Trial Outcomes & Findings for Safety, PK/PD (Pharmacokinetics/Pharmacodynamics) and Efficacy of ACP-001 Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD) (NCT NCT01947907)
NCT ID: NCT01947907
Last Updated: 2017-01-19
Results Overview
Number of subjects with positive results for anti-hGH binding antibodies at two consecutive post-dose visits
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
53 participants
Primary outcome timeframe
Visit 2 - Visit 5
Results posted on
2017-01-19
Participant Flow
Participant milestones
| Measure |
ACP-001, Dose-level 1
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
12
|
14
|
14
|
13
|
|
Overall Study
COMPLETED
|
12
|
14
|
14
|
13
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety, PK/PD (Pharmacokinetics/Pharmacodynamics) and Efficacy of ACP-001 Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)
Baseline characteristics by cohort
| Measure |
ACP-001, Dose-level 1
n=12 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
n=13 Participants
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
Total
n=53 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
12 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
13 Participants
n=4 Participants
|
53 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Continuous
|
7.98 years
STANDARD_DEVIATION 2.889 • n=5 Participants
|
8.24 years
STANDARD_DEVIATION 2.13 • n=7 Participants
|
7.32 years
STANDARD_DEVIATION 2.784 • n=5 Participants
|
7.53 years
STANDARD_DEVIATION 2.483 • n=4 Participants
|
7.77 years
STANDARD_DEVIATION 2.57 • n=21 Participants
|
|
Gender
Female
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
15 Participants
n=21 Participants
|
|
Gender
Male
|
9 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
10 Participants
n=4 Participants
|
38 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
13 Participants
n=4 Participants
|
53 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Region of Enrollment
Russian Federation
|
4 participants
n=5 Participants
|
4 participants
n=7 Participants
|
7 participants
n=5 Participants
|
6 participants
n=4 Participants
|
21 participants
n=21 Participants
|
|
Region of Enrollment
Romania
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
2 participants
n=21 Participants
|
|
Region of Enrollment
Hungary
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
0 participants
n=4 Participants
|
1 participants
n=21 Participants
|
|
Region of Enrollment
Egypt
|
2 participants
n=5 Participants
|
4 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
7 participants
n=21 Participants
|
|
Region of Enrollment
Ukraine
|
1 participants
n=5 Participants
|
4 participants
n=7 Participants
|
1 participants
n=5 Participants
|
1 participants
n=4 Participants
|
7 participants
n=21 Participants
|
|
Region of Enrollment
Belarus
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
1 participants
n=4 Participants
|
7 participants
n=21 Participants
|
|
Region of Enrollment
Greece
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
2 participants
n=5 Participants
|
0 participants
n=4 Participants
|
3 participants
n=21 Participants
|
|
Region of Enrollment
Turkey
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
2 participants
n=4 Participants
|
3 participants
n=21 Participants
|
|
Region of Enrollment
Poland
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
0 participants
n=4 Participants
|
1 participants
n=21 Participants
|
|
Region of Enrollment
Bulgaria
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
1 participants
n=21 Participants
|
|
Baseline IGF-1 SDS (standard deviation score)
|
-2.034 Standard Deviation Score
STANDARD_DEVIATION 0.7429 • n=5 Participants
|
-2.017 Standard Deviation Score
STANDARD_DEVIATION 0.7713 • n=7 Participants
|
-2.19 Standard Deviation Score
STANDARD_DEVIATION 0.7169 • n=5 Participants
|
-2.502 Standard Deviation Score
STANDARD_DEVIATION 0.896 • n=4 Participants
|
-2.19 Standard Deviation Score
STANDARD_DEVIATION 0.782 • n=21 Participants
|
|
Baseline Height SDS
|
-3.05 Standard Deviation Score
STANDARD_DEVIATION 1.127 • n=5 Participants
|
-2.75 Standard Deviation Score
STANDARD_DEVIATION 0.383 • n=7 Participants
|
-3.17 Standard Deviation Score
STANDARD_DEVIATION 1.04 • n=5 Participants
|
-3.27 Standard Deviation Score
STANDARD_DEVIATION 1.077 • n=4 Participants
|
-3.06 Standard Deviation Score
STANDARD_DEVIATION 0.905 • n=21 Participants
|
|
Screening Peak GH Values
|
5.09 ng/mL
STANDARD_DEVIATION 3.169 • n=5 Participants
|
5.16 ng/mL
STANDARD_DEVIATION 2.598 • n=7 Participants
|
4.44 ng/mL
STANDARD_DEVIATION 2.770 • n=5 Participants
|
5.15 ng/mL
STANDARD_DEVIATION 3.068 • n=4 Participants
|
4.95 ng/mL
STANDARD_DEVIATION 2.829 • n=21 Participants
|
PRIMARY outcome
Timeframe: Visit 2 - Visit 5Population: Safety analysis set includes all patients who receive at least one dose of planned study medication
Number of subjects with positive results for anti-hGH binding antibodies at two consecutive post-dose visits
Outcome measures
| Measure |
ACP-001, Dose-level 1
n=12 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
n=13 Participants
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
|---|---|---|---|---|
|
Incidence of Anti-hGH Binding Antibody Formation
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
PRIMARY outcome
Timeframe: Visit 2 - Visit 5Number of subjects with positive results for anti-hGH neutralizing antibodies at two consecutive post-dose visits
Outcome measures
| Measure |
ACP-001, Dose-level 1
n=12 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
n=13 Participants
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
|---|---|---|---|---|
|
Incidence of Anti-hGH Neutralizing Antibody Formation
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
PRIMARY outcome
Timeframe: Start of study treatment through Visit 5 (Week 27)Assessment of local tolerability was performed by examining injection sites (by the investigator during study visits) and on the basis of anamnestic data and records in the Patient Diary. Assessments included pain, redness, bruising, swelling, and itching. Every subject was counted only once within each symptom category.
Outcome measures
| Measure |
ACP-001, Dose-level 1
n=12 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
n=13 Participants
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
|---|---|---|---|---|
|
Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)
|
7 Number of subjects with any symptom
|
6 Number of subjects with any symptom
|
6 Number of subjects with any symptom
|
6 Number of subjects with any symptom
|
PRIMARY outcome
Timeframe: 0 hours to 168 hours at Visit 3 (Week 13)As part of the following endpoint: PK profile of serum hGH from ACP-001 treated patients compared between ACP-001 dose groups and to the pharmacokinetic (PK) profile of hGH from the daily rhGH groups during visit 1 and 3. Uncorrected Cmax (maximum value of concentration) values at Visit 3 (Week 13)
Outcome measures
| Measure |
ACP-001, Dose-level 1
n=12 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
n=13 Participants
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
|---|---|---|---|---|
|
Cmax of hGH
|
12.558 ng/mL
Standard Deviation 8.678
|
13.418 ng/mL
Standard Deviation 9.428
|
31.8 ng/mL
Standard Deviation 17.499
|
16.612 ng/mL
Standard Deviation 12.777
|
PRIMARY outcome
Timeframe: 0 hours to 168 hours at Visit 3 (Week 13)As part of the following endpoint: PK profile of serum hGH from ACP-001 treated patients compared between ACP-001 dose groups and to the PK profile of hGH from the daily rhGH group during Visit 1 and Visit 3 Uncorrected AUC0-168h (area under the curve from 0h to 168h) values at Visit 3 (Week 13)
Outcome measures
| Measure |
ACP-001, Dose-level 1
n=12 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
n=13 Participants
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
|---|---|---|---|---|
|
AUC0-168h of hGH
|
696.34 h*ng/mL
Standard Deviation 410.096
|
787.41 h*ng/mL
Standard Deviation 483.169
|
2167.43 h*ng/mL
Standard Deviation 1064.729
|
556.88 h*ng/mL
Standard Deviation 412.618
|
PRIMARY outcome
Timeframe: 0 hours to 168 hours at Visit 3 (Week 13)As part of the following endpoint: PD profile of serum IGF-1 during Visit 1 and Visit 3 compared between the ACP-001 dose groups and to the daily rhGH group Uncorrected E-Trough (the pre-dose efficacy response) values at Week 13
Outcome measures
| Measure |
ACP-001, Dose-level 1
n=12 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
|---|---|---|---|---|
|
E-Trough of IGF-1
|
97.17 ng/mL
Standard Deviation 50.53
|
156 ng/mL
Standard Deviation 76.235
|
167.83 ng/mL
Standard Deviation 74.01
|
—
|
PRIMARY outcome
Timeframe: 0 hours to 168 hours at Visit 3 (Week 13)As part of the following endpoint: PD profile of serum IGF-1 during V1 and V3 compared between the ACP-001 dose groups and to the daily rhGH group
Outcome measures
| Measure |
ACP-001, Dose-level 1
n=12 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
|---|---|---|---|---|
|
Emax of IGF-1
|
209.5 ng/mL
Standard Deviation 120.189
|
276 ng/mL
Standard Deviation 126.632
|
289.92 ng/mL
Standard Deviation 129.469
|
—
|
PRIMARY outcome
Timeframe: 0 hours to 168 hours at Visit 3 (Week 13)As part of the following endpoint: PD profile of serum IGF-1 during V1 and V3 compared between the ACP-001 dose groups and to the rhGH group. Uncorrected AUEC0-168 (area under the efficacy curve from 0h-168h) values at Week 13
Outcome measures
| Measure |
ACP-001, Dose-level 1
n=12 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
|---|---|---|---|---|
|
AUEC0-168h of IGF-1
|
28526.19 h*ng/mL
Standard Deviation 15756.44
|
35591.94 h*ng/mL
Standard Deviation 17068.59
|
36066.01 h*ng/mL
Standard Deviation 17379.44
|
—
|
SECONDARY outcome
Timeframe: Baseline to 6 months (Visit 5)Annualized HV during treatment with ACP-001 or daily rhGH at the end of 6 months, for each ACP-001 dose group and for the daily rhGH dose group
Outcome measures
| Measure |
ACP-001, Dose-level 1
n=12 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
n=14 Participants
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
n=13 Participants
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
|---|---|---|---|---|
|
Annualized Height Velocity
|
11.93 cm/year
Standard Deviation 4.066
|
12.89 cm/year
Standard Deviation 3.464
|
13.85 cm/year
Standard Deviation 4.009
|
11.64 cm/year
Standard Deviation 3.592
|
Adverse Events
ACP-001, Dose-level 1
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
ACP-001, Dose-level 2
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
ACP-001, Dose-level 3
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Human Growth Hormone
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
ACP-001, Dose-level 1
n=12 participants at risk
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
n=14 participants at risk
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
n=14 participants at risk
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
n=13 participants at risk
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Inguinal hernia
|
8.3%
1/12 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
Other adverse events
| Measure |
ACP-001, Dose-level 1
n=12 participants at risk
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 2
n=14 participants at risk
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
ACP-001, Dose-level 3
n=14 participants at risk
Once weekly subcutaneous injection of ACP-001
ACP-001: Once weekly subcutaneous injection
|
Human Growth Hormone
n=13 participants at risk
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone
|
|---|---|---|---|---|
|
Immune system disorders
Food allergy
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
General disorders
Fatigue
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
General disorders
Hyperthermia
|
8.3%
1/12 • Number of events 2 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
General disorders
Pyrexia
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
23.1%
3/13 • Number of events 3 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Injury, poisoning and procedural complications
Heat exhaustion
|
8.3%
1/12 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Injury, poisoning and procedural complications
Open wound
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Injury, poisoning and procedural complications
Procedural dizziness
|
8.3%
1/12 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Investigations
Blood triglycerides
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Congenital, familial and genetic disorders
Thalassemia beta
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.7%
1/13 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.7%
1/13 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
14.3%
2/14 • Number of events 2 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Blood and lymphatic system disorders
Iron deficiency anemia
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
14.3%
2/14 • Number of events 2 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.7%
1/13 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Nervous system disorders
Headache
|
16.7%
2/12 • Number of events 2 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.7%
1/13 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Eye disorders
Strabismus
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Ear and labyrinth disorders
Ear pain
|
8.3%
1/12 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.7%
1/13 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Ear and labyrinth disorders
Middle ear inflammation
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.7%
1/13 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Gastrointestinal disorders
Anal pruritis
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Gastrointestinal disorders
Inguinal hernia
|
8.3%
1/12 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Gastrointestinal disorders
Nausea
|
8.3%
1/12 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Gastrointestinal disorders
Vomiting
|
8.3%
1/12 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.7%
1/13 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Endocrine disorders
Hypothyroidism
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.7%
1/13 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Endocrine disorders
Secondary hypothyroidism
|
8.3%
1/12 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Metabolism and nutrition disorders
Decreased appetite
|
8.3%
1/12 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Infections and infestations
Bronchitis
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
14.3%
2/14 • Number of events 2 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.7%
1/13 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Infections and infestations
Nasopharyngitis
|
8.3%
1/12 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.7%
1/13 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Infections and infestations
Respiratory tract infection
|
8.3%
1/12 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Infections and infestations
Rhinitis
|
8.3%
1/12 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Infections and infestations
Tonsilitis
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Infections and infestations
Tooth abcess
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.7%
1/13 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Infections and infestations
Tracheitis
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
|
Infections and infestations
Varicella
|
0.00%
0/12 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/14 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
7.1%
1/14 • Number of events 1 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
0.00%
0/13 • From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place