McCune-Albright SyndromePhase 2NCT00055302
Arimidex in McCune Albright Syndrome
A Phase 2 study testing Arimidex 1 mg in people with McCune-Albright Syndrome, run by AstraZeneca at 7 sites in 4 countries.
Trial summary
The primary objective of this study is to evaluate the safety and efficacy of anastrozole 1 mg given once daily in subjects with McCune-Albright Syndrome. Read more about this study
- Who is it for?
- Ages Up to 10 years, women only
- Conditions the study is for
- McCune-Albright Syndrome
- What is being tested?
- Arimidex 1 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 7 sitesin 4 countries
- Study dates
- Aug 2002to Aug 2015
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 Up to 10 years
- Women only
- No healthy volunteers
- Child
Who may be able to join
- informed written consent of parent/legal guardian and subject assent (as needed by local requirements)
- females less than or equal to 10 years of age
- diagnosed with McCune-Albright Syndrome
- have progressive precocious puberty
- Exclusion Criteria: Any one of the following is regarded as a criterion for exclusion from the study:
- any prior treatment of MAS associated with progressive precocious puberty with a third generation aromatase inhibitor (anastrozole, letrozole, exemestane) in which no clinical response was seen
Show all 9Show fewer
- concomitant treatment of precocious puberty associated with MAS, with the exception of bisphosphonates for polyostotic fibrous dysplasia and LHRH analogues in the case of central precocious puberty
- liver function tests at screening visit (AST, ALT) > or = 3x the upper limit of the reference range for age
- known hypersensitivity to any component of study medication
Who cannot join
None listed.
Trial locations and contacts
7 sites in 4 countries.
7 sites in 4 countries
Germany3 sites
Research Site
Berlin
Research Site
Erlangen
Research Site
Osnabrück
About this study
Official title: An Open-label Study Evaluating the Safety and Efficacy of Anastrozole™ (ARIMIDEX) in the Treatment of Precocious Puberty in Girls With McCune-Albright Syndrome
The primary objective of this study is to evaluate the safety and efficacy of anastrozole 1 mg given once daily in subjects with McCune-Albright Syndrome.
- Conditions
- McCune-Albright Syndrome
- Being tested
- Arimidex 1 mg
- Keywords
- vaginal bleeding, advanced bone age, fibrous dysplasia, MAS, McCune-Albright Syndrome
- Medical subject headings
- Fibrous Dysplasia, Polyostotic, Uterine Hemorrhage, Fibrous Dysplasia of Bone
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Arimidex 1 mgDrug
Arimidex (anastrozole) 1mg once daily by mouth
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Not described Receives: Drug: Arimidex 1 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
The efficacy of study treatment will be assessed based on the change from baseline measurements relating to vaginal bleeding, bone age, and growth velocity
Measured: 12 months or until the subject demonstrates the lack of efficacy bases upon progression of primary endpoints or experiences serious drug-related toxicity requiring withdrawal
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2003 (estimated)
- Study startAug 2002
- Primary completionFeb 2006
- Study completionAug 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for McCune-Albright SyndromeStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- AstraZeneca Arimidex Medical Science Director, MD, Study director, AstraZeneca
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00055302
- Sponsor's protocol ID
- 1033IL/0046
- Other IDs
- D5394C00046
- Status last verified
- Aug 2015
- First submitted
- 25 Feb 2003
- Last updated
- 1 Sep 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Study Evaluating the Safety and Efficacy of Anastrozole™ (ARIMIDEX) in the Treatment of Precocious Puberty in Girls With McCune-Albright Syndrome
- ClinicalTrials.gov ID
- NCT00055302
- Acronym / sponsor ID
- 1033IL/0046
- Conditions
- McCune-Albright Syndrome
- Keywords
- vaginal bleeding, advanced bone age, fibrous dysplasia, MAS, McCune-Albright Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- Aug 2002
- Primary completion
- Feb 2006 (actual)
- Study completion
- Aug 2015 (actual)
- First posted
- 26 Feb 2003 (estimated)
- Results
- Not posted in the source record
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 evaluate the safety and efficacy of anastrozole 1 mg given once daily in subjects with McCune-Albright Syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: Arimidex 1 mg |
Who may qualify
- Ages
- Up to 10 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- informed written consent of parent/legal guardian and subject assent (as needed by local requirements)
- females less than or equal to 10 years of age
- diagnosed with McCune-Albright Syndrome
- have progressive precocious puberty
- Exclusion Criteria: Any one of the following is regarded as a criterion for exclusion from the study:
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| Arimidex 1 mgArimidex (anastrozole) 1mg once daily by mouth | Drug | 1 |
Primary outcome
The efficacy of study treatment will be assessed based on the change from baseline measurements relating to vaginal bleeding, bone age, and growth velocity
Time frame: 12 months or until the subject demonstrates the lack of efficacy bases upon progression of primary endpoints or experiences serious drug-related toxicity requiring withdrawal
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2015.
- CSR-1033IL-0046.pdfLink provided by the sponsor.
Related trials for McCune-Albright Syndrome
RecruitingObservational study
Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome
McCune-Albright Syndrome
Not yet recruitingPhase 2
(Z)-Endoxifen in Girls With MAS-Associated Peripheral Precocious Puberty
Precocious Puberty, Gonadotropin-dependent
RecruitingObservational study
Fibrous Dysplasia: An Epidemiological and Correlational Evaluation of Multimodal Data
Fibrous Dysplasia, Fibrous Dysplasia of Bone
Active, not recruitingObservational study
Assessment of Femoral Failure Load and Fracture Risk in Rare Bone Disorders Using MEKANOS Tool. Case Study of Fibrous Dysplasia of Bone/McCune Albright Syndrome
Fibrous Dysplasia/McCune-Albright Syndrome
RecruitingObservational study
Fibrous Dysplasia, McCune-Albright Syndrome Patient Registry
Fibrous Dysplasia, McCune Albright Syndrome
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.
AstraZeneca Arimidex Medical Science Director, MD study director
AstraZeneca
7 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Arimidex 1 mg in people with McCune-Albright Syndrome, run by AstraZeneca at 7 sites in 4 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 9 inclusion and 0 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 1 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Germany, France, Italy, United Kingdom. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The efficacy of study treatment will be assessed based on the change from baseline measurements relating to vaginal bleeding, bone age, and growth velocity (time frame: 12 months or until the subject demonstrates the lack of efficacy bases upon progression of primary endpoints or experiences serious drug-related toxicity requiring withdrawal). 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 1 Sep 2015.