Clinical trial NCT04736602: Efficacy and Safety Study of Triptorelin 3-Month Formulation in Chinese Children With Central Precocious Puberty.
Official title: An Open-label, Multicentre, Single Arm Study to Assess the Efficacy and Safety of Triptorelin 3-month Formulation in Chinese Children With Central Precocious Puberty
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Central Precocious Puberty
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2021-02-03
Enrollment: 32
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 this research was to confirm the effectiveness and safety of the study drug, Triptorelin pamoate 15mg 3-month formulation, in a Chinese population of Central Precocious Puberty (CPP) children.


Eligibility criteria: Inclusion Criteria:

* Onset of development of secondary sex characteristics before 8 and 9 years in girls and boys, respectively breast development in girls or testicular enlargement in boys according to the Tanner method: Stage II
* Pubertal response of LH to GnRH stimulation test (stimulated peak LH ≥5 IU/L)
* Difference between bone age (BA) (according to Greulich and Pyle method) and chronological age (CA) \>1 year
* Girls with Tanner staging ≥2 for breast development and enlarged uterine length and several follicles with diameter \>4 mm in the ovary at Screening visit; boys who have testicular volume ≥4 mL at Screening visit
* Age \< 9 years old for girls and \< 10 years old for boys at initiation of triptorelin treatment
* Weight at least 20 kg
* Subjects will qualify for the extension phase if they sign the corresponding specific consent form, are still benefiting from treatment at the end the primary study and have not experienced any unacceptable safety issues.

Exclusion Criteria:

* Gonadotropin-independent (peripheral) precocious puberty: extra pituitary 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
* Evidence of renal (creatinine \>1.5 x upper limit of normal (ULN)) or hepatic impairment (bilirubin \>1.5 x ULN or alanine aminotransferase (ALT)/aspartate transaminase (AST) \>3 x ULN)
* Any other condition or chronic illness or treatment possibly interfering with growth or other study endpoints (e.g. chronic steroid use except topical steroids, renal failure, diabetes, moderate to severe scoliosis)
* Prior or current therapy with a GnRH agonist (GnRHa), medroxyprogesterone acetate, growth hormone or insulin-like growth factor-1 (IGF-1)
* Diagnosis of short stature, i.e. \>2.25 standard deviation (SD) below the mean height for age
* Major medical or psychiatric illness that could interfere with study visits
* Known hypersensitivity to any of the test materials or related compounds
* 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 After Gonadotropin-Releasing Hormone (GnRH) Stimulation
timeFrame: At Month 3
Secondary outcomes:
measure: Change From Baseline in Basal LH and Follicle-Stimulating Hormone (FSH) Serum Levels
timeFrame: Baseline (Day 1) and at Months 3, 6, 9 and 12
measure: Percentage of Participants With LH Suppression After GnRH Stimulation
timeFrame: At Months 6 and 12
measure: Change From Baseline in Peak LH and FSH Level After GnRH Stimulation
timeFrame: Baseline (Day 1) and at Months 3, 6 and 12
measure: Percentage of Participants With Prepubertal Levels of Sex Steroids
timeFrame: At Months 3, 6, 9 and 12
measure: Change From Baseline in Estradiol Levels
timeFrame: Baseline (Day 1) and at Months 3, 6 ,9 and 12
measure: Change From Baseline in Testosterone Levels
timeFrame: Baseline (Day 1) and at Months 3, 6 ,9 and 12
measure: Percentage of Participants With Change From Baseline in Pubertal Stage
timeFrame: Baseline (Day 1) and at Months 6 and 12
measure: Percentage of Participants With Stabilized Pubertal Stage Compared to Baseline
timeFrame: Baseline (Day 1) and at Months 6 and 12
measure: Change From Baseline in Auxological Parameter: Height
timeFrame: Baseline (Day 1) and at Months 3, 6, 9 and 12
measure: Change From Baseline in Auxological Parameter: Weight
timeFrame: Baseline (Day 1) and at Months 3, 6, 9 and 12
measure: Change From Baseline in Auxological Parameter: Growth Velocity
timeFrame: Baseline (Day 1) and at Months 3, 6, 9 and 12
measure: Change From Baseline in Auxological Parameter: Body Mass Index (BMI)
timeFrame: Baseline (Day 1) and at Months 3, 6, 9 and 12
measure: Change From Baseline in Bone Age (BA)
timeFrame: Baseline (Day 1) and at Months 6 and 12
measure: Change From Baseline Difference Between BA and Chronological Age (CA)
timeFrame: Baseline (Day 1) and at Months 6 and 12
measure: Change From Baseline in Uterine Length
timeFrame: Baseline (Day 1) and at Months 6 and12
measure: Change From Baseline of Testicular Volume
timeFrame: Baseline (Day 1) and at Months 6 and 12
Other outcomes:
Locations:
city: Chengdu
country: China
facility: Chengdu Women's and Children's Central Hospital
city: Nanchang
country: China
facility: Jiangxi Provincial Children's Hospital
city: Nanjing
country: China
facility: Children's Hospital of Nanjing
city: Tangshan
country: China
facility: Tangshan Maternal & Child Health Hospital
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: Henan Children's Hospital, Zhengzhou Children's Hospital
Study officials:
affiliation: Ipsen
name: Ipsen Medical Director
role: STUDY_DIRECTOR
References: