Clinical trial NCT01009905: An Observational Study (Registry) Assessing Treatment Outcomes and Safety for Children and Adults Who Are Prescribed Norditropin® (Human Growth Hormone)
Official title: Registry of Patients Being Treated With Norditropin®, Recombinant Human Growth Hormone
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Growth Hormone Disorder
Pituitary Dwarfism
Hypopituitarism
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2009-11-09
Enrollment: 22960
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients from both general and specialty practice settings, existing and new users of growth hormones, who have been deemed appropriate to receive Norditropin® as part of routine out-patient care by the prescribing physician.

Brief summary: This study is conducted in the United States of America (USA). The aim of this observational study is to collect data concerning the treatment outcomes and safety for children and adults who are prescribed Norditropin®. Specific objectives include: 1) developing models defining the relationship of Norditropin dose to changes in insulin-like growth factor (IGF-I) and treatment outcomes, accounting for independent factors such as age, gender and puberty and 2) determining the relative predictive values of peak growth hormone (GH) and IGF-I levels and other factors before treatment to clinical outcomes.


Eligibility criteria: Inclusion Criteria:

* Informed consent obtained
* Children or adult patients treated with Norditropin® for an appropriate condition as prescribed by their physician

Exclusion Criteria:

* Patients not being treated with Norditropin®
* Patients who have discontinued treatment with Norditropin®
* Known or suspected allergy to Norditropin® or related products
* Contraindications for somatropin treatment consistent with the Prescribing Information for Norditropin®

Primary outcomes:
measure: Change in height, measured as the height standard deviation score (HSDS), from the measurement made by the physician at study entry to the most recent clinic observation, end of GH treatment, de-registration, or change of clinic.
timeFrame: measurement made by physician at study entry, annually, or more frequently at physician discretion during the study.
measure: Change in waist/hip circumference ratio for adult patients
timeFrame: measurement made by physician at study entry, annually, or more frequently at physician discretion during the study
Secondary outcomes:
measure: Short-term: Height, Height velocity, Height velocity standard deviation score (SDS), predicted adult height, height SDS for bone age, Proportion achieving height SDS > -2 at end of observational duration period
timeFrame: measurement made by physician at study entry, annually, or more frequently at physician discretion during the study.
measure: Final Height (or Adult height), Final Height SDS, near-adult height, near-adult height SDS, Final Height - Target Height, Final height - predicted adult height, height age
timeFrame: measurement made by physician at study entry, annually, or more frequently at physician discretion during the study.
measure: Final Height SDS - Target Height SDS, Final height SDS - predicted adult height SDS
timeFrame: measurement made by physician at study entry, annually, or more frequently at physician discretion during the study.
measure: Proportion achieving HSDS of more than -2 at final height
timeFrame: measurement made by physician at study entry, annually, or more frequently at physician discretion during the study.
measure: Weight, waist circumference, and hip circumference for adult patients
timeFrame: measurement made by physician at study entry, annually, or more frequently at physician discretion during the study
Other outcomes:
Locations:
city: Plainsboro
country: United States
facility: Novo Nordisk Investigational Site
state: New Jersey
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452), MD
role: STUDY_DIRECTOR
References:
citation: Ross JL, Lee PA, Gut R, Germak J. Attaining genetic height potential: Analysis of height outcomes from the ANSWER Program in children treated with growth hormone over 5 years. Growth Horm IGF Res. 2015 Dec;25(6):286-93. doi: 10.1016/j.ghir.2015.08.006. Epub 2015 Aug 22.
citation: Lee PA, Savendahl L, Oliver I, Tauber M, Blankenstein O, Ross J, Snajderova M, Rakov V, Pedersen BT, Christesen HT. Comparison of response to 2-years' growth hormone treatment in children with isolated growth hormone deficiency, born small for gestational age, idiopathic short stature, or multiple pituitary hormone deficiency: combined results from two large observational studies. Int J Pediatr Endocrinol. 2012 Jul 12;2012(1):22. doi: 10.1186/1687-9856-2012-22.
citation: Hoybye C, Savendahl L, Christesen HT, Lee P, Pedersen BT, Schlumpf M, Germak J, Ross J. The NordiNet(R) International Outcome Study and NovoNet(R) ANSWER Program(R): rationale, design, and methodology of two international pharmacoepidemiological registry-based studies monitoring long-term clinical and safety outcomes of growth hormone therapy (Norditropin(R)). Clin Epidemiol. 2013 Apr 26;5:119-27. doi: 10.2147/CLEP.S42602. Print 2013.
citation: Savendahl L, Blankenstein O, Oliver I, Christesen HT, Lee P, Pedersen BT, Rakov V, Ross J. Gender influences short-term growth hormone treatment response in children. Horm Res Paediatr. 2012;77(3):188-94. doi: 10.1159/000337570. Epub 2012 Apr 12.
citation: Biller BMK, Hoybye C, Carroll P, Gordon MB, Birkegard AC, Kelepouris N, Nedjatian N, Weber MM. Pregnancy outcomes in women receiving growth hormone replacement therapy enrolled in the NordiNet(R) International Outcome Study (IOS) and the American Norditropin(R) Studies: Web-Enabled Research (ANSWER) Program. Pituitary. 2021 Aug;24(4):611-621. doi: 10.1007/s11102-021-01138-3. Epub 2021 Mar 12.
citation: Savendahl L, Polak M, Backeljauw P, Blair JC, Miller BS, Rohrer TR, Hokken-Koelega A, Pietropoli A, Kelepouris N, Ross J. Long-Term Safety of Growth Hormone Treatment in Childhood: Two Large Observational Studies: NordiNet IOS and ANSWER. J Clin Endocrinol Metab. 2021 May 13;106(6):1728-1741. doi: 10.1210/clinem/dgab080.
citation: Angulo M, Abuzzahab MJ, Pietropoli A, Ostrow V, Kelepouris N, Tauber M. Outcomes in children treated with growth hormone for Prader-Willi syndrome: data from the ANSWER Program(R) and NordiNet(R) International Outcome Study. Int J Pediatr Endocrinol. 2020 Nov 10;2020(1):20. doi: 10.1186/s13633-020-00090-6.
citation: Miller BS, Ross J, Ostrow V. Height outcomes in children with growth hormone deficiency and idiopathic short stature treated concomitantly with growth hormone and aromatase inhibitor therapy: data from the ANSWER program. Int J Pediatr Endocrinol. 2020;2020:19. doi: 10.1186/s13633-020-00089-z. Epub 2020 Oct 6.
citation: Weber MM, Gordon MB, Hoybye C, Jorgensen JOL, Puras G, Popovic-Brkic V, Molitch ME, Ostrow V, Holot N, Pietropoli A, Biller BMK. Growth hormone replacement in adults: Real-world data from two large studies in US and Europe. Growth Horm IGF Res. 2020 Feb;50:71-82. doi: 10.1016/j.ghir.2019.09.002. Epub 2019 Oct 26.
citation: Savendahl L, Polak M, Backeljauw P, Blair J, Miller BS, Rohrer TR, Pietropoli A, Ostrow V, Ross J. Treatment of Children With GH in the United States and Europe: Long-Term Follow-Up From NordiNet(R) IOS and ANSWER Program. J Clin Endocrinol Metab. 2019 Oct 1;104(10):4730-4742. doi: 10.1210/jc.2019-00775.
citation: Rose SR, Reeves G, Gut R, Germak J. Attention-Deficit/Hyperactivity Disorder Medication Treatment Impact on Response to Growth Hormone Therapy: Results from the ANSWER Program, a Non-Interventional Study. J Pediatr. 2015 Dec;167(6):1389-96. doi: 10.1016/j.jpeds.2015.08.036. Epub 2015 Sep 26.
citation: Lee PA, Ross J, Germak JA, Gut R. Effect of 4 years of growth hormone therapy in children with Noonan syndrome in the American Norditropin Studies: Web-Enabled Research (ANSWER) Program(R) registry. Int J Pediatr Endocrinol. 2012 Jun 8;2012(1):15. doi: 10.1186/1687-9856-2012-15.