A Non-interventional, Observational, Registry-based Study to Investigate Long-term Safety and Clinical Parameters of Somapacitan Treatment in Paediatric Patients With Growth Hormone Deficiency During Routine Clinical Practice

The aim of this study is to look at the long-term safety and clinical parameters of somapacitan in paediatric participants with growth hormone deficiency under routine clinical practice conditions. The study population will include 400 paediatric growth hormone deficient participants from the Global Registry for Novel Therapies in Rare Bone and Endocrine Conditions (GloBE-Reg) treated with once-weekly somapacitan and fulfilling the eligibility criteria of the study. The total duration of the study is planned to 10 years consisting of a 5-year recruitment period in the GLoBE-Reg followed by a 5-year follow-up period.

Start Date01 Dec 2024

Sponsor / Collaborator

Novo Nordisk A/S

NCT06109935 / Enrolling by invitationNot Applicable

A Multi-centre, Prospective, Open Label, Single-arm, Non-interventional Post-marketing Study to Investigate the Long-term Safety and Clinical Parameters of Sogroya® Treatment in Children With Short Stature Due to Growth Hormone Deficiency Where Epiphysial Discs Are Not Closed Under Normal Clinical Practice Conditions in Japan Special Use-results Surveillance on Long Term Use of Sogroya® in Children With Short Stature Due to Growth Hormone Deficiency (GHD) Where Epiphysial Discs Are Not Closed

The purpose of the study is to investigate the safety and effectiveness of Sogroya® in children with short stature due to growth hormone deficiency where epiphysial discs are not closed under real-world clinical practice in Japan. The study will last for about 1 year (at shortest) to 3 years (at longest) depending on when the participant takes part in the study. The participant will be asked to answer questionnaire(s) about how they feel about the growth hormone (GH) product treatment once during the study (at about 3 months after starting the Sogroya® treatment) and about 3 months after starting the Sogroya® treatment.

Start Date04 Oct 2023

Sponsor / Collaborator

Novo Nordisk A/S

CTIS2022-501055-87-01 / Not yet recruiting

A study evaluating the safety and efficacy of once-weekly dosing of somapacitan in a basket study design in paediatric participants with short stature either born small of gestational age or with Turner syndrome, Noonan syndrome or idiopathic short stature - NN8640-4469

Start Date19 Jun 2023

Sponsor / Collaborator

Novo Nordisk A/S

100 Clinical Results associated with Somapacitan

100 Translational Medicine associated with Somapacitan

100 Patents (Medical) associated with Somapacitan

8,789

Literatures (Medical) associated with Somapacitan

01 Mar 2025·GROWTH HORMONE & IGF RESEARCH

Identification and differentiation of somapacitan, a long-acting growth hormone derivative, and recombinant human growth hormone in equine plasma by LC-HRMS for doping control purpose

Article

Author: Leung, Gary Ngai-Wa ; Shimizu, Yoshibumi ; Hirano-Kodaira, Misato ; Sugai-Bannai, Michiko ; Saito, Kazunobu

Somapacitan, a long-acting growth hormone derivative, and recombinant human growth hormone (rhGH) are protein-based drugs generally used to treat growth disorders and GH deficiency in humans. Due to their potential to enhance the horse performance, the use of these drugs is prohibited at-all-times by the International Federation of Horseracing Authorities for horseracing and the Fédération Equestre Internationale for equestrian sports. In this study, we developed a test method for the identification and differentiation of somapacitan and rhGH in equine plasma by using liquid chromatography high-resolution mass spectrometry (LC-HRMS). The method involved C4 solid-phase extraction after ammonium sulfate precipitation, followed by chloroform/methanol precipitation, trypsin digestion, and analysis by LC-HRMS. The discriminative identification of somapacitan and rhGH was successfully achieved through the detection of their respective unique T10 peptide fragments. Noteworthy, the T10 peptide fragment of somapacitan was detected with its side chain structure remaining intact. The limit of identification (confirmation) (LOI) was determined to be 5 ng/mL for somapacitan and 2 ng/mL for rhGH in equine plasma, while the limit of detection (LOD) was 5 ng/mL for somapacitan and 1 ng/mL for rhGH. Furthermore, using the peptide fragments T1, T8, and T9 (which are shared between somapacitan and rhGH), the presence of somapacitan in equine plasma could be confirmed with higher sensitivity, achieving down to LOIs of 2 ng/mL and LODs of 1 ng/mL. The method was validated with respect to specificity, identification capability, robustness, precision, and reproducibility, paving the way for the analysis of post-administration samples from our already planned administration trials and future potential positive cases.

01 Jan 2025·AMERICAN JOURNAL OF KIDNEY DISEASES

Patient, Parental, and Health Professional Perspectives on Growth in Children With CKD

Article

Author: Jaure, Allison ; Samuel, Susan ; Alexander, Stephen I ; Eddy, Allison A ; Hughes, Anastasia ; Yap, Hui-Kim ; Carter, Simon A ; Groothoff, Jaap ; Dart, Allison ; Torrisi, Luca G ; Kausman, Joshua ; Bockenhauer, Detlef ; Guha, Chandana ; Wu, Justin G ; Sinha, Aditi ; Walker, Amanda ; Craig, Jonathan C ; Furth, Susan L

RATIONALE & OBJECTIVE:

Growth failure is a common problem among children with chronic kidney disease (CKD). Reduced height is associated with psychosocial burden, social stigma, and impaired quality of life. This study describes the aspects of growth impairment that are most impactful from the perspectives of children with CKD, their parents, and health professionals.

STUDY DESIGN:

Qualitative study.

SETTINGS & PARTICIPANTS:

120 children with CKD (aged 8-21 years), 250 parents, and 445 health professionals from 53 countries who participated in 16 focus groups, 2 consensus workshops, and a Delphi survey.

ANALYTICAL APPROACH:

A thematic analysis of all qualitative data concerning growth from the Standardized Outcomes in Nephrology-Children and Adolescents (SONG-Kids) initiative.

RESULTS:

We identified 5 themes: diminishing psychological well-being (compared to and judged by peers, tired of explaining to others, damaging self-esteem), constrained life participation and enjoyment (deprived of normal school experiences, excluded from sports or competing at a disadvantage, impaired quality of life in adulthood); grappling with impacts of symptoms and treatment (difficulty understanding short stature and accessing help, lack of appetite, uncertainty regarding bone pains, medication side effects, burden of growth hormone treatment); facilitating timely interventions and optimizing outcomes (early indicator of disease, assessing management, maximizing transplant outcomes, minimizing morbidity); and keeping growth and health priorities in perspective (quality of life and survival of utmost priority, achieved adequate height).

LIMITATIONS:

Only English-speaking participants were included.

CONCLUSIONS:

Impaired growth may diminish psychological well-being, self-esteem, and participation in daily activities for children with CKD. Balancing different treatments that can affect growth complicates decision making. These findings may inform the psychosocial support needed by children with CKD and their caregivers to address concerns about growth.

PLAIN-LANGUAGE SUMMARY:

Children with chronic kidney disease (CKD) are often much shorter than their peers and may experience poorer mental health and quality of life. To understand the specific important issues on how growth impairment affects these children, we collected qualitative data from the Standardized Outcomes in Nephrology-Children and Adolescents (SONG-Kids) initiative and analyzed perspectives on growth from patients, parents, and health professionals. These data revealed impaired psychological health, reduced enjoyment during school and sports, difficulty dealing with medication side effects and growth hormone treatment, and concerns related to tracking health status and kidney transplant outcomes. These findings may inform the psychosocial support needed by children with CKD and their caregivers to address concerns about growth and overall health.

31 Dec 2024·GYNECOLOGICAL ENDOCRINOLOGY

Effects of growth hormone therapy on the onset and progression of pubertal development in girls with idiopathic short stature

Review

Author: Zhu, Feng ; Zhu, Jieqian ; Zhang, Yu ; Huang, Lina ; Xu, Qi ; Huang, Lingxiao

OBJECTIVE:

The aim of this study was to explore the impact of growth hormone (GH) therapy on the onset and progression of puberty in girls with idiopathic short stature.

METHODS:

This study included 541 girls aged between 4.5 and 10.6 years who were receiving GH treatment, monitored over a 22-year follow-up period. Of these, 126 girls have been followed up to the onset of menarche. The participants were divided into two groups: a ISS control group (n = 66) and a group receiving daily GH treatment at a dose of 0.15 iu/kg (n = 60). We assessed the pubertal development and GH usage of these girls every three months.

RESULTS:

(1) There was no significant difference in the onset of puberty between the growth hormone (GH) treatment group and the control group; however, the average duration of puberty was longer in the treatment group compared to the control group. (2) During puberty, there were no significant differences in height growth between the treated and untreated groups. (3) The duration of GH treatment showed a significant negative correlation with the age at onset of gonadal development and the age at menarche in females within the treatment group.

CONCLUSION:

GH treatment does not seem to accelerate the onset of puberty but may extend its duration, without significantly impacting height growth during puberty. Additionally, longer GH treatment duration is linked to earlier gonadal development and menarche in females.

News (Medical) associated with Somapacitan

05 Sep 2024

·www.echemi.com

Safety of Novo Nordisk's Growth Hormone Injection Under Scrutiny: Weekly Dosing Raises New Concerns

According to the news published on the official website of China's National Medical Products Administration (CDE) on September 5, Novo Nordisk's market access application for Parsi growth hormone injection (Sogroya) has been officially accepted. Pasil auxin injection contains the active ingredient somapacitan, which is a long-acting variant of human growth hormone (hGH). Somapacitan modifies natural hGH and enhances its affinity with plasma albumin, thus adapting to a weekly dosing regimen. Somapacitan was originally approved by the U.S. Food and Drug Administration (FDA) on September 1, 2020 to treat growth hormone deficiency in adults. Through April 2023, the FDA further expanded the indication for somapacitan to include the treatment of growth hormone deficiency in children 2.5 years of age and older. According to the Pharmaceutical Rubik's Cube database, Novo Nordisk initiated a Phase III clinical study (CTR20211271) in China in June 2021 for pediatric patients with slow growth due to insufficient growth hormone secretion, which was completed in January this year. The primary objective of the study was to evaluate the efficacy and safety of once-weekly administration of somapacitan versus once-daily administration of Norditropin in children with growth hormone deficiency in China. There were also positive results from the previous REAL4 study, a multicenter, randomized, open-label Phase III clinical study to evaluate the efficacy and safety of somapacitan in the treatment of growth hormone deficiency in preadolescent children. In the REAL4 study, patients were randomized in a 2:1 ratio to receive a weekly subcutaneous injection of 0.16 mg/kg somapacitan (n=132) or a daily subcutaneous injection of 0.034 mg/kg norditropin (n=68). The results showed that patients receiving weekly somapacitan had an annualized growth rate of 11.2cm/ year, which was not significantly different from 11.7cm/ year in patients receiving once-daily Norditropin, meeting the primary study endpoint of non-inferior efficacy. In addition, somapacitan has been shown to be well tolerated, with similar safety and tolerability profiles to Norditropin.

Phase 3Clinical ResultDrug ApprovalNDA

01 Jul 2024

·www.globenewswire.com

Soleno Therapeutics Announces Submission of New Drug Application to the U.S. FDA for DCCR (Diazoxide Choline) Extended-Release Tablets for the Treatment of Prader-Willi Syndrome

June 28, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS) in individuals four years and older who have hyperphagia. “Submission of the DCCR NDA to the FDA marks a significant milestone not only for Soleno, but for people living with PWS,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “This regulatory submission advances us one important step closer to our goal of bringing to market a new therapeutic for individuals with PWS that addresses the life-threatening hyperphagia and other key aspects of this serious and rare condition. We look forward to working with the FDA throughout the review process. We extend sincere gratitude to the team at Soleno, investigators, study site teams, advocacy organizations and most importantly, the individuals with PWS and their families who were instrumental in completing our DCCR development program.” DCCR has Breakthrough and Fast Track Designations in the U.S., as well as Orphan Drug Designation for PWS in the U.S. and E.U. The FDA has 60 days to determine whether the NDA is accepted for review. Soleno has requested Priority Review of the NDA, which would provide a target review period of six months by the FDA after the NDA has been accepted. The Prader-Willi Syndrome Association USA estimates that PWS occurs in one in every 15,000 live births. The hallmark symptom of this disorder is hyperphagia, a chronic and life-threatening feeling of intense, persistent hunger, food pre-occupation, extreme drive to food seek and consume food that severely diminish the quality of life for individuals with PWS and their families. Additional characteristics of PWS include behavioral problems, cognitive disabilities, low muscle tone, short stature (when not treated with growth hormone), the accumulation of excess body fat, developmental delays, and incomplete sexual development. Hyperphagia can lead to significant mortality (e.g., stomach rupture, choking, accidental death due to food seeking behavior) and longer term, co-morbidities such as diabetes, obesity, and cardiovascular disease. In a global survey conducted by the Foundation for Prader-Willi Research, 96.5% of respondents (parents and caregivers) rated hyperphagia and 92.9% rated body composition as either the most important or a very important symptom to be relieved by a new medicine. There are currently no approved therapies to treat the hyperphagia/appetite, metabolic, cognitive function, or behavioral aspects of the disorder. DCCR is a novel, proprietary extended-release dosage form containing diazoxide choline, the crystalline salt of diazoxide and is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of individuals in a few rare diseases in neonates, infants, children and adults, but is not approved for use in PWS. Soleno conceived of and established extensive patent protection for the therapeutic use of diazoxide, diazoxide choline and DCCR in individuals with PWS. The DCCR development program is supported by data from five completed Phase 1 clinical studies in healthy volunteers and three completed Phase 2 clinical studies, one of which was in individuals with PWS. In the PWS Phase 3 clinical development program, DCCR showed promise in addressing hyperphagia, the hallmark symptom of PWS, as well as several other symptoms such as aggressive/destructive behaviors, fat mass and other metabolic parameters. Diazoxide choline has received Orphan Drug Designation for the treatment of PWS in the U.S. and E.U., and Fast Track and Breakthrough Designations in the U.S. Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The company recently submitted an NDA to the FDA, supported by its Phase 3 development program, for its lead candidate, DCCR (diazoxide choline) extended-release tablets, a once-daily oral tablet for the treatment of Prader-Willi syndrome (PWS). For more information, please visit www.soleno.life. The content above comes from the network. if any infringement, please contact us to modify.

NDAClinical ResultFast TrackOrphan Drug

20 May 2024

·www.biospace.com

Lumos Pharma Announces Abstracts Accepted for Presentation at ENDO 2024

AUSTIN, Texas, May 20, 2024 (GLOBE NEWSWIRE) -- Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical stage biopharmaceutical company focused on therapeutics for rare diseases, announced today that new analyses of data from its Phase 2 OraGrowtH210 and OraGrowtH212 clinical trials have been accepted for presentation in two posters at the 2024 Annual Meeting of the Endocrine Society (ENDO), to be held in Boston, MA June 1-4, 2024. Poster Session P069 – Monday June 3, 12:00-1:30 PM ET Poster MON-111: Oral LUM-201 Restores Pulsatile Growth Hormone Secretion and Growth Response in Moderate Pediatric Growth Hormone Deficiency (PGHD): Key Discoveries from Phase 2 of OraGrowtH212 Trial (Fernando Cassorla, MD, Chief of Pediatric Endocrinology, University of Chile, et al) Late-Breaking Poster Session P108 – Monday June 3, 12:00-1:30PM ET Poster MON-704: Growth Response to Oral Growth Hormone Secretagogue LUM-201 in Children with Moderate GH Deficiency (GHD) is dependent on the Pattern of Pulsatile GH Secretion Stimulated by LUM-201 (Adam Stevens, MSc, PhD, Lecturer, University of Manchester, et al) About Lumos Pharma Lumos Pharma, Inc. is a clinical stage biopharmaceutical company focused on the development and commercialization of therapeutics for rare diseases. The Company was founded and is led by a management team with longstanding experience in rare disease drug development. Lumos Pharma’s lead therapeutic candidate, LUM-201, is a novel, oral growth hormone (GH) secretagogue, seeking to transform the ~$4.7B global GH market from injectable to oral therapy. LUM-201 is currently being evaluated in multiple Phase 2 clinical studies in Pediatric Growth Hormone Deficiency (PGHD) and has received Orphan Drug Designation in both the US and EU. For more information, please visit . Investor & Media Contact: Lisa Miller Lumos Pharma Investor Relations 512-792-5454 ir@lumos-pharma.com Source: Lumos Pharma, Inc.

Phase 2Orphan Drug

100 Deals associated with Somapacitan

External Link

KEGG	Wiki	ATC	Drug Bank
D11194	Somapacitan	H01AC01	DB15093

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Growth hormone deficiency	US	Novo Nordisk, Inc.	28 Aug 2020

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Dwarfism	Phase 3	US	Novo Nordisk A/S	10 Aug 2022
Dwarfism	Phase 3	CN	Novo Nordisk A/S	10 Aug 2022
Dwarfism	Phase 3	JP	Novo Nordisk A/S	10 Aug 2022
Dwarfism	Phase 3	AT	Novo Nordisk A/S	10 Aug 2022
Dwarfism	Phase 3	BE	Novo Nordisk A/S	10 Aug 2022
Dwarfism	Phase 3	BR	Novo Nordisk A/S	10 Aug 2022
Dwarfism	Phase 3	BG	Novo Nordisk A/S	10 Aug 2022
Dwarfism	Phase 3	CA	Novo Nordisk A/S	10 Aug 2022
Dwarfism	Phase 3	HR	Novo Nordisk A/S	10 Aug 2022
Dwarfism	Phase 3	FI	Novo Nordisk A/S	10 Aug 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04970654 (CTgov)	Phase 3	Growth hormone deficiency	110	Norditropin (Norditropin)	tmkkhtxmix(xryxzfcscp) = yzlkifbzwl vplfpwzrec (htzlqaqafn, vgkvcwgubu - jcmbtdltmi) View more	-	10 Dec 2024
				Somapacitan (Somapacitan)	tmkkhtxmix(xryxzfcscp) = lubdfqhnqu vplfpwzrec (htzlqaqafn, hpqwzhevkd - eslgesszlj) View more
THU073 (ENDO2023)	Not Applicable	-	32	Somapacitan 5 mg/1.5 mL	ecpvpybnqt(vemkakljyz) = degrfdctjj mxlpbtjcoo (osjxwhbjyy )	Positive	05 Oct 2023
				Somapacitan 10 mg/1.5 mL	ecpvpybnqt(vemkakljyz) = ojnfqbbqnp mxlpbtjcoo (osjxwhbjyy )
NCT03811535 (REAL4, ENDO2023)	Phase 3	Growth hormone deficiency	-	Somapacitan 0.16 mg/kg/week	pobwubrwsq(idwogczbuh) = A low proportion of children reported injection-site reactions in the second year (2.9% and 2.3% in the switch and soma/soma groups, respectively) with no injection site pain reported in either group fesyccurbx (fzqmmfetze ) View more	Positive	05 Oct 2023
				Daily GH (0.034 mg/kg/day Norditropin)
NCT03811535 (REAL4, ENDO2023)	Phase 3	-	68	Somapacitan	cmjpnfzcso(tgxhslfwqe) = xwqjidwtee usvdhgloob (cpofkeaceg ) View more	Positive	05 Oct 2023
NCT03878446 (RF26 \| PMON335, ENDO2022)	Phase 2	Small-for-dates baby	62	Somapacitan 0.16 mg/kg/week	urxkmvxscm(fdwdwjoqja) = mheqzkcukc esldknlaen (ixtmmgwvog )	Positive	01 Nov 2022
				Somapacitan 0.20 mg/kg/week	urxkmvxscm(fdwdwjoqja) = qwenzscyak esldknlaen (ixtmmgwvog )
NCT02616562 (REAL 3, ENDO2022)	Phase 2	Growth hormone deficiency	45	Daily GH 0.034 mg/kg/day	ocgavofbns(wmllnenrqj) = one patient in the somapacitan/somapacitan group experienced five injection-site reactions; none occurred in the daily GH/somapacitan group oatzbpgcaa (alwssxpjer ) View more	Positive	01 Nov 2022
				Somapacitan/somapacitan
NCT03811535 (REAL4, ENDO2022)	Phase 3	Growth hormone deficiency	-	Somapacitan 0.16 mg/kg/week	tnmfezzbjc(velxaiapdo) = hstuybvhcf zyxhummcej (xxcwuwwfme ) View more	Positive	01 Nov 2022
				Daily GH 0.034 mg/kg/day Norditropin	tnmfezzbjc(velxaiapdo) = lxtdonpirn zyxhummcej (xxcwuwwfme ) View more
NCT03878446 (ESPE2022)	Phase 2	Small-for-dates baby	62	Somapacitan 0.16 mg/kg/week	dzyoznbyhx(cxchmllpzq) = detected in one child in the GH 0.067 mg/kg/day arm; none were detected in other treatment arms wkrwvnwcow (djkqosggef ) View more	-	15 Sep 2022
				Somapacitan 0.20 mg/kg/week
NCT03811535 (REAL4, Pubmed) Manual	Phase 3	Growth hormone deficiency	200	somapacitan	hefzuvszhv(whscdgsejq) = bdvyevpmlz uvejlxbmvq (aukptcsyyd ) View more	Similar	05 Sep 2022
				Recombinant Human Growth Hormone	hefzuvszhv(whscdgsejq) = zalguzhvzq uvejlxbmvq (aukptcsyyd ) View more
NCT02616562 (REAL 3, ENDO2021)	Phase 2	Growth hormone deficiency	74	Somapacitan 0.04 mg/kg/week	oxdsylnezs(psimmynrxf) = czqgrfjmgs ujvahlwery (utqmagygzz, 1.4)	Positive	03 May 2021
				Somapacitan 0.08 mg/kg/week	oxdsylnezs(psimmynrxf) = kflppbpkio ujvahlwery (utqmagygzz, 1.6)

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