Clinical trial NCT05341115: A Study of Leuprolide Acetate Depot in Children With Central Precocious Puberty
Official title: An Open Label, Multicenter, Single-arm and Prospective Study to Assess the Efficacy and Safety of Leuprorelin 3M in the Treatment of Central Precocious Puberty (CPP)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Central Precocious Puberty
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2022-04-22
Enrollment: 80
Sex: ALL
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The main aim is to see how leuprolide works to treat central precocious puberty in children. Participants will receive an injection of leuprorelin acetate depot 11.25 mg every 12 weeks during 6 months and will visit their study clinic 6 times to complete some assessments.

Detailed description: The drug being tested in this study is called leuprorelin acetate depot 3M. Leuprorelin acetate depot 3M will be tested to treat children who have central precocious puberty. This study will look at the efficacy and safety of leuprorelin acetate depot 3M in the treatment of CPP.

The study will enroll approximately 80 participants with CPP. Participants with a bodyweight of ≥ 20 kg will receive the recommended dose of leuprorelin acetate depot 3M in a 24 weeks Treatment Period followed by a 12 weeks Post-treatment follow-up period. Participants will be assigned to the following drug administration:

• Leuprorelin Acetate Depot 3M 11.25 mg

Participants will receive leuprorelin acetate depot 3M 11.25 mg as subcutaneous (SC) injection on Weeks 0, 12, and 24. The gonadotropin-releasing hormone agonist (GnRHa) stimulation, basal luteinizing hormone (LH), and follicle-stimulating hormone (FSH) levels will be tested pre-dose of every SC injection of the study drug or at premature termination.

This multi-center trial will be conducted in China. The overall time to participate in this study is 38 weeks. Participants will make a follow-up visit to the site at approximately 12 weeks after the last dose of study treatment.

Eligibility criteria: Inclusion Criteria:

1. Maximum age to participate is this study is up to 10 years (child). Early appearance of secondary sexual characteristics: Girls ≤8 years, Boys ≤9years
2. Body weight ≥20 kg
3. According to the National Consensus Statement in China (2015), CPP is diagnosed when secondary sexual characteristics appeared before the age of 8 years in girls and 9 years in boys, a peak LH level \>5.0 IU/L with LH/FSH \>0.6 in stimulating test; evidence of gonadal development by ultrasonography (multiple ovarian follicles ≥4 mm in any ovary or uterine enlargement in females or testicular volume ≥4 mL in males); advanced bone age (BA) ≥1 year; linear growth acceleration with higher growth velocity (GV) than normal children. BA is determined by Greulich and Pyle standards or TW3 standards at screening.

Exclusion Criteria:

1. The participant has received GnRHa treatment in a previous clinical study or as a therapeutic agent.
2. The participant has a history or clinical manifestations of significant adrenal or thyroid diseases or intracranial tumor OR has a history of malignant disease.
3. The participant has a history of hypersensitivity or allergies to leuprorelin, or related compounds including any excipients of the compound.
4. The participant has a diagnosis of peripheral precocious puberty.

Primary outcomes:
measure: Percentage of Participants with Peak Luteinizing Hormone (LH) Suppression in Gonadotropin-Releasing Hormone (GnRH) Stimulation at Week 24
timeFrame: Week 24
Secondary outcomes:
measure: Percentage of Participants with Tanner Stage Regression or No Progression at Week 24
timeFrame: Baseline and Week 24
measure: Concentrations of Basal Luteinizing Hormone (LH) and Follicle Stimulating Hormone (FSH)
timeFrame: Baseline, Week 24 and 36
measure: Percentage of Participants With Decreased Ratio of Bone Age Over Chronological Age at Week 24
timeFrame: Baseline and Week 24
measure: Percentage of Participants with Decreased First Morning Voided (FMV) Urinary Gonadotropin (Gn) at Week 24
timeFrame: Baseline and Week 24
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAE)
timeFrame: From first dose of study drug up to 12 weeks post last dose or early termination Visit (ET) (Up to approximately 38 weeks)
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Children's Hospital, Capital Medical University
state: Beijing
city: Guangzhou
country: China
facility: the First A liated Hospital of Sun Yat-sen University
state: Guangdong
city: Wuhan
country: China
facility: The Hospital attached by the Torigji Medical College, Huazhong Science and Technology University.
state: Hubei
city: Jinan
country: China
facility: Provincial Hospital Affiliated to Shandong First Medical University
state: Shandong
city: Shanghai
country: China
facility: Shanghai Children's Hospital
state: Shanghai
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: