilson K, Worth C, Worthington S, and Banerjee I

Subjects
Child, Infant, Humans, Child, Preschool, Consensus, Pancreatectomy, United Kingdom, Congenital Hyperinsulinism diagnosis, Congenital Hyperinsulinism genetics, Congenital Hyperinsulinism therapy
Abstract

Congenital hyperinsulinism (CHI) is a condition characterised by severe and recurrent hypoglycaemia in infants and young children caused by inappropriate insulin over-secretion. CHI is of heterogeneous aetiology with a significant genetic component and is often unresponsive to standard medical therapy options. The treatment of CHI can be multifaceted and complex, requiring multidisciplinary input. It is important to manage hypoglycaemia in CHI promptly as the risk of long-term neurodisability arising from neuroglycopaenia is high. The UK CHI consensus on the practice and management of CHI was developed to optimise and harmonise clinical management of patients in centres specialising in CHI as well as in non-specialist centres engaged in collaborative, networked models of care. Using current best practice and a consensus approach, it provides guidance and practical advice in the domains of diagnosis, clinical assessment and treatment to mitigate hypoglycaemia risk and improve long term outcomes for health and well-being., Competing Interests: IB has received honoraria for advisory opinion from Merck and Diurnal Pharmaceuticals. He is the UK CI for clinical trials funded by Zealand Pharmaceuticals and has received grant funding from Merck, Diurnal and Crinetics Pharmaceuticals. IB is the Chair of the BSPED-NIHR Clinical Studies Group and the Chair of the ESPE Communications Committee. He is a co-opted member of the NICE Head Injury Update Committee. IB has advisory roles with several patient organisations including the Pituitary Foundation, Living with CAH, CHC and CHI. MGS has received funding/speaker honorium from Novo Nordisk, Sandoz, Pfizer and received honorarium for consultancies/advisory boards from Novo Nordisk, Pfizer and Merck. RK has received grant funding and honoraria for advisory opinion from Merck. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2023 Shaikh, Lucas-Herald, Dastamani, Salomon Estebanez, Senniappan, Abid, Ahmad, Alexander, Avatapalle, Awan, Blair, Boyle, Chesover, Cochrane, Craigie, Cunjamalay, Dearman, De Coppi, Erlandson-Parry, Flanagan, Gilbert, Gilligan, Hall, Houghton, Kapoor, McDevitt, Mohamed, Morgan, Nicholson, Nikiforovski, O'Shea, Shah, Wilson, Worth, Worthington and Banerjee.)

Published
2023
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