Clinical Trial: A Study to Assess the Safety and Efficacy of Leuprorelin in Central Precocious Puberty Among Chinese Participants

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Observational

Official Title: An Observational, Retrospective Study to Evaluate the Long Term Safety and Effectiveness of Leuprorelin in the Treatment of Central Precocious Puberty

Brief Summary: The purpose of this study is to assess long-term safety and efficacy of leuprorelin in the treatment of Central Precocious Puberty (CPP) in Chinese participants.

Detailed Summary:

The drug being tested in this study is called leuprorelin. Leuprorelin is being researched 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 patients. Participants will be assigned to following groups based on the dose received per body weight:

  • Leuprorelin low dose group
  • Leuprorelin high dose group

All participants who have received leuprorelin 30 μg/kg to <90 μg/kg or 90 μg/kg to 180 μg/kg per body weight, injection, subcutaneously, every 4 weeks up to at least 9 months during the index period from September 1st 1998 to September 30th 2017 will be observed.

This multi-center trial will be conducted in China. Data will be collected over period of 19 months.


Sponsor: Takeda

Current Primary Outcome:

  • Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline up to 19 years ]
    An Adverse Event (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. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. An SAE is defined as any untoward medical occurrence or effect that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect or is medically important due to other reasons than the above mentioned criteria.
  • Percentage of Participants who had Regression or no Progression in Tanner Staging [ Time Frame: Last observation prior to entry into follow up phase in index period (up to 19 years) ]
    Tanner assessment score is used to document the stage of development of secondary sexual characteristics. Female pubertal development staged by pubic hair development and breast size; male pubertal development staged by size of the genitalia and development of pubic hair. Rated in 5 stages: stage 1 (no development) to 5 (adult-like development in quantity and size). Regression or no progression is defined as negative change (improvement) or no change in Tanner score at last observation prior to entry into fo

    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • Percentage of Participants With Luteinizing Hormone (LH) and Follicle Stimulating Hormone (FSH) Suppression to Pre-pubertal Level [ Time Frame: Last observation prior to entry into follow up phase in index period (up to 19 years) ]
      A lab test is conducted to measure LH and FSH levels. Levels are compared to individual pre-puberty levels.
    • Percentage of Participants With Estradiol or Testosterone Level Suppression to Pre-pubertal Level [ Time Frame: Last observation prior to entry into follow up phase in index period (up to 19 years) ]
      A lab test is conducted to measure estradiol and testosterone levels. Levels are compared to individual pre-puberty levels.
    • Percentage of Participants With a Decrease From Baseline in the Ratio of Bone age to Chronological age (BA/CA ratio) [ Time Frame: Last observation prior to entry into follow up phase in index period (up to 19 years) ]
      Bone age (BA) is estimated locally using an X-ray (bone scan). Chronological age (CA) at the date of corresponding X-ray (Date of X-ray - Date of birth)/365.25. Ratio of BA/CA is calculated at each annual study visit.
    • Percentage of Participants With an Increase From Baseline in Predicted Adult Height [ Time Frame: Last observation prior to entry into follow up phase in index period (up to 19 years) ]
      Predicted adult height is calculated using the Bayley-Pinneau charts with skeletal age assessment (SAA). SAA was calculated using the Greulich and Pyle (GP) or Tanner and Whitehouse (TW) method. The height of the participants were measured at the site. Standard deviation score (SDS) report the number of standard deviations from the mean for age and sex for an individual measurement (normal range: -2 to +2 SDS). Height SDS is derived by subtracting the population mean from individual's height value and then dividing that difference by the population standard deviation to derive Bayley-Pinneau charts.
    • Change From Baseline in Laboratory Parameters During the Index Period [ Time Frame: Baseline and up to 19 years ]
      Any abnormal findings in laboratory parameters is collected throughout the study.
    • Percentage of Participants With Polycystic Ovarian Syndrome [ Time Frame: Baseline up to 19 years ]
      Polycystic ovarian syndrome is assessed using pelvic ultrasonography.
    • Percentage of Participants With Long Term Effect on Reproduction [ Time Frame: Baseline up to 19 years ]
      Long term effect on reproduction is assessed by observing fertility condition (ovarian volume and follicle counts in females or testicular volume in males).
    • Change From Baseline in Bone Mineral Density During the Treatment Phase [ Time Frame: Baseline and up to 19 years ]
      Bone mineral density is determined using dual energy X-ray bone densitometry. Change relative to baseline in participant's bone mineral density measured throughout study.
    • Mean Change From end of Treatment Phase in Bone Mineral Density at Yearly Intervals of the Follow up Phase [ Time Frame: End of treatment phase and each yearly interval of the follow up phase in index period (up to 19 years) ]
      Bone mineral density is determined using dual energy X-ray bone densitometry. Change relative to baseline in participant's bone mineral density measured throughout study.
    • Change From Baseline in Body Mass Index (BMI) During the Treatment Phase [ Time Frame: Baseline and up to 19 years ]
      The BMI is weight in kilograms divided by height in meters squared (kg/m^2). Change relative to baseline in participant's BMI measured throughout study.
    • Change From end of Treatment Phase in BMI During at Yearly Intervals of the Follow up Phase [ Time Frame: End of treatment phase and each yearly interval of the follow up phase in index period (up to 19 years) ]
      The BMI is weight in kilograms divided by height in meters squared (kg/m^2). Change relative to baseline in participant's BMI measured throughout study.
    • Percentage of Participants With Injection Site Reaction [ Time Frame: Baseline up to 19 years ]


    Original Secondary Outcome: Same as current

    Information By: Takeda

    Dates:
    Date Received: December 13, 2016
    Date Started: May 22, 2017
    Date Completion: February 27, 2018
    Last Updated: May 18, 2017
    Last Verified: May 2017