Clinical Trial Results:
An Open label, Multicenter, Single-arm and Prospective Study to Assess the Efficacy and Safety of Leuprorelin 3M in the Treatment of CPP
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Summary
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EudraCT number |
2022-002471-11 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
10 Mar 2025
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Results information
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Results version number |
v1(current) |
This version publication date |
23 Oct 2025
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First version publication date |
23 Oct 2025
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
Leuprorelin-4002
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT05341115 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Takeda (China) International Trading Co., Ltd.
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Sponsor organisation address |
37F, New Bund Center, NO.555 West Haiyang Rd, Pudong New Area, Shanghai, China, 200124
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Public contact |
Study Director, Takeda, TrialDisclosures@takeda.com
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Scientific contact |
Study Director, Takeda, TrialDisclosures@takeda.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
10 Mar 2025
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
10 Mar 2025
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The main aim of this study was to investigate the efficacy and safety of leuprolide acetate depot 11.25 milligrams (mg) 3-month formulations for the treatment of central precocious puberty (CPP) in children in China.
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Protection of trial subjects |
Each participant or their legally authorised representative signed an informed consent form (ICF) before participating in the study.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
14 Mar 2023
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
China: 79
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Worldwide total number of subjects |
79
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EEA total number of subjects |
0
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
79
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
A total of 79 participants took part in the study at 5 investigative sites in China from 12 March 2023 to 10 March 2025. | ||||||||||||
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Pre-assignment
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Screening details |
A total of 79 participants with a diagnosis of CPP received leuprorelin acetate depot 11.25 mg. | ||||||||||||
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Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||
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Arms
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Arm title
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Leuprorelin Acetate Depot 3M 11.25 mg | ||||||||||||
Arm description |
Participants with CPP having body weight greater than or equal to (≥)20 kilograms (kg) received the recommended dose of leuprorelin acetate depot 11.25 mg subcutaneous administration (SC) every 12 weeks based on the standard of 30～180 micrograms (μg)/kg/4weeks for the 24-week Treatment Period. It was not recommended to exceed the dose above 180 μg/kg. | ||||||||||||
Arm type |
Experimental | ||||||||||||
Investigational medicinal product name |
Leuprorelin Acetate
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Investigational medicinal product code |
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Other name |
Leuprorelin-4002
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Pharmaceutical forms |
Powder and solvent for solution for injection
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Routes of administration |
Subcutaneous use
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Dosage and administration details |
Leuprorelin acetate depot 11.25 mg SC every 12 weeks based on the standard of 30～180μg/kg/4weeks for the 24-week Treatment Period.
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Baseline characteristics reporting groups
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Reporting group title |
Leuprorelin Acetate Depot 3M 11.25 mg
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Reporting group description |
Participants with CPP having body weight greater than or equal to (≥)20 kilograms (kg) received the recommended dose of leuprorelin acetate depot 11.25 mg subcutaneous administration (SC) every 12 weeks based on the standard of 30～180 micrograms (μg)/kg/4weeks for the 24-week Treatment Period. It was not recommended to exceed the dose above 180 μg/kg. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Leuprorelin Acetate Depot 3M 11.25 mg
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Reporting group description |
Participants with CPP having body weight greater than or equal to (≥)20 kilograms (kg) received the recommended dose of leuprorelin acetate depot 11.25 mg subcutaneous administration (SC) every 12 weeks based on the standard of 30～180 micrograms (μg)/kg/4weeks for the 24-week Treatment Period. It was not recommended to exceed the dose above 180 μg/kg. | ||
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End point title |
Percentage of Participants with Peak Luteinizing Hormone (LH) Suppression in Gonadotropin-Releasing Hormone (GnRH) Stimulation at Week 24 [1] | ||||||||
End point description |
The LH suppression was defined as LH peak value in GnRH stimulation ≤3.0 international units per liter (IU/L). The enrolled population set included all the eligible participants enrolled in this study, i.e., all participants enrolled in this study who met the inclusion criteria and did not meet any of the exclusion criteria, regardless of whether they received the study drug.
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End point type |
Primary
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End point timeframe |
Week 24
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive analysis was planned for this endpoint. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Percentage of Participants with Tanner Stage Regression or No Progression at Week 24 | ||||||||
End point description |
Tanner Stage was used to measure pubertal development. Tanner Stage is based on progression through 5-stages. Progression was defined as either breast/genitals or pubic hair score being increased compared with baseline score. Otherwise, the status was classified as regression or no progression. Baseline was defined as the assessment prior to the first dose of study drug. The enrolled population set included all the eligible participants enrolled in this study, i.e., all participants enrolled in this study who met the inclusion criteria and did not meet any of the exclusion criteria, regardless of whether they received the study drug.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 24
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| No statistical analyses for this end point | |||||||||
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End point title |
Concentrations of Basal Luteinizing Hormone (LH) and Follicle Stimulating Hormone (FSH) | ||||||||||||||||||||
End point description |
Plasma LH and FSH basal concentrations were assessed. The enrolled population set included all the eligible participants enrolled in this study, i.e., all participants enrolled in this study who met the inclusion criteria and did not meet any of the exclusion criteria, regardless of whether they received the study drug. 'n' denotes number of participants with data available for analysis at the specified category and time-point.
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End point type |
Secondary
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End point timeframe |
Baseline, Weeks 24 and 36
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| No statistical analyses for this end point | |||||||||||||||||||||
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End point title |
Percentage of Participants With Decreased Ratio of Bone Age Over Chronological Age at Week 24 | ||||||||
End point description |
Bone age was determined by Greulich and Pyle standards or Tanner-Whitehouse 3 (TW3) standards. The enrolled population set included all the eligible participants enrolled in this study, i.e., all participants enrolled in this study who met the inclusion criteria and did not meet any of the exclusion criteria, regardless of whether they received the study drug.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 24
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| No statistical analyses for this end point | |||||||||
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End point title |
Percentage of Participants with Decreased First Morning Voided (FMV) Urinary Gonadotropin (Gn) at Week 24 | ||||||||||||
End point description |
Percentage of participants with reductions from baseline FMV Gn urinary LH and urinary FSH values at Week 24 were reported. The enrolled population set included all the eligible participants enrolled in this study, i.e., all participants enrolled in this study who met the inclusion criteria and did not meet any of the exclusion criteria, regardless of whether they received the study drug.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 24
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Number of Participants With Treatment-Emergent Adverse Events (TEAE) | ||||||
End point description |
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (eg, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Safety population set included all participants who had been under treatment with leuprorelin or who were first prescribed leuprorelin, received at least one dose and completed one follow-up visit.
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End point type |
Secondary
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End point timeframe |
From first dose of study drug up to 12 weeks post last dose or early termination Visit (ET) (up to approximately 38 weeks)
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| No statistical analyses for this end point | |||||||
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Adverse events information
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Timeframe for reporting adverse events |
From first dose of study drug up to 12 weeks post last dose or early termination Visit (ET) (up to approximately 38 weeks)
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Adverse event reporting additional description |
The safety population set included all included participants who had been under treatment with leuprorelin or who were first prescribed leuprorelin, received at least one dose and completed one follow-up visit.
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Assessment type |
Systematic | ||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||
Dictionary version |
28.0
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Reporting groups
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Reporting group title |
Leuprorelin Acetate Depot 3M 11.25 mg
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Reporting group description |
Participants with CPP having body weight ≥20 kg received the recommended dose of leuprorelin acetate depot 11.25 mg SC every 12 weeks based on the standard of 30～180 μg/kg/4weeks for the 24-week Treatment Period. It was not recommended to exceed the dose above 180 μg/kg. | ||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
