Efficacy, Safety, and Pharmacokinetics of Leuprolide Mesylate in Subjects with Central Precocious Puberty

Eligibility Criteria

• 1. Females aged 2 to 8 years (inclusive) or males aged 2 to 9 years (inclusive).
• 2. Confirmed diagnosis of CPP within 12 months of Baseline Visit (Day 0) but have not received prior GnRHa treatment for CPP.
• 3. Pubertal-type LH response at 60 minutes post GnRHa stimulation test before treatment initiation \> 5 mIU/mL.
• 4. Clinical evidence of puberty, defined as Tanner stage ≥ 2 for breast development in females or testicular volume ≥ 4 mL in males.
• 5. Willing and able to participate in the study.
• 6. Difference between bone age (Greulich and Pyle method) and chronological age ≥ 1 year.
• 7. Bone age \< 13 years for girls and \< 14 years for boys.
• 8. Signed Institutional Review Board/Independent Ethics Committee (IRB/IEC)-approved informed consent form (ICF) by one or both parents (per IRB/IEC requirements), by the custodial parent(s) or by the legal guardian(s) (if required).
• 9. Signed Assent by patients as per IRB/IEC requirements.
• 1. Gonadotropin-independent (peripheral) precocious puberty: extra pituitary secretion of gonadotropins or gonadotropin-independent gonadal or adrenal sex steroid secretion. This includes true CPP triggered by other conditions, such as congenital adrenal hyperplasia.
• 2. Prior or current GnRH treatment for CPP.
• 3. Non-progressing isolated premature thelarche.
• 4. Presence of an unstable intracranial tumor or an intracranial tumor requiring neurosurgery or cerebral irradiation. Patients with hamartomas or adenomas not requiring surgery are eligible.
• 5. Any other condition, chronic illness or treatment that, in the opinion of the Investigator, may interfere with growth or other study endpoints (e.g., chronic steroid use \[except mild topical steroids\], renal failure, diabetes, moderate to severe scoliosis, previously treated intracranial tumor).
• 6. Prior or current therapy with medroxyprogesterone acetate, growth hormone or insulin-like growth factor-1 (IGF-1).
• 7. Major medical or psychiatric illness that could interfere with study visits.
• 8. Diagnosis of short stature (i.e., 2.25 standard deviations (SD) below the mean height for age).
• 9. Positive urine pregnancy test.
• 10. Known hypersensitivity to GnRH or related compounds.
• 11. Any other medical condition or serious intercurrent illness that, in the opinion of the Investigator, may make it undesirable for the patients to participate in the study.
• 12. Any other condition(s) which could significantly interfere with Protocol compliance.
• 13. Treatment with an investigational product within 5 half-lives of that product in prior clinical studies before the baseline visit (Day 0).
• 14. Known history of seizures, epilepsy, and/or central nervous system disorders that may be associated with seizures or convulsions.
• 15. Prior (within 6 months of Baseline (Day 0)) or current use of medications that, per Investigator opinion, have been associated with seizures or convulsions.