Clinical trial NCT02993926: A Study to Assess the Safety and Efficacy of Enantone (Leuprorelin) in Central Precocious Puberty (CPP) Among Chinese Participants
Official title: An Observational, Retrospective Study to Evaluate the Long Term Safety and Effectiveness of Leuprorelin in the Treatment of Central Precocious Puberty
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Central Precocious Puberty
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2016-12-15
Enrollment: 108
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants diagnosed with CPP will be observed.

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.

Detailed description: The drug being evaluated in this study is called Enantone (leuprorelin). Enantone is used to treat children who have CPP. This study will look at long term safety and efficacy of leuprorelin in the treatment of Chinese participants with CPP.

The study will enroll approximately 300 participants.

All participants who have received leuprorelin 30 mcg/kg to \<90 mcg/kg or 90 mcg/kg to 180 mcg/kg per body weight, injection, subcutaneously, every 4 weeks up to at least 9 continuous months during the index period from September 1st 1998 to September 30th 2018 will be observed.

This multi-center trial will be conducted in China. Data will be collected over period of 20 months.

Eligibility criteria: Inclusion Criteria:

1. Has diagnosis of idiopathic CPP.
2. 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).
3. Has initiated and completed treatment during the index period from September 1st 1998 to September 30th 2018.
4. 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.

Exclusion Criteria:

1. Has been treated with leuprorelin acetate or any other GnRHa for conditions other than CPP.
2. Has used any other GnRHa products for CPP treatment prior to initiation of enantone therapy.
3. CPP participants with identified etiology, such as brain tumor or cranial irradiation.

Primary outcomes:
measure: Number of Participants With at Least One Treatment Emergent Adverse Event (TEAE) and Serious Adverse Event (SAE) During Enantone Treatment Phase
timeFrame: 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)
measure: Number of Participants With at Least One Treatment Emergent Adverse (TEAE) and Serious Adverse Event (SAE) During Follow-up Phase
timeFrame: 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)
measure: Percentage of Participants Who Had Regression or No Progression in Tanner Staging at the End of Enantone Treatment Phase
timeFrame: The mean duration of Enantone exposure was 22.3 months, ranging from 10.1 to 52.4 months
measure: Percentage of Participants Who Had Regression or No Progression in Tanner Staging at the End of Follow-Up Phase
timeFrame: 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:
measure: 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
timeFrame: The mean duration of Enantone exposure was 22.3 months, ranging from 10.1 to 52.4 months
measure: 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
timeFrame: 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
measure: Percentage of Participants With Value, for Estradiol or Testosterone, Suppressed Below Upper Limit Value (ULV) at the End of Enantone Treatment Phase
timeFrame: The mean duration of Enantone exposure was 22.3 months, ranging from 10.1 to 52.4 months
measure: Percentage of Participants With Value, for Estradiol or Testosterone, Suppressed Below Upper Limit Value (ULV) at the End of Follow-Up Phase
timeFrame: 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
measure: Percentage of Participants With Decease of Ratio of Bone Age to Chronological Age at the End of Enantone Treatment Phase
timeFrame: The mean duration of Enantone exposure was 22.3 months, ranging from 10.1 to 52.4 months
measure: Percentage of Participants With Decease of Ratio of Bone Age to Chronological Age at the End of Follow-up Phase
timeFrame: No longer treated for CPP group - Month: 27 (766- 855 days) post last dose of Enantone; Treated with Non-Enantone GnRHa group - Month 18 (496-585 days) post last dose of Enantone
Other outcomes:
Locations:
city: Wuhan
country: China
facility: The Children's Hospital, Zhejiang University School of Medicine
state: Hubei
city: Changsha
country: China
facility: Childrens Hospital of Hunan province
state: Hunan
city: Nanjing
country: China
facility: Jiangsu Province Hosptial
state: Jiangsu
city: Nanchang
country: China
facility: Children's Hospital of Jiangxi province
state: Jiangxi
city: Shanghai
country: China
facility: Children's Hospital of Shanghai
state: Shanghai
city: Hangzhou
country: China
facility: The Children's Hospital, Zhejiang University School of Medicine
state: Zhejiang
Study officials:
affiliation: Takeda
name: Medical Director Clinical Science
role: STUDY_DIRECTOR
References: