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

Primary Purpose

Central Precocious Puberty

Status

Completed

Phase

Phase 3

Locations

China

Study Type

Interventional

Intervention

Triptorelin Pamoate

About this trial

This is an interventional treatment trial for Central Precocious Puberty

Eligibility Criteria

undefined - 10 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

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

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Triptorelin formulation for Intramuscular injection (IM).

Arm Description

Outcomes

Primary Outcome Measures

Proportion of children with LH (Luteinising Hormone) suppression defined as stimulated peak LH ≤5 IU/L after GnRH (Gonadotropin-releasing Hormone) stimulation.

Secondary Outcome Measures

Proportion of children with LH response to GnRH test.

Change from basal serum LH levels.

Change from baseline in basal FSH (Follicle-stimulating Hormone) levels

Change from baseline in peak serum LH levels after the GnRH stimulation test

Change from baseline in peak serum FSH levels after the GnRH stimulation test

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

Change from baseline in height-for-age Z-score

Change from baseline in height-for-age percentile

Change from baseline in growth velocity

Proportion of children in whom the BA/CA (Bone Age/Chronological Age) ratio did not rise (X ray).

Change in the ratio BA/CA

Proportion of children who achieve stabilisation of sexual maturation compared to baseline stage using Tanner method.

Proportion of girls with regression of uterine length

Clinical assessment with transabdominal ultrasound

Proportion of boys with absence of progression of testis volumes

Clinical assessment with orchidometer.

Change in BMI (Body Mass Index).

Change in weight.

Incidence of TEAEs (treatment-emergent adverse events), including local tolerability at the injection site.

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

Change in clinical safety laboratory haematology parameters (Complete blood count).

Change in clinical safety laboratory urinalysis parameters (Specific gravity, pH, glucose, protein, blood, ketones, bilirubin, urobilinogen, nitrite, leukocyte esterase by dipstick).

Change in physical examination.

A complete physical examination will include, assessments of the cardiovascular, respiratory, gastrointestinal and neurological systems. Height and weight.

Change in vital signs: Change in heart rate.

Change in vital signs: Change in blood pressure.

Sparse plasma triptorelin concentrations

Full Information

NCT ID

NCT05029622

First Posted

August 9, 2021

Last Updated

March 15, 2023

Sponsor

Ipsen

1. Study Identification

Unique Protocol Identification Number

NCT05029622

Brief Title

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

2. Study Status

Record Verification Date

March 2023

Overall Recruitment Status

Completed

Study Start Date

August 10, 2021 (Actual)

Primary Completion Date

August 21, 2022 (Actual)

Study Completion Date

February 13, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Ipsen

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Central Precocious Puberty

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

66 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Triptorelin formulation for Intramuscular injection (IM).

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Triptorelin Pamoate

Intervention Description

Triptorelin 6-month formulation for IM on day 1 and Month 6.

Primary Outcome Measure Information:

Title

Proportion of children with LH (Luteinising Hormone) suppression defined as stimulated peak LH ≤5 IU/L after GnRH (Gonadotropin-releasing Hormone) stimulation.

Time Frame

At month 6.

Secondary Outcome Measure Information:

Title

Proportion of children with LH response to GnRH test.

Time Frame

At months 3 and month 12.

Title

Change from basal serum LH levels.

Time Frame

At months 3, 6, 9 and 12.

Title

Change from baseline in basal FSH (Follicle-stimulating Hormone) levels

Time Frame

At months 3,6, 9 and 12

Title

Change from baseline in peak serum LH levels after the GnRH stimulation test

Time Frame

At months 3, 6 and 12.

Title

Change from baseline in peak serum FSH levels after the GnRH stimulation test

Time Frame

At months 3, 6 and 12.

Title

Proportion of children with pre-pubertal levels of sex steroids.

Description

Defined as oestradiol ≤20 pg/mL in girls or testosterone ≤30 ng/dL in boys

Time Frame

Months 3, 6, 9 and 12.

Title

Change from baseline in height-for-age Z-score

Time Frame

At months 6 and 12.

Title

Change from baseline in height-for-age percentile

Time Frame

At months 6 and 12.

Title

Change from baseline in growth velocity

Time Frame

At months 6 and 12.

Title

Proportion of children in whom the BA/CA (Bone Age/Chronological Age) ratio did not rise (X ray).

Time Frame

At months 6 and 12.

Title

Change in the ratio BA/CA

Time Frame

At months 6 and 12.

Title

Proportion of children who achieve stabilisation of sexual maturation compared to baseline stage using Tanner method.

Time Frame

At months 6 and 12.

Title

Proportion of girls with regression of uterine length

Description

Clinical assessment with transabdominal ultrasound

Time Frame

At months 6 and 12.

Title

Proportion of boys with absence of progression of testis volumes

Description

Clinical assessment with orchidometer.

Time Frame

At month 6 and 12.

Title

Change in BMI (Body Mass Index).

Time Frame

At months 6 and 12.

Title

Change in weight.

Time Frame

At months 6 and 12.

Title

Incidence of TEAEs (treatment-emergent adverse events), including local tolerability at the injection site.

Time Frame

1 year, including immediately and 2 hours after triptorelin injection.

Title

Description

Time Frame

At month 3, 6, 9 and 12.

Title

Change in clinical safety laboratory haematology parameters (Complete blood count).

Time Frame

At month 3, 6, 9 and 12.

Title

Change in clinical safety laboratory urinalysis parameters (Specific gravity, pH, glucose, protein, blood, ketones, bilirubin, urobilinogen, nitrite, leukocyte esterase by dipstick).

Time Frame

At month 3, 6, 9 and 12.

Title

Change in physical examination.

Description

A complete physical examination will include, assessments of the cardiovascular, respiratory, gastrointestinal and neurological systems. Height and weight.

Time Frame

At day 1, months 3, 6, 9 and 12.

Title

Change in vital signs: Change in heart rate.

Time Frame

At day 1, months 3, 6, 9 and 12.

Title

Change in vital signs: Change in blood pressure.

Time Frame

At day 1, months 3, 6, 9 and 12.

Title

Sparse plasma triptorelin concentrations

Time Frame

At day 1, months 3, 6 and 12.

10. Eligibility

Sex

All

Maximum Age & Unit of Time

10 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Ipsen Medical Director

Organizational Affiliation

Ipsen

Official's Role

Study Director

Facility Information:

Facility Name

Beijing Children's Hospital, Capital Medical University

City

Beijing

ZIP/Postal Code

100045

Country

China

Facility Name

No.1 Hospital of Jilin University (Bethune first hospital of Jilin University)

City

Changchun

ZIP/Postal Code

130021

Country

China

Facility Name

Children's Hospital of Fudan University

City

Changhai

ZIP/Postal Code

201102

Country

China

Facility Name

Hunan children's hospital

City

Changsha

ZIP/Postal Code

410007

Country

China

Facility Name

Chengdu Women's & Children's Central Hospital

City

Chengdu

ZIP/Postal Code

610073

Country

China

Facility Name

Shandong Provincial Hospital

City

Jinan

ZIP/Postal Code

250021

Country

China

Facility Name

Linyi Maternal and Child Health Care Hospital

City

Linyi

ZIP/Postal Code

276016

Country

China

Facility Name

Jiangxi Provincial Children's Hospital

City

Nanchang

ZIP/Postal Code

330006

Country

China

Facility Name

Pingxiang Maternity and Child Care

City

Pingxiang

ZIP/Postal Code

337000

Country

China

Facility Name

Children's Hospital of Soochow University

City

Suzhou

ZIP/Postal Code

215031

Country

China

Facility Name

Wuhan Children's Hospital Tongji Medical College Huazhong University of Science & Technology

City

Wuhan

ZIP/Postal Code

430015

Country

China

Facility Name

Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology

City

Wuhan

ZIP/Postal Code

430030

Country

China

Facility Name

Wuxi children's Hospital

City

Wuxi

ZIP/Postal Code

214023

Country

China

Facility Name

Zhengzhou Children's Hospital , Henan Children's Hospital

City

Zhengzhou

ZIP/Postal Code

450018

Country

China

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants. Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.

IPD Sharing Time Frame

Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and EU or after the primary manuscript describing the results has been accepted for publication, whichever is later.

IPD Sharing Access Criteria

Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).

IPD Sharing URL

https://vivli.org/members/ourmembers/

Learn more about this trial

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

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