Clinical trial NCT00660010: Study of Lupron Depot In The Treatment of Central Precocious Puberty
Official title: Study of Lupron Depot In The Treatment of Central Precocious Puberty
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Puberty, Precocious
Keywords: CPP
central precocious puberty
pediatrics
suppression of LH
Lupron
leuprolide acetate
depot formulation
GnRHa
GnRH agonist
GNRH analog
LH
Tanner staging
Lead sponsor: Abbott
Sponsor class: INDUSTRY
First posted: 2008-04-17
Enrollment: 55
Sex: ALL
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine if Lupron (leuprolide acetate) is safe and effective in treating children with Central Precocious Puberty (CPP), and to assess long term effects of leuprolide acetate treatment after therapy is discontinued.

Detailed description: This study includes a Prestudy Period; a treatment period where subjects will receive treatment every 4 weeks until the initiation of puberty (age 12 for males and age 11 for females); a follow-up period where subjects will be observed every 6 months until physical and laboratory observations are at pubertal levels, then every 12 months for 5 years; lastly a final follow-up questionnaire is given to all subjects when they are at least 18 years old.

At the treatment visits, efficacy assessments are Tanner staging (suppression of breast development in females and genital development in males), gonadotropins (LH and FSH), sex steroids (estradiol in females and testosterone in males), ratio of bone age to chronological age, adult height compared to a standard population and predicted mature height, and age and time to regular menses in females. This protocol will also capture data from the final questionnaire about female reproductive status at adulthood including the presence of regular menses and number of pregnancies.

Eligibility criteria: Inclusion Criteria:

* Clinical diagnosis of isosexual central precocious puberty with onset of Tanner scores of Stage II for breast or pubic hair earlier than age 8.0 years in girls or Stage II for pubic hair or genitalia earlier than 9.0 years in boys.
* Confirmation of diagnosis by a pubertal response to a gonadotropin-releasing hormone (GnRH) stimulation test (LH \> 10 U/L at baseline).
* Chronological age less than 9.0 years in girls or less than 10.0 years in boys at time of first dosing.
* Bone age advanced at least 1 year beyond the chronological age at entry into the study.
* The condition may be idiopathic or secondary to another lesion. If secondary, therapy of the primary condition will have been undertaken and stabilized.
* No evidence of abnormal pituitary, adrenal, thyroid and gonadal function except for premature secretion of gonadotropins.

Exclusion Criteria:

* Irradiation to the central nervous system.
* Prior therapy with medroxyprogesterone acetate and/or with any GnRH analog (including prior treatment with daily subcutaneous and depot formulations of leuprolide acetate).

Primary outcomes:
measure: Percentage of Subjects (n/N) With Suppression of Clinical Sexual Characteristics According to Tanner Staging (Breast Development in Females)
timeFrame: Week 4, Week 48 (Year 1), yearly for 5 years (Week 240), and Final Visit
measure: Percentage of Subjects (n/N) With Suppression of Clinical Sexual Characteristics According to Tanner Staging (Genital Development in Males)
timeFrame: Week 4, Week 48 (Year 1), yearly for 5 years (Week 240), and Final Visit
Secondary outcomes:
measure: Mean Peak Stimulated Luteinizing Hormone (LH) and Follicle Stimulating Hormone (FSH) Concentrations
timeFrame: Baseline, Weeks 4, 12, 24, 48 (Year 1), yearly for 5 years (Week 240), and Final Visit
measure: Mean Stimulated Estradiol Concentrations in Females
timeFrame: Baseline, Weeks 4, 12, 24, 48 (Year 1), yearly for 5 years (Week 240), and Final Visit
measure: Mean Stimulated Testosterone Concentrations in Males
timeFrame: Baseline, Weeks 4, 12, 24, 48 (Year 1), yearly for 5 years (Week 240), and Final Visit
measure: Mean Ratio of Bone Age to Chronological Age
timeFrame: Week 24 and Week 48 (Year 1), yearly for 5 years (Week 240), and Final Visit
Other outcomes:
measure: Posttreatment Height (ht.) Compared to Standard Population and as Predicted From Ht. at Baseline (BL)
timeFrame: Final ht. (measured or provided for final questionnaire in subjects >= 18 years of age) or near final adult ht. (<1 cm/year or bone age > 14 years for females or > 15 years for males)
measure: Mean Time to or Mean Age at Regular Menses in Females After Treatment
timeFrame: Posttreatment during the follow-up period (subjects observed every 6 months until physical and laboratory observations are at pubertal levels)
measure: Number of Female Subjects Who Reported Regular Menses at Adulthood
timeFrame: Posttreatment data were collected from the final adult questionnaire (subjects >= 18 years of age)
measure: Number of Subjects Who Reported Pregnancies at Final Questionnaire
timeFrame: Posttreatment data were collected from the final adult questionnaire (subjects >= 18 years of age)
measure: Number of Pregnancies Reported by Subjects at Final Questionnaire
timeFrame: Posttreatment data were collected from the final adult questionnaire (subjects >= 18 years of age)
Locations:
city: Phoenix
country: United States
facility: Site Reference ID/Investigator# 46673
state: Arizona
city: San Francisco
country: United States
facility: Site Reference ID/Investigator# 46671
state: California
city: Stanford
country: United States
facility: Site Reference ID/Investigator# 14921
state: California
city: Aurora
country: United States
facility: Site Reference ID/Investigator# 14343
state: Colorado
city: St. Petersburg
country: United States
facility: Site Reference ID/Investigator# 14341
state: Florida
city: Indianapolis
country: United States
facility: Site Reference ID/Investigator# 14342
state: Indiana
city: Baltimore
country: United States
facility: Site Reference ID/Investigator# 46672
state: Maryland
city: Springfield
country: United States
facility: Site Reference ID/Investigator# 46668
state: Massachusetts
city: Hershey
country: United States
facility: Site Reference ID/Investigator# 14344
state: Pennsylvania
Study officials:
affiliation: Abbott
name: Kristof Chwalisz, MD
role: STUDY_DIRECTOR
References:
citation: Lee PA, Neely EK, Fuqua J, Yang D, Larsen LM, Mattia-Goldberg C, Chwalisz K. Efficacy of Leuprolide Acetate 1-Month Depot for Central Precocious Puberty (CPP): Growth Outcomes During a Prospective, Longitudinal Study. Int J Pediatr Endocrinol. 2011;2011(1):7. doi: 10.1186/1687-9856-2011-7. Epub 2011 Jul 12.