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Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome Clinical Trials Resources presented on Clinical Trials Search is not meant to be a substitute for proven health advice, calls or treatment with a real medical. We aren't mDs. Always consult your doctor on Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome conditions. Clinical Trials Search.org is a website dedicated to listing clinical research studies in human subjects. Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome Clinical research trials and Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome healthcare trials take place in a lot of of localities throughout the U.S.. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials typically assess the effectiveness of new does drugs. The function of the studies / projects is to figure out specific human medical questions. Clinical trials are a popular means for doctors, government agencies, and private sector corporations to find cures for all varieties of conditions, like Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome. Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome Clinical Trials and other clinical trials allow volunteers to access health treatment options before they are available to the masses. Many times the subjects receive professional assistance for free, and every now and again they are compensated for their time. Sometimes there is a cost for a Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome clinical trial. Human subjects often obtain the finest healthcare possible for their Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome condition. Hazards are a reality, nevertheless, and might include additional or frequent dr. calls, health hazards (potentially life-jeopardizing), and/or the treatment being uneffective. Trials are federally regulated with stern guidelines to protect clinical trials patients.
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Home > "E" Clinical Trials Conditions > Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome  Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome
Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome
For Condition: McCune Albright Syndrome,Polyostotic Fibrous Dysplasia,Precocious Puberty
Status: Recruiting
Sponsor(s): National Institute of Dental and Craniofacial Research (NIDCR) ,
Synopsis: This study will test the safety and effectiveness of letrozole in treating precocious (early) puberty in girls with McCune-Albright syndrome (MAS). The physical changes of puberty, such as breast enlargement, menstruation and growth spurt, as well as the emotional changes of this developmental stage, usually begin in girls between the ages of 8 and 14. Girls with MAS, however, often begin puberty before age 7. In MAS, large ovarian cysts produce high levels of estrogens (female hormones) that cause the changes of puberty. Children with MAS also have polyostotic fibrous dysplasia (PFD), a disease of bones that, depending on what parts of the skeleton are affected, can lead to broken bones or disfigurement of the head, face, arms and legs, or can cause pressure on nerves and blood vessels. Many children with MAS have cafe-au-lait spots (increased pigmentation) on areas of their skin as well. Letrozole is an estrogen-lowering drug that has been approved for treating women with breast and other cancers. Although the drug has not been tested or approved for use in children, some pediatric specialists have given it to girls with precocious puberty and MAS and found that it improves their condition without harmful side effects. This study will examine whether letrozole can lower estrogen in girls with MAS and arrest puberty. It will also study the drug's effects on substances involved in bone growth, including calcium, phosphate and amino acids. Girls 1 to 8 years old with MAS may be eligible for this study. Patients who were enrolled in NIH protocol 98-D-0145 (Screening and Natural History of Patients with Polyostotic Fibrous Dysplasia and the McCune-Albright syndrome) are also eligible. Participants will be admitted to the hospital for 2 to 3 days every 3 months for 15 months, for a total of 6 visits. They will undergo a complete history and physical examination and routine blood and urine tests every visit, as well as evaluations of their general health, growth and bone development, endocrine system (hormone-secreting glands) status and PFD status. A hand X-ray will be taken at the first visit and every 6 months to measure bone age advance. The children will begin taking letrozole at the second visit and continue the drug for 6 months. They will be evaluated after 3 months and 6 months on the drug (visits 3 and 4), and again after 3 months and 6 months after stopping treatment (visits 5 and 6). Parents of children who weigh more than 18 kilograms (about 40 pounds) may be asked if extra blood may be drawn after 3 months (visit 3) and 6 months (visit 4) of treatment to measure letrozole levels. The blood will be drawn before the morning dose and at 0.5, 1, 1.5, 2, 3, 4, 6, 8, and 24 hours after the dose through an indwelling needle placed in the vein for 8 to 24 hours. Parents will keep a record of all episodes of menstrual bleeding and any other symptoms or complaints. Children who respond well to therapy (decreased menses, slowed breast development, slowed growth and bone age advance) will be offered another 12 months of letrozole treatment.
Details: Girls ages 1 - 8 y with the McCune-Albright syndrome (MAS) and girls with other conditions characterized by precocious puberty due to estrogen hypersecretion from ovarian cysts will be eligible for this pilot study. Patients who have previously enrolled in Protocol 98-D-0145 (Screening and natural history of patients with polyostotic fibrous dysplasia and the McCune-Albright Syndrome) will also be eligible. Patients will be treated with letrozole, a potent, nonsteroidal aromatase inhibitor, to suppress their elevated serum estrogen levels. We will confirm the safety and efficacy of letrozole, and study its effectiveness in controlling the elevated sex steroid levels, and the advanced rates of linear growth, bone maturation, and pubertal progression in these patients. We will also study the effect of decreased estrogen levels on the status of their polyostotic fibrous dysplasia by measuring serum and urine values for bone biomarkers, including calcium, phosphate, organic amino acids, and vitamin D metabolites, which are known to be abnormal in many patients with MAS. Patients will act as their own controls. We will compare serum and urine parameters of pubertal progression and bone biomarkers before, during, and after discontinuation of letrozole. This trial will be carried out in parallel with in-vitro and in-vivo laboratory studies using an animal model of fibrous dysplasia. In this model, osteogenic precursor cells from patient bone biopsies will be cultured in a hydroxyapatite/tricalcium phosphate matrix and transplanted into immunocompromised mice. We anticipate that our laboratory findings will complement the care of our patients, resulting in more effective treatment for the precocious puberty and the bone disease in children with MAS. Because our initial studies have indicated that letrozole is effective in treating precocious puberty in MAS patients, this protocol also enrolls girls who have a related condition, gonadotropin-independent precocious puberty without the bone disease polyostotic fibrous dysplasia. We also believe that this study complements the recent FDA and NIH mandates that children be included in the evaluation of pharmaceutical products and in federally funded clinical research studies.
Eligibility:
Study Type: Interventional, Treatment, Safety
Minimum Age/Maximum Age: /
Genders: Female
Protocol Entry Criteria: INCLUSION CRITERIA Patients will be girls age 1-8 years with precocious puberty (menses, and/or breast development, and/or BA greater than plus 2SD [for chronologic age] and/or linear growth rate greater than plus 2SD) and polyostotic fibrous dysplasia due to MAS. All ethnic groups will be included. EXCLUSION CRITERIA Boys with MAS will be excluded. Patients with clinically significant hepatic and/or renal impairment will be excluded.
Total Enrollment: 25
Location and Contact Information:
National Institute of Dental And Craniofacial Research (NIDCR) *Recruiting*
Bethesda, Maryland, 20892
United States
Recruiting Penelope Feuillan 3015940309
Additional Information:
Study ID Numbers: 000183; 00-D-0183
Study Start Date: August 3, 2000
Record last reviewed: June 4, 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00006174
Other Precocious Puberty Studies:
1. Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome
2. Testolactone for the Treatment of Girls with LHRH Resistant Precocious Puberty
3. Study of Gonadotropin-Releasing Hormone Pulse Frequency in Sexual Maturation and in the Menstrual Cycle
4. Purification of Testis-Stimulating Factor in Precocious Puberty
5. Treatment of Boys with Precocious Puberty
Related Studies:
Other Precocious Puberty Clinical Trials
Other Maryland Clinical Trials
Other Bethesda Clinical Trials
Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome
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