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

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

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00909844

Acronym

DECAPUB

Enrollment

Registered

2009-05-29

Start date

2008-04-30

Completion date

2016-01-31

Last updated

2019-01-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Precocious Puberty

Brief summary

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.

Interventions

DRUGTriptorelin (I.N.N.)

Decapeptyl® SR 11.25mg

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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)	Months 12, 24, 36, 48 and Final Visit (if applicable; up to 63 months)	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.

Secondary

Measure	Time frame	Description
Levels of Oestradiol in Girls or Testosterone in Boys Both Measured by Radioimmunoassay (RIA)	Months -6, 0, 12, 36 and Final Visit (up to 63 months)	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.
Percentage of Patients With a Suppressed Follicle Stimulating Hormone (FSH) Response to GnRH Test	Months -6, 0 and 36	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.
Body Mass Index (BMI) for Chronological Age Variation	Months -6, 0, 12, 24, 36, 48 and Final Visit (up to 63 months)	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.
BMI Standard Deviation (SD) Score for Chronological Age Variation	Months -6, 0, 12, 24, 36, 48 and Final Visit (up to 63 months)	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.
Auxological Parameters Variations: Height SD Score	Months -6, 0, 12, 24, 36, 48 and Final Visit (up to 63 months)	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.
Percentage of Patients With a Suppressed Luteinizing Hormone (LH) Response to Gonadotropin-Releasing Hormone (GnRH) Test	Months -6, 0 and 36	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.
Auxological Parameters Variations: Weight Variation	Months -6, 0, 12, 24, 36, 48 and Final Visit (up to 63 months)	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.
Predicted Adult Height SD Score	Months -6, 0, 12 and Final Visit (up to 63 months)	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.
Bone Age Maturation	Months -6, 0 and Final Visit (up to 63 months)	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.
Percentage of Girls With a Uterine Length < 36 Millimetres (mm)	Months -6, 0, 12, 24, 36 and Final Visit (up to 63 months)	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.
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)	Months 12, 24, 36, 48 and Final Visit (if applicable; up to 63 months)	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.
Auxological Parameters Variations: Growth Velocity SD Score	Months -6, 0, 12, 24, 36, 48 and Final Visit (up to 63 months)	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.

Countries

France

Participant flow

Recruitment details

The study was designed as a multicentre study and included 10 investigational sites in France. This follow up study was to start on the day of the last visit (Month 6) of the phase III 2-54-52014-143 study and was to end when the Investigator judged that the patient had completed his/her treatment, i.e. at around 11 years in girls and 13 in boys.

Pre-assignment details

A maximum of 35 patients could be included in this study (i.e. the number of patients who had completed the phase III 2-54-52014-143 study). A total of 35 patients were screened and enrolled in this current study (2-54-52014-159).

Participants by arm

Arm	Count
Triptorelin Pamoate 11.25 mg 11.25 mg triptorelin pamoate (prolonged release formulation) was administered via intramuscular (i.m.) injection once every 3 months from Baseline until end of the study treatment.	35
Total	35

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Adverse Event	1
Overall Study	Lost to Follow-up	2
Overall Study	Withdrawal by Subject	1

Baseline characteristics

Characteristic	Triptorelin Pamoate 11.25 mg
Age, Continuous	8.73 years STANDARD_DEVIATION 1.07
Sex: Female, Male Female	34 Participants
Sex: Female, Male Male	1 Participants
Weight at Pretreatment	32.4 kilogram (kg) STANDARD_DEVIATION 6.9

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	16 / 35
serious Total, serious adverse events	3 / 35

Outcome results

Primary

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.

Time frame: Months 12, 24, 36, 48 and Final Visit (if applicable; up to 63 months)

Population: Analysis performed on female patients in the ITT population, consisting of all enrolled female patients who received at least one injection of study treatment in this follow up study.

Arm	Measure	Group	Value (NUMBER)
Triptorelin Pamoate 11.25 mg	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)	Compared to Pretreatment (Month -6)	61.8 percentage of patients
Triptorelin Pamoate 11.25 mg	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)	Compared to Baseline (Month 0)	52.9 percentage of patients

Secondary

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.