Back to results

Efficacy and Safety Study of Triptorelin 3-Month Formulation in Chinese Children With Central Precocious Puberty.

Primary Purpose

Central Precocious Puberty

Status

Completed

Phase

Phase 3

Locations

China

Study Type

Interventional

Intervention

Triptorelin pamoate 15mg

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:

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.

Sites / Locations

Chengdu Women's and Children's Central Hospital
Jiangxi Provincial Children's Hospital
Children's Hospital of Nanjing
Tangshan Maternal & Child Health Hospital
Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
Wuxi Children's Hospital
Henan Children's Hospital, Zhengzhou Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Triptorelin Pamoate 15mg for injection

Arm Description

Triptorelin will be injected at day 1 and month 3. If participants are willing to enter extension phase, two additional Triptorelin injections will be given at month 6 and month 9.

Outcomes

Primary Outcome Measures

Proportion of children with Luteinising Hormone (LH) suppression defined as stimulated peak LH ≤3 IU/L after Gonadotropin-releasing Hormone (GnRH) stimulation

Secondary Outcome Measures

Basal Luteinising Hormone (LH) and Follicle Stimulating Hormone (FSH) serum levels

Peak LH after GnRH stimulation test

Peak FSH levels after GnRH stimulation test

Oestradiol or testocterone serum concentration

Pubertal stage (Tanner Method)

Auxological parameters (height, growth velocity, weight, BMI)

Bone Age

Gonad development (Uterine length or testis volume)

Proportion of children with LH suppression defined as stimulated peak LH ≤3 IU/L after GnRH stimulation

Proportion of children with pre-pubertal levels of sex steroids (defined as oestradiol ≤20 pg/mL in girls and testosterone ≤0.3 ng/mL in boys)

Proportion of children with stabilised pubertal stage compared to baseline stage using the Tanner method

Full Information

NCT ID

NCT04736602

First Posted

January 29, 2021

Last Updated

October 12, 2022

Sponsor

Ipsen

1. Study Identification

Unique Protocol Identification Number

NCT04736602

Brief Title

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

2. Study Status

Record Verification Date

October 2022

Overall Recruitment Status

Completed

Study Start Date

March 27, 2021 (Actual)

Primary Completion Date

February 9, 2022 (Actual)

Study Completion Date

September 3, 2022 (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 this study is to observe whether the Triptorelin pamoate 15mg (3-month formulation) effectiveness in Chinese population of CPP children has the same or similar trend with that in overseas CPP population. This is measured by assessing the proportion of children who have a suppressed Luteinizing Hormone (LH) response to Gonadotropin Releasing Hormone (GnRH) test performed 3 months after injection of triptorelin.

Detailed Description

For patients enter extension phase, two additional Triptorelin injections will be given at month 6 and month 9 visits.

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

32 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Triptorelin Pamoate 15mg for injection

Arm Type

Experimental

Arm Description

Triptorelin will be injected at day 1 and month 3. If participants are willing to enter extension phase, two additional Triptorelin injections will be given at month 6 and month 9.

Intervention Type

Drug

Intervention Name(s)

Triptorelin pamoate 15mg

Intervention Description

Intramuscular injection (IM)

Primary Outcome Measure Information:

Title

Proportion of children with Luteinising Hormone (LH) suppression defined as stimulated peak LH ≤3 IU/L after Gonadotropin-releasing Hormone (GnRH) stimulation

Time Frame

3 months after first injection of study drug (for main study)

Secondary Outcome Measure Information:

Title

Basal Luteinising Hormone (LH) and Follicle Stimulating Hormone (FSH) serum levels

Time Frame

Baseline, Month 3 and Month 6 (End of Study of main study), for the extension phase study: month 9 and month 12

Title

Peak LH after GnRH stimulation test

Time Frame

Baseline, Month 3 and Month 6 (End of Study of main study), for the extension phase study: month 12]

Title

Peak FSH levels after GnRH stimulation test

Time Frame

Baseline, Month 3 and Month 6 (End of Study of main study), for the extension phase study: month 12.

Title

Oestradiol or testocterone serum concentration

Time Frame

Baseline, Month 3 and Month 6 (End of Study of main study), for the extension phase study month 9 and month 12.

Title

Pubertal stage (Tanner Method)

Time Frame

Baseline and Month 6 (End of Study of main study) and month 12 for the extension phase study.

Title

Auxological parameters (height, growth velocity, weight, BMI)

Time Frame

Baseline, Month 3 and Month 6 (End of Study of main study), for the extension phase study: month 9 and 12.

Title

Bone Age

Time Frame

Baseline and Month 6 (End of Study of main study) and month 12 for the extension phase study.

Title

Gonad development (Uterine length or testis volume)

Time Frame

Baseline and Month 6 (End of Study of main study) and month 12 for the extension phase study.

Title

Proportion of children with LH suppression defined as stimulated peak LH ≤3 IU/L after GnRH stimulation

Time Frame

Month 6 of main study and at month 12 for the extension phase study.

Title

Proportion of children with pre-pubertal levels of sex steroids (defined as oestradiol ≤20 pg/mL in girls and testosterone ≤0.3 ng/mL in boys)

Time Frame

Month 3 and Month 6 for the main study and for the extension phase study: month 9 and month 12.

Title

Proportion of children with stabilised pubertal stage compared to baseline stage using the Tanner method

Time Frame

Month 6 for the main study and month 12 for the extension phase study.

10. Eligibility

Sex

All

Maximum Age & Unit of Time

10 Years

Accepts Healthy Volunteers

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.

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

Chengdu Women's and Children's Central Hospital

City

Chengdu

ZIP/Postal Code

610073

Country

China

Facility Name

Jiangxi Provincial Children's Hospital

City

Nanchang

ZIP/Postal Code

330006

Country

China

Facility Name

Children's Hospital of Nanjing

City

Nanjing

ZIP/Postal Code

210008

Country

China

Facility Name

Tangshan Maternal & Child Health Hospital

City

Tangshan

ZIP/Postal Code

063000

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

Henan Children's Hospital, Zhengzhou 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

Efficacy and Safety Study of Triptorelin 3-Month Formulation in Chinese Children With Central Precocious Puberty.

We'll reach out to this number within 24 hrs