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Phase 2 N=14 Treatment

Pediatrics Testotoxicosis Study [Bicalutamide Anastrozole Treatment for Testotoxicosis]

Puberty, Precocious

Enrolled (actual)
14
Serious AEs
0.0%
Results posted
Jul 2009
Primary outcome: Primary: Change in Growth Rate (cm/Year) — -1.62 cm/year — p=0.278

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Bicalutamide (Drug); Anastrozole (Drug)
Age
Pediatric · 2+ yrs
Sex
Male
Sponsor
AstraZeneca
Primary completion
May 2008

Outcome Measures

OutcomeResultp-value
PRIMARY
Change in Growth Rate (cm/Year)
-0.70
PRIMARY
Change in Growth Rate (SD Units)
-0.14
SECONDARY
Change in Growth Rate (cm/Year)
-0.70
SECONDARY
Change in Growth Rate (SD Units)
-0.14
SECONDARY
Change in Bone Age Maturation Rate (cm/Year)
-2.03; -2.29
SECONDARY
Change in Bone Age to Chronological Age Ratio
-0.09; -0.24
SECONDARY
Number of Patients With Height Between 5th and 95th Percentile
3; 3; 3; 3
SECONDARY
Change in Predicted Adult Height (PAH)
6.21
SECONDARY
Change in Average Testicular Volume
1.46; 2.69

Summary

The primary objective of this study is to investigate whether bicalutamide given in combination with anastrozole once daily for 12 months is effective in treating testotoxicosis in boys. Testotoxicosis is a condition that causes early puberty in boys including growth in height, and development of muscles and sexual organs .

Eligibility Criteria

Inclusion Criteria

  • Provision of written informed consent of parent/legal guardian and subject assent (as needed by local requirements)
  • Male aged 2 years and over
  • Diagnosis of testotoxicosis based on the following:
  • Clinical features of Progressive sexual precocity documented by Tanner staging and evidence of symmetrical testicular enlargement
  • Clinical features of significantly advanced bone age (defined as bone age of at least 12 months beyond chronological age)
  • Pubertal levels of serum testosterone
  • Prepubertal levels of serum gonadotropins
  • Lack of an increase in serum gonadotropin levels following GnRH stimulation
  • Other pathology excluded by:
  • Undetectable plasma b human chorionic gonadotropin (bHCG). Samples with values below the LOQ will be reported as " 6 months prior to study enrollment. Additionally for subjects who have previously received ketoconazole or spironolactone treatment, a documented reliable height measurement taken immediately prior to beginning this treatment.

(Note: for subjects who received such previous treatment only a single assessment is needed if it was taken immediately prior to beginning treatment and > 6 months prior to study entry)

  • Subjects should be free of endocrine or other effects of previous treatment for testotoxicosis prior to study entry: to ensure this there should be 15 days or 4 drug half lives (whichever is the longer) washout period from prior medication for testotoxicosis.

Exclusion Criteria

  • Evidence of central precocious puberty as demonstrated by GnRH stimulation test
  • Serum concentration of total or direct bilirubin, GGT, AST or ALT greater than 1.5 times the upper limit of normal for age
  • Serum concentration of creatinine greater than 1.5 times the upper limit of normal for age
  • Any concomitant medical condition that, in the opinion of the investigator, may expose a subject to an unacceptable level of safety risk or that affects subject compliance
  • Known hypersensitivity to any of the study medications
  • Participation in a clinical study at the time of enrollment
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00094328). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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