Randomized Clinical Trial of Two Different Initial Growth Hormone Doses in Children

NCT06103513 | Unknown Phase 3 DMC FDA Drug

• 1 other identifier: 23-0027
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

A prospective, randomized, open-label single-blinded study of 50 subjects with growth hormone deficiency, ages 5 to 15 years in which 25 subjects will initiate rhGH therapy at 0.3mg/kg/week and the remaining 25 subjects will initiate their rhGH treatment at 0.2 mg/kg/week for the first 12 months of treatment. Safety parameters, height velocity, and adult height prediction by bone age determination will be assessed at 4-month intervals for 1 year following the initiation of rhGH therapy.

Conditions

Growth Disorders; Growth Failure; Growth Hormone Treatment; Growth

Keywords

Growth Hormone

Outcome Measures

Primary Outcomes (1)

• Annualized Growth Velocity (GV) in the first year after treatment

  Height (cm) at 0- month visit; height (cm) at 12-month visit

  I year

Secondary Outcomes (5)

• IGF1- 0,4,8,12 months

  12 Months

• IGFBP3 - 0,4,8,12 months

  12 Months

• HbA1c - 4,8,12 months

  12 Months

• Total T4 or Free T4 - 0,12 months

  12 Months

• TSH - 0, 12 months

  12 Months

Study Arms (2)

Arm 1: Growth hormone 0.2 mg/kg/week

ACTIVE COMPARATOR

Twenty-five subjects will initiate rhGH therapy at 0.2 mg/kg/week for the first 12 months of treatment

Drug: Somatropin

Arm 2: Growth hormone 0.3 mg/kg/week

ACTIVE COMPARATOR

Twenty-five subjects will initiate rhGH therapy at 0.3 mg/kg/week for the first 12 months of treatment

Drug: Somatropin

Interventions

Somatropin DRUG

The treatment of children with subcutaneous recombinant human growth hormone (rhGH) is the current gold standard of care for children with diagnosed GHD. This study will serve only to investigate the optimal dose of treatment in the first year of treatment with rhGH.

Also known as: Norditropin, Nutropin AQ, Genotropin, Zomacton, Humatrope, Omnitrope.

Arm 1: Growth hormone 0.2 mg/kg/week; Arm 2: Growth hormone 0.3 mg/kg/week

Eligibility Criteria

• Age: 5 Years - 15 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• Provision of signed and dated informed consent form
• Stated willingness to comply with all study procedures and availability for the duration of the study
• Male or female, aged 5-15 years
• In good general health as evidenced by medical history or diagnosed with growth hormone deficiency
• Ability to take subcutaneous GH injections nightly

You may not qualify if:

• Subjects will be excluded if they have GH resistance, or syndromic short stature such as Prader Willi syndrome and Turner syndrome. Patients will also be excluded if they have active malignancies, or systemic illnesses such as heart failure, kidney failure, or liver failure.

Sponsors & Collaborators

• Northwell Health: lead

Study Sites (1)

Northwell Health

New York, New York, 11042, United States

RECRUITING

Related Publications (19)

• Backelijaw P, Dattani M, Cohen P Rosenfeld R, (2014).Chapter 10: Disorders of Growth Hormone/ Insulin Like Growth Factor Secretion and Action. Shreiner, J (Ed.). Pediatric Endocrinology: Marc Sperling. (chapter 10, page 364, 366). Philadelphia, Saunders

  BACKGROUND

• Grumbach MM, Bin-Abbas BS, Kaplan SL. The growth hormone cascade: progress and long-term results of growth hormone treatment in growth hormone deficiency. Horm Res. 1998;49(# Suppl 2):41-57. No abstract available.

  PMID: 9716827 BACKGROUND

• Blethen SL, MacGillivray MH. A risk-benefit assessment of growth hormone use in children. Drug Saf. 1997 Nov;17(5):303-16. doi: 10.2165/00002018-199717050-00003.

  PMID: 9391774 BACKGROUND

• Christ ER, Cummings MH, Jackson N, Stolinski M, Lumb PJ, Wierzbicki AS, Sonksen PH, Russell-Jones DL, Umpleby AM. Effects of growth hormone (GH) replacement therapy on low-density lipoprotein apolipoprotein B100 kinetics in adult patients with GH deficiency: a stable isotope study. J Clin Endocrinol Metab. 2004 Apr;89(4):1801-7. doi: 10.1210/jc.2003-031474.

  PMID: 15070948 BACKGROUND

• Locatelli V, Bianchi VE. Effect of GH/IGF-1 on Bone Metabolism and Osteoporsosis. Int J Endocrinol. 2014;2014:235060. doi: 10.1155/2014/235060. Epub 2014 Jul 23.

  PMID: 25147565 BACKGROUND

• Boguszewski CL, Meister LH, Zaninelli DC, Radominski RB. One year of GH replacement therapy with a fixed low-dose regimen improves body composition, bone mineral density and lipid profile of GH-deficient adults. Eur J Endocrinol. 2005 Jan;152(1):67-75. doi: 10.1530/eje.1.01817.

  PMID: 15762189 BACKGROUND

• Hardin DS. Treatment of short stature and growth hormone deficiency in children with somatotropin (rDNA origin). Biologics. 2008 Dec;2(4):655-61. doi: 10.2147/btt.s2252.

  PMID: 19707446 BACKGROUND

• Straetemans S, Thomas M, Craen M, Rooman R, De Schepper J; BESPEED. Poor growth response during the first year of growth hormone treatment in short prepubertal children with growth hormone deficiency and born small for gestational age: a comparison of different criteria. Int J Pediatr Endocrinol. 2018;2018:9. doi: 10.1186/s13633-018-0064-3. Epub 2018 Oct 22.

  PMID: 30377433 BACKGROUND

• Reiter EO, Price DA, Wilton P, Albertsson-Wikland K, Ranke MB. Effect of growth hormone (GH) treatment on the near-final height of 1258 patients with idiopathic GH deficiency: analysis of a large international database. J Clin Endocrinol Metab. 2006 Jun;91(6):2047-54. doi: 10.1210/jc.2005-2284. Epub 2006 Mar 14.

  PMID: 16537676 BACKGROUND

• de Ridder MA, Stijnen T, Hokken-Koelega AC. Prediction of adult height in growth-hormone-treated children with growth hormone deficiency. J Clin Endocrinol Metab. 2007 Mar;92(3):925-31. doi: 10.1210/jc.2006-1259. Epub 2006 Dec 19.

  PMID: 17179199 BACKGROUND

• Wit JM, Ranke MB, Albertsson-Wikland K, Carrascosa A, Rosenfeld RG, Van Buuren S, Kristrom B, Schoenau E, Audi L, Hokken-Koelega AC, Bang P, Jung H, Blum WF, Silverman LA, Cohen P, Cianfarani S, Deal C, Clayton PE, de Graaff L, Dahlgren J, Kleintjens J, Roelants M. Personalized approach to growth hormone treatment: clinical use of growth prediction models. Horm Res Paediatr. 2013;79(5):257-70. doi: 10.1159/000351025. Epub 2013 May 28.

  PMID: 23735882 BACKGROUND

• Kristrom B, Dahlgren J, Niklasson A, Nierop AF, Albertsson-Wikland K. The first-year growth response to growth hormone treatment predicts the long-term prepubertal growth response in children. BMC Med Inform Decis Mak. 2009 Jan 12;9:1. doi: 10.1186/1472-6947-9-1.

  PMID: 19138407 BACKGROUND

• MacGillivray MH, Baptista J, Johanson A. Outcome of a four-year randomized study of daily versus three times weekly somatropin treatment in prepubertal naive growth hormone-deficient children. Genentech Study Group. J Clin Endocrinol Metab. 1996 May;81(5):1806-9. doi: 10.1210/jcem.81.5.8626839.

  PMID: 8626839 BACKGROUND

• Ranke MB, Schweizer R, Wollmann HA, Schwarze P. Dosing of growth hormone in growth hormone deficiency. Horm Res. 1999;51 Suppl 3:70-4. doi: 10.1159/000053165.

  PMID: 10592447 BACKGROUND

• Ranke MB, Lindberg A, Chatelain P, Wilton P, Cutfield W, Albertsson-Wikland K, Price DA. Derivation and validation of a mathematical model for predicting the response to exogenous recombinant human growth hormone (GH) in prepubertal children with idiopathic GH deficiency. KIGS International Board. Kabi Pharmacia International Growth Study. J Clin Endocrinol Metab. 1999 Apr;84(4):1174-83. doi: 10.1210/jcem.84.4.5634.

  PMID: 10199749 BACKGROUND

• Sudfeld H, Kiese K, Heinecke A, Bramswig JH. Prediction of growth response in prepubertal children treated with growth hormone for idiopathic growth hormone deficiency. Acta Paediatr. 2000 Jan;89(1):34-7. doi: 10.1080/080352500750029022.

  PMID: 10677054 BACKGROUND

• Kristrom B, Aronson AS, Dahlgren J, Gustafsson J, Halldin M, Ivarsson SA, Nilsson NO, Svensson J, Tuvemo T, Albertsson-Wikland K. Growth hormone (GH) dosing during catch-up growth guided by individual responsiveness decreases growth response variability in prepubertal children with GH deficiency or idiopathic short stature. J Clin Endocrinol Metab. 2009 Feb;94(2):483-90. doi: 10.1210/jc.2008-1503. Epub 2008 Nov 11.

  PMID: 19001519 BACKGROUND

• Radetti G, Buzi F, Paganini C, Pilotta A, Felappi B. Treatment of GH-deficient children with two different GH doses: effect on final height and cost-benefit implications. Eur J Endocrinol. 2003 May;148(5):515-8. doi: 10.1530/eje.0.1480515.

  PMID: 12720533 BACKGROUND

• Cho WK, Ahn MB, Kim EY, Cho KS, Jung MH, Suh BK. Predicting First-Year Growth in Response to Growth Hormone Treatment in Prepubertal Korean Children with Idiopathic Growth Hormone Deficiency: Analysis of Data from the LG Growth Study Database. J Korean Med Sci. 2020 May 18;35(19):e151. doi: 10.3346/jkms.2020.35.e151.

  PMID: 32419399 BACKGROUND

MeSH Terms

Conditions

Growth Disorders; Failure to Thrive

Interventions

Human Growth Hormone; Growth Hormone

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Benjamin U. Nwosu, MD

  NORTHWELL HEALTH, INC.

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jennifer Apsan, MD

CONTACT

516-472-3750; japsan@northwell.edu

Rashida Talib, MPH

CONTACT

516-472-3631; rtalib@northwell.edu

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 17, 2023
• First Posted: October 26, 2023
• Study Start: December 10, 2023
• Primary Completion: June 10, 2025
• Study Completion: December 10, 2025
• Last Updated: December 13, 2023

Record last verified: 2023-10

Locations

US (1)