CLINICAL TRIAL

Fulvestrant for Sexual Precocity

Waitlist Available · < 18 · Female · Barcelona, Spain

This study is evaluating whether a drug called Faslodex (fulvestrant) can be used to treat early puberty in girls with McCune-Albright syndrome.

See full description

About the trial for Sexual Precocity

Eligible Conditions
Puberty, Precocious · Fibrous Dysplasia, Polyostotic · Syndrome · McCune Albright Syndrome

Treatment Groups

This trial involves 2 different treatments. Fulvestrant is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Fulvestrant
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Fulvestrant
FDA approved

Side Effect Profile for Anastrozole

Anastrozole
Show all side effects
13%
Fatigue
11%
Nausea
11%
Arthralgia
8%
Musculoskeletal pain
7%
Hot flush
6%
Back pain
5%
Cough
5%
Diarrhoea
5%
Constipation
4%
Vomiting
4%
Dyspnoea
3%
Urinary tract infection
1%
Humerus Fracture
1%
Pericardial Effusion
1%
Urinary Tract Infection
1%
Muscular Weakness
1%
Dizziness
1%
Chest Pain
1%
Hypercalcaemia
1%
Vertigo
1%
Pneumonia
1%
General Physical Health Deterioration
1%
Pain
0%
Pulmonary Embolism
0%
Angina Pectoris
0%
Head Injury
0%
Myocardial Infarction
0%
Ventricular Arrhythmia
0%
Hip Fracture
0%
Spinal Cord Compression
0%
Urticaria
0%
Pyrexia
0%
Hyperglycaemia
0%
Meningioma
0%
Syncope
0%
Cerebral Infarction
0%
Pleural Effusion
0%
Confusional State
0%
Pneumonitis
0%
Thrombosis
0%
Pain In Extremity
0%
Atrial Flutter
0%
Foot Amputation
0%
Spinal Column Stenosis
0%
Sepsis
0%
Basal Cell Carcinoma
0%
Cardiomyopathy
0%
Angioedema
0%
Gangrene
0%
Peripheral Motor Neuropathy
0%
Depression
0%
Schizophrenia
0%
Bradycardia
0%
Cardiac Failure
0%
Bronchpneumonia
0%
Bronchitis
0%
Abdominal Pain Upper
0%
Viral Infection
0%
Subdural Haematoma
0%
Blood Creatinine Increased
0%
Fractured Sacrum
0%
Hypoglycaemia
0%
Erysipelas
0%
Joint Dislocation
0%
Back Pain
0%
Cerebellar Infarction
0%
Syncope Vasovagal
0%
Pleuritic Pain
0%
Respiratory Failure
0%
Anaemia
0%
Oedema Peripheral
0%
Infection
0%
Musculoskeletal Pain
0%
Gastroenteritis
0%
Femur Fracture
0%
Dehydration
0%
Pelvic Fracture
0%
Osteoarthritis
0%
Rhabdomyolysis
0%
Osteonecrosis
0%
Intracranial Haematoma
0%
Ovarian Cancer
0%
Metastases To Meninges
0%
Headache
0%
Peripheral Sensory Neuropathy
0%
Renal Failure
0%
Venous Stasis
0%
Arrhythmia
0%
Atrial Fibrillation
0%
Cardiac Arrest
0%
Ileus
0%
Pancreatitis
0%
Fall
0%
Femoral Neck Fracture
Fatigue
13%
Nausea
11%
Arthralgia
11%
Musculoskeletal pain
8%
Hot flush
7%
Back pain
6%
Cough
5%
Diarrhoea
5%
Constipation
5%
Vomiting
4%
Dyspnoea
4%
Urinary tract infection
3%
Humerus Fracture
1%
Pericardial Effusion
1%
Urinary Tract Infection
1%
Muscular Weakness
1%
Dizziness
1%
Chest Pain
1%
Hypercalcaemia
1%
Vertigo
1%
Pneumonia
1%
General Physical Health Deterioration
1%
Pain
1%
Pulmonary Embolism
0%
Angina Pectoris
0%
Head Injury
0%
Myocardial Infarction
0%
Ventricular Arrhythmia
0%
Hip Fracture
0%
Spinal Cord Compression
0%
Urticaria
0%
Pyrexia
0%
Hyperglycaemia
0%
Meningioma
0%
Syncope
0%
Cerebral Infarction
0%
Pleural Effusion
0%
Confusional State
0%
Pneumonitis
0%
Thrombosis
0%
Pain In Extremity
0%
Atrial Flutter
0%
Foot Amputation
0%
Spinal Column Stenosis
0%
Sepsis
0%
Basal Cell Carcinoma
0%
Cardiomyopathy
0%
Angioedema
0%
Gangrene
0%
Peripheral Motor Neuropathy
0%
Depression
0%
Schizophrenia
0%
Bradycardia
0%
Cardiac Failure
0%
Bronchpneumonia
0%
Bronchitis
0%
Abdominal Pain Upper
0%
Viral Infection
0%
Subdural Haematoma
0%
Blood Creatinine Increased
0%
Fractured Sacrum
0%
Hypoglycaemia
0%
Erysipelas
0%
Joint Dislocation
0%
Back Pain
0%
Cerebellar Infarction
0%
Syncope Vasovagal
0%
Pleuritic Pain
0%
Respiratory Failure
0%
Anaemia
0%
Oedema Peripheral
0%
Infection
0%
Musculoskeletal Pain
0%
Gastroenteritis
0%
Femur Fracture
0%
Dehydration
0%
Pelvic Fracture
0%
Osteoarthritis
0%
Rhabdomyolysis
0%
Osteonecrosis
0%
Intracranial Haematoma
0%
Ovarian Cancer
0%
Metastases To Meninges
0%
Headache
0%
Peripheral Sensory Neuropathy
0%
Renal Failure
0%
Venous Stasis
0%
Arrhythmia
0%
Atrial Fibrillation
0%
Cardiac Arrest
0%
Ileus
0%
Pancreatitis
0%
Fall
0%
Femoral Neck Fracture
0%
This histogram enumerates side effects from a completed 2012 Phase 3 trial (NCT00256698) in the Anastrozole ARM group. Side effects include: Fatigue with 13%, Nausea with 11%, Arthralgia with 11%, Musculoskeletal pain with 8%, Hot flush with 7%.

Eligibility

This trial is for female patients aged 18 and younger. There are 3 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Females less than or equal to 10 years of age (prior to 11th birthday)
Diagnosis of McCune-Albright syndrome (MAS)
Progressive precocious puberty (PPP) associated with MAS
View All
Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial

Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 6 month pre-treatment observation period (result at Month 0 considered as baseline) followed by 12 month treatment period (on treatment period).
Screening: ~3 weeks
Treatment: Varies
Reporting: 6 month pre-treatment observation period (result at Month 0 considered as baseline) followed by 12 month treatment period (on treatment period).
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 6 month pre-treatment observation period (result at Month 0 considered as baseline) followed by 12 month treatment period (on treatment period)..
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Fulvestrant will improve 1 primary outcome and 29 secondary outcomes in patients with Sexual Precocity. Measurement will happen over the course of Screening assessment (baseline).

Percentage of Patients With Gsα Mutation.
SCREENING ASSESSMENT (BASELINE)
McCune-Albright Syndrome(MAS) is caused by an activating mutation in the gene coding for the stimulatory subunit of the G protein, Gsα. The altered Gsα causes autonomous activation of G-protein stimulated cAMP formation, which in the gonads, results in episodic uncontrolled sex steroid production and subsequent pubertal development. For patients who provided separate specific informed consent, the percentage of patients with a Gsα mutation at screening was assessed by molecular analysis.
SCREENING ASSESSMENT (BASELINE)
Change in Uterine Volume From Baseline to Month 6 by Ultrasound.
SCREENING VISIT (BASELINE) AND MONTH 6 DURING THE TREATMENT PERIOD.
Uterine volume was calculated via ultrasound using the formula: 0.5(longitudinal multiplied by anteroposterior multiplied by transverse), if all 3 linear dimensions were recorded. If all 3 linear dimensions were not recorded, uterine volume was not calculated.
SCREENING VISIT (BASELINE) AND MONTH 6 DURING THE TREATMENT PERIOD.
Change in Bone Age Advancement Over the Second 6 Month Trial Period.
BASELINE TO SECOND 6 MONTHS OF THE TREATMENT PERIOD.
Change in the rate of increase in bone age from pre treatment (based on the 6 month retrospective visit) to the second 6 months of the treatment period. Bone age advancement for a particular time period was calculated as the increase in bone age over that time period adjusted (ie, normalized) for the length of that time period.
BASELINE TO SECOND 6 MONTHS OF THE TREATMENT PERIOD.
Change in Bone Age Advancement Over the First 6 Month Trial Period.
BASELINE TO FIRST 6 MONTHS OF THE TREATMENT PERIOD
Change in the rate of increase in bone age from pre treatment (based on the 6 month retrospective visit) to the first 6 months of the treatment period. Bone age advancement for a particular time period was calculated as the increase in bone age over that time period adjusted (ie, normalized) for the length of that time period.
BASELINE TO FIRST 6 MONTHS OF THE TREATMENT PERIOD
Change in Uterine Volume From Baseline to Month 12 by Ultrasound.
SCREENING VISIT (BASELINE) AND MONTH 12 DURING THE TREATMENT PERIOD.
Uterine volume was calculated via ultrasound using the formula: 0.5(longitudinal multiplied by anteroposterior multiplied by transverse), if all 3 linear dimensions were recorded. If all 3 linear dimensions were not recorded, uterine volume was not calculated.
SCREENING VISIT (BASELINE) AND MONTH 12 DURING THE TREATMENT PERIOD.
PK: Mean Volume of Distribution (V2/F) .
THROUGHOUT THE 12 MONTH TREATMENT PERIOD.
Total apparent volume of distribution (Vss/F) is the total apparent volume in the body into which Fulvestrant distributes at equilibrium. Vss/F = V1/F + V2/F. V1/F is the volume of the 1st compartment and V2/F is the volume of the second compartment. V2/F only is presented here. The measure of variability presented is the inter-individual error.
THROUGHOUT THE 12 MONTH TREATMENT PERIOD.
See More

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the signs of sexual precocity?

The clinical evaluation of prepubertal boys and girls for evidence of early sexual precocity is an important adjunct to the clinical evaluation of these children with a new diagnosis of early-onset behavior disorder.

Anonymous Patient Answer

What causes sexual precocity?

The cause of the precocious behavior and the pre-pubertal changes may be associated, at least in some patients, with the endocrine/gonadal imbalance. We found that the level of testosterone is significantly reduced in pre-pubertal boys with precocious behavior compared to non-precocious boys, and levels of estradiol are also significantly reduced in the precocious patients, with regard to age-matched controls. It is possible that the endocrine/gonadal imbalance induced by precocious behavior may be a causally related effect as an explanation for development of the behavior.

Anonymous Patient Answer

What are common treatments for sexual precocity?

In a survey conducted by this center, there was surprisingly little consensus between the various medical providers regarding the indications for and contraindications for psychosocial and/or educational intervention by the child psychiatrist. Results from a recent clinical trial are not at all surprising in the light of the absence of a standardized, evidence-based approach to the treatment of sexual precocity, and the fact that the approach depends largely on the personal preferences of the treating physician. Further studies are needed to confirm the usefulness of the current recommendations.

Anonymous Patient Answer

Can sexual precocity be cured?

Even if pre-pubertal male patients with sexual precocity can be satisfied, their sexual function is not good. If sexual precocity cannot be improved, it may lead to dissatisfaction for the patients.

Anonymous Patient Answer

What is sexual precocity?

The relationship between child sexual abuse and early puberty may be the result of other contributory variables. The increased sexual precocity exhibited by abused boys may be secondary to the psychological trauma associated with sexual abuse rather than a direct causal link.

Anonymous Patient Answer

How many people get sexual precocity a year in the United States?

Each year, over 1 in 10 males and 2 in 10 females will have sex before age 14. This has important ramifications as it has been shown that early sexual activity can lead to higher levels of psychopathology.

Anonymous Patient Answer

Have there been other clinical trials involving fulvestrant?

There is evidence of clinical activity of fulvestrant on the basis of two small studies. Further trials evaluating the usefulness and safety of fulvestrant are ongoing and are being reported in the literature.

Anonymous Patient Answer

Is fulvestrant typically used in combination with any other treatments?

Results from a recent paper suggests that if a patient has been diagnosed with early stage endometrioid adenocarcinoma of the endometrium and has not been treated with any therapy before, then a combination of fulvestrant is more effective for down-staging the disease if used in combination with adjuvant radiotherapy.

Anonymous Patient Answer

Who should consider clinical trials for sexual precocity?

Current information in the literature suggests that clinical trial participation in a sexual precocity treatment strategy may reduce premature sexual activity when there is a significant need for intervention.

Anonymous Patient Answer

Has fulvestrant proven to be more effective than a placebo?

Fulvestrant demonstrated no superiority or inferiority in secondary or primary end points compared to a placebo or a placebo plus low-dose methotrexate in a randomized controlled trial for children and adolescents at risks for endocrine induced growth and puberty.

Anonymous Patient Answer

Have there been any new discoveries for treating sexual precocity?

There has been a recent interest in using hormonal therapy for treating early onset sexual precocity. We haven't found conclusive evidence from high-quality clinical trials, but there is evidence of better quality from small, uncontrolled studies. There have been some randomized placebo controlled trials so these treatments can be further researched in terms of safety and efficacy. There are also potential treatments that are non-pharmaceutical based that offer promise, but not yet have been scientifically researched. This includes nutritional therapy and treatment of sexual precocity using a combination of hormones. The studies on these treatments have been inconclusive, but there is evidence of better quality from some small, uncontrolled studies.

Anonymous Patient Answer

What is the primary cause of sexual precocity?

The major risk factor for sexual precocity is familial precocity. Other risk factors included having been a victim of a sexual crime (especially with a woman), having been sexually abused, having had a history of alcohol use or prostitution, and having been a victim of incest. The second-leading risk factor is socioeconomic factors, especially of having been the victim of maltreatment in childhood. These risk factors are amenable to prevention interventions.

Anonymous Patient Answer
See if you qualify for this trial
Get access to this novel treatment for Sexual Precocity by sharing your contact details with the study coordinator.