Clinical trial NCT05493709: Efficacy, Safety, and Pharmacokinetics of Leuprolide Mesylate in Subjects With Central Precocious Puberty
Official title: An Open-label, Single Arm, Multicenter, Phase III Study on the Efficacy, Safety, and Pharmacokinetics of FP-001 42 mg Controlled Release in Patients With Central (Gonadotropin-Dependent) Precocious Puberty (Casppian Study)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Puberty; Precocious, Central
Lead sponsor: Foresee Pharmaceuticals Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: QPS Holdings LLC
name: Changchun GeneScience Pharmaceutical Co., Ltd.
First posted: 2022-08-09
Enrollment: 93
Sex: ALL
Minimum age (years): 2
Maximum age (years): 9
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion 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.

Exclusion Criteria:

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.

Primary outcomes:
measure: Efficacy of Leuprolide Mesylate (FP-001 42 mg)
timeFrame: 48 weeks
Secondary outcomes:
measure: Effect of FP-001 42 mg on bone age progression
timeFrame: 24 and 48 weeks
measure: Effect of FP-001 42 mg on growth rate
timeFrame: 48 weeks
measure: Effect of FP-001 42 mg on physical signs of puberty
timeFrame: 48 weeks
measure: Effect of FP-001 42 mg on suppression of physical signs of puberty
timeFrame: 48 weeks
measure: Acute-On-Chronic (AOC) phenomenon of serum testosterone and LH
timeFrame: 48 weeks
Other outcomes:
Locations:
city: Tucson
country: United States
facility: Arizona University
state: Arizona
city: San Diego
country: United States
facility: Rady Children's Hospital- San Diego
state: California
city: Jacksonville
country: United States
facility: Nemours Children's Health Center
state: Florida
city: Saint Petersburg
country: United States
facility: Johns Hopkins - All Children's Hospital
state: Florida
city: Idaho Falls
country: United States
facility: Rocky Mountain Clinical Research
state: Idaho
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Reno
country: United States
facility: Renown Health
state: Nevada
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's
state: Texas
city: Charlottesville
country: United States
facility: Virginia University
state: Virginia
city: Tacoma
country: United States
facility: Multicare Health System
state: Washington
city: Hefei
country: China
facility: The Second Hospital of Anhui Medical University
state: Anhui
city: Beijing
country: China
facility: Children's Hospital affiliated to Capital Institute of Pediatrics
state: Chaoyang District
city: Xiamen
country: China
facility: The first Affiliated Hospital of Xiamen University
state: Fujian
city: Guangzhou City
country: China
facility: Pearl River Hospital, Southern Medical University
state: Guangdong
city: Guangzhou City
country: China
facility: Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University
state: Guangdong
city: Guangzhou
country: China
facility: The First Affiliated Hospital, Sun Yat-sen University
state: Guangdong
city: Guangzhou
country: China
facility: The Third Affiliated Hospital, Sun Yat-Sen University
state: Guangdong
city: Wuhan
country: China
facility: Tongji Hospital, Tongji Medical College of HUST
state: Hubei
city: Wuhan
country: China
facility: Wuhan Children's Hospital, Tongji Medical College of HUST
state: Hubei
city: Changsha
country: China
facility: Hunan Children's Hospital
state: Hunan
city: Suzhou
country: China
facility: Children's Hospital of Soochow University
state: Jiangsu
city: Nanchang
country: China
facility: Jiangxi Provincial Children's Hospital
state: Jiangxi
city: Changchun City
country: China
facility: The First Bethune Hospital of Jilin University
state: Jilin
city: Shenyang City
country: China
facility: Shengjing Hospital of China Medical University
state: Liaoning
city: Taiyuan
country: China
facility: Children's Hospital of Shanxi
state: Shanxi
city: Chengdu
country: China
facility: Chengdu Women's and Children's Central Hospital
state: Sichuan
city: Hangzhou City
country: China
facility: The Children&#39;s Hospital, Zhejiang University School of Medicine
state: Zhejiang
city: Jiaxing
country: China
facility: The First Hospital of Jiaxing Affiliated Hospital of Jiaxing University
state: Zhejiang
city: Ningbo
country: China
facility: Ningbo Women & Children's Hospital
state: Zhejiang
city: Beijing
country: China
facility: Beijing Children's Hospital, Capital Medical University
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
city: Shanghai
country: China
facility: Children's Hospital of Shanghai
city: Shanghai
country: China
facility: Shanghai Children's Medical Center
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
city: San Juan
country: Puerto Rico
facility: University Pediatric Hospital
city: Changhua City
country: Taiwan
facility: Changhua Christian Hospital
city: Kaohsiung City
country: Taiwan
facility: Chang Gung Memorial Hospital-Kaohsiung branch
city: Taichung City
country: Taiwan
facility: China Medical University Hospital
city: Tainan City
country: Taiwan
facility: National Cheng Kung University Hospital
city: Taipei City
country: Taiwan
facility: Mackay Memorial Hospital
city: Taoyuan City
country: Taiwan
facility: LinKou Chang-Gung Memorial Hospital (CGMH-LK)
Study officials:
affiliation: Foresee Pharma
name: Susan Whitaker
role: STUDY_DIRECTOR
References: