Efficacy, Safety, and Pharmacokinetics of Leuprolide Mesylate in Subjects With Central Precocious Puberty
- Conditions
- Puberty; Precocious, Central
- Interventions
- Registration Number
- NCT05493709
- Lead Sponsor
- Foresee Pharmaceuticals Co., Ltd.
- Brief Summary
The study will evaluate if Leuprolide Mesylate is safe and effective in the treatment of subjects with central (gonadotropin-dependent) precocious puberty, when administered as two injections six months apart.
- Detailed Description
This is a multi-center, open-label, single-arm study. All subjects will be pediatric patients with central precocious puberty judged to be candidates for GnRH (gonadotropin releasing hormone) analog therapy, and all will receive two injections of FP-001 42 mg six-month apart in an unblinded fashion.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 93
- Females aged 2 to 8 years (inclusive) or males aged 2 to 9 years (inclusive).
- Confirmed diagnosis of CPP within 12 months of Baseline Visit (Day 0) but have not received prior GnRHa treatment for CPP.
- Pubertal-type LH response at 60 minutes post GnRHa stimulation test before treatment initiation > 5 mIU/mL.
- Clinical evidence of puberty, defined as Tanner stage β₯ 2 for breast development in females or testicular volume β₯ 4 mL in males.
- Willing and able to participate in the study.
- Difference between bone age (Greulich and Pyle method) and chronological age β₯ 1 year.
- Bone age < 13 years for girls and < 14 years for boys.
- 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).
- Signed Assent by patients as per IRB/IEC requirements.
- 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.
- Prior or current GnRH treatment for CPP.
- Non-progressing isolated premature thelarche.
- 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.
- 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).
- Prior or current therapy with medroxyprogesterone acetate, growth hormone or insulin-like growth factor-1 (IGF-1).
- Major medical or psychiatric illness that could interfere with study visits.
- Diagnosis of short stature (i.e., 2.25 standard deviations (SD) below the mean height for age).
- Positive urine pregnancy test.
- Known hypersensitivity to GnRH or related compounds.
- 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.
- Any other condition(s) which could significantly interfere with Protocol compliance.
- Treatment with an investigational product within 5 half-lives of that product in prior clinical studies before the baseline visit (Day 0).
- Known history of seizures, epilepsy, and/or central nervous system disorders that may be associated with seizures or convulsions.
- Prior (within 6 months of Baseline (Day 0)) or current use of medications that, per Investigator opinion, have been associated with seizures or convulsions.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description FP-001 42 mg Leuprolide Mesylate, Subcutaneous injection of 42 mg Leuprolide All subjects will be pediatric patients with central precocious puberty. They will be injected twice with a depot formulation containing 42 mg of Leuprolide. The first dose on day 0 the second dose on week 24 (six months apart).
- Primary Outcome Measures
Name Time Method Efficacy of Leuprolide Mesylate (FP-001 42 mg) 48 weeks The percentage of patients with serum LH concentrations \< 4 mIU/mL 60 minutes following an abbreviated GnRHa stimulation test at Visit 6 (Week 24).
- Secondary Outcome Measures
Name Time Method Effect of FP-001 42 mg on bone age progression 24 and 48 weeks Evaluate the changes in bone age progression from the baseline to Weeks 24 and 48 using centralized analysis of wrist x-ray
Effect of FP-001 42 mg on suppression of physical signs of puberty 48 weeks Evaluate the percentage of patients with suppression of physical signs of puberty
Acute-On-Chronic (AOC) phenomenon of serum testosterone and LH 48 weeks Evaluate The proportion of subjects exhibiting "acute-on-chronic" phenomenon (i.e., related to the second dose of FP-001 42 mg)
Effect of FP-001 42 mg on growth rate 48 weeks Evaluate the changes in growth rate and bone age advancement relative to chronological age from baseline to end of study using height in meters
Effect of FP-001 42 mg on physical signs of puberty 48 weeks Evaluate the change in physical signs of puberty as measure by Tanner stages from baseline to end of study
Trial Locations
- Locations (43)
Arizona University
πΊπΈTucson, Arizona, United States
Rady Children's Hospital- San Diego
πΊπΈSan Diego, California, United States
Nemours Children's Health Center
πΊπΈJacksonville, Florida, United States
Johns Hopkins - All Children's Hospital
πΊπΈSaint Petersburg, Florida, United States
Rocky Mountain Clinical Research
πΊπΈIdaho Falls, Idaho, United States
Indiana University
πΊπΈIndianapolis, Indiana, United States
University of Minnesota
πΊπΈMinneapolis, Minnesota, United States
Renown Health
πΊπΈReno, Nevada, United States
University of Texas Southwestern Medical Center
πΊπΈDallas, Texas, United States
Cook Children's
πΊπΈFort Worth, Texas, United States
Virginia University
πΊπΈCharlottesville, Virginia, United States
Multicare Health System
πΊπΈTacoma, Washington, United States
The Second Hospital of Anhui Medical University
π¨π³Hefei, Anhui, China
Children's Hospital affiliated to Capital Institute of Pediatrics
π¨π³Beijing, Chaoyang District, China
The first Affiliated Hospital of Xiamen University
π¨π³Xiamen, Fujian, China
Pearl River Hospital, Southern Medical University
π¨π³Guangzhou City, Guangdong, China
Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University
π¨π³Guangzhou City, Guangdong, China
The First Affiliated Hospital, Sun Yat-sen University
π¨π³Guangzhou, Guangdong, China
The Third Affiliated Hospital, Sun Yat-Sen University
π¨π³Guangzhou, Guangdong, China
Tongji Hospital, Tongji Medical College of HUST
π¨π³Wuhan, Hubei, China
Wuhan Children's Hospital, Tongji Medical College of HUST
π¨π³Wuhan, Hubei, China
Hunan Children's Hospital
π¨π³Changsha, Hunan, China
Children's Hospital of Soochow University
π¨π³Suzhou, Jiangsu, China
Jiangxi Provincial Children's Hospital
π¨π³Nanchang, Jiangxi, China
The First Bethune Hospital of Jilin University
π¨π³Changchun City, Jilin, China
Shengjing Hospital of China Medical University
π¨π³Shenyang City, Liaoning, China
Children's Hospital of Shanxi
π¨π³Taiyuan, Shanxi, China
Chengdu Women's and Children's Central Hospital
π¨π³Chengdu, Sichuan, China
The Children's Hospital, Zhejiang University School of Medicine
π¨π³Hangzhou City, Zhejiang, China
The First Hospital of Jiaxing Affiliated Hospital of Jiaxing University
π¨π³Jiaxing, Zhejiang, China
Ningbo Women & Children's Hospital
π¨π³Ningbo, Zhejiang, China
Beijing Children's Hospital, Capital Medical University
π¨π³Beijing, China
Children's Hospital of Fudan University
π¨π³Shanghai, China
Children's Hospital of Shanghai
π¨π³Shanghai, China
Shanghai Children's Medical Center
π¨π³Shanghai, China
Tianjin Medical University General Hospital
π¨π³Tianjin, China
University Pediatric Hospital
π΅π·San Juan, Puerto Rico
Changhua Christian Hospital
π¨π³Changhua City, Taiwan
Chang Gung Memorial Hospital-Kaohsiung branch
π¨π³Kaohsiung City, Taiwan
China Medical University Hospital
π¨π³Taichung City, Taiwan
National Cheng Kung University Hospital
π¨π³Tainan City, Taiwan
Mackay Memorial Hospital
π¨π³Taipei City, Taiwan
LinKou Chang-Gung Memorial Hospital (CGMH-LK)
π¨π³Taoyuan City, Taiwan