Central Precocious PubertyPhase 3NCT04736602
Efficacy and Safety Study of Triptorelin 3-Month Formulation in Chinese Children With Central Precocious Puberty.
A Phase 3 study testing Triptorelin pamoate 15mg in people with Central Precocious Puberty, run by Ipsen at 7 sites in China.
Trial summary
The purpose of this research was to confirm the effectiveness and safety of the study drug, Triptorelin pamoate 15mg 3-month formulation, in a Chinese population of Central Precocious Puberty (CPP) children. Read more about this study
- Who is it for?
- Ages Up to 10 years
- Conditions the study is for
- Central Precocious Puberty
- What is being tested?
- Triptorelin pamoate 15mg
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 7 sitesChina
- Study dates
- 27 Mar 2021to 3 Sep 2022
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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)
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- 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.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
China7 sites
Chengdu Women's and Children's Central Hospital
Chengdu, 610073
Jiangxi Provincial Children's Hospital
Nanchang, 330006
Children's Hospital of Nanjing
Nanjing, 210008
Tangshan Maternal & Child Health Hospital
Tangshan, 063000
Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
Wuhan, 430030
Wuxi Children's Hospital
Wuxi, 214023
Henan Children's Hospital, Zhengzhou Children's Hospital
Zhengzhou, 450018
About this study
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
The purpose of this research was to confirm the effectiveness and safety of the study drug, Triptorelin pamoate 15mg 3-month formulation, in a Chinese population of Central Precocious Puberty (CPP) children.
- Conditions
- Central Precocious Puberty
- Being tested
- Triptorelin pamoate 15mg
- Medical subject headings
- Puberty, Precocious
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Triptorelin pamoate 15mgDrug
Intramuscular injection (IM)
| Study group | Role | What they receive |
|---|---|---|
| Triptorelin Pamoate 15mg for injection | Experimental | Triptorelin was injected at day 1 and month 3.
If participants were willing to enter the extension phase, two additional Triptorelin injections were given at month 6 and month 9. Receives: Drug: Triptorelin pamoate 15mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Luteinising Hormone (LH) Suppression After Gonadotropin-Releasing Hormone (GnRH) Stimulation
The LH suppression was defined as stimulated peak LH ≤3 International Units/Liter (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.
Measured: At Month 3
Secondary outcomes (16)
Change From Baseline in Basal LH and Follicle-Stimulating Hormone (FSH) Serum Levels
Basal LH and FSH serum concentrations were analyzed centrally. Change from baseline was defined as the value for LH and FSH 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.
Measured: Baseline (Day 1) and at Months 3, 6, 9 and 12
Percentage of Participants With LH Suppression After GnRH Stimulation
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).
Measured: At Months 6 and 12
Change From Baseline in Peak LH and FSH Level After GnRH Stimulation
A synthetic GnRH 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. 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.
Measured: Baseline (Day 1) and at Months 3, 6 and 12
Percentage of Participants With Prepubertal Levels of Sex Steroids
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)/milliliter (mL) in female participants and testosterone ≤0.3 nanogram (ng)/mL in male participants.
Measured: At Months 3, 6, 9 and 12
Change From Baseline in Estradiol Levels
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.
Measured: Baseline (Day 1) and at Months 3, 6 ,9 and 12
Change From Baseline in Testosterone Levels
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.
Measured: Baseline (Day 1) and at Months 3, 6 ,9 and 12
Percentage of Participants With Change From Baseline in Pubertal Stage
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.
Measured: Baseline (Day 1) and at Months 6 and 12
Percentage of Participants With Stabilized Pubertal Stage Compared to Baseline
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.
Measured: Baseline (Day 1) and at Months 6 and 12
Change From Baseline in Auxological Parameter: Height
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.
Measured: Baseline (Day 1) and at Months 3, 6, 9 and 12
Change From Baseline in Auxological Parameter: Weight
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.
Measured: Baseline (Day 1) and at Months 3, 6, 9 and 12
Change From Baseline in Auxological Parameter: Growth Velocity
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.
Measured: Baseline (Day 1) and at Months 3, 6, 9 and 12
Change From Baseline in Auxological Parameter: Body Mass Index (BMI)
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.
Measured: Baseline (Day 1) and at Months 3, 6, 9 and 12
Change From Baseline in Bone Age (BA)
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.
Measured: Baseline (Day 1) and at Months 6 and 12
Change From Baseline Difference Between BA and Chronological Age (CA)
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.
Measured: Baseline (Day 1) and at Months 6 and 12
Change From Baseline in Uterine Length
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.
Measured: Baseline (Day 1) and at Months 6 and12
Change From Baseline of Testicular Volume
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.
Measured: Baseline (Day 1) and at Months 6 and 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2021
- Study start27 Mar 2021
- Primary completion9 Feb 2022
- Study completion3 Sep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Central Precocious PubertyStudies of the same condition, open and under way.
- More trials run by IpsenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04736602
- Sponsor's protocol ID
- D-CN-52014-243
- Status last verified
- Feb 2024
- First submitted
- 29 Jan 2021
- Last updated
- 1 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 1 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04736602
- Acronym / sponsor ID
- D-CN-52014-243
- Conditions
- Central Precocious Puberty
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Study start
- 27 Mar 2021 (actual)
- Primary completion
- 9 Feb 2022 (actual)
- Study completion
- 3 Sep 2022 (actual)
- First posted
- 3 Feb 2021 (actual)
- Results
- Posted 1 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this research was to confirm the effectiveness and safety of the study drug, Triptorelin pamoate 15mg 3-month formulation, in a Chinese population of Central Precocious Puberty (CPP) children.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Triptorelin Pamoate 15mg for injection | Experimental | Drug: Triptorelin pamoate 15mgTriptorelin was injected at day 1 and month 3. If participants were willing to enter the extension phase, two additional Triptorelin injections were given at month 6 and month 9. |
Who may qualify
- Ages
- Up to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (10 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Triptorelin pamoate 15mgIntramuscular injection (IM) | Drug | Triptorelin Pamoate 15mg for injection |
Primary outcome
Percentage of Participants With Luteinising Hormone (LH) Suppression After Gonadotropin-Releasing Hormone (GnRH) Stimulation
Time frame: At Month 3
The LH suppression was defined as stimulated peak LH ≤3 International Units/Liter (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.
16 secondary outcomes
Change From Baseline in Basal LH and Follicle-Stimulating Hormone (FSH) Serum Levels
Time frame: Baseline (Day 1) and at Months 3, 6, 9 and 12
Percentage of Participants With LH Suppression After GnRH Stimulation
Time frame: At Months 6 and 12
Change From Baseline in Peak LH and FSH Level After GnRH Stimulation
Time frame: Baseline (Day 1) and at Months 3, 6 and 12
Percentage of Participants With Prepubertal Levels of Sex Steroids
Time frame: At Months 3, 6, 9 and 12
Change From Baseline in Estradiol Levels
Time frame: Baseline (Day 1) and at Months 3, 6 ,9 and 12
Change From Baseline in Testosterone Levels
Time frame: Baseline (Day 1) and at Months 3, 6 ,9 and 12
Percentage of Participants With Change From Baseline in Pubertal Stage
Time frame: Baseline (Day 1) and at Months 6 and 12
Percentage of Participants With Stabilized Pubertal Stage Compared to Baseline
Time frame: Baseline (Day 1) and at Months 6 and 12
Change From Baseline in Auxological Parameter: Height
Time frame: Baseline (Day 1) and at Months 3, 6, 9 and 12
Change From Baseline in Auxological Parameter: Weight
Time frame: Baseline (Day 1) and at Months 3, 6, 9 and 12
Change From Baseline in Auxological Parameter: Growth Velocity
Time frame: Baseline (Day 1) and at Months 3, 6, 9 and 12
Change From Baseline in Auxological Parameter: Body Mass Index (BMI)
Time frame: Baseline (Day 1) and at Months 3, 6, 9 and 12
Change From Baseline in Bone Age (BA)
Time frame: Baseline (Day 1) and at Months 6 and 12
Change From Baseline Difference Between BA and Chronological Age (CA)
Time frame: Baseline (Day 1) and at Months 6 and 12
Change From Baseline in Uterine Length
Time frame: Baseline (Day 1) and at Months 6 and12
Change From Baseline of Testicular Volume
Time frame: Baseline (Day 1) and at Months 6 and 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ipsen Medical Director study director
Ipsen
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Triptorelin pamoate 15mg in people with Central Precocious Puberty, run by Ipsen at 7 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants With Luteinising Hormone (LH) Suppression After Gonadotropin-Releasing Hormone (GnRH) Stimulation (time frame: At Month 3). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Aug 2024.