Aromatase Inhibitors, Alone And In Combination With Growth Hormone In Adolescent Boys With Idiopathic Short Stature
ThrasherAI
A Randomized Controlled Trial Of The Use Of Aromatase Inhibitors, Alone And In Combination With Growth Hormone In Adolescent Boys With Idiopathic Short Stature
1 other identifier
interventional
76
2 countries
4
Brief Summary
When treating very short children in puberty we are time-limited, as sex hormones cause the growth plates to fuse and growth to end. Growth Hormone (GH), plus drugs that stop puberty, increase height potential, but leave children sexually infantile at a critical time in development. Human and animal data show that estrogen, in females and males, is a principal regulator of the fusion of the growth plate in puberty. Using aromatase inhibitors (AIs), which block testosterone to estrogen conversion, in boys with different growth disorders, we have shown that AIs may have beneficial effects enhancing height potential in growth-retarded males, without affecting their puberty. However, no direct comparison of the effect of AIs alone vs. conventional GH treatment has been done to date. This study will assess the effect of AIs alone, GH alone and combination treatment in enhancing height potential in adolescent boys with idiopathic short stature.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Nov 2010
Longer than P75 for phase_3
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 1, 2010
CompletedFirst Submitted
Initial submission to the registry
November 2, 2010
CompletedFirst Posted
Study publicly available on registry
November 25, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
September 1, 2016
CompletedResults Posted
Study results publicly available
June 7, 2018
CompletedAugust 7, 2018
July 1, 2018
5.8 years
November 2, 2010
October 29, 2017
July 9, 2018
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Change in Height
Differences in height gains
0 to 24 months
Change in Predicted Height
Primary efficacy end point: change in predicted height (cm) from baseline at 24 months based on change in bone age (years)
0 to 24 months
Secondary Outcomes (7)
Change in Bone Density z Score Adjusted for Height
0 to 24 months
Change in Lean Body Mass
0 to 24 months
Change in Body Mass Index
0 to 24 months
Change in IGF-I Concentrations
0 to 24 months
Change in Testosterone
0 to 24 months
- +2 more secondary outcomes
Study Arms (3)
Aromatase Inhibitor
ACTIVE COMPARATORAnastrozole 1mg or Letrozole 2.5mg daily orally for 2 to 3 years
Growth Hormone
ACTIVE COMPARATORSomatropin 0.3mg/kg/week divided daily subcutaneously for 2 to 3 years
Aromatase Inhibitor and Growth Hormone
ACTIVE COMPARATORAnastrozole 1mg or Letrozole 2.5mg orally daily for 2 to 3 years and Somatropin 0.3mg/kg/week divided daily subcutaneously for 2 to 3 years
Interventions
Eligibility Criteria
You may qualify if:
- Males: Ages: 12 - less than 18 years.
- Bone age less than 14 ½ years at study initiation.
- Presence of puberty.
- Idiopathic short stature will be defined as a short child equal or less than -2SD for height, with normal GH responses to stimuli (\> or = 5ng/ml to at least 2 secretagogues) or a normal IGF-I and BP-3, normal body proportions and no other identifiable growth pathology.
- Accurate growth data for at least 6 months at baseline is available.
You may not qualify if:
- Chronic illnesses.
- Chronic use of glucocorticosteroids.
- Previous use of hormonal treatment with AIs, sex steroids or GH in the preceding 6 months.
- Birth weight small for gestational age (SGA).
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Nemours Children's Cliniclead
- Thrasher Research Fundcollaborator
- Genentech, Inc.collaborator
- Novartiscollaborator
- AstraZenecacollaborator
- Pfizercollaborator
Study Sites (4)
Nemours Children's Clinic
Jacksonville, Florida, 32207, United States
Nemours Children's Clinic
Orlando, Florida, 32801, United States
Nemours Children's Clinic- Jefferson
Philadelphia, Pennsylvania, 19107, United States
Veronica Mericq, MD
Santiago, Chile
Related Publications (1)
Mauras N, Ross JL, Gagliardi P, Yu YM, Hossain J, Permuy J, Damaso L, Merinbaum D, Singh RJ, Gaete X, Mericq V. Randomized Trial of Aromatase Inhibitors, Growth Hormone, or Combination in Pubertal Boys with Idiopathic, Short Stature. J Clin Endocrinol Metab. 2016 Dec;101(12):4984-4993. doi: 10.1210/jc.2016-2891. Epub 2016 Oct 6.
PMID: 27710241RESULT
MeSH Terms
Interventions
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Dr. Nelly Mauras
- Organization
- Nemours Children's Clinic
Study Officials
- PRINCIPAL INVESTIGATOR
Nelly Mauras, MD
Nemours Children's Clinic Jacksonville
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Chief, Division of Endocrinology, Diabetes & Metabolism
Study Record Dates
First Submitted
November 2, 2010
First Posted
November 25, 2010
Study Start
November 1, 2010
Primary Completion
September 1, 2016
Study Completion
September 1, 2016
Last Updated
August 7, 2018
Results First Posted
June 7, 2018
Record last verified: 2018-07