A Study to Assess the Efficacy and Safety of the Triptorelin 6-month Formulation in Paediatric Participants With Central Precocious Puberty.

Central Precocious Puberty Clinical Trial

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05029622
• Other study ID #: D-CN-52014-244
• Status: Completed
• Phase: Phase 3
• Start date: August 10, 2021
• Est. completion date: February 13, 2023

Study information

• Verified date: March 2023
• Source: Ipsen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 66
• Est. completion date: February 13, 2023
• Est. primary completion date: August 21, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 10 Years

Eligibility

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.

Related Conditions & MeSH terms

• Central Precocious Puberty
• Puberty, Precocious

Intervention

Drug:
• Triptorelin Pamoate
Triptorelin 6-month formulation for IM on day 1 and Month 6.

Locations

Country	Name	City	State
China	Beijing Children's Hospital, Capital Medical University	Beijing
China	No.1 Hospital of Jilin University (Bethune first hospital of Jilin University)	Changchun
China	Children's Hospital of Fudan University	Changhai
China	Hunan children's hospital	Changsha
China	Chengdu Women's & Children's Central Hospital	Chengdu
China	Shandong Provincial Hospital	Jinan
China	Linyi Maternal and Child Health Care Hospital	Linyi
China	Jiangxi Provincial Children's Hospital	Nanchang
China	Pingxiang Maternity and Child Care	Pingxiang
China	Children's Hospital of Soochow University	Suzhou
China	Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology	Wuhan
China	Wuhan Children's Hospital Tongji Medical College Huazhong University of Science & Technology	Wuhan
China	Wuxi children's Hospital	Wuxi
China	Zhengzhou Children's Hospital , Henan Children's Hospital	Zhengzhou

Sponsors (1)

Lead Sponsor	Collaborator
Ipsen

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of children with LH (Luteinising Hormone) suppression defined as stimulated peak LH =5 IU/L after GnRH (Gonadotropin-releasing Hormone) stimulation.		At month 6.
Secondary	Proportion of children with LH response to GnRH test.		At months 3 and month 12.
Secondary	Change from basal serum LH levels.		At months 3, 6, 9 and 12.
Secondary	Change from baseline in basal FSH (Follicle-stimulating Hormone) levels		At months 3,6, 9 and 12
Secondary	Change from baseline in peak serum LH levels after the GnRH stimulation test		At months 3, 6 and 12.
Secondary	Change from baseline in peak serum FSH levels after the GnRH stimulation test		At months 3, 6 and 12.
Secondary	Proportion of children with pre-pubertal levels of sex steroids.	Defined as oestradiol =20 pg/mL in girls or testosterone =30 ng/dL in boys	Months 3, 6, 9 and 12.
Secondary	Change from baseline in height-for-age Z-score		At months 6 and 12.
Secondary	Change from baseline in height-for-age percentile		At months 6 and 12.
Secondary	Change from baseline in growth velocity		At months 6 and 12.
Secondary	Proportion of children in whom the BA/CA (Bone Age/Chronological Age) ratio did not rise (X ray).		At months 6 and 12.
Secondary	Change in the ratio BA/CA		At months 6 and 12.
Secondary	Proportion of children who achieve stabilisation of sexual maturation compared to baseline stage using Tanner method.		At months 6 and 12.
Secondary	Proportion of girls with regression of uterine length	Clinical assessment with transabdominal ultrasound	At months 6 and 12.
Secondary	Proportion of boys with absence of progression of testis volumes	Clinical assessment with orchidometer.	At month 6 and 12.
Secondary	Change in BMI (Body Mass Index).		At months 6 and 12.
Secondary	Change in weight.		At months 6 and 12.
Secondary	Incidence of TEAEs (treatment-emergent adverse events), including local tolerability at the injection site.		1 year, including immediately and 2 hours after triptorelin injection.
Secondary	Change in clinical safety laboratory: blood biochemistry parameters. (Creatinine, Non fasting Glucose, Aspartate Aminotransferase (AST), Alanine Aminotransferase (ALT), Alkaline Phosphatase, Total and direct bilirubin, Calcium, Phosphorous),	Any abnormal laboratory test results or other safety assessments, including those that worsen from baseline, considered clinically significant in the medical and scientific judgment of the investigator	At month 3, 6, 9 and 12.
Secondary	Change in clinical safety laboratory haematology parameters (Complete blood count).		At month 3, 6, 9 and 12.
Secondary	Change in clinical safety laboratory urinalysis parameters (Specific gravity, pH, glucose, protein, blood, ketones, bilirubin, urobilinogen, nitrite, leukocyte esterase by dipstick).		At month 3, 6, 9 and 12.
Secondary	Change in physical examination.	A complete physical examination will include, assessments of the cardiovascular, respiratory, gastrointestinal and neurological systems. Height and weight.	At day 1, months 3, 6, 9 and 12.
Secondary	Change in vital signs: Change in heart rate.		At day 1, months 3, 6, 9 and 12.
Secondary	Change in vital signs: Change in blood pressure.		At day 1, months 3, 6, 9 and 12.
Secondary	Sparse plasma triptorelin concentrations		At day 1, months 3, 6 and 12.