Puberty, PrecociousPhase 2BATTNCT00094328
Pediatrics Testotoxicosis Study [Bicalutamide Anastrozole Treatment for Testotoxicosis]
A Phase 2 study testing Bicalutamide and Anastrozole in people with Puberty, Precocious, run by AstraZeneca at 15 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 13 years, men only
- Conditions the study is for
- Puberty, Precocious
- What is being tested?
- Bicalutamide and Anastrozole
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 15 sitesin 6 countries
- Study dates
- 22 Nov 2004to 6 Dec 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 13 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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
Show all 17Show fewer
- 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.
- (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.
Who cannot join
- 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
Trial locations and contacts
15 sites in 6 countries.
15 sites in 6 countries
United States9 sites
Research Site
Birmingham, Alabama, 35233
Research Site
Greenville, South Carolina, 29615
Research Site
Indianapolis, Indiana, 46202
Research Site
Jacksonville, Florida, 32207
Research Site
Minneapolis, Minnesota, 55416
Research Site
Philadelphia, Pennsylvania, 19134
Research Site
Spokane, Washington, 99204
Research Site
Temple, Texas, 76508
Research Site
Tulsa, Oklahoma, 74136
About this study
Official title: An Open-label, Non-comparative, Multi-centre Study to Assess the Efficacy and Safety of Bicalutamide When Used in Combination With Anastrozole for the Treatment of Gonadotropin-independent Precocious Puberty in Boys With Testotoxicosis
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
- Being tested
- Bicalutamide, Anastrozole
- Keywords
- Testotoxicosis, Familial Male-limited Precocious Puberty (FMPP)
- Medical subject headings
- Puberty, Precocious, Familial Testotoxicosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BicalutamideDrug
Also called Casodex
oral
AnastrozoleDrug
Also called Arimidex, ZD1033
oral
| Study group | Role | What they receive |
|---|---|---|
| Bicalutamide with Anastrozole | Other | Bicalutamide in combination with Anastrozole Receives: Drug: Bicalutamide; Drug: Anastrozole |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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).
Measured: Assessed after 12 months treatment
Secondary outcomes (7)
Change in Growth Rate (cm/Year)
Change in growth rate after 6 months of treatment relative to the growth rate during the ≥6 months pre-study period.
Measured: Assessed after 6 months treatment
Change in Growth Rate (SD Units)
Change in growth rate after 6 months of treatment relative to the growth rate during the ≥6 months pre-study period.
Measured: Assessed after 6 months treatment
Change in Bone Age Maturation Rate (cm/Year)
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.
Measured: Assessed after 6 and 12 months treatment
Change in Bone Age to Chronological Age Ratio
Change in bone age to chronological age ratio after 6 and 12 months treatment relative to the baseline ratio for all patients.
Measured: Assessed after 6 and 12 months of treatment
Number of Patients With Height Between 5th and 95th Percentile
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.
Measured: Assessed after 3, 6, 9 and 12 months of treatment
Change in Predicted Adult Height (PAH)
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.
Measured: Assessed after 12 months treatment
Change in Average Testicular Volume
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.
Measured: Assessed after 6 and 12 months of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2004 (estimated)
- Study start22 Nov 2004
- Primary completion22 May 2008
- Study completion6 Dec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Puberty, PrecociousStudies of the same condition, open and under way.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yuri E Rukazenkov, MD, Study director, AstraZeneca
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00094328
- Sponsor's protocol ID
- D6873C00047
- Other IDs
- BATT
- Acronym
- BATT
- Status last verified
- Jun 2018
- First submitted
- 16 Oct 2004
- Last updated
- 26 Jun 2018
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Non-comparative, Multi-centre Study to Assess the Efficacy and Safety of Bicalutamide When Used in Combination With Anastrozole for the Treatment of Gonadotropin-independent Precocious Puberty in Boys With Testotoxicosis
- ClinicalTrials.gov ID
- NCT00094328
- Acronym / sponsor ID
- BATT / D6873C00047
- Conditions
- Puberty, Precocious
- Keywords
- Testotoxicosis, Familial Male-limited Precocious Puberty (FMPP)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- 22 Nov 2004 (actual)
- Primary completion
- 22 May 2008 (actual)
- Study completion
- 6 Dec 2017 (actual)
- First posted
- 18 Oct 2004 (estimated)
- Results
- Posted 9 Jul 2009
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 .
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Bicalutamide with Anastrozole | Other | Drug: Bicalutamide, Drug: AnastrozoleBicalutamide in combination with Anastrozole |
Who may qualify
- Ages
- 2 to 13 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (17 listed)
- 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)
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BicalutamideAlso: Casodexoral | Drug | Bicalutamide with Anastrozole |
| AnastrozoleAlso: Arimidex, ZD1033oral | Drug | Bicalutamide with Anastrozole |
Primary outcome
Change in Growth Rate (cm/Year)
Time frame: Assessed after 12 months treatment
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).
Change in Growth Rate (SD Units)
Time frame: Assessed after 12 months treatment
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).
7 secondary outcomes
Change in Growth Rate (cm/Year)
Time frame: Assessed after 6 months treatment
Change in Growth Rate (SD Units)
Time frame: Assessed after 6 months treatment
Change in Bone Age Maturation Rate (cm/Year)
Time frame: Assessed after 6 and 12 months treatment
Change in Bone Age to Chronological Age Ratio
Time frame: Assessed after 6 and 12 months of treatment
Number of Patients With Height Between 5th and 95th Percentile
Time frame: Assessed after 3, 6, 9 and 12 months of treatment
Change in Predicted Adult Height (PAH)
Time frame: Assessed after 12 months treatment
Change in Average Testicular Volume
Time frame: Assessed after 6 and 12 months of treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- CSR-D6873C00047.pdfLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yuri E Rukazenkov, MD study director
AstraZeneca
15 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Bicalutamide and Anastrozole in people with Puberty, Precocious, run by AstraZeneca at 15 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Jun 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, India, Canada, France, Russia and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Jul 2009). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Growth Rate (cm/Year) (time frame: Assessed after 12 months treatment). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jun 2018.