Once-Weekly somapacitan vs daily GH in children with GH deficiency: Results from a randomized phase 2 trial

Yuta Chiba; Yusuke Fujisawa; Yumiko Terada; Tomoko Yoshida; Kenichi Kinjo; Atsushi Tsukamura; Shinobu Ida; Yuri Etani; Yasuko Shoji; Masanobu Kawai; Hisakazu Nakajima; Jun Mori; Shota Fukuhara; Keiichi Shigehara; Hidechika Morimoto; Yusuke Tsuma; Yasuhiro Kawabe; Takeshi Ota; Kenichi Kashimada; Ryuichi Nakagawa; Atsumi Tsuji; Risa Nomura; Kei Takasawa; Takeru Yamauchi; Kanako Ishii; Naoko Toda; Kazuhiro Ohkubo; Tohru Yorifuji; Yuki Hosokawa; Rie Kawakita; Yukiko Hashimoto; Azumi Sakakibara; Shinji Higuchi; Shun Soneda; Kenichiro Ogushi; Shuichi Yatsuga; Yasutoshi Koga; Takako Matsumoto; Miyuki Kitamura; Ricard Nergardh; Mojca Zerjav Tansek; Serap Turan; Abdullah Bereket; Zeynep Atay; Azad Akbarzade; Olena Bolshova; Mykola Tronko; Olga Vyshnevskaya; Natalia Sprynchuk; Iryna Lukashuk; Natalia Muz; Tatyana Marchenko; Nataliya Chorna; Mariana Konovalova; Liliya Zelinska; Lawrence Silverman; Barbara Cerame; Sunita Cheruvu; Daisy Chin; Laurie Ebner-Lyon; Marie Fox; Marianna Nicolette-Gentile; Kristin Sabanosh; Harold Starkman; Ian Marshall; Mariam Gangat; Sadana Balachandar; Philippe Backeljauw; Andrew Dauber; Leah Tyzinski; Luis Zamora Siliezar; Jacqueline P. Velasco; Judith L. Ross; Martha Bardsley; Karen Kowal; Gad B. Kletter; Britney G. Frazier; Kathryn Garrison

doi:https://doi.org/10.1210/clinem/dgz310

Once-Weekly somapacitan vs daily GH in children with GH deficiency: Results from a randomized phase 2 trial

, , Yuta Chiba, Yusuke Fujisawa, Yumiko Terada, Tomoko Yoshida, Kenichi Kinjo, Atsushi Tsukamura, Shinobu Ida, Yuri Etani, Yasuko Shoji, Masanobu Kawai, Hisakazu Nakajima, Jun Mori, Shota Fukuhara, Keiichi Shigehara, Hidechika Morimoto, Yusuke Tsuma, Yasuhiro Kawabe, Takeshi OtaKenichi Kashimada, Ryuichi Nakagawa, Atsumi Tsuji, Risa Nomura, Kei Takasawa, Takeru Yamauchi, Kanako Ishii, Naoko Toda, Kazuhiro Ohkubo, Tohru Yorifuji, Yuki Hosokawa, Rie Kawakita, Yukiko Hashimoto, Azumi Sakakibara, Shinji Higuchi, Shun Soneda, Kenichiro Ogushi, Shuichi Yatsuga, Yasutoshi Koga, Takako Matsumoto, Miyuki Kitamura, Ricard Nergardh, Mojca Zerjav Tansek, Serap Turan, Abdullah Bereket, Zeynep Atay, Azad Akbarzade, Olena Bolshova, Mykola Tronko, Olga Vyshnevskaya, Natalia Sprynchuk, Iryna Lukashuk, Natalia Muz, Tatyana Marchenko, Nataliya Chorna, Mariana Konovalova, Liliya Zelinska, Lawrence Silverman, Barbara Cerame, Sunita Cheruvu, Daisy Chin, Laurie Ebner-Lyon, Marie Fox, Marianna Nicolette-Gentile, Kristin Sabanosh, Harold Starkman, Ian Marshall, Mariam Gangat, Sadana Balachandar, Philippe Backeljauw, Andrew Dauber, Leah Tyzinski, Luis Zamora Siliezar, Jacqueline P. Velasco, Judith L. Ross, Martha Bardsley, Karen Kowal, Gad B. Kletter, Britney G. Frazier, Kathryn Garrison

Ben-Gurion University of the Negev, Tel Aviv University, Technion - Israel Institute of Technology

Research output: Contribution to journal › Article › peer-review

Abstract

Context: Daily growth hormone (GH) injections can be burdensome for patients and carers. Somapacitan is a long-acting, reversible albumin-binding GH derivative in development for once-weekly administration in patients with growth hormone deficiency (GHD). Objective: The objective of this study is to evaluate the efficacy, safety, and tolerability of onceweekly somapacitan vs once-daily GH. Design: REAL 3 is a multicenter, randomized, controlled, double-blind (somapacitan doses), phase 2 study with a 26-week main and 26-week extension phase (NCT02616562). Setting: This study took place at 29 sites in 11 countries. Patients: Fifty-nine GH treatment-naive prepubertal children with GHD were randomly assigned; 58 completed the trial. Interventions: Interventions comprised 3 somapacitan doses (0.04 [n = 16], 0.08 [n = 15], or 0.16 mg/kg/wk [n = 14]) and daily GH (0.034 mg/kg/d [n = 14]), administered subcutaneously. Main Outcome Measures: The primary end point was height velocity (HV) at week 26. Secondary efficacy end points included HV SD score (SDS) and insulin-like growth factor-I (IGF-I) SDS. Results: At week 26, mean (SD) annualized HV for the somapacitan groups was 8.0 (2.0), 10.9 (1.9), and 12.9 (3.5) cm/year, respectively, vs 11.4 (3.3) cm/year for daily GH; estimated treatment difference (somapacitan 0.16 mg/kg/week-daily GH): 1.7 [95% CI -0.2 to 3.6] cm/year. HV was sustained at week 52, and significantly greater with somapacitan 0.16 mg/kg/week vs daily GH. Mean (SD) change from baseline in HV SDS at week 52 was 4.72 (2.79), 6.14 (3.36), and 8.60 (3.15) for the somapacitan groups, respectively, vs 7.41 (4.08) for daily GH. Model-derived mean (SD) IGF-I SDS for the somapacitan groups was -1.62 (0.86), -1.09 (0.78), and 0.31 (1.06), respectively, vs -0.40 (1.50) observed for daily GH. Safety and tolerability were consistent with the profile of daily GH. Conclusions: In children with GHD, once-weekly somapacitan 0.16 mg/kg/week provided the closest efficacy match with similar safety and tolerability to daily GH after 26 and 52 weeks of treatment. A short visual summary of our work is available (1).

Original language	American English
Pages (from-to)	E1847-E1861
Journal	Journal of Clinical Endocrinology and Metabolism
Volume	105
Issue number	4
DOIs	https://doi.org/10.1210/clinem/dgz310
State	Published - 1 Apr 2020

Keywords

Biomarkers/analysis
Child
Child, Preschool
Double-Blind Method
Drug Administration Schedule
Dwarfism, Pituitary/drug therapy
Female
Follow-Up Studies
Growth hormone
Growth hormone deficiency
Growth hormone replacement therapy
Human Growth Hormone/administration & dosage
Humans
Insulin-Like Growth Factor Binding Protein 3/analysis
Insulin-Like Growth Factor I/analysis
Long-acting growth hormone
Male
Prognosis
Somapacitan
Treatment burden

All Science Journal Classification (ASJC) codes

Endocrinology, Diabetes and Metabolism
Biochemistry
Endocrinology
Clinical Biochemistry
Biochemistry, medical

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

Access to Document

https://doi.org/10.1210/clinem/dgz310

Cite this

, Chiba, Y., Fujisawa, Y., Terada, Y., Yoshida, T., Kinjo, K., Tsukamura, A., Ida, S., Etani, Y., Shoji, Y., Kawai, M., Nakajima, H., Mori, J., Fukuhara, S., Shigehara, K., Morimoto, H., Tsuma, Y., Kawabe, Y., ... Garrison, K. (2020). Once-Weekly somapacitan vs daily GH in children with GH deficiency: Results from a randomized phase 2 trial. Journal of Clinical Endocrinology and Metabolism, 105(4), E1847-E1861. https://doi.org/10.1210/clinem/dgz310

@article{eec2dde152aa49c0aa092bc7cddcc68d,

title = "Once-Weekly somapacitan vs daily GH in children with GH deficiency: Results from a randomized phase 2 trial",

abstract = "Context: Daily growth hormone (GH) injections can be burdensome for patients and carers. Somapacitan is a long-acting, reversible albumin-binding GH derivative in development for once-weekly administration in patients with growth hormone deficiency (GHD). Objective: The objective of this study is to evaluate the efficacy, safety, and tolerability of onceweekly somapacitan vs once-daily GH. Design: REAL 3 is a multicenter, randomized, controlled, double-blind (somapacitan doses), phase 2 study with a 26-week main and 26-week extension phase (NCT02616562). Setting: This study took place at 29 sites in 11 countries. Patients: Fifty-nine GH treatment-naive prepubertal children with GHD were randomly assigned; 58 completed the trial. Interventions: Interventions comprised 3 somapacitan doses (0.04 [n = 16], 0.08 [n = 15], or 0.16 mg/kg/wk [n = 14]) and daily GH (0.034 mg/kg/d [n = 14]), administered subcutaneously. Main Outcome Measures: The primary end point was height velocity (HV) at week 26. Secondary efficacy end points included HV SD score (SDS) and insulin-like growth factor-I (IGF-I) SDS. Results: At week 26, mean (SD) annualized HV for the somapacitan groups was 8.0 (2.0), 10.9 (1.9), and 12.9 (3.5) cm/year, respectively, vs 11.4 (3.3) cm/year for daily GH; estimated treatment difference (somapacitan 0.16 mg/kg/week-daily GH): 1.7 [95% CI -0.2 to 3.6] cm/year. HV was sustained at week 52, and significantly greater with somapacitan 0.16 mg/kg/week vs daily GH. Mean (SD) change from baseline in HV SDS at week 52 was 4.72 (2.79), 6.14 (3.36), and 8.60 (3.15) for the somapacitan groups, respectively, vs 7.41 (4.08) for daily GH. Model-derived mean (SD) IGF-I SDS for the somapacitan groups was -1.62 (0.86), -1.09 (0.78), and 0.31 (1.06), respectively, vs -0.40 (1.50) observed for daily GH. Safety and tolerability were consistent with the profile of daily GH. Conclusions: In children with GHD, once-weekly somapacitan 0.16 mg/kg/week provided the closest efficacy match with similar safety and tolerability to daily GH after 26 and 52 weeks of treatment. A short visual summary of our work is available (1).",

keywords = "Biomarkers/analysis, Child, Child, Preschool, Double-Blind Method, Drug Administration Schedule, Dwarfism, Pituitary/drug therapy, Female, Follow-Up Studies, Growth hormone, Growth hormone deficiency, Growth hormone replacement therapy, Human Growth Hormone/administration & dosage, Humans, Insulin-Like Growth Factor Binding Protein 3/analysis, Insulin-Like Growth Factor I/analysis, Long-acting growth hormone, Male, Prognosis, Somapacitan, Treatment burden",

author = "Lars S{\"a}vendahl and Tadej Battelino and Meryl Brod and Rasmussen, {Michael H{\o}jby} and Reiko Horikawa and Juul, {Rasmus Vestergaard} and Paul Saenger and Dieter Furthner and Bettina Piringer and Lorenz Auer-Hackenberg and Klaus Schmitt and Marlene Reitmayr and Bronstein, {Marcello Delano} and Lima, {Francisco Samuel Magalhaes} and Martin Wabitsch and Carsten Posovszky and Volker Bottcher and Alexander Mann and Eli Hershkovitz and Alon Haim and Neta Lowenthal and Orit Hamiel and Levin, {Sharon Sheinvald} and Kineret Mazor-Aronovitch and Michal Ben-Ami and Shraga, {Yael Levy} and Dalit Modan and Noah Gruber and Moshe Phillip and Yael Lebenthal and Ariel Tenenbaum and Alon Eliakim and Nitzan Dror and Ruby Haviv and Nehama Zuckerman-Levin and Naim Shehadeh and Liav Givon and Ameer Elemy and Miriam Marji and Vardit Gepstein and Praveen, {V. P.} and P. Aswin and Nithiya Abraham and Rajesh Khadgawat and Yashdeep Gupta and Vaman Khadilkar and Anuradha Khadilkar and Sagar Lad and Yasuhiro Naiki and Yasuko Ogiwara and Yuta Chiba and Yusuke Fujisawa and Yumiko Terada and Tomoko Yoshida and Kenichi Kinjo and Atsushi Tsukamura and Shinobu Ida and Yuri Etani and Yasuko Shoji and Masanobu Kawai and Hisakazu Nakajima and Jun Mori and Shota Fukuhara and Keiichi Shigehara and Hidechika Morimoto and Yusuke Tsuma and Yasuhiro Kawabe and Takeshi Ota and Kenichi Kashimada and Ryuichi Nakagawa and Atsumi Tsuji and Risa Nomura and Kei Takasawa and Takeru Yamauchi and Kanako Ishii and Naoko Toda and Kazuhiro Ohkubo and Tohru Yorifuji and Yuki Hosokawa and Rie Kawakita and Yukiko Hashimoto and Azumi Sakakibara and Shinji Higuchi and Shun Soneda and Kenichiro Ogushi and Shuichi Yatsuga and Yasutoshi Koga and Takako Matsumoto and Miyuki Kitamura and Ricard Nergardh and Tansek, {Mojca Zerjav} and Serap Turan and Abdullah Bereket and Zeynep Atay and Azad Akbarzade and Olena Bolshova and Mykola Tronko and Olga Vyshnevskaya and Natalia Sprynchuk and Iryna Lukashuk and Natalia Muz and Tatyana Marchenko and Nataliya Chorna and Mariana Konovalova and Liliya Zelinska and Lawrence Silverman and Barbara Cerame and Sunita Cheruvu and Daisy Chin and Laurie Ebner-Lyon and Marie Fox and Marianna Nicolette-Gentile and Kristin Sabanosh and Harold Starkman and Ian Marshall and Mariam Gangat and Sadana Balachandar and Philippe Backeljauw and Andrew Dauber and Leah Tyzinski and Siliezar, {Luis Zamora} and Velasco, {Jacqueline P.} and Ross, {Judith L.} and Martha Bardsley and Karen Kowal and Kletter, {Gad B.} and Frazier, {Britney G.} and Kathryn Garrison",

note = "Publisher Copyright: {\textcopyright} Endocrine Society 2020.",

year = "2020",

month = apr,

day = "1",

doi = "https://doi.org/10.1210/clinem/dgz310",

language = "American English",

volume = "105",

pages = "E1847--E1861",

journal = "Journal of Clinical Endocrinology and Metabolism",

issn = "0021-972X",

publisher = "Endocrine Society",

number = "4",

}

Chiba, Y, Fujisawa, Y, Terada, Y, Yoshida, T, Kinjo, K, Tsukamura, A, Ida, S, Etani, Y, Shoji, Y, Kawai, M, Nakajima, H, Mori, J, Fukuhara, S, Shigehara, K, Morimoto, H, Tsuma, Y, Kawabe, Y, Ota, T, Kashimada, K, Nakagawa, R, Tsuji, A, Nomura, R, Takasawa, K, Yamauchi, T, Ishii, K, Toda, N, Ohkubo, K, Yorifuji, T, Hosokawa, Y, Kawakita, R, Hashimoto, Y, Sakakibara, A, Higuchi, S, Soneda, S, Ogushi, K, Yatsuga, S, Koga, Y, Matsumoto, T, Kitamura, M, Nergardh, R, Tansek, MZ, Turan, S, Bereket, A, Atay, Z, Akbarzade, A, Bolshova, O, Tronko, M, Vyshnevskaya, O, Sprynchuk, N, Lukashuk, I, Muz, N, Marchenko, T, Chorna, N, Konovalova, M, Zelinska, L, Silverman, L, Cerame, B, Cheruvu, S, Chin, D, Ebner-Lyon, L, Fox, M, Nicolette-Gentile, M, Sabanosh, K, Starkman, H, Marshall, I, Gangat, M, Balachandar, S, Backeljauw, P, Dauber, A, Tyzinski, L, Siliezar, LZ, Velasco, JP, Ross, JL, Bardsley, M, Kowal, K & Kletter, GB & Frazier, BG & Garrison, K 2020, 'Once-Weekly somapacitan vs daily GH in children with GH deficiency: Results from a randomized phase 2 trial', Journal of Clinical Endocrinology and Metabolism, vol. 105, no. 4, pp. E1847-E1861. https://doi.org/10.1210/clinem/dgz310

TY - JOUR

T1 - Once-Weekly somapacitan vs daily GH in children with GH deficiency

T2 - Results from a randomized phase 2 trial

AU - Sävendahl, Lars

AU - Battelino, Tadej

AU - Brod, Meryl

AU - Rasmussen, Michael Højby

AU - Horikawa, Reiko

AU - Juul, Rasmus Vestergaard

AU - Saenger, Paul

AU - Furthner, Dieter

AU - Piringer, Bettina

AU - Auer-Hackenberg, Lorenz

AU - Schmitt, Klaus

AU - Reitmayr, Marlene

AU - Bronstein, Marcello Delano

AU - Lima, Francisco Samuel Magalhaes

AU - Wabitsch, Martin

AU - Posovszky, Carsten

AU - Bottcher, Volker

AU - Mann, Alexander

AU - Hershkovitz, Eli

AU - Haim, Alon

AU - Lowenthal, Neta

AU - Hamiel, Orit

AU - Levin, Sharon Sheinvald

AU - Mazor-Aronovitch, Kineret

AU - Ben-Ami, Michal

AU - Shraga, Yael Levy

AU - Modan, Dalit

AU - Gruber, Noah

AU - Phillip, Moshe

AU - Lebenthal, Yael

AU - Tenenbaum, Ariel

AU - Eliakim, Alon

AU - Dror, Nitzan

AU - Haviv, Ruby

AU - Zuckerman-Levin, Nehama

AU - Shehadeh, Naim

AU - Givon, Liav

AU - Elemy, Ameer

AU - Marji, Miriam

AU - Gepstein, Vardit

AU - Praveen, V. P.

AU - Aswin, P.

AU - Abraham, Nithiya

AU - Khadgawat, Rajesh

AU - Gupta, Yashdeep

AU - Khadilkar, Vaman

AU - Khadilkar, Anuradha

AU - Lad, Sagar

AU - Naiki, Yasuhiro

AU - Ogiwara, Yasuko

AU - Chiba, Yuta

AU - Fujisawa, Yusuke

AU - Terada, Yumiko

AU - Yoshida, Tomoko

AU - Kinjo, Kenichi

AU - Tsukamura, Atsushi

AU - Ida, Shinobu

AU - Etani, Yuri

AU - Shoji, Yasuko

AU - Kawai, Masanobu

AU - Nakajima, Hisakazu

AU - Mori, Jun

AU - Fukuhara, Shota

AU - Shigehara, Keiichi

AU - Morimoto, Hidechika

AU - Tsuma, Yusuke

AU - Kawabe, Yasuhiro

AU - Ota, Takeshi

AU - Kashimada, Kenichi

AU - Nakagawa, Ryuichi

AU - Tsuji, Atsumi

AU - Nomura, Risa

AU - Takasawa, Kei

AU - Yamauchi, Takeru

AU - Ishii, Kanako

AU - Toda, Naoko

AU - Ohkubo, Kazuhiro

AU - Yorifuji, Tohru

AU - Hosokawa, Yuki

AU - Kawakita, Rie

AU - Hashimoto, Yukiko

AU - Sakakibara, Azumi

AU - Higuchi, Shinji

AU - Soneda, Shun

AU - Ogushi, Kenichiro

AU - Yatsuga, Shuichi

AU - Koga, Yasutoshi

AU - Matsumoto, Takako

AU - Kitamura, Miyuki

AU - Nergardh, Ricard

AU - Tansek, Mojca Zerjav

AU - Turan, Serap

AU - Bereket, Abdullah

AU - Atay, Zeynep

AU - Akbarzade, Azad

AU - Bolshova, Olena

AU - Tronko, Mykola

AU - Vyshnevskaya, Olga

AU - Sprynchuk, Natalia

AU - Lukashuk, Iryna

AU - Muz, Natalia

AU - Marchenko, Tatyana

AU - Chorna, Nataliya

AU - Konovalova, Mariana

AU - Zelinska, Liliya

AU - Silverman, Lawrence

AU - Cerame, Barbara

AU - Cheruvu, Sunita

AU - Chin, Daisy

AU - Ebner-Lyon, Laurie

AU - Fox, Marie

AU - Nicolette-Gentile, Marianna

AU - Sabanosh, Kristin

AU - Starkman, Harold

AU - Marshall, Ian

AU - Gangat, Mariam

AU - Balachandar, Sadana

AU - Backeljauw, Philippe

AU - Dauber, Andrew

AU - Tyzinski, Leah

AU - Siliezar, Luis Zamora

AU - Velasco, Jacqueline P.

AU - Ross, Judith L.

AU - Bardsley, Martha

AU - Kowal, Karen

AU - Kletter, Gad B.

AU - Frazier, Britney G.

AU - Garrison, Kathryn

PY - 2020/4/1

Y1 - 2020/4/1

N2 - Context: Daily growth hormone (GH) injections can be burdensome for patients and carers. Somapacitan is a long-acting, reversible albumin-binding GH derivative in development for once-weekly administration in patients with growth hormone deficiency (GHD). Objective: The objective of this study is to evaluate the efficacy, safety, and tolerability of onceweekly somapacitan vs once-daily GH. Design: REAL 3 is a multicenter, randomized, controlled, double-blind (somapacitan doses), phase 2 study with a 26-week main and 26-week extension phase (NCT02616562). Setting: This study took place at 29 sites in 11 countries. Patients: Fifty-nine GH treatment-naive prepubertal children with GHD were randomly assigned; 58 completed the trial. Interventions: Interventions comprised 3 somapacitan doses (0.04 [n = 16], 0.08 [n = 15], or 0.16 mg/kg/wk [n = 14]) and daily GH (0.034 mg/kg/d [n = 14]), administered subcutaneously. Main Outcome Measures: The primary end point was height velocity (HV) at week 26. Secondary efficacy end points included HV SD score (SDS) and insulin-like growth factor-I (IGF-I) SDS. Results: At week 26, mean (SD) annualized HV for the somapacitan groups was 8.0 (2.0), 10.9 (1.9), and 12.9 (3.5) cm/year, respectively, vs 11.4 (3.3) cm/year for daily GH; estimated treatment difference (somapacitan 0.16 mg/kg/week-daily GH): 1.7 [95% CI -0.2 to 3.6] cm/year. HV was sustained at week 52, and significantly greater with somapacitan 0.16 mg/kg/week vs daily GH. Mean (SD) change from baseline in HV SDS at week 52 was 4.72 (2.79), 6.14 (3.36), and 8.60 (3.15) for the somapacitan groups, respectively, vs 7.41 (4.08) for daily GH. Model-derived mean (SD) IGF-I SDS for the somapacitan groups was -1.62 (0.86), -1.09 (0.78), and 0.31 (1.06), respectively, vs -0.40 (1.50) observed for daily GH. Safety and tolerability were consistent with the profile of daily GH. Conclusions: In children with GHD, once-weekly somapacitan 0.16 mg/kg/week provided the closest efficacy match with similar safety and tolerability to daily GH after 26 and 52 weeks of treatment. A short visual summary of our work is available (1).

AB - Context: Daily growth hormone (GH) injections can be burdensome for patients and carers. Somapacitan is a long-acting, reversible albumin-binding GH derivative in development for once-weekly administration in patients with growth hormone deficiency (GHD). Objective: The objective of this study is to evaluate the efficacy, safety, and tolerability of onceweekly somapacitan vs once-daily GH. Design: REAL 3 is a multicenter, randomized, controlled, double-blind (somapacitan doses), phase 2 study with a 26-week main and 26-week extension phase (NCT02616562). Setting: This study took place at 29 sites in 11 countries. Patients: Fifty-nine GH treatment-naive prepubertal children with GHD were randomly assigned; 58 completed the trial. Interventions: Interventions comprised 3 somapacitan doses (0.04 [n = 16], 0.08 [n = 15], or 0.16 mg/kg/wk [n = 14]) and daily GH (0.034 mg/kg/d [n = 14]), administered subcutaneously. Main Outcome Measures: The primary end point was height velocity (HV) at week 26. Secondary efficacy end points included HV SD score (SDS) and insulin-like growth factor-I (IGF-I) SDS. Results: At week 26, mean (SD) annualized HV for the somapacitan groups was 8.0 (2.0), 10.9 (1.9), and 12.9 (3.5) cm/year, respectively, vs 11.4 (3.3) cm/year for daily GH; estimated treatment difference (somapacitan 0.16 mg/kg/week-daily GH): 1.7 [95% CI -0.2 to 3.6] cm/year. HV was sustained at week 52, and significantly greater with somapacitan 0.16 mg/kg/week vs daily GH. Mean (SD) change from baseline in HV SDS at week 52 was 4.72 (2.79), 6.14 (3.36), and 8.60 (3.15) for the somapacitan groups, respectively, vs 7.41 (4.08) for daily GH. Model-derived mean (SD) IGF-I SDS for the somapacitan groups was -1.62 (0.86), -1.09 (0.78), and 0.31 (1.06), respectively, vs -0.40 (1.50) observed for daily GH. Safety and tolerability were consistent with the profile of daily GH. Conclusions: In children with GHD, once-weekly somapacitan 0.16 mg/kg/week provided the closest efficacy match with similar safety and tolerability to daily GH after 26 and 52 weeks of treatment. A short visual summary of our work is available (1).

KW - Biomarkers/analysis

KW - Child

KW - Child, Preschool

KW - Double-Blind Method

KW - Drug Administration Schedule

KW - Dwarfism, Pituitary/drug therapy

KW - Female

KW - Follow-Up Studies

KW - Growth hormone

KW - Growth hormone deficiency

KW - Growth hormone replacement therapy

KW - Human Growth Hormone/administration & dosage

KW - Humans

KW - Insulin-Like Growth Factor Binding Protein 3/analysis

KW - Insulin-Like Growth Factor I/analysis

KW - Long-acting growth hormone

KW - Male

KW - Prognosis

KW - Somapacitan

KW - Treatment burden

UR - http://www.scopus.com/inward/record.url?scp=85081945845&partnerID=8YFLogxK

U2 - https://doi.org/10.1210/clinem/dgz310

DO - https://doi.org/10.1210/clinem/dgz310

M3 - Article

C2 - 31917835

SN - 0021-972X

VL - 105

SP - E1847-E1861

JO - Journal of Clinical Endocrinology and Metabolism

JF - Journal of Clinical Endocrinology and Metabolism

IS - 4

ER -

Once-Weekly somapacitan vs daily GH in children with GH deficiency: Results from a randomized phase 2 trial

Abstract

Keywords

All Science Journal Classification (ASJC) codes

UN SDGs

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