A Study to Assess the Safety and Efficacy of Enantone (Leuprorelin) in Central Precocious Puberty (CPP) Among Chinese Participants
An Observational, Retrospective Study to Evaluate the Long Term Safety and Effectiveness of Leuprorelin in the Treatment of Central Precocious Puberty
1 other identifier
observational
108
1 country
6
Brief Summary
The purpose of this study is to evaluate the long-term safety and efficacy of Enantone in the treatment of CPP in Chinese participants.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Jun 2017
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 13, 2016
CompletedFirst Posted
Study publicly available on registry
December 15, 2016
CompletedStudy Start
First participant enrolled
June 24, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 30, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
September 30, 2018
CompletedResults Posted
Study results publicly available
October 24, 2019
CompletedMarch 16, 2022
March 1, 2022
1.3 years
December 13, 2016
September 30, 2019
March 7, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (4)
Number of Participants With at Least One Treatment Emergent Adverse Event (TEAE) and Serious Adverse Event (SAE) During Enantone Treatment Phase
A TEAE is any untoward medical occurrence in a subject administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
During treatment with and up to 30 days post last dose of Enantone (the mean duration of Enantone exposure was 22.3 months, ranging from 10.1 to 52.4 months)
Number of Participants With at Least One Treatment Emergent Adverse (TEAE) and Serious Adverse Event (SAE) During Follow-up Phase
A TEAE is any untoward medical occurrence in a subject administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
Mean duration of follow-up=8.75 months (range: 1.9-29.5 months) for No longer treated for CPP group; 10.80 months (range: 2.8-20.5 months) for Treated with Non-Enantone GnRHa group after treatment with Enantone (while on another GnRHa)
Percentage of Participants Who Had Regression or No Progression in Tanner Staging at the End of Enantone Treatment Phase
Tanner Stage is used to measure pubertal development. Female (F) and male (M) participants were evaluated for breast development and genital development respectively and both genders for pubic hair development. Tanner Stage is based on progression through 5-stages. Participants were classified as having progression if either breast/genitals or pubic hair progression were present. Otherwise participant is classified as regression or no progression.
The mean duration of Enantone exposure was 22.3 months, ranging from 10.1 to 52.4 months
Percentage of Participants Who Had Regression or No Progression in Tanner Staging at the End of Follow-Up Phase
Tanner Stage is used to measure pubertal development. Female (F) and male (M) participants were evaluated for breast development and genital development respectively and both genders for pubic hair development. Tanner Stage is based on progression through 5-stages. Participants were classified as having progression if either breast/genitals or pubic hair progression were present. Otherwise participant is classified as regression or no progression.
No longer treated for CPP group - Month: 27 (766- 855 days) post last dose of Enantone; Treated with Non-Enantone GnRHa group - Month 21 (586- 675 days) post last dose of Enantone
Secondary Outcomes (6)
Percentage of Participants With Post Stimulation Test Peak Values, for Luteinizing Hormone (LH) and Follicle Stimulating Hormone (FSH), Suppressed Below Upper Limit Value (ULV) at the End of Enantone Treatment Phase
The mean duration of Enantone exposure was 22.3 months, ranging from 10.1 to 52.4 months
Percentage of Participants With Post Stimulation Test Peak Values, for Luteinizing Hormone (LH) and Follicle Stimulating Hormone (FSH), Suppressed Below Upper Limit Value (ULV) at the End of Follow-Up Phase
No longer treated for CPP group - Month: 27 (766- 855 days) post last dose of Enantone; Treated with Non-Enantone GnRHa group - Month 21 (586- 675 days) post last dose of Enantone
Percentage of Participants With Value, for Estradiol or Testosterone, Suppressed Below Upper Limit Value (ULV) at the End of Enantone Treatment Phase
The mean duration of Enantone exposure was 22.3 months, ranging from 10.1 to 52.4 months
Percentage of Participants With Value, for Estradiol or Testosterone, Suppressed Below Upper Limit Value (ULV) at the End of Follow-Up Phase
No longer treated for CPP group-Month: 27 (766-855 days) post last dose of Enantone; Treated with Non-Enantone GnRHa group-Month 21 (586-675 days) post last dose of Enantone
Percentage of Participants With Decease of Ratio of Bone Age to Chronological Age at the End of Enantone Treatment Phase
The mean duration of Enantone exposure was 22.3 months, ranging from 10.1 to 52.4 months
- +1 more secondary outcomes
Study Arms (3)
Treatment Phase: Enantone
Participants with CPP who were treated with Enantone (≥ 30 μg/kg up to 180 μg/kg) for at least 9 continuous months and who initiated and received the last dose of treatment during the index period from 01 September 1998 to 30 September 2018 (the mean duration of Enantone exposure was 22.3 months, ranging from 10.1 to 52.4 months).
Follow Up: Participants No longer Treated for CPP
Participants who had completed their CPP during the treatment phase with Enantone and were no longer on treatment in the follow-up phase (the mean duration of follow up was 8.75 months with a range of 1.9 to 29.5 months).
Follow Up: Treated with Non-Enantone GnRHa after Enantone
Participants who were continuing their CPP treatment with a non-Enantone gonadotropin releasing hormone agonist (GnRHa) after treatment with Enantone in the follow-up phase (the mean duration of follow up while on another GnRHa was 10.80 months with a range of 2.8 to 20.5 months, and the mean duration of follow up after stopping treatment was 4.26 months with a range of 0.0 \[i.e. 1 day\] to 12 months).
Interventions
A non-Enantone GnRH agonist
Eligibility Criteria
Participants diagnosed with CPP will be observed.
You may qualify if:
- Has diagnosis of idiopathic CPP.
- Has been treated with leuprorelin acetate (Enantone) for at least 9 continuous months of therapy with either a stable dose of high dose (greater than or equal to \[\>=\] 90 mcg/kg up to 180 mcg/kg) or low dose (\< 90 mcg/kg down to 30 mcg/kg).
- Has initiated and completed treatment during the index period from September 1st 1998 to September 30th 2018.
- Have the following information prior to initiation of enantone and at least one record of each of the following parameters at the end of enantone treatment in the medical records: Tanner staging, estradiol or testosterone level, and FSH and LH level. The participant should have at least one record of bone age prior to the initiation gonadotropin releasing hormone analogs (GnRHa) therapy with enantone to support the diagnosis of CPP. In addition, should have at least one record of bone age during treatment with enantone.
You may not qualify if:
- Has been treated with leuprorelin acetate or any other GnRHa for conditions other than CPP.
- Has used any other GnRHa products for CPP treatment prior to initiation of enantone therapy.
- CPP participants with identified etiology, such as brain tumor or cranial irradiation.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Takedalead
Study Sites (6)
The Children's Hospital, Zhejiang University School of Medicine
Wuhan, Hubei, 430030, China
Childrens Hospital of Hunan province
Changsha, Hunan, 410007, China
Jiangsu Province Hosptial
Nanjing, Jiangsu, 210036, China
Children's Hospital of Jiangxi province
Nanchang, Jiangxi, 330006, China
Children's Hospital of Shanghai
Shanghai, Shanghai Municipality, 200040, China
The Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310053, China
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
The enrollment of only 100 participants did not support the performance of subgroup analysis of outcomes by dose. Retrospective observational data is not controlled and therefore robust analysis could not be performed.
Results Point of Contact
- Title
- Medical Director
- Organization
- Takeda
Study Officials
- STUDY DIRECTOR
Medical Director Clinical Science
Takeda
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- RETROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 13, 2016
First Posted
December 15, 2016
Study Start
June 24, 2017
Primary Completion
September 30, 2018
Study Completion
September 30, 2018
Last Updated
March 16, 2022
Results First Posted
October 24, 2019
Record last verified: 2022-03
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, CSR
- Access Criteria
- IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.