Pediatrics Testotoxicosis Study [Bicalutamide Anastrozole Treatment for Testotoxicosis]

NCT00094328 | Completed Phase 2 Results

• 2 other identifiers: D6873C00047BATT
• Study Type: interventional
• Target: 14
• Locations: 6 countries
• Sites: 15

Brief 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 .

Conditions

Puberty, Precocious

Keywords

Testotoxicosis; Familial Male-limited Precocious Puberty (FMPP)

Outcome Measures

Primary Outcomes (2)

• Change in Growth Rate (cm/Year)

  Change in growth rate after 12 months relative to the growth rate during the ≥6 month pre-study period, based on raw height data (cm/year).

  Assessed after 12 months treatment

• Change in Growth Rate (SD Units)

  Change in growth rate after 12 months relative to the growth rate during the ≥6 month pre-study period, calculated after adjustment for the chronological age of the patient (expressed as a standard deviation \[SD\] score).

  Assessed after 12 months treatment

Secondary Outcomes (7)

• Change in Growth Rate (cm/Year)

  Assessed after 6 months treatment

• Change in Growth Rate (SD Units)

  Assessed after 6 months treatment

• Change in Bone Age Maturation Rate (cm/Year)

  Assessed after 6 and 12 months treatment

• Change in Bone Age to Chronological Age Ratio

  Assessed after 6 and 12 months of treatment

• Number of Patients With Height Between 5th and 95th Percentile

  Assessed after 3, 6, 9 and 12 months of treatment

Study Arms (1)

Bicalutamide with Anastrozole

OTHER

Bicalutamide in combination with Anastrozole

Drug: Bicalutamide; Drug: Anastrozole

Interventions

Bicalutamide DRUG

oral

Also known as: Casodex

Bicalutamide with Anastrozole

Anastrozole DRUG

oral

Also known as: Arimidex, ZD1033

Bicalutamide with Anastrozole

Eligibility Criteria

• Age: 2 Years - 13 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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 "\<10 IU/L" which in the clinical setting equate to 'undetectable'.
• Normal levels of 17-hydroxyprogesterone (17-OHP)
• Normal levels of dehydroepiandrosterone sulphate (DHEAS)
• Naive to anti androgen receptor therapy:
• (Note: Ketoconazole and Spironolactone are considered acceptable as is prior use of anastrozole or other aromatase inhibitors)
• A documented reliable height measurement taken \> 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• AstraZeneca: lead

Study Sites (15)

Research Site

Birmingham, Alabama, 35233, United States

Location

Research Site

Jacksonville, Florida, 32207, United States

Location

Research Site

Indianapolis, Indiana, 46202, United States

Location

Research Site

Minneapolis, Minnesota, 55416, United States

Location

Research Site

Tulsa, Oklahoma, 74136, United States

Location

Research Site

Philadelphia, Pennsylvania, 19134, United States

Location

Research Site

Greenville, South Carolina, 29615, United States

Location

Research Site

Temple, Texas, 76508, United States

Location

Research Site

Spokane, Washington, 99204, United States

Location

Research Site

London, Ontario, N6A 4G5, Canada

Location

Research Site

Montpellier, 34295, France

Location

Research Site

Chennai, 600020, India

Location

Research Site

New Dehli, 110029, India

Location

Research Site

Moscow, 117036, Russia

Location

Research Site

London, WC1N 3JH, United Kingdom

Location

Related Links

• CSR-D6873C00047.pdf

MeSH Terms

Conditions

Puberty, Precocious; Familial Testotoxicosis

Interventions

bicalutamide; Anastrozole

Condition Hierarchy (Ancestors)

Gonadal Disorders; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Nitriles; Organic Chemicals; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Gerard Lynch
• Organization: AstraZeneca

AZTrial_Results_Posting@astrazeneca.com

Study Officials

• Yuri E Rukazenkov, MD

  AstraZeneca

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 16, 2004
• First Posted: October 18, 2004
• Study Start: November 22, 2004
• Primary Completion: May 22, 2008
• Study Completion: December 6, 2017
• Last Updated: June 26, 2018
• Results First Posted: July 9, 2009

Record last verified: 2018-06

Locations

FR (1); US (9); IN (2); GB (1); RU (1); CA (1)