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Trial record 2 of 3 for:    "Precocious Puberty" | "Steroid Synthesis Inhibitors"

Pediatrics Testotoxicosis Study [Bicalutamide Anastrozole Treatment for Testotoxicosis] (BATT)

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ClinicalTrials.gov Identifier: NCT00094328
Recruitment Status : Completed
First Posted : October 18, 2004
Results First Posted : July 9, 2009
Last Update Posted : June 26, 2018
Sponsor:
Information provided by (Responsible Party):
AstraZeneca

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Puberty, Precocious
Interventions Drug: Bicalutamide
Drug: Anastrozole
Enrollment 14
Recruitment Details The first patient was enrolled on 22 November 2004 and the last patient completed the 12 months visit on 7 May 2008. Patients were allocated treatment at 9 centres in 3 countries: India, the UK and the USA. Care for two patients, transferred from one US to a new approved US centre, therefore, patients were treated at 10 centres in total
Pre-assignment Details Of the 24 patients enrolled, 10 failed eligibility criteria and were classed as screening failures while the remaining 14 patients were allocated treatment.
Arm/Group Title Open Label Bicalutamide With Anastrozole
Hide Arm/Group Description Patients with testotoxicosis (familial male-limited precocious puberty) were given study drugs (bicalutamide in combination with anastrozole) orally once-daily for 12 months. The dosing of bicalutamide and anastrozole was independently tailored for each patient.
Period Title: Overall Study
Started 14 [1]
Completed 13 [2]
Not Completed 1
Reason Not Completed
Lost to Follow-up             1
[1]
Received treatment
[2]
Completed 12 months on treatment
Arm/Group Title Open Label Bicalutamide With Anastrozole
Hide Arm/Group Description Patients with testotoxicosis (familial male-limited precocious puberty) were given study drugs (bicalutamide in combination with anastrozole) orally once-daily for 12 months. The dosing of bicalutamide and anastrozole was independently tailored for each patient.
Overall Number of Baseline Participants 14
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 14 participants
3.5
(2 to 9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants
Female
0
   0.0%
Male
14
 100.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Race Number Analyzed 14 participants
Caucasian
12
  85.7%
Black
1
   7.1%
Other
1
   7.1%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Ethnic group Number Analyzed 14 participants
Hispanic/Latino
1
   7.1%
African/American
1
   7.1%
Asian
3
  21.4%
Not Applicable
9
  64.3%
1.Primary Outcome
Title Change in Growth Rate (cm/Year)
Hide Description 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).
Time Frame Assessed after 12 months treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All treated (AT) set
Arm/Group Title Open Label Bicalutamide With Anastrozole
Hide Arm/Group Description:
Patients with testotoxicosis (familial male-limited precocious puberty) were given study drugs (bicalutamide in combination with anastrozole) orally once-daily for 12 months. The dosing of bicalutamide and anastrozole was independently tailored for each patient.
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: cm/year
-1.62  (5.13)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Open Label Bicalutamide With Anastrozole
Comments The primary efficacy parameter, change in growth rate (cm/year) after 12 months relative to the baseline growth rate was analysed using a one sample t-test. A 95% 2-sided confidence interval was calculated for the mean change in growth rate.
Type of Statistical Test Other
Comments One sample t-test
Statistical Test of Hypothesis P-Value 0.278
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -1.62
Confidence Interval (2-Sided) 95%
-4.72 to 1.48
Estimation Comments [Not Specified]
2.Primary Outcome
Title Change in Growth Rate (SD Units)
Hide Description 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).
Time Frame Assessed after 12 months treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All treated (AT) set
Arm/Group Title Open Label Bicalutamide With Anastrozole
Hide Arm/Group Description:
Patients with testotoxicosis (familial male-limited precocious puberty) were given study drugs (bicalutamide in combination with anastrozole) orally once-daily for 12 months. The dosing of bicalutamide and anastrozole was independently tailored for each patient.
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: SD units
-0.07  (1.78)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Open Label Bicalutamide With Anastrozole
Comments The primary efficacy parameter, change in growth rate (SD units) after 12 months relative to the baseline growth rate was analysed using a one sample t-test. A 95% 2-sided confidence interval was calculated for the mean change in growth rate.
Type of Statistical Test Other
Comments One sample t-test
Statistical Test of Hypothesis P-Value 0.882
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Median Difference (Final Values)
Estimated Value -0.07
Confidence Interval (2-Sided) 95%
-1.15 to 1.00
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Change in Growth Rate (cm/Year)
Hide Description Change in growth rate after 6 months of treatment relative to the growth rate during the ≥6 months pre-study period.
Time Frame Assessed after 6 months treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All treated (AT) set
Arm/Group Title Open Label Bicalutamide With Anastrozole
Hide Arm/Group Description:
Patients with testotoxicosis (familial male-limited precocious puberty) were given study drugs (bicalutamide in combination with anastrozole) orally once-daily for 12 months. The dosing of bicalutamide and anastrozole was independently tailored for each patient.
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: cm/year
-0.70  (5.77)
4.Secondary Outcome
Title Change in Growth Rate (SD Units)
Hide Description Change in growth rate after 6 months of treatment relative to the growth rate during the ≥6 months pre-study period.
Time Frame Assessed after 6 months treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All treated (AT) set
Arm/Group Title Open Label Bicalutamide With Anastrozole
Hide Arm/Group Description:
Patients with testotoxicosis (familial male-limited precocious puberty) were given study drugs (bicalutamide in combination with anastrozole) orally once-daily for 12 months. The dosing of bicalutamide and anastrozole was independently tailored for each patient.
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: SD units
-0.14  (1.67)
5.Secondary Outcome
Title Change in Bone Age Maturation Rate (cm/Year)
Hide Description Radiographs were used to assess the bone age at ≥6 months pre-study, baseline, 6 and 12 months. The rate of change in bone age at baseline was calculated from a radiograph taken at least 6 months prior to study enrolment. The change in bone maturation after 6 months of treatment was calculated relative to the rate of change in bone age during the ≥ 6 months pre-study period.
Time Frame Assessed after 6 and 12 months treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Calculated on All treated analysis set for those patients who had a 6-month pre study radiograph.
Arm/Group Title Open Label Bicalutamide With Anastrozole
Hide Arm/Group Description:
Patients with testotoxicosis (familial male-limited precocious puberty) were given study drugs (bicalutamide in combination with anastrozole) orally once-daily for 12 months. The dosing of bicalutamide and anastrozole was independently tailored for each patient.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: cm/year
After 6 months treatment -2.03  (0.38)
After 12 months treatment -2.29  (0.51)
6.Secondary Outcome
Title Change in Bone Age to Chronological Age Ratio
Hide Description Change in bone age to chronological age ratio after 6 and 12 months treatment relative to the baseline ratio for all patients.
Time Frame Assessed after 6 and 12 months of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All treated (AT) set
Arm/Group Title Open Label Bicalutamide With Anastrozole
Hide Arm/Group Description:
Patients with testotoxicosis (familial male-limited precocious puberty) were given study drugs (bicalutamide in combination with anastrozole) orally once-daily for 12 months. The dosing of bicalutamide and anastrozole was independently tailored for each patient.
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: Ratio
After 6 months treatment -0.09  (0.14)
After 12 months treatment -0.24  (0.18)
7.Secondary Outcome
Title Number of Patients With Height Between 5th and 95th Percentile
Hide Description The number of patients whose height lies between the 5th and 95th percentiles (using the percentile tables on the WHO database) for chronological age at the 12 month assessment.
Time Frame Assessed after 3, 6, 9 and 12 months of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All treated (AT) set
Arm/Group Title Open Label Bicalutamide With Anastrozole
Hide Arm/Group Description:
Patients with testotoxicosis (familial male-limited precocious puberty) were given study drugs (bicalutamide in combination with anastrozole) orally once-daily for 12 months. The dosing of bicalutamide and anastrozole was independently tailored for each patient.
Overall Number of Participants Analyzed 13
Measure Type: Number
Unit of Measure: Participants
After 3 months treatment 3
After 6 months treatment 3
After 9 months treatment 3
After 12 months treatment 3
8.Secondary Outcome
Title Change in Predicted Adult Height (PAH)
Hide Description Radiographs are used to assess the bone age, the change in predicted adult height (PAH) is calculated from the bone age using the Bayley and Pinneau Method. The change in PAH is be calculated by subtracting the PAH at baseline from the PAH at 12 months.
Time Frame Assessed after 12 months treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Calculated on All treated analysis set, however, if bone age is less than 6 years or bone age is less than 7 years and bone age>=(chronological age-1) then PAH cannot be calculated using the Bayley and Pinneau method.
Arm/Group Title Open Label Bicalutamide With Anastrozole
Hide Arm/Group Description:
Patients with testotoxicosis (familial male-limited precocious puberty) were given study drugs (bicalutamide in combination with anastrozole) orally once-daily for 12 months. The dosing of bicalutamide and anastrozole was independently tailored for each patient.
Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: cm
6.21  (3.93)
9.Secondary Outcome
Title Change in Average Testicular Volume
Hide Description Testicular volume of both testes was measured using either ultrasound or an orchidometer. Testicular volume was measured at baseline and at 6 and 12 months. The change in testicular volume from baseline was calculated for the left and right testicle as well as the average across both testes by subtracting the baseline volume from the volumes at 6 and 12 months within each patient.
Time Frame Assessed after 6 and 12 months of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All treated (AT) set
Arm/Group Title Open Label Bicalutamide With Anastrozole
Hide Arm/Group Description:
Patients with testotoxicosis (familial male-limited precocious puberty) were given study drugs (bicalutamide in combination with anastrozole) orally once-daily for 12 months. The dosing of bicalutamide and anastrozole was independently tailored for each patient.
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: mL
After 6 months treatment 1.46  (2.29)
After 12 months treatment 2.69  (2.51)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Open Label Bicalutamide With Anastrozole
Hide Arm/Group Description Patients with testotoxicosis (familial male-limited precocious puberty) were given study drugs (bicalutamide in combination with anastrozole) orally once-daily for 12 months. The dosing of bicalutamide and anastrozole was independently tailored for each patient.
All-Cause Mortality
Open Label Bicalutamide With Anastrozole
Affected / at Risk (%)
Total   0/14 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Open Label Bicalutamide With Anastrozole
Affected / at Risk (%)
Total   0/14 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Open Label Bicalutamide With Anastrozole
Affected / at Risk (%)
Total   13/14 (92.86%) 
Blood and lymphatic system disorders   
Eosinophilia  1  1/14 (7.14%) 
Lymphadenopathy  1  1/14 (7.14%) 
Microcytosis  1  1/14 (7.14%) 
Endocrine disorders   
Precocious Puberty  1  6/14 (42.86%) 
Eye disorders   
Conjunctivitis  1  2/14 (14.29%) 
Gastrointestinal disorders   
Abdominal Pain  1  2/14 (14.29%) 
Abdominal Pain Upper  1  1/14 (7.14%) 
Diarrhoea  1  1/14 (7.14%) 
Nausea  1  2/14 (14.29%) 
Vomiting  1  5/14 (35.71%) 
General disorders   
Asthenia  1  1/14 (7.14%) 
Fatigue  1  1/14 (7.14%) 
Pyrexia  1  3/14 (21.43%) 
Immune system disorders   
Seasonal Allergy  1  1/14 (7.14%) 
Infections and infestations   
Croup Infectious  1  2/14 (14.29%) 
Ear Infection  1  1/14 (7.14%) 
Furuncle  1  1/14 (7.14%) 
Gastroenteritis  1  2/14 (14.29%) 
Labyrinthitis  1  1/14 (7.14%) 
Lower Respiratory Tract Infection  1  1/14 (7.14%) 
Nasopharyngitis  1  2/14 (14.29%) 
Otitis Externa  1  1/14 (7.14%) 
Pyoderma  1  1/14 (7.14%) 
Respiratory Tract Infection Viral  1  1/14 (7.14%) 
Rhinitis  1  1/14 (7.14%) 
Sinusitis  1  1/14 (7.14%) 
Staphylococcal Abscess  1  1/14 (7.14%) 
Tonsillitis  1  2/14 (14.29%) 
Upper Respiratory Tract Infection  1  2/14 (14.29%) 
Varicella  1  1/14 (7.14%) 
Viral Infection  1  1/14 (7.14%) 
Injury, poisoning and procedural complications   
Fall  1  1/14 (7.14%) 
Skin Laceration  1  1/14 (7.14%) 
Sunburn  1  1/14 (7.14%) 
Investigations   
Alanine Aminotransferase Increased  1  1/14 (7.14%) 
Aspartate Aminotransferase Increased  1  1/14 (7.14%) 
Metabolism and nutrition disorders   
Decreased Appetite  1  1/14 (7.14%) 
Musculoskeletal and connective tissue disorders   
Musculoskeletal Chest Pain  1  1/14 (7.14%) 
Nervous system disorders   
Headache  1  3/14 (21.43%) 
Psychiatric disorders   
Crying  1  1/14 (7.14%) 
Reproductive system and breast disorders   
Breast Pain  1  1/14 (7.14%) 
Breast Tenderness  1  2/14 (14.29%) 
Gynaecomastia  1  7/14 (50.00%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  1/14 (7.14%) 
Stridor  1  1/14 (7.14%) 
Skin and subcutaneous tissue disorders   
Acne  1  2/14 (14.29%) 
Cafe Au Lait Spots  1  1/14 (7.14%) 
Skin Hyperpigmentation  1  1/14 (7.14%) 
1
Term from vocabulary, MedDRA 10.0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The PI agrees to collaborate with AstraZeneca on the contents and formation of any publication or disclosure and to pay due consideration to comments and opinions offered. AstraZeneca have 60 days for final manuscript review and may require that submission for publication be delayed for a further 90 days in order to file patent applications.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Gerard Lynch
Organization: AstraZeneca
EMail: AZTrial_Results_Posting@astrazeneca.com
Layout table for additonal information
Responsible Party: AstraZeneca
ClinicalTrials.gov Identifier: NCT00094328     History of Changes
Other Study ID Numbers: D6873C00047
BATT
First Submitted: October 16, 2004
First Posted: October 18, 2004
Results First Submitted: May 19, 2009
Results First Posted: July 9, 2009
Last Update Posted: June 26, 2018