Effects of Triptorelin Pamoate in Children With Precocious Puberty - Follow up Study

Study Overview
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Brief Summary
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The purpose of the protocol is to assess the efficacy of triptorelin 11.25 mg with respect to the proportion of children who maintain a regression or stabilisation of sexual maturity until the end of the study.

Official Title
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Follow-up of the Phase III, Multicentre, Non Comparative, One Single Group, Open Study to Assess the Long-term Efficacy and Tolerability of Pamoate of Triptorelin 11.25 mg in Children With Precocious Puberty

Conditions
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Precocious Puberty

Intervention / Treatment
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* Drug: Triptorelin (I.N.N.)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The child must have completed study 2-54-52014-143 The child must have an effective response to 2 injections of triptorelin 11.25 mg according to investigator's evaluation with no significant treatment side effects Exclusion Criteria: The patient has a known hypersensitivity to any of the test materials or related compounds The patient is unable or unwilling to comply fully with the protocol
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Triptorelin<br> | Drug: Triptorelin (I.N.N.)<br>* Decapeptyl® SR 11.25mg<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Children With a Stabilisation or Regression of Tanner Pubertal Stage at the End of the Study (Final Visit), Compared to Pretreatment (Month -6) and Baseline (Month 0) | The primary objective was to assess efficacy of triptorelin pamoate 11.25 mg with respect to percentage of children maintaining a regression or stabilisation of sexual maturity (based on Tanner breast [girls] or genital [boys] pubertal stage) until end of study. Study treatment lasted until end of the therapeutic period; visits for Months 36 and 48 were optional since a child may have already finished the study at a prior visit. The Final Visit only occurred if the child did not end the study by a complete visit such as at Months 24, 36 or 48. Results are presented only for percentage of girls with regression or stabilisation of Tanner breast pubertal stage (n=34). Since only one boy was included in the study, results for this outcome measure were listed only and no statistical analysis was performed. Please also note additional post-hoc analysis for regression or stabilisation of Tanner breast pubertal stage which applied the variable Last Visit on Treatment instead of Final Visit. | Months 12, 24, 36, 48 and Final Visit (if applicable; up to 63 months) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Patients With a Suppressed Luteinizing Hormone (LH) Response to Gonadotropin-Releasing Hormone (GnRH) Test | A suppressed LH response to the GnRH test was defined as a stimulated peak of LH ≤3 international units per litre (IU/L). Percentage of patients who had a suppressed LH response to the GnRH test is reported. It should be noted that almost no hormonal data was collected after Baseline; all data analysed is presented. | Months -6, 0 and 36 | 
| Levels of Oestradiol in Girls or Testosterone in Boys Both Measured by Radioimmunoassay (RIA) | Mean levels of oestradiol in girls or testosterone in boys are reported. It should be noted that almost no hormonal data was collected after Baseline; all data analysed is presented. | Months -6, 0, 12, 36 and Final Visit (up to 63 months) | 
| Percentage of Patients With a Suppressed Follicle Stimulating Hormone (FSH) Response to GnRH Test | A suppressed FSH response to the GnRH test was defined as a stimulated peak of FSH ≤3 IU/L. Percentage of patients who had a suppressed FSH response to the GnRH test is reported. It should be noted that almost no hormonal data was collected after Baseline; all data analysed is presented. | Months -6, 0 and 36 | 
| Body Mass Index (BMI) for Chronological Age Variation | Mean changes of BMI from Pretreatment and from Baseline are reported. It should be noted that only limited patient data was collected after Baseline; all data analysed is presented. | Months -6, 0, 12, 24, 36, 48 and Final Visit (up to 63 months) | 
| BMI Standard Deviation (SD) Score for Chronological Age Variation | Mean changes of BMI SD score from Pretreatment and from Baseline are reported. SD score is a standard term used in growth studies and represents standard deviations calculated as the patient value minus the mean divided by the SD. SD scores vary depending on the age and sex of the child. It should be noted that only limited patient data was collected after Baseline; all data analysed is presented. | Months -6, 0, 12, 24, 36, 48 and Final Visit (up to 63 months) | 
| Auxological Parameters Variations: Height SD Score | Mean changes of height SD score from Pretreatment and from Baseline are reported. SD score is a standard term used in growth studies and represents standard deviations calculated as the patient value minus the mean divided by the SD. SD scores vary depending on the age and sex of the child. It should be noted that only limited patient data was collected after Baseline; all data analysed is presented. | Months -6, 0, 12, 24, 36, 48 and Final Visit (up to 63 months) | 
| Auxological Parameters Variations: Growth Velocity SD Score | Mean changes of growth velocity SD score from Pretreatment and from Baseline are reported. SD score is a standard term used in growth studies and represents standard deviations calculated as the patient value minus the mean divided by the SD. SD scores vary depending on the age and sex of the child. It should be noted that only limited patient data was collected after Baseline; all data analysed is presented. | Months -6, 0, 12, 24, 36, 48 and Final Visit (up to 63 months) | 
| Auxological Parameters Variations: Weight Variation | Mean changes of weight from Pretreatment and from Baseline are reported. It should be noted that only limited patient data was collected after Baseline; all data analysed is presented. | Months -6, 0, 12, 24, 36, 48 and Final Visit (up to 63 months) | 
| Predicted Adult Height SD Score | Mean change of predicted adult height SD score from Pretreatment and from Baseline are reported. SD score is a standard term used in growth studies and represents standard deviations calculated as the patient value minus the mean divided by the SD. SD scores vary depending on the age and sex of the child. It should be noted that only limited patient data was collected after Baseline; all data analysed is presented. Also note that data for this endpoint was analysed for girls only. | Months -6, 0, 12 and Final Visit (up to 63 months) | 
| Bone Age Maturation | Mean change in difference between bone age and chronological age from Pretreatment and from Baseline are reported. It should be noted that only limited patient data was collected after Baseline; all data analysed is presented. | Months -6, 0 and Final Visit (up to 63 months) | 
| Percentage of Girls With a Uterine Length < 36 Millimetres (mm) | Percentage of girls who had a uterine length < 36 mm are reported. It should be noted that only limited patient data was collected after Baseline; all data analysed is presented. | Months -6, 0, 12, 24, 36 and Final Visit (up to 63 months) | 
| Percentage of Children With a Stabilisation or Regression of Tanner Pubic Hair Pubertal Stage at the End of the Study (Final Visit), Compared to Pretreatment (Month -6) and Baseline (Month 0) | Pubic hair was measured by the Tanner method on a scale of 1 to 6. A low grade (i.e. 1) corresponds to a pre-pubertal stage and a high grade (i.e. 5 or 6) to an adult stage. Percentage of patients who had stabilisation or regression (no change in grade or a reduced grade) of Tanner pubic hair pubertal stage is reported. Study treatment was to last until the end of the therapeutic period; visits for Months 36 and 48 were optional because if the girl was already 11 and the boy already 13, they would have finished the study at a prior visit. The Final Visit was to occur only if the child did not end the study by a complete visit such as at Months 24, 36 or 48. Please also note the additional post-hoc analysis for percentage of children with a stabilisation or regression of Tanner pubic hair pubertal stage which applied the variable Last Visit on Treatment instead of Final Visit. | Months 12, 24, 36, 48 and Final Visit (if applicable; up to 63 months) |