Clinical trial NCT05029622: A Study to Assess the Efficacy and Safety of the Triptorelin 6-month Formulation in Paediatric Participants With Central Precocious Puberty.
Official title: A Phase III, Open-label, Multicentre, Single Arm Study to Assess the Efficacy and Safety of the Triptorelin 6-month Formulation in Chinese Paediatric Participants With Central Precocious Puberty.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Central Precocious Puberty
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2021-08-31
Enrollment: 66
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 purpose of the protocol is to assess the efficacy of the triptorelin 6 month PR (Prolonged Release) formulation in suppressing LH (Luteinising hormone) levels to prepubertal levels (defined as a peak LH ≤5 IU/L) after i.v. GnRH (Gonadotropin-releasing Hormone) stimulation at Month 6 (Day 169) in Chinese children with CPP (Central Precocious Puberty).


Eligibility criteria: Inclusion Criteria:

* Participant is less than 9 years old for girls and less than 10 years old for boys at initiation of triptorelin treatment or at the time of signing the informed consent.
* Participant must present evidence of CPP documented by:
* Onset of development of secondary sex characteristics (breast development in girls or testicular enlargement in boys according to the Tanner method: Stage II) before the age of 8 years in girls and 9 years in boys.
* Pubertal response of LH to GnRH stimulation test (stimulated peak LH ≥6 IU/L) in both sexes.
* Difference between bone age (BA) and CA \>1 year.
* Girls with Tanner staging ≥2 for breast development and who have enlarged uterine length and/or ovarian volume and at last 2 follicles with diameter \>4 mm in the ovary observed by pelvic type B ultrasound at the Screening visit; boys who have testicular volume ≥4 mL observed by testicular orchidometer at the Screening visit.
* Girls who have already had menophania/menarche must have a negative highly sensitive (urine) pregnancy test as required by local regulations within 24 hours before the first dose of study intervention and should not be at risk of pregnancy throughout the study period.

Exclusion Criteria:

* Gonadotropin-independent (peripheral) precocious puberty: extrapituitary secretion of gonadotropins or gonadotropin-independent gonadal or adrenal sex steroid secretion.
* Non-progressing isolated premature thelarche.
* Presence of an unstable intracranial tumour or an intracranial tumour requiring neurosurgery or cerebral irradiation. Participants with hamartomas not requiring surgery are eligible.
* Prior or current therapy with a GnRHa (Gonadotropin-releasing Hormone Agonist) , medroxyprogesterone acetate, growth hormone or insulin-like growth factor 1 (IGF 1).Use of anticoagulants (heparin and coumarin derivatives) within the 2 weeks prior to the Screening visit.

Primary outcomes:
measure: Percentage of Participants With Luteinising Hormone (LH) Suppression
timeFrame: At Month 6
Secondary outcomes:
measure: Percentage of Participants With LH Response to GnRH Test
timeFrame: At Months 3 and 12
measure: Change From Baseline in Basal Serum LH and Follicle-Stimulating Hormone (FSH) Levels
timeFrame: Baseline and at Months 3, 6, 9 and 12
measure: Change From Baseline in Peak Serum LH and FSH Level After the GnRH Stimulation Test
timeFrame: Baseline and at Months 3, 6 and 12
measure: Percentage of Participants With LH Response (Peak LH <=5 IU/L) From Month 6 to Month 12
timeFrame: Month 6 to Month 12
measure: Percentage of Participants With Prepubertal Levels of Sex Steroids
timeFrame: At Months 3, 6, 9 and 12
measure: Change From Baseline in Mean Height for Age (Z-Score)
timeFrame: Baseline and at Months 6 and 12
measure: Change From Baseline in Percentile for Height for Age
timeFrame: Baseline and at Months 6 and 12
measure: Change From Baseline in Growth Velocity
timeFrame: Baseline and at Months 6 and 12
measure: Percentage of Participants With Bone Age (BA)/Chronological Age (CA) Ratio Not Risen
timeFrame: At Months 6 and 12
measure: Change From Baseline in BA:CA Ratio
timeFrame: Baseline, Months 6 and 12
measure: Percentage of Participants With Stabilized Pubertal Stage
timeFrame: At Months 6 and 12
measure: Percentage of Participants With Regression of Uterine Length
timeFrame: At Months 6 and 12
measure: Percentage of Participants With Absence of Progression of Testis Volumes
timeFrame: At Months 6 and 12
measure: Change From Baseline in Body Mass Index (BMI)
timeFrame: Baseline and at Months 6 and 12
measure: Change From Baseline in Weight
timeFrame: Baseline and at Months 6 and 12
measure: Plasma Concentrations of Triptorelin
timeFrame: Day 1, 4 hours post-injection; Month 3; Month 6, predose; Month 6, 4 hours post-injection; and Month 12
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Children's Hospital, Capital Medical University
city: Changchun
country: China
facility: No.1 Hospital of Jilin University (Bethune first hospital of Jilin University)
city: Changhai
country: China
facility: Children's Hospital of Fudan University
city: Changsha
country: China
facility: Hunan children's hospital
city: Chengdu
country: China
facility: Chengdu Women's & Children's Central Hospital
city: Jinan
country: China
facility: Shandong Provincial Hospital
city: Linyi
country: China
facility: Linyi Maternal and Child Health Care Hospital
city: Nanchang
country: China
facility: Jiangxi Provincial Children's Hospital
city: Pingxiang
country: China
facility: Pingxiang Maternity and Child Care
city: Suzhou
country: China
facility: Children's Hospital of Soochow University
city: Wuhan
country: China
facility: Wuhan Children's Hospital Tongji Medical College Huazhong University of Science & Technology
city: Wuhan
country: China
facility: Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
city: Wuxi
country: China
facility: Wuxi children's Hospital
city: Zhengzhou
country: China
facility: Zhengzhou Children's Hospital , Henan Children's Hospital
Study officials:
affiliation: Ipsen
name: Ipsen Medical Director
role: STUDY_DIRECTOR
References: