A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)

• Sponsor: Ascendis Pharma Endocrinology Division A/S (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02781727
• Collaborator: (none)
• Enrollment: 162
• Locations: 53
• Arms: 2
• Actual Duration (Months): 25.1
• Patients Per Site: 3.1
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

A 52 week trial of TransCon hGH, a long-acting growth hormone product, versus human growth hormone therapy. TransCon hGH will be given once-a-week, human growth hormone (hGH) will be given daily. Approximately 150 prepubertal, hGH-treatment naïve children (males and females) with GHD will be included. Randomization will occur in a 2:1 ratio (TransCon hGH : Genotropin). This is a global trial that will be conducted in Armenia, Australia, Belarus, Bulgaria, Georgia, Greece, Italy, New Zealand, Poland, Romania, Russia, Turkey, Ukraine, and the United States.

Condition or Disease	Intervention/Treatment	Phase
Growth Hormone Deficiency, Pediatric; hGH (Human Growth Hormone); Endocrine System Diseases; Hormones; Pituitary Diseases	Drug: Once weekly subcutaneous injection of TransCon hGH; Drug: Once daily subcutaneous injection of Genotropin	Phase 3

Study Design

• Study Type: Interventional
• Actual Enrollment: 162 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Multicenter, Phase 3, Randomized, Open-label, Active-controlled, Parallel-group Trial Investigating the Safety, Tolerability, and Efficacy of TransCon hGH Administered Once a Week Versus Standard Daily hGH Replacement Therapy Over 52 Weeks in Prepubertal Children With Growth Hormone Deficiency (GHD)
• Actual Study Start Date: Dec 13, 2016
• Actual Primary Completion Date: Jan 17, 2019
• Actual Study Completion Date: Jan 17, 2019

Arms and Interventions

Arm	Intervention/Treatment
Experimental: TransCon hGH Once weekly subcutaneous injection of TransCon hGH	Drug: Once weekly subcutaneous injection of TransCon hGH; Once weekly subcutaneous injection
Active Comparator: human growth hormone (Genotropin) Once daily subcutaneous injection of Genotropin	Drug: Once daily subcutaneous injection of Genotropin; Once daily subcutaneous injection

Outcome Measures

Primary Outcome Measures

1. Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups [52 weeks]

   Annualized height velocity (AHV) at 52 weeks for weekly lonapegsomatropin (TransCon hGH) and daily hGH treatment groups

Secondary Outcome Measures

1. Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability] [52 Weeks]

   Number of participants with Treatment-Emergent Adverse Events for the weekly lonapegsomatropin and daily hGH treatment groups

2. Annualized Height Velocity Over 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups [Week 5, Week 13, Week 26, Week 39 and Week 52]

   Annualized height velocity (AHV) over 52 weeks for weekly lonapegsomatropin and daily hGH treatment groups. AHV by visit was determined by ANCOVA model with multiple imputation. For each imputed data set, an ANCOVA model with by visit AHV as the dependent variable, treatment and gender as factors, baseline age, baseline peak GH levels (log transformed) at stimulation test, and baseline height SDS - average parental height SDS as covariates were fitted.

3. Change in Height Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups [Week 5, Week 13, Week 26, Week 39 and Week 52]

   Height Standard Deviation Score (SDS) is the number of standard deviations above or below the mean height for age and sex. Height SDS was derived using the LMS method as ((Height/M)^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from 2000 CDC growth charts for the United States. A Standard Deviation Score of 0 represents the population mean. A higher change from baseline in Height Standard Deviation Score (SDS) indicates a better outcome. The change from baseline in height SDS by visit was determined by ANCOVA model and included baseline age, peak GH levels (log transformed) at stimulation test and baseline height SDS as covariates, as well as treatment and gender as factors.

4. Average IGF-1 Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups [Week 13, Week 26, Week 39, and Week 52]

   IGF-1 Standard Deviation Score (SDS) is the number of standard deviations above or below the mean Insulin-like Growth Factor 1 (IGF-1) level for age and sex. IGF-1 SDS was derived using the LMS method as ((IGF-1/M)^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from Bidlingmaier et al. (2014). A Standard Deviation Score of 0 represents the population mean. Average IGF-1 SDS by visit was determined by ANCOVA. The ANCOVA model included baseline age, peak GH levels (log transformed) at stimulation test, baseline IGF-1 SDS as covariates, as well as treatment and gender as factors. Modeled values begin at Week 13 corresponding with achievement of IGF-1 steady state. Average IGF-1 SDS values by visit for the Lonapegsomatropin group were derived from a population pharmacodynamic model; the average IGF-1 SDS values for the Genotropin group are represented by observed values.

5. Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation [Start of study treatment through Week 52]

   Number of participants with treatment emergent anti-hGH binding antibody formation during the 52 week study. All samples were negative for anti-hGH neutralizing antibodies.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 3 Years to 12 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Prepubertal children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage 1 (Tanner 1982) aged:

• Boys: 3-12 years, inclusive

• Girls: 3-11 years, inclusive

• Impaired height (HT) defined as at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS ≤ -2.0) according to the 2000 CDC Growth Charts for the United States Methods and Development, available at http://www.cdc.gov/growthcharts/

• Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH level of ≤10 ng/mL, determined with a validated assay

• Bone age (BA) at least 6 months less than chronological age

• Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1)

• Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject (if the subject is able to read, understand, and sign)

Exclusion Criteria:

• Children with a body weight below 12 kg

• Prior exposure to recombinant hGH or IGF-1 therapy

• Children with past or present intracranial tumor growth as confirmed by a sellar MRI scan (with contrast) at Screening (MRI results from up to 6 months prior to Screening may be accepted)

• Children with psychosocial dwarfism

• Children with idiopathic short stature

• History or presence of malignant disease; any evidence of present tumor growth

• Closed epiphyses

• Major medical conditions and/or presence of contraindication to hGH treatment

• Participation in any other trial of an investigational agent within 3 months prior to Screening

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Ascendis Pharma Investigational Site	Birmingham	Alabama	United States	35233
2	Ascendis Pharma Investigational Site	Little Rock	Arkansas	United States	72202
3	Ascendis Pharma Investigational Site	Los Angeles	California	United States	90048
4	Ascendis Pharma Investigational Site	Orange	California	United States	92868
5	Ascendis Pharma Investigational Site	Centennial	Colorado	United States	80112
6	Ascendis Pharma Investigational Site	Jacksonville	Florida	United States	32207
7	Ascendis Pharma Investigational Site	Orlando	Florida	United States	32806
8	Ascendis Pharma Investigational Site	Saint Paul	Minnesota	United States	55102
9	Ascendis Pharma Investigational Site	Jackson	Mississippi	United States	39216
10	Ascendis Pharma Investigational Site	Lebanon	New Hampshire	United States	03756
11	Ascendis Pharma Investigational Site	Mineola	New York	United States	11501
12	Ascendis Pharma Investigational Site	Oklahoma City	Oklahoma	United States	73104
13	Ascendis Pharma Investigational Site	Portland	Oregon	United States	97239
14	Ascendis Pharma Investigational Site	Dallas	Texas	United States	75235
15	Ascendis Pharma Investigational Site	Fort Worth	Texas	United States	76104
16	Ascendis Pharma Investigational Site	Tacoma	Washington	United States	98405
17	Ascendis Pharma Investigational Site	Yerevan		Armenia	0075
18	Ascendis Pharma Investigational Site	Clayton		Australia	3168
19	Ascendis Pharma Investigational Site	Minsk		Belarus	220020
20	Ascendis Pharma Investigational Site	Varna		Bulgaria	9010
21	Ascendis Pharma Investigational Site	Tbilisi		Georgia	0144
22	Ascendis Pharma Investigational Site	Tbilisi		Georgia	0159
23	Ascendis Pharma Investigational Site	Tbilisi		Georgia	0162
24	Ascendis Pharma Investigational Site	Athens		Greece	11527
25	Ascendis Pharma Investigational Site	Milano		Italy	20157
26	Ascendis Pharma Investigational Site	Roma		Italy	00165
27	Ascendis Pharma Investigational Site	Grafton		New Zealand	1023
28	Ascendis Pharma Investigatonal Site	Gdańsk		Poland	80-952
29	Ascendis Pharma Investigational Site	Warszawa		Poland	02-691
30	Ascendis Pharma Investigational Site	Iaşi		Romania	700111
31	Ascendis Pharma Investigational Site	Izhevsk		Russian Federation	426009
32	Ascendis Pharma Investigational Site	Kazan		Russian Federation	420138
33	Ascendis Pharma Investigational Site	Krasnoyarsk		Russian Federation	620022
34	Ascendis Pharma Investigational Site	Moscow		Russian Federation	125373
35	Ascendis Pharma Investigational Site	Moscow		Russian Federation	127994
36	Ascendis Pharma Investigational Site	Nizhny Novgorod		Russian Federation	603136
37	Ascendis Pharma Investigational Site	Novosibirsk		Russian Federation	630048
38	Ascendis Pharma Investigational Site	Omsk		Russian Federation	644001
39	Ascendis Pharma Investigational Site	Saint Petersburg		Russian Federation	191144
40	Ascendis Pharma Investigational Site	Saint Petersburg		Russian Federation	194100
41	Ascendis Pharma Investigational Site	Samara		Russian Federation	443079
42	Ascendis Pharma Investigational Site	Saratov		Russian Federation	410054
43	Ascendis Pharma Investigational Site	Tomsk		Russian Federation	634050
44	Ascendis Pharma Investigational Site	Ufa		Russian Federation	450008
45	Ascendis Pharma Investigational Site	Vologda		Russian Federation	160022
46	Ascendis Pharma Investigational Site	Voronezh		Russian Federation	394024
47	Ascendis Pharma Investigational Site	İzmir		Turkey	35100
48	Ascendis Pharma Investigational Site	Melikgazi		Turkey	38039
49	Ascendis Pharma Investigational Site	Trabzon		Turkey	61080
50	Ascendis Pharma Investigational Site	Kharkov		Ukraine	61093
51	Ascendis Pharma Investigational Site	Kyiv		Ukraine	01021
52	Ascendis Pharma Investigational Site	Kyiv		Ukraine	04114
53	Ascendis Pharma Investigational Site	Odesa		Ukraine	65031

Sponsors and Collaborators

• Ascendis Pharma Endocrinology Division A/S

Investigators

• Study Director: Michael Beckert, MD, Ascendis Pharma A/S
• Study Director: Aimee D Shu, MD, Ascendis Pharma, Inc.

Study Documents (Full-Text)

• Study Protocol - Sep 12, 2017
• Statistical Analysis Plan - Feb 19, 2019

More Information

Publications

None provided.

• Responsible Party: Ascendis Pharma Endocrinology Division A/S
• ClinicalTrials.gov Identifier: NCT02781727
• Other Study ID Numbers: TransCon hGH CT-301; 2016-001145-11
• First Posted: May 24, 2016
• Last Update Posted: Jan 4, 2022
• Last Verified: Dec 1, 2021

• Keywords provided by Ascendis Pharma Endocrinology Division A/S: Human Growth Hormone; hGH; GHD; rHGH; Pediatric Growth Hormone Deficiency; Long Acting Growth Hormone; Somatropin; Prodrug; Growth Failure; Growth Hormone Replacement Therapy; Sustained Release; Sustained Release Growth Hormone; Growth Hormone Deficiency
• Additional relevant MeSH terms: Dwarfism, Pituitary; Pituitary Diseases; Endocrine System Diseases

Study Results

Participant Flow

Arm/Group Title	Lonapegsomatropin	Daily hGH
Arm/Group Description	Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH)	Once daily subcutaneous injection of Genotropin
Period Title: Pre-dosing Period
STARTED	106	56
COMPLETED	105	56
NOT COMPLETED	1	0
Period Title: Pre-dosing Period
STARTED	105	56
COMPLETED	104	55
NOT COMPLETED	1	1

Baseline Characteristics

Arm/Group Title	Lonapegsomatropin	Daily hGH	Total
Arm/Group Description	Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH)	Once daily subcutaneous injection of Genotropin	Total of all reporting groups
Overall Participants	105	56	161
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	8.5 (2.7)	8.5 (2.8)	8.5 (2.7)
Age, Customized (Count of Participants)
<6 years	25 23.8%	14 25%	39 24.2%
≥6 years	80 76.2%	42 75%	122 75.8%
Sex: Female, Male (Count of Participants)
Female	19 18.1%	10 17.9%	29 18%
Male	86 81.9%	46 82.1%	132 82%
Race/Ethnicity, Customized (Count of Participants)
Asian	1 1%	0 0%	1 0.6%
Black or African American	2 1.9%	1 1.8%	3 1.9%
White	100 95.2%	52 92.9%	152 94.4%
Other	2 1.9%	3 5.4%	5 3.1%
Race/Ethnicity, Customized (Count of Participants)
Hispanic or Latino	5 4.8%	2 3.6%	7 4.3%
Not Hispanic or Latino	100 95.2%	54 96.4%	154 95.7%
Height (cm) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [cm]	112.93 (14.09)	112.15 (15.29)	112.66 (14.48)
Height SDS (standard deviation score) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [standard deviation score]	-2.89 (0.85)	-3.00 (0.90)	-2.93 (0.87)
Weight (kg) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [kg]	21.01 (6.54)	21.20 (6.67)	21.08 (6.56)
Body Mass Index (BMI) (kg/m^2) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [kg/m^2]	16.06 (1.78)	16.46 (2.17)	16.20 (1.93)
BMI SDS (standard deviation score) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [standard deviation score]	-0.32 (0.95)	-0.14 (1.07)	-0.25 (0.99)
IGF-1 SDS (standard deviation score) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [standard deviation score]	-2.08 (0.88)	-1.96 (0.98)	-2.04 (0.92)

Outcome Measures

1. Primary Outcome

Title	Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Description	Annualized height velocity (AHV) at 52 weeks for weekly lonapegsomatropin (TransCon hGH) and daily hGH treatment groups
Time Frame	52 weeks

Outcome Measure Data

Analysis Population Description

The Intention-to-Treat (ITT) population included all randomized subjects who had received at least 1 dose of active treatment.

Arm/Group Title	Lonapegsomatropin	Daily hGH
Arm/Group Description	Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH)	Once daily subcutaneous injection of Genotropin
Measure Participants	105	56
Least Squares Mean (Standard Error) [cm/year]	11.17 (0.23)	10.31 (0.30)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Lonapegsomatropin, Daily hGH
	Comments	ANCOVA model with multiple imputation. For each imputed data set, an ANCOVA model with by visit AHV as the dependent variable; treatment and gender as factors; and baseline age, baseline peak GH levels (log transformed) at stimulation test, and baseline height SDS - average parental height SDS as covariates were fitted.
	Type of Statistical Test	Non-Inferiority
	Comments	Non-inferiority comparison with a non-inferiority margin of 2 cm/year, followed by a test of superiority if non-inferiority is established.
Statistical Test of Hypothesis	p-Value	0.0088
	Comments	P-value is based on a test of superiority
	Method	ANCOVA with multiple imputation
	Comments	two-sided

2. Secondary Outcome

Title	Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
Description	Number of participants with Treatment-Emergent Adverse Events for the weekly lonapegsomatropin and daily hGH treatment groups
Time Frame	52 Weeks

Outcome Measure Data

Analysis Population Description

The safety analysis population included all randomized subjects who had received at least 1 dose of active treatment.

Arm/Group Title	Lonapegsomatropin	Daily hGH
Arm/Group Description	Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH)	Once daily subcutaneous injection of Genotropin
Measure Participants	105	56
TEAEs	81 77.1%	39 69.6%
TEAEs related to study drug	12 11.4%	10 17.9%
SAEs	1 1%	1 1.8%
SAEs related to study drug	0 0%	0 0%

3. Secondary Outcome

Title	Annualized Height Velocity Over 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Description	Annualized height velocity (AHV) over 52 weeks for weekly lonapegsomatropin and daily hGH treatment groups. AHV by visit was determined by ANCOVA model with multiple imputation. For each imputed data set, an ANCOVA model with by visit AHV as the dependent variable, treatment and gender as factors, baseline age, baseline peak GH levels (log transformed) at stimulation test, and baseline height SDS - average parental height SDS as covariates were fitted.
Time Frame	Week 5, Week 13, Week 26, Week 39 and Week 52

Outcome Measure Data

Analysis Population Description

The Intention-to-Treat (ITT) population included all randomized subjects who had received at least 1 dose of active treatment.

Arm/Group Title	Lonapegsomatropin	Daily hGH
Arm/Group Description	Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH)	Once daily subcutaneous injection of Genotropin
Measure Participants	105	56
Week 5	13.54 (1.07)	12.83 (1.37)
Week 13	13.28 (0.49)	12.22 (0.63)
Week 26	12.65 (0.32)	11.21 (0.42)
Week 39	11.89 (0.26)	10.90 (0.33)
Week 52	11.17 (0.23)	10.31 (0.30)

4. Secondary Outcome

Title	Change in Height Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Description	Height Standard Deviation Score (SDS) is the number of standard deviations above or below the mean height for age and sex. Height SDS was derived using the LMS method as ((Height/M)^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from 2000 CDC growth charts for the United States. A Standard Deviation Score of 0 represents the population mean. A higher change from baseline in Height Standard Deviation Score (SDS) indicates a better outcome. The change from baseline in height SDS by visit was determined by ANCOVA model and included baseline age, peak GH levels (log transformed) at stimulation test and baseline height SDS as covariates, as well as treatment and gender as factors.
Time Frame	Week 5, Week 13, Week 26, Week 39 and Week 52

Outcome Measure Data

Analysis Population Description

The Intention-to-Treat (ITT) population included all randomized subjects who had received at least 1 dose of active treatment.

Arm/Group Title	Lonapegsomatropin	Daily hGH
Arm/Group Description	Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH)	Once daily subcutaneous injection of Genotropin
Measure Participants	105	56
Week 5	0.13 (0.02)	0.12 (0.02)
Week 13	0.38 (0.02)	0.33 (0.03)
Week 26	0.68 (0.03)	0.58 (0.04)
Week 39	0.92 (0.03)	0.80 (0.04)
Week 52	1.10 (0.04)	0.96 (0.05)

5. Secondary Outcome

Title	Average IGF-1 Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Description	IGF-1 Standard Deviation Score (SDS) is the number of standard deviations above or below the mean Insulin-like Growth Factor 1 (IGF-1) level for age and sex. IGF-1 SDS was derived using the LMS method as ((IGF-1/M)^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from Bidlingmaier et al. (2014). A Standard Deviation Score of 0 represents the population mean. Average IGF-1 SDS by visit was determined by ANCOVA. The ANCOVA model included baseline age, peak GH levels (log transformed) at stimulation test, baseline IGF-1 SDS as covariates, as well as treatment and gender as factors. Modeled values begin at Week 13 corresponding with achievement of IGF-1 steady state. Average IGF-1 SDS values by visit for the Lonapegsomatropin group were derived from a population pharmacodynamic model; the average IGF-1 SDS values for the Genotropin group are represented by observed values.
Time Frame	Week 13, Week 26, Week 39, and Week 52

Outcome Measure Data

Analysis Population Description

The Intention-to-Treat (ITT) population included all randomized subjects who had received at least 1 dose of active treatment.

Arm/Group Title	Lonapegsomatropin	Daily hGH
Arm/Group Description	Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH)	Once daily subcutaneous injection of Genotropin
Measure Participants	105	56
Week 13	0.31 (0.09)	-0.60 (0.11)
Week 26	0.46 (0.08)	-0.51 (0.10)
Week 39	0.59 (0.09)	-0.30 (0.11)
Week 52	0.72 (0.09)	-0.02 (0.12)

6. Secondary Outcome

Title	Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation
Description	Number of participants with treatment emergent anti-hGH binding antibody formation during the 52 week study. All samples were negative for anti-hGH neutralizing antibodies.
Time Frame	Start of study treatment through Week 52

Outcome Measure Data

Analysis Population Description

The safety analysis population included all randomized subjects who had received at least 1 dose of active treatment.

Arm/Group Title	Lonapegsomatropin	Daily hGH
Arm/Group Description	Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH)	Once daily subcutaneous injection of Genotropin
Measure Participants	105	56
Count of Participants [Participants]	6 5.7%	2 3.6%

Adverse Events

Time Frame	From the first trial-related activity after the subject signed the informed consent until the end of the post-treatment follow-up period (up to week 52)
Adverse Event Reporting Description	All adverse events (AEs) were collected in response to a general question about the subject's well-being and any possible changes from the previous visit, but were not specifically solicited. AEs, including any serious adverse events, were collected through the end of trial (ie, the Week 52 Visit 6). AEs ongoing at Visit 6 or the time of premature trial discontinuation were followed until the event was resolved or deemed stable by the investigator.
Arm/Group Title	Lonapegsomatropin		Daily hGH
Arm/Group Description	Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH)		Once daily subcutaneous injection of Genotropin
All Cause Mortality
	Lonapegsomatropin		Daily hGH
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/105 (0%)		0/56 (0%)
Serious Adverse Events
	Lonapegsomatropin		Daily hGH
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/105 (1%)		1/56 (1.8%)
Infections and infestations
Appendicitis	1/105 (1%)		0/56 (0%)
Injury, poisoning and procedural complications
Concussion	0/105 (0%)		1/56 (1.8%)
Other (Not Including Serious) Adverse Events
	Lonapegsomatropin		Daily hGH
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	81/105 (77.1%)		39/56 (69.6%)
Endocrine disorders
Secondary hypothyroidism	7/105 (6.7%)		3/56 (5.4%)
Gastrointestinal disorders
Vomiting	9/105 (8.6%)		3/56 (5.4%)
Diarrhea	6/105 (5.7%)		3/56 (5.4%)
General disorders
Pyrexia	16/105 (15.2%)		5/56 (8.9%)
Infections and infestations
Nasopharyngitis	12/105 (11.4%)		8/56 (14.3%)
Pharyngitis	10/105 (9.5%)		10/56 (17.9%)
Upper respiratory tract infection	6/105 (5.7%)		5/56 (8.9%)
Respiratory tract infection	7/105 (6.7%)		3/56 (5.4%)
Nervous system disorders
Headache	13/105 (12.4%)		7/56 (12.5%)
Respiratory, thoracic and mediastinal disorders
Cough	10/105 (9.5%)		4/56 (7.1%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.