Study of Leuprolide Acetate Injectable Suspension in the Treatment of Central Precocious Puberty

Tolmar Inc.64 enrolled

Overview

This study determines the effectiveness of leuprolide acetate 45 mg for injectable suspension for treatment of children with Central Precocious Puberty.

Leuprolide acetate is a GnRH agonist that inhibits pituitary gonadotropin secretion by binding to the GnRH receptors and blocking downstream hormone synthesis. The steady decrease in hormone synthesis (LH and FSH) leads to a suppression of testicular and ovarian steroidogenesis. In children with CPP, this steady decrease in hormone synthesis disrupts the progression of puberty.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Precocious Puberty, Central

Interventions

Leuprolide Acetate 45 mgDRUG

Subcutaneous injection

Eligibility

Sex: ALLMin age: 2 YearsMax age: 9 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Females age 2 to 8 years (inclusive) or males age 2 to 9 years (inclusive) * Confirmed diagnosis of CPP within 12 months of Baseline Visit (Day 0) but have not received prior GnRH agonist treatment for CPP * Pubertal-type LH response following an abbreviated GnRHa stimulation test before treatment initiation * Clinical evidence of puberty, defined as Tanner stage ≥ 2 for breast development in females or testicular volume ≥ 4 mL in males * Difference between bone age (Greulich and Pyle method) and chronological age ≥ 1 year Exclusion Criteria: * Gonadotropin-independent (peripheral) precocious puberty * Prior or current GnRH treatment for CPP * Prior or current therapy with medroxyprogesterone acetate, growth hormone or insulin-like growth factor-1 (IGF-1) * Diagnosis of short stature (ie, 2.25 standard deviations (SD) below the mean height for age) * Known history of seizures, epilepsy, and/or central nervous system disorders that may be associated with seizures or convulsions * Any other medical condition or serious intercurrent illness that, in the opinion of the Investigator, may make it undesirable for the subject to participate in the study

Locations (20)

University of California, San Diego

San Diego, California, United States

Outcomes

Primary Outcomes

Percentage of Participants With Suppression of Peak-Stimulated Luteinizing Hormone at 6 Months.

Luteinizing Hormone (LH) suppression is defined as peak-stimulated LH \<4 IU/L. Peak stimulated LH refers to the maximum LH concentration measured 30 minutes after a gonadotropin-releasing hormone agonst (GnRHa) stimulation test.

Time frame: 6 months

Secondary Outcomes

Percentage of Subjects With Suppression of Luteinizing Hormone Measured by Blood Levels.

Percentage of subjects with suppressed serum LH concentrations(\<4 IU/L) 30 minutes post GnRHa stimulation test at all assessed timepoints.

Time frame: Week 12, Week 24, Week 36, and Week 48

Changes in Height Velocity (Growth Rate)

Changes in height velocity (growth rate) at all study timepoints after Screening to end of study. Growth velocity is defined for each visit as change from baseline / \[(number of weeks since baseline)/52\]. Week 48: Change from Week 24 growth velocity is defined as change from week 24 to week 48 / \[(number of weeks since week 24)/52\].

Time frame: Week 4, Week 12, Week 20, Week 24, Week 36, Week 44, and Week 48

Bone Age Ratio to Chronological Age at Time of Measurement

Bone Age Ratio to Chronological Age at Time of Measurement is bone age/age at bone age assessment.

Time frame: Week 24 and Week 48

Percent Change From Baseline in Height

The percent change from baseline in height at each available post-baseline measurement. Percent change is defined as (((change from Baseline)/(Baseline)) x 100).

Time frame: Week 4, Week 12, Week 20, Week 24, Week 36, Week 44, and Week 48

Tanner Scores: Boys - Development of External Genitalia

Data from ClinicalTrials.gov

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Joe DiMaggio Children's Hospital

Hollywood, Florida, United States

Nemours Children's Clinic

Jacksonville, Florida, United States

Nemours Children's Hospital

Orlando, Florida, United States

Riley Hospital for Children at Indiana University Health

Indianapolis, Indiana, United States

University of Minnesota

Minneapolis, Minnesota, United States

Cincinnati Children's Hospital Medical Center, Endocrine

Cincinnati, Ohio, United States

University of Oklahoma College of Medicine

Tulsa, Oklahoma, United States

Seattle Children's

Seattle, Washington, United States

MultiCare Institute for Research and Innovation

Tacoma, Washington, United States

...and 10 more locations

Sexual development (physical signs) in puberty were assessed by Tanner staging, a system developed by Marshall and Tanner to categorize pubertal maturation. Tanner stages are commonly used to categorize pubertal maturation in terms of sequence, timing and tempo. Tanner Stages: the minimum is Stage 1 = pre-pubertal physical characteristics and the maximum is Stage 5 = fully matured (adult) physical characteristics.

Time frame: Baseline, Week 12, Week 24, Week 36, and Week 48

Tanner Scores: Boys - Development of External Genitalia (Change From Baseline)

Time frame: Week 12, Week 24, Week 36, and Week 48

Tanner Scores: Boys and Girls - Pubic Hair

Time frame: Baseline, Week 12, Week 24, Week 36, and Week 48

Tanner Scores: Boys and Girls - Pubic Hair (Change From Baseline)

Time frame: Week 12, Week 24, Week 36, and Week 48

Tanner Scores: Girls - Breast Development

Time frame: Baseline, Week 12, Week 24, Week 36, and Week 48

Tanner Scores: Girls - Breast Development (Change From Baseline)

Time frame: Week 12, Week 24, Week 36, and Week 48