Dataset Information

Treatment Burden of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Randomized Study.

ABSTRACT:

Context

Somatrogon is a long-acting recombinant human growth hormone treatment developed as a once-weekly treatment for pediatric patients with growth hormone deficiency (GHD).

Objective

Evaluate patient and caregiver perceptions of the treatment burden associated with the once-weekly somatrogon injection regimen vs a once-daily Somatropin injection regimen.

Methods

Pediatric patients (≥3 to <18 years) with GHD receiving once-daily somatropin at enrollment were randomized 1:1 to Sequence 1 (12 weeks of once-daily Somatropin, then 12 weeks of once-weekly somatrogon) or Sequence 2 (12 weeks of once-weekly somatrogon, then 12 weeks of once-daily Somatropin). Treatment burden was assessed using validated questionnaires completed by patients and caregivers. The primary endpoint was the difference in mean overall life interference (LI) total scores after each 12-week treatment period (somatrogon vs Somatropin), as assessed by questionnaires.

Results

Of 87 patients randomized to Sequence 1 (n = 43) or 2 (n = 44), 85 completed the study. Once-weekly somatrogon had a significantly lower treatment burden than once-daily Somatropin, based on mean overall LI total scores after somatrogon (8.63) vs Somatropin (24.13) treatment (mean difference -15.49; 2-sided 95% CI -19.71, -11.27; P < .0001). Once-weekly somatrogon was associated with greater convenience, higher satisfaction with treatment experience, and less LI. The incidence of treatment-emergent adverse events (TEAEs) for Somatropin and somatrogon was 44.2% and 54.0%, respectively. No severe or serious AEs were reported.

Conclusion

In pediatric patients with GHD, once-weekly somatrogon had a lower treatment burden and was associated with a more favorable treatment experience than once-daily Somatropin.

SUBMITTER: Maniatis AK

PROVIDER: S-EPMC9463876 | biostudies-literature | 2022 Oct

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Treatment Burden of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Randomized Study.

Maniatis Aristides K AK Carakushansky Mauri M Galcheva Sonya S Prakasam Gnanagurudasan G Fox Larry A LA Dankovcikova Adriana A Loftus Jane J Palladino Andrew A AA de Los Angeles Resa Maria M Turich Taylor Carrie C Dattani Mehul T MT Lebl Jan J

Journal of the Endocrine Society 20220910 10

<h4>Context</h4>Somatrogon is a long-acting recombinant human growth hormone treatment developed as a once-weekly treatment for pediatric patients with growth hormone deficiency (GHD).<h4>Objective</h4>Evaluate patient and caregiver perceptions of the treatment burden associated with the once-weekly somatrogon injection regimen vs a once-daily Somatropin injection regimen.<h4>Methods</h4>Pediatric patients (≥3 to <18 years) with GHD receiving once-daily somatropin at enrollment were randomized 1 ...[more]

PMID: 36101713

Similar Datasets

Project description:Abstract Background Somatrogon is a long-acting recombinant human growth hormone (hGH) currently being developed as a once-weekly subcutaneous injectable treatment for pediatric patients with growth hormone deficiency (GHD). A recent global Phase 3 study compared the efficacy and safety of somatrogon administered once weekly with Genotropin administered once daily in pediatric patients with GHD (ClinicalTrials.gov: NCT02968004). Aims To understand and describe physicians’ experiences using once-weekly somatrogon injections compared with once-daily hGH injections during the Phase 3 study. Methods In this quantitative, cross-sectional, observational study, a 14-item multiple choice questionnaire was administered to physicians who were clinical investigators in the Phase 3 study. The questionnaire was designed to assess physicians’ experiences, preferences and satisfaction regarding the use of the once-weekly somatrogon regimen compared with the standard once-daily hGH injection regimen. Survey results were analyzed quantitatively using descriptive statistics. Results A total of 24 physicians who were part of the global Phase 3 study (mean age [SD]: 58.5 [10.1] years; 10 males, 14 females) in 12 different countries completed the on-line survey. Compared with daily injections, most physicians preferred somatrogon (n=18, 75.0%) and reported that somatrogon was more convenient (n=19, 79.2%), less burdensome (n=19, 79.2%) and that they would be more likely to prescribe somatrogon to patients in the future (n=20, 83.3%). In terms of being more likely to support positive long-term growth outcomes, 37.5% (n=9) of physicians selected somatrogon; the remaining physicians (n=15, 62.5%) reported no difference between treatments. The proportion of physicians who were satisfied or very satisfied with somatrogon was 95.8% (n=23), compared with 87.5% (n=21) for daily hGH injections. Most physicians (n=15, 62.5%) reported that daily hGH injections required more effort/much more effort to monitor adherence, compared with somatrogon. Some physicians (n=7, 29.2%) reported that explaining the device instructions to patient/caregivers required more/much more effort for somatrogon compared with daily hGH injections; more than half (n=14, 58.3%) reported no difference in effort required for either regimen. Physicians were divided as to which treatment needed more/much more effort to: (i) explain the injection regimen (n=7 [29.2%] daily injection; n=7 [29.2%] no difference; n=10 [41.7%] somatrogon), (ii) explain what to do if an injection is missed (n=7 [29.2%] daily injection; n=9 [37.5%] no difference; n=8 [33.3%] somatrogon), and (iii) address patient/caregiver questions (n=8 [33.3%] daily injection; n=11 [45.8%] no difference; n=5 [20.8%] somatrogon). Conclusions Overall, physicians from the global Phase 3 study were positive about their experience using somatrogon for the treatment of children with GHD. Physicians generally preferred the once-weekly somatrogon injection regimen, with most physicians reporting somatrogon to be more convenient and less burdensome to patients, indicating that they would be more likely to prescribe somatrogon to their patients in the future. Presentation: Monday, June 13, 2022 12:30 p.m. - 2:30 p.m.

Project description:Abstract Growth hormone (GH) replacement therapy currently requires daily injections. Somapacitan is a long-acting GH-derivative being developed for once-weekly (OW) use in children and adults with GH deficiency (GHD). A phase 2, multinational, randomized, open-label, controlled trial (ClinicalTrials.gov: NCT02616562) investigated the efficacy and safety of OW somapacitan compared with daily GH (Norditropin® FlexPro®). GH-treatment-naïve prepubertal children with GH deficiency received 0.04 (n=16), 0.08 (n=15) or 0.16 mg/kg/wk (n=14) subcutaneous (sc) OW somapacitan, or sc GH 0.034 mg/kg/day (0.24 mg/kg/wk; n=14) for 52 wks, followed by a 104-wk safety extension with all patients on somapacitan receiving 0.16 mg/kg/wk while GH dose was unaltered. Safety endpoints included frequency of adverse events (AEs), including injection-site reactions, and occurrence of anti-somapacitan/anti-human growth hormone (hGH) antibodies. The 52-wk efficacy and safety results have been reported. We report here safety results at 104 wks’ total treatment. Number of AEs (% of patients) was as follows: OW somapacitan 0.04/0.16 mg/kg/wk, 51 (75%); 0.08/0.16 mg/kg/wk, 89 (80%); 0.16/0.16 mg/kg/wk, 89 (100%); and for daily GH, 82 (100%). Nasopharyngitis, influenza, allergic rhinitis and gastroenteritis were the most common AEs across all treatment groups. Pyrexia was more common in the OW somapacitan 0.04/0.16 mg/kg/wk (43.8%) and 0.08/0.16 mg/kg/wk (26.7%) groups vs the higher-dose OW somapacitan and daily GH groups (7.1% each). Most AEs were mild to moderate and unlikely related to treatment. Ten serious AEs were reported in five (8.5%) children and unlikely related to treatment, except two AEs of moderate severity during the first 26 wks: generalized edema and vomiting in one child on 0.16 mg/kg/wk OW somapacitan, rated as probably related to treatment although she was also given intravenous antibiotics for suspected infection. Injection-site reactions were reported in the 0.04/0.16 mg/kg/wk (n=2) and 0.16/0.16 mg/kg/wk (n=1) OW somapacitan groups and in the daily GH group (n=1). Four children (0.04/0.16 mg/kg/wk) and one child (0.16/0.16 mg/kg/wk) had transient anti-somapacitan antibodies; one child (0.16/0.16 mg/kg/wk) had low-titer anti-somapacitan antibodies at two consecutive visits; in one child (daily GH group) with persistent low-titer anti-hGH antibodies, treatment was discontinued at wk 52. All antibodies were non-neutralizing. In conclusion, OW somapacitan was well tolerated at all doses, with no new safety or tolerability issues identified after up to 104 wks of treatment. The frequency, severity and type of AEs were similar in the OW somapacitan and daily GH treatment groups except for pyrexia, which was unlikely related to treatment and more frequently reported in the lowest dose somapacitan group.

Project description: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). The objective of this study is to evaluate the efficacy, safety, and tolerability of once-weekly somapacitan vs once-daily GH. 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). This study took place at 29 sites in 11 countries. Fifty-nine GH treatment-naive prepubertal children with GHD were randomly assigned; 58 completed the trial. 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. 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. 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. 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).

Dataset Information

Treatment Burden of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Randomized Study.

Context

Objective

Methods

Results

Conclusion

Publications

Treatment Burden of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Randomized Study.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets