Trial Outcomes & Findings for A Study to Assess the Efficacy and Safety of the Triptorelin 6-month Formulation in Paediatric Participants With Central Precocious Puberty. (NCT NCT05029622)

Last Updated: 2025-02-19

Results Overview

LH response was defined as a peak LH \<=5 international units per liter (IU/L) after intravenous (IV) gonadotropin-releasing hormone (GnRH) stimulation. The GnRH stimulation test was performed by using an 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. Percentage of response was calculated by number of participants in the specified category divided by number of participants in the analysis population multiplied by 100.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

66 participants

Primary outcome timeframe

At Month 6

Results posted on

2025-02-19

Participant Flow

This Phase 3, open-label, single arm, study was conducted in children with central precocious puberty (CPP) at 12 investigational sites in China from 25 August 2021 to 13 February 2023.

This study consisted of screening period (up to 28 days) and study duration of minimum 12 months and up to 13 months including screening period.

Participant milestones

Participant milestones
Measure	All Participants Participants received triptorelin pamoate 22.5 milligrams (mg) intramuscular (IM) injection on Day 1.
Overall Study STARTED	66
Overall Study COMPLETED	65
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	All Participants Participants received triptorelin pamoate 22.5 milligrams (mg) intramuscular (IM) injection on Day 1.
Overall Study Lost to Follow-up	1

Baseline Characteristics

A Study to Assess the Efficacy and Safety of the Triptorelin 6-month Formulation in Paediatric Participants With Central Precocious Puberty.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Age, Continuous	7.5 years STANDARD_DEVIATION 0.9 • n=5 Participants
Sex: Female, Male Female	62 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Race/Ethnicity, Customized Chinese	66 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	0 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	66 Participants n=5 Participants

PRIMARY outcome

Timeframe: At Month 6

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Percentage of Participants With Luteinising Hormone (LH) Suppression	100 percentage of participants Interval 94.6 to 100.0

SECONDARY outcome

Timeframe: At Months 3 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

LH response was defined as a peak LH \<=5 IU/L after IV GnRH stimulation. The GnRH stimulation test was performed by using an 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. Percentage of response was calculated by number of participants in the specified category divided by number of participants in the analysis population multiplied by 100.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Percentage of Participants With LH Response to GnRH Test Month 3	97.0 percentage of participants Interval 89.5 to 99.6
Percentage of Participants With LH Response to GnRH Test Month 12	98.5 percentage of participants Interval 91.8 to 100.0

SECONDARY outcome

Timeframe: Baseline and at Months 3, 6, 9 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

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 study treatment administered.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Change From Baseline in Basal Serum LH and Follicle-Stimulating Hormone (FSH) Levels LH, Month 3	-1.6420 IU/L Standard Deviation 2.4287
Change From Baseline in Basal Serum LH and Follicle-Stimulating Hormone (FSH) Levels LH, Month 6	-1.8015 IU/L Standard Deviation 2.4109
Change From Baseline in Basal Serum LH and Follicle-Stimulating Hormone (FSH) Levels LH, Month 9	-1.6307 IU/L Standard Deviation 2.3396
Change From Baseline in Basal Serum LH and Follicle-Stimulating Hormone (FSH) Levels LH, Month 12	-1.8504 IU/L Standard Deviation 2.4524
Change From Baseline in Basal Serum LH and Follicle-Stimulating Hormone (FSH) Levels FSH, Month 3	-2.9938 IU/L Standard Deviation 1.9450
Change From Baseline in Basal Serum LH and Follicle-Stimulating Hormone (FSH) Levels FSH, Month 6	-2.4871 IU/L Standard Deviation 2.0414
Change From Baseline in Basal Serum LH and Follicle-Stimulating Hormone (FSH) Levels FSH, Month 9	-2.8586 IU/L Standard Deviation 1.9310
Change From Baseline in Basal Serum LH and Follicle-Stimulating Hormone (FSH) Levels FSH, Month 12	-2.5348 IU/L Standard Deviation 1.9162

SECONDARY outcome

Timeframe: Baseline and at Months 3, 6 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

Peak 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 study treatment administered. Blood samples were collected prior to gonadorelin injection (timepoint T0) and at 30 minutes (T30), 60 minutes (T60) and 90 minutes (T90) after a single IV injection of gonadorelin. A suppressed LH response to GnRH stimulation test was defined as peak serum LH \<=5 IU/L among the four timepoints (T0, T30, T60 and T90). The FSH response to GnRH stimulation was the peak serum FSH level among the four timepoints (T0, T30, T60 and T90).

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Change From Baseline in Peak Serum LH and FSH Level After the GnRH Stimulation Test LH, Month 3	-26.0075 IU/L Standard Deviation 19.8863
Change From Baseline in Peak Serum LH and FSH Level After the GnRH Stimulation Test LH, Month 6	-26.2867 IU/L Standard Deviation 20.3004
Change From Baseline in Peak Serum LH and FSH Level After the GnRH Stimulation Test LH, Month 12	-26.6065 IU/L Standard Deviation 20.5680
Change From Baseline in Peak Serum LH and FSH Level After the GnRH Stimulation Test FSH, Month 3	-11.7297 IU/L Standard Deviation 5.1787
Change From Baseline in Peak Serum LH and FSH Level After the GnRH Stimulation Test FSH, Month 6	-10.0656 IU/L Standard Deviation 5.0160
Change From Baseline in Peak Serum LH and FSH Level After the GnRH Stimulation Test FSH, Month 12	-10.1645 IU/L Standard Deviation 5.2442

SECONDARY outcome

Timeframe: Month 6 to Month 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

Peak LH serum concentration was analyzed centrally. LH response was defined as a peak LH \<=5 IU/L after IV GnRH stimulation. The GnRH stimulation test was performed by using an IV injection of gonadorelin (synthetic GnRH) to stimulate gonadotrophin release and blood samples were collected after the gonadorelin injection for central assessment of peak LH levels. Percentage of response was calculated by number of participants in the specified category divided by number of participants in the analysis population multiplied by 100.

Outcome measures

Outcome measures
Measure	All Participants n=65 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Percentage of Participants With LH Response (Peak LH <=5 IU/L) From Month 6 to Month 12	98.5 percentage of participants Interval 91.8 to 100.0

SECONDARY outcome

Timeframe: At Months 3, 6, 9 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

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 \<=30 nanogram (ng)/ deciliter (dL) in male participants. Percentage of response was calculated by number of participants in the specified category divided by number of participants in the analysis population multiplied by 100.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Percentage of Participants With Prepubertal Levels of Sex Steroids Month 3	97.0 percentage of participants Interval 89.5 to 99.6
Percentage of Participants With Prepubertal Levels of Sex Steroids Month 6	100 percentage of participants Interval 94.6 to 100.0
Percentage of Participants With Prepubertal Levels of Sex Steroids Month 9	86.4 percentage of participants Interval 75.7 to 93.6
Percentage of Participants With Prepubertal Levels of Sex Steroids Month 12	98.5 percentage of participants Interval 91.8 to 100.0

SECONDARY outcome

Timeframe: Baseline and at Months 6 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

Z-scores are calculated using Centers for Disease Control and Prevention (CDC) Growth Charts. Change from baseline was defined as the values for Z-score at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to study treatment administration. Negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Change From Baseline in Mean Height for Age (Z-Score) Month 6	0.0734 Z-score Standard Deviation 0.1433
Change From Baseline in Mean Height for Age (Z-Score) Month 12	0.0204 Z-score Standard Deviation 0.1567

SECONDARY outcome

Timeframe: Baseline and at Months 6 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

Z-scores were calculated using CDC growth charts, that contained Box-Cox transformation (L), the median (M) and the generalized coefficient of variation (S). Percentile was obtained using the following equation M (1 + LSZ) \*\* (1/L), where \*\* indicated an exponent, such that m (1+LSZ)\*\* (1/L) meant raising (1+LSZ) to the (1/L)th power and then multiplying the M. Z was the Z-score that corresponds to the percentile. Negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline was defined as the values for percentile of Z-score at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first study treatment administered.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Change From Baseline in Percentile for Height for Age Month 6	1.4675 percentiles of Z-score Standard Deviation 2.8638
Change From Baseline in Percentile for Height for Age Month 12	0.2810 percentiles of Z-score Standard Deviation 3.2237

SECONDARY outcome

Timeframe: Baseline and at Months 6 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

Growth velocity analysis was part of auxological parameter. 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 study treatment administered.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Change From Baseline in Growth Velocity Month 6	-3.938 centimeter (cm)/year Standard Deviation 6.075
Change From Baseline in Growth Velocity Month 12	-4.798 centimeter (cm)/year Standard Deviation 5.577

SECONDARY outcome

Timeframe: At Months 6 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

BA was determined using X-rays of the hand and wrist by Greulich and Pyle method. CA was calculated as (visit date -birth date + 1)/365.25. Percentage of response was calculated as n/N\*100, where n was number of participants in the specified category and N was number of participants in the analysis population.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Percentage of Participants With Bone Age (BA)/Chronological Age (CA) Ratio Not Risen Month 6	95.5 percentage of participants Interval 87.3 to 99.1
Percentage of Participants With Bone Age (BA)/Chronological Age (CA) Ratio Not Risen Month 12	92.4 percentage of participants Interval 83.2 to 97.5

SECONDARY outcome

Timeframe: Baseline, Months 6 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

BA was determined using X-rays of the hand and wrist by Greulich and Pyle method. CA was calculated as (visit date -birth date + 1)/365.25. Change from baseline was defined as the values for BA:CA ratio at indicated timepoints minus the value at baseline. Baseline was defined as the last non-missing measurement taken prior to the first study treatment administered.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Change From Baseline in BA:CA Ratio Month 6	-0.04 ratio Standard Deviation 0.06
Change From Baseline in BA:CA Ratio Month 12	-0.06 ratio Standard Deviation 0.07

SECONDARY outcome

Timeframe: At Months 6 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

Pubertal stage parameters were analyzed using Tanner method. Pubertal stage parameters included genital development stage (GDS) in male participants, breast development stage (BDS) in female participants and pubic hair development (PHD) stage in both sexes. Percentage of response was calculated by number of participants in the specified category divided by number of participants in the analysis population multiplied by 100.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Percentage of Participants With Stabilized Pubertal Stage BDS for female participants, Month 6	98.4 percentage of participants Interval 91.3 to 100.0
Percentage of Participants With Stabilized Pubertal Stage GDS for male participants, Month 6	100 percentage of participants Interval 39.8 to 100.0
Percentage of Participants With Stabilized Pubertal Stage PHD for female participants, Month 6	91.9 percentage of participants Interval 82.2 to 97.3
Percentage of Participants With Stabilized Pubertal Stage PHD for male participants, Month 6	100 percentage of participants Interval 39.8 to 100.0
Percentage of Participants With Stabilized Pubertal Stage BDS for female participants, Month 12	93.5 percentage of participants Interval 84.3 to 98.2
Percentage of Participants With Stabilized Pubertal Stage GDS for male participants, Month 12	100 percentage of participants Interval 39.8 to 100.0
Percentage of Participants With Stabilized Pubertal Stage PHD for female participants, Month 12	87.1 percentage of participants Interval 76.1 to 94.3
Percentage of Participants With Stabilized Pubertal Stage PHD for male participants, Month 12	75.0 percentage of participants Interval 19.4 to 99.4

SECONDARY outcome

Timeframe: At Months 6 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the female participants analyzed were reported.

Uterine length was determined by transabdominal ultrasound. Percentage of response was calculated by number of participants in the specified category divided by number of participants in the analysis population multiplied by 100.

Outcome measures

Outcome measures
Measure	All Participants n=62 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Percentage of Participants With Regression of Uterine Length Month 6	64.5 percentage of participants Interval 51.3 to 76.3
Percentage of Participants With Regression of Uterine Length Month 12	64.5 percentage of participants Interval 51.3 to 76.3

SECONDARY outcome

Timeframe: At Months 6 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the male participants analyzed were reported.

Testis volume was a clinical assessment with orchidometer. Percentage of response was calculated by number of participants in the specified category divided by number of participants in the analysis population multiplied by 100.

Outcome measures

Outcome measures
Measure	All Participants n=4 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Percentage of Participants With Absence of Progression of Testis Volumes Month 12	75.0 percentage of participants Interval 19.4 to 99.4
Percentage of Participants With Absence of Progression of Testis Volumes Month 6	75.0 percentage of participants Interval 19.4 to 99.4

SECONDARY outcome

Timeframe: Baseline and at Months 6 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

BMI analysis was part of auxological parameter assessment. 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 study treatment administered.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Change From Baseline in Body Mass Index (BMI) Month 6	0.282 kilogram (kg)/meter square Standard Deviation 0.882
Change From Baseline in Body Mass Index (BMI) Month 12	1.195 kilogram (kg)/meter square Standard Deviation 1.196

SECONDARY outcome

Timeframe: Baseline and at Months 6 and 12

Population: The ITT population consisted of all participants who received at least 1 dose of study intervention. Only data from the participants analyzed were reported.

Weight analysis was part of auxological parameter assessment. 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 study treatment administered.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Change From Baseline in Weight Month 6	2.020 kg Standard Deviation 1.927
Change From Baseline in Weight Month 12	5.146 kg Standard Deviation 2.816

SECONDARY outcome

Timeframe: Day 1, 4 hours post-injection; Month 3; Month 6, predose; Month 6, 4 hours post-injection; and Month 12

Population: The Pharmacokinetic (PK) set consisted of all participants who received at 1 dose of study treatment and had at least 1 valid triptorelin concentration. Only data from the participants analyzed were reported.

Blood samples were collected at specified timepoints.

Outcome measures

Outcome measures
Measure	All Participants n=66 Participants Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
Plasma Concentrations of Triptorelin Day 1, 4 hours post-injection	41.8 ng/mL Geometric Coefficient of Variation 56.9
Plasma Concentrations of Triptorelin Month 3	0.0434 ng/mL Geometric Coefficient of Variation 76.8
Plasma Concentrations of Triptorelin Month 6, predose	0.0251 ng/mL Geometric Coefficient of Variation 648.7
Plasma Concentrations of Triptorelin Month 6, 4 hours post-injection	24.7 ng/mL Geometric Coefficient of Variation 66.0
Plasma Concentrations of Triptorelin Month 12	0.0223 ng/mL Geometric Coefficient of Variation 110.3

Adverse Events

All Participants

Serious events: 0 serious events

Other events: 59 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	All Participants n=66 participants at risk Participants received triptorelin pamoate 22.5 mg IM injection on Day 1.
General disorders Pyrexia	13.6% 9/66 • Number of events 9 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
General disorders Influenza like illness	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Reproductive system and breast disorders Vaginal haemorrhage	3.0% 2/66 • Number of events 2 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Reproductive system and breast disorders Nocturnal emission	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Respiratory, thoracic and mediastinal disorders Cough	10.6% 7/66 • Number of events 7 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	4.5% 3/66 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	3.0% 2/66 • Number of events 2 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Respiratory, thoracic and mediastinal disorders Allergic cough	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Psychiatric disorders Listless	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Investigations Weight increased	13.6% 9/66 • Number of events 9 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Investigations Urinary occult blood positive	3.0% 2/66 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Investigations Alanine aminotransferase increased	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Investigations Aspartate aminotransferase increased	1.5% 1/66 • Number of events 2 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Investigations Eosinophil count increased	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Investigations Gamma-glutamyltransferase increased	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Investigations Liver function test abnormal	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Investigations Myoglobin blood increased	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Injury, poisoning and procedural complications Scratch	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Blood and lymphatic system disorders Anaemia	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Blood and lymphatic system disorders Leukocytosis	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Eye disorders Asthenopia	3.0% 2/66 • Number of events 2 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Eye disorders Refraction disorder	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Eye disorders Xerophthalmia	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Gastrointestinal disorders Abdominal pain	4.5% 3/66 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Gastrointestinal disorders Mouth ulceration	3.0% 2/66 • Number of events 6 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Gastrointestinal disorders Abdominal pain upper	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Gastrointestinal disorders Anal fissure	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Gastrointestinal disorders Constipation	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Gastrointestinal disorders Diarrhoea	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Gastrointestinal disorders Gastritis	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Gastrointestinal disorders Nausea	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Gastrointestinal disorders Vomiting	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Hepatobiliary disorders Hepatic steatosis	3.0% 2/66 • Number of events 2 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Skin and subcutaneous tissue disorders Urticaria	3.0% 2/66 • Number of events 2 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Skin and subcutaneous tissue disorders Rash	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Skin and subcutaneous tissue disorders Dermatitis	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Skin and subcutaneous tissue disorders Eczema	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Renal and urinary disorders Haematuria	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Upper respiratory tract infection	47.0% 31/66 • Number of events 45 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations COVID-19	15.2% 10/66 • Number of events 10 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Suspected COVID-19	6.1% 4/66 • Number of events 4 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Bronchitis	6.1% 4/66 • Number of events 4 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Tonsillitis	4.5% 3/66 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Rhinitis	4.5% 3/66 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Respiratory tract infection	3.0% 2/66 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Sinusitis	3.0% 2/66 • Number of events 2 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Urinary tract infection	3.0% 2/66 • Number of events 2 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Acarodermatitis	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Helicobacter infection	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Localised infection	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Nasopharyngitis	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Otitis media	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Acute sinusitis	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Infections and infestations Gastroenteritis	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Metabolism and nutrition disorders Overweight	10.6% 7/66 • Number of events 7 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Metabolism and nutrition disorders Obesity	4.5% 3/66 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Metabolism and nutrition disorders Hyperglycaemia	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.
Metabolism and nutrition disorders Vitamin D deficiency	1.5% 1/66 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study treatment (Day 1) up to a maximum of approximately 344 days. The Safety population consisted of all participants who received at least 1 dose of study treatment and have at least 1 post-baseline safety assessment.

Additional Information

Medical Director

Ipsen

Phone: see email

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER