Clinical Trial Results:
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
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Summary
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EudraCT number |
2022-002963-31 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
03 Sep 2022
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Results information
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Results version number |
v1(current) |
This version publication date |
20 Mar 2023
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First version publication date |
20 Mar 2023
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
D-CN-52014-243
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT04736602 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Ipsen Pharma
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Sponsor organisation address |
65, quai Georges Gorse, Boulogne Billancourt, France, 92100
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Public contact |
Medical Director, Ipsen Pharma, clinical.trials@ipsen.com
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Scientific contact |
Medical Director, Ipsen Pharma, clinical.trials@ipsen.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
03 Sep 2022
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
03 Sep 2022
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The purpose of this study was to assess the efficacy of the triptorelin 3-month prolonged release (PR) formulation in suppressing luteinising hormone (LH) levels to pre-pubertal levels [defined as a peak LH ≤3 international units per liter (IU/L)] after intravenous (IV) gonadotropin-releasing hormone (GnRH) stimulation at Month 3 in Chinese children with central precocious puberty (CPP).
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Protection of trial subjects |
The study was conducted under the provisions of the Declaration of Helsinki, in accordance with the Good Clinical Practice of China and in compliance with ethics committee and informed consent regulations.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
27 Mar 2021
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
China: 32
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Worldwide total number of subjects |
32
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EEA total number of subjects |
0
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
32
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
This prospective, Phase 3, open-label, single arm, 2-phase (main study phase and an extension phase) study was conducted in children with central precocious puberty (CPP) at 6 investigational sites in China. | ||||||||||
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Pre-assignment
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Screening details |
This study had two phases: main study phase and extension phase. Study consisted of screening period (up to 28 days), main study phase (6 months) and an extension phase (6 months). A total of 32 participants were enrolled in this study. | ||||||||||
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Period 1
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Period 1 title |
Main study phase
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Is this the baseline period? |
Yes | ||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||
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Arms
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Arm title
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All Participants | ||||||||||
Arm description |
Participants received triptorelin pamoate 15 milligrams (mg) intramuscular (IM) injection on Day 1 visit and Month 3 visit (Day 91) during the main study phase. Participants had an option to continue with the triptorelin pamoate in extension phase of the study. As per Investigator decision, eligible participants continued to receive IM injections of triptorelin pamoate on Month 6 (Day 182) and Month 9 (Day 271) during the extension phase. | ||||||||||
Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
Triptorelin pamoate
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Investigational medicinal product code |
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Other name |
Diphereline®
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Pharmaceutical forms |
Powder and solvent for suspension for injection
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Routes of administration |
Intramuscular use
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Dosage and administration details |
Triptorelin pamoate was administered as an IM injection of 4 milliliter (mL) sterile lyophilizate of microparticles and 2 mL of sterile solvent, containing 15 mg dose.
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Period 2
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Period 2 title |
Extension phase
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Is this the baseline period? |
No | ||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||
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Arms
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Arm title
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All Participants | ||||||||||
Arm description |
Participants received triptorelin pamoate 15 mg IM injection on Day 1 visit and Month 3 visit (Day 91) during the main study phase. Participants had an option to continue with the triptorelin pamoate in extension phase of the study. As per Investigator decision, eligible participants continued to receive IM injections of triptorelin pamoate on Month 6 (Day 182) and Month 9 (Day 271) during the extension phase. | ||||||||||
Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
Triptorelin pamoate
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Investigational medicinal product code |
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Other name |
Diphereline®
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Pharmaceutical forms |
Powder and solvent for suspension for injection
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Routes of administration |
Intramuscular use
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Dosage and administration details |
Triptorelin pamoate was administered as an IM injection of 4 mL sterile lyophilizate of microparticles and 2 mL of sterile solvent, containing 15 mg dose.
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Baseline characteristics reporting groups
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Reporting group title |
All Participants
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Reporting group description |
Participants received triptorelin pamoate 15 milligrams (mg) intramuscular (IM) injection on Day 1 visit and Month 3 visit (Day 91) during the main study phase. Participants had an option to continue with the triptorelin pamoate in extension phase of the study. As per Investigator decision, eligible participants continued to receive IM injections of triptorelin pamoate on Month 6 (Day 182) and Month 9 (Day 271) during the extension phase. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
All Participants
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Reporting group description |
Participants received triptorelin pamoate 15 milligrams (mg) intramuscular (IM) injection on Day 1 visit and Month 3 visit (Day 91) during the main study phase. Participants had an option to continue with the triptorelin pamoate in extension phase of the study. As per Investigator decision, eligible participants continued to receive IM injections of triptorelin pamoate on Month 6 (Day 182) and Month 9 (Day 271) during the extension phase. | ||
Reporting group title |
All Participants
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Reporting group description |
Participants received triptorelin pamoate 15 mg IM injection on Day 1 visit and Month 3 visit (Day 91) during the main study phase. Participants had an option to continue with the triptorelin pamoate in extension phase of the study. As per Investigator decision, eligible participants continued to receive IM injections of triptorelin pamoate on Month 6 (Day 182) and Month 9 (Day 271) during the extension phase. | ||
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End point title |
Percentage of Participants With LH Suppression After GnRH Stimulation [1] | ||||||||
End point description |
The LH suppression was defined as stimulated peak LH ≤3 IU/L. The GnRH stimulation test was performed by using an intravenous (IV) injection of gonadorelin (synthetic GnRH) to stimulate gonadotrophin release and blood samples were collected after the gonadorelin injection for central assessment of serum LH levels. The modified ITT (mITT) population consisted of all treated participants with at least 1 baseline and month 3 post-baseline assessment of the primary efficacy endpoint.
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End point type |
Primary
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End point timeframe |
At Month 3
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No additional statistical analysis was prespecified for this endpoint. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Change From Baseline in Basal LH and Follicle-Stimulating Hormone (FSH) Serum Levels | ||||||||||||||||||||||||
End point description |
Basal LH and FSH serum concentrations were analyzed centrally. Change from baseline was defined as the values for LH and FSH at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first dose administered. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data from the participants analyzed were reported. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 3, 6, 9 and 12
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| No statistical analyses for this end point | |||||||||||||||||||||||||
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End point title |
Percentage of Participants With LH Suppression After GnRH Stimulation | ||||||||||||
End point description |
A synthetic GnRH (gonadorelin) was used for gonadotrophin stimulation. Blood samples were collected prior to gonadorelin injection (timepoint T0) and at 30 minutes (T30), 60 minutes (T60) and 90 minutes (T90) (±5 minutes at each timepoint) after a single IV injection of gonadorelin. A suppressed LH response to GnRH stimulation test was defined as peak LH ≤3 IU/L among the 4 timepoints (T0, T30, T60 and T90). The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
At Months 6 and 12
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Change From Baseline in Peak LH and FSH Level After GnRH Stimulation | ||||||||||||||||||||
End point description |
A synthetic GnRH were used for gonadotrophin stimulation. Blood samples were collected prior to gonadorelin injection (timepoint T0) and at 30 minutes (T30), 60 minutes (T60) and 90 minutes (T90) (±5 minutes at each timepoint) after a single IV injection of gonadorelin. The FSH response to GnRH stimulation was the peak FSH level among the 4 timepoints (T0, T30, T60 and T90). The LH response to GnRH stimulation test was defined as peak LH ≤3 IU/L among the 4 timepoints T0, T30, T60 and T90). Baseline was defined as the last non-missing measurement taken prior to the first dose administered. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data from the participants analyzed were reported. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 3, 6 and 12
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| No statistical analyses for this end point | |||||||||||||||||||||
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End point title |
Percentage of Participants With Prepubertal Levels of Sex Steroids | ||||||||||||||||
End point description |
Prepubertal sex steroids assessment included estradiol in female participants and testosterone in male participants. Prepubertal sex steroids levels were defined as: estradiol ≤20 picogram (pg)/mL in female participants and testosterone ≤0.3 nanogram (ng)/mL in male participants. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data from the participants analyzed were reported. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
At Months 3, 6, 9 and 12
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| No statistical analyses for this end point | |||||||||||||||||
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End point title |
Change From Baseline in Estradiol Levels | ||||||||||||||||
End point description |
Estradiol serum concentration was analyzed centrally. Change from baseline was defined as the value for estradiol levels at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first dose administered. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data for female participants were analyzed. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 3, 6 ,9 and 12
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| No statistical analyses for this end point | |||||||||||||||||
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End point title |
Change From Baseline in Testosterone Levels | ||||||||||||||||
End point description |
Testosterone serum concentration was analyzed centrally. Change from baseline was defined as the value for testosterone levels at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first dose administered. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data for male participants were analyzed.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 3, 6 ,9 and 12
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| No statistical analyses for this end point | |||||||||||||||||
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End point title |
Percentage of Participants With Change From Baseline in Pubertal Stage | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Pubertal stage parameters were analyzed using Tanner method. Pubertal stage parameters included genital stage in male participants, breast stage in female participants and pubic hair stage in both sexes. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data from the participants analyzed were reported. Here, ‘n’ = number of participants analyzed at specific time point. Breast development stage (BDS), Genital development stage (GDS), Pubic hair development (PHD), Month 6 (M6), Month 12 (M12).
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 6 and 12
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End point title |
Percentage of Participants With Stabilized Pubertal Stage Compared to Baseline | ||||||||||||||||||||||||
End point description |
Pubertal stage parameters were analyzed using Tanner method. Pubertal stage parameters included genital stage in male participants, breast stage in female participants and pubic hair stage in both sexes. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 6 and 12
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| No statistical analyses for this end point | |||||||||||||||||||||||||
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End point title |
Change From Baseline in Auxological Parameter: Height | ||||||||||||||||
End point description |
Auxological parameter including height was analyzed. Change from baseline was defined as the value for each auxological parameter at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first dose administered. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data from the participants analyzed were reported. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 3, 6, 9 and 12
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| No statistical analyses for this end point | |||||||||||||||||
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End point title |
Change From Baseline in Auxological Parameter: Weight | ||||||||||||||||
End point description |
Auxological parameter including weight was analyzed. Change from baseline was defined as the value for each auxological parameter at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first dose administered. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data from the participants analyzed were reported. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 3, 6, 9 and 12
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| No statistical analyses for this end point | |||||||||||||||||
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End point title |
Change From Baseline in Auxological Parameter: Growth Velocity | ||||||||||||||||
End point description |
Auxological parameter including growth velocity was analyzed. Change from baseline was defined as the value for each auxological parameter at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first dose administered. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data from the participants analyzed were reported. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 3, 6, 9 and 12
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| No statistical analyses for this end point | |||||||||||||||||
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End point title |
Change From Baseline in Auxological Parameter: Body Mass Index (BMI) | ||||||||||||||||
End point description |
Auxological parameter including BMI was analyzed. Change from baseline was defined as the value for each auxological parameter at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first dose administered. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data from the participants analyzed were reported. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 3, 6, 9 and 12
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| No statistical analyses for this end point | |||||||||||||||||
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End point title |
Change From Baseline in Bone Age (BA) | ||||||||||||
End point description |
BA was determined using X-rays of the hand and wrist. Change from baseline was defined as the value for BA at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first dose administered. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data from the participants analyzed were reported. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 6 and 12
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Change From Baseline Difference Between BA and Chronological Age (CA) | ||||||||||||
End point description |
BA was determined using X-rays of the hand and wrist. Change from baseline was defined as the difference between BA and CA value at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first dose administered. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data from the participants analyzed were reported. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 6 and 12
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Change From Baseline in Uterine Length | ||||||||||||
End point description |
Uterine length was determined by type B ultrasound. Change from baseline was defined as the value of uterine length at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first dose administered. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data from the female participants analyzed were reported. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 6 and12
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Change From Baseline of Testicular Volume | ||||||||||||||||
End point description |
Testicular volume was determined by type B ultrasound. Change from baseline was defined as the value of testicular volume at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first dose administered. The mITT population consisted of all treated participants with at least 1 baseline and Month 3 post-baseline assessment of the primary efficacy endpoint. Only data from the male participants analyzed were reported. 9999 indicates that standard deviation could not be calculated as only 1 participant was analyzed. Here, ‘n’ = number of participants analyzed at specific time point.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1) and at Months 6 and 12
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| No statistical analyses for this end point | |||||||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to end of extension phase, a maximum of approximately 368 days.
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Adverse event reporting additional description |
Safety population consisted of all participants who received at least 1 dose of study medication and had at least 1 post-baseline safety assessment.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
25.0
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Reporting groups
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Reporting group title |
All Participants
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Reporting group description |
Participants received triptorelin pamoate 15 mg IM injection on Day 1 visit and Month 3 visit (Day 91) during the main study phase. Participants had an option to continue with the triptorelin pamoate in extension phase of the study. As per Investigator decision, eligible participants continued to receive IM injections of triptorelin pamoate on Month 6 (Day 182) and Month 9 (Day 271) during the extension phase. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 0% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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28 Oct 2020 |
Introduction of extension study available for participants. Clarified how safety profile were analyzed. Extra assessments were removed since the population was at very low risk of pregnancy. For participant centricity to reduced blood draw burden and removed the requirement for children to fast. Measurement of uterine length as measurement of gonad development was normal clinical practice. For participant centricity, increasing the larger window was maximize flexibility for children at school. To clarify timing of this assessment. |
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16 Mar 2021 |
Added information regarding the extension phase and provide other minor changes to improved clarity regarding the main and extension phases of the study. Amended to provided distinction between the main study phase and the extension study phase. Added the requirement of reporting serious adverse events with the electronic data collection tool. The blood chemistry was to be done at site local lab, it may be different practice in different sites. |
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09 Jul 2021 |
Additional ±5 minutes time window for blood sample collection time point for GnRH stimulation test was added. Added temporary discontinuation due to COVID-19. Added the definition of lost follow-up and the action to be taken. Added requirement to record information on subjects who failed screening. Added medication or procedure that are not permitted. Added operational requirements for on-site administration. |
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Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
