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A Safety, Pharmacokinetic and Pharmacodynamic Study of ACP-001 (TransCon hGH) in Adults With Growth Hormone Deficiency

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ClinicalTrials.gov Identifier: NCT01247675
Recruitment Status : Completed
First Posted : November 24, 2010
Results First Posted : March 9, 2017
Last Update Posted : March 9, 2017
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Adult Growth Hormone Deficiency
Interventions Drug: ACP-001 (TransCon hGH)
Drug: Omnitrope
Enrollment 37
Recruitment Details  
Pre-assignment Details  
Arm/Group Title ACP-001, 0.02 mg hGH/kg/wk ACP-001, 0.04 mg hGH/kg/wk ACP-001, 0.08 mg hGH/kg/wk Omnitrope, 0.04 mg hGH/kg/wk
Hide Arm/Group Description ACP-001 (TransCon hGH): subcutaneous, weekly injection equivalent to 0.02 mg hGH/kg/wk over 4 weeks. Prior to randomization, study subjects entered a 14 to 21 day wash out period following cessation of daily growth hormone therapy. ACP-001 (TransCon hGH): subcutaneous, weekly injection equivalent to 0.04 mg hGH/kg/wk over 4 weeks. Prior to randomization, study subjects entered a 14 to 21 day wash out period following cessation of daily growth hormone therapy. ACP-001 (TransCon hGH): subcutaneous, weekly injection equivalent to 0.08 mg hGH/kg/wk over 4 weeks. Prior to randomization, study subjects entered a 14 to 21 day wash out period following cessation of daily growth hormone therapy. Human Growth Hormone: subcutaneous, daily injection of Omnitrope equivalent to 0.04 mg/kg/wk over 4 weeks. Prior to randomization, study subjects entered a wash out period of 14 to 21 days following cessation of daily growth hormone therapy.
Period Title: Overall Study
Started 10 10 9 8
Completed 8 8 9 8
Not Completed 2 2 0 0
Arm/Group Title ACP-001, 0.02 mg hGH/kg/wk ACP-001, 0.04 mg hGH/kg/wk ACP-001, 0.08 mg hGH/kg/wk Omnitrope, 0.04 mg hGH/kg/wk Total
Hide Arm/Group Description ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.02 mg hGH/kg/wk for 4 weeks ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.04 mg hGH/kg/wk for 4 weeks ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.08 mg hGH/kg/wk for 4 weeks Human Growth Hormone: s.c., daily injection equivalent to 0.04 mg hGH/kg/wk for 4 weeks Total of all reporting groups
Overall Number of Baseline Participants 10 10 9 8 37
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 10 participants 10 participants 9 participants 8 participants 37 participants
55.7  (12.6) 45.9  (15.0) 51.6  (18.1) 44.0  (15.7) 49.5  (15.4)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 10 participants 9 participants 8 participants 37 participants
Female
5
  50.0%
6
  60.0%
5
  55.6%
3
  37.5%
19
  51.4%
Male
5
  50.0%
4
  40.0%
4
  44.4%
5
  62.5%
18
  48.6%
Weight  
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 10 participants 10 participants 9 participants 8 participants 37 participants
82.3  (19.8) 79.6  (17.0) 92.5  (20.5) 74.7  (16.6) 82.4  (18.9)
Body Mass Index  
Mean (Standard Deviation)
Unit of measure:  Kg/m²
Number Analyzed 10 participants 10 participants 9 participants 8 participants 37 participants
27.6  (4.4) 27.9  (4.4) 30.7  (4.4) 25.7  (4.4) 28.0  (4.6)
1.Primary Outcome
Title Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)
Hide Description Assessment of local tolerability was performed by examining injection sites by the investigator during study visits, and on the basis of records in the Patient Diary. Assessments included erythema, swelling, or pain.
Time Frame Start of study treatment through Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
All patients who were randomized and received at least one dose of test product were included in the Safety analysis.
Arm/Group Title ACP-001, 0.02 mg hGH/kg/wk ACP-001, 0.04 mg hGH/kg/wk ACP-001, 0.08 mg hGH/kg/wk Omnitrope, 0.04 mg hGH/kg/wk
Hide Arm/Group Description:
ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.02 mg hGH/kg/wk for 4 weeks
ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.04 mg hGH/kg/wk for 4 weeks
ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.08 mg hGH/kg/wk for 4 weeks
Human Growth Hormone: s.c., daily injection of Omnitrope equivalent to 0.04 mg hGH/kg/wk for 4 weeks
Overall Number of Participants Analyzed 10 10 9 8
Measure Type: Number
Unit of Measure: Number of subjects with any symptom
3 3 2 1
2.Primary Outcome
Title Incidence of Treatment Emergent Anti-hGH Binding Antibody Formation
Hide Description Number of subjects with treatment emergent anti-hGH binding antibodies
Time Frame Start of study treatment through Day 42
Hide Outcome Measure Data
Hide Analysis Population Description
All patients who were randomized and received at least one dose of test product were included in the Safety analysis.
Arm/Group Title ACP-001, 0.02 mg hGH/kg/wk ACP-001, 0.04 mg hGH/kg/wk ACP-001, 0.08 mg hGH/kg/wk Omnitrope, 0.04 mg hGH/kg/wk
Hide Arm/Group Description:
ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.02 mg hGH/kg/wk for 4 weeks
ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.04 mg hGH/kg/wk for 4 weeks
ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.08 mg hGH/kg/wk for 4 weeks
Human Growth Hormone: s.c., daily injection of Omnitrope equivalent to 0.04 mg hGH/kg/wk for 4 weeks
Overall Number of Participants Analyzed 10 10 9 8
Measure Type: Number
Unit of Measure: Participants
0 0 0 0
3.Secondary Outcome
Title Cmax of hGH
Hide Description

As part of the following endpoint:

Pharmacokinetic (PK) profile of serum human Growth Hormone (hGH) from ACP-001 treated dose groups compared to the PK profile of hGH from the daily Omnitrope treated group.

Cmax (maximum value of concentration) values at Week 4
Time Frame Days 22 to 29
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic and pharmacodynamic analysis was performed on all patients who had at least one measurement of the primary variable and who had attended the Day 28 study visit. Two patients in Cohort 2 (ACP-001, 0.04 mg hGH/kg/wk) were withdrawn from the study before Day 28 and were therefore excluded from the analysis.
Arm/Group Title ACP-001, 0.02 mg hGH/kg/wk ACP-001, 0.04 mg hGH/kg/wk ACP-001, 0.08 mg hGH/kg/wk Omnitrope, 0.04 mg hGH/kg/wk
Hide Arm/Group Description:
ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.02 mg hGH/kg/wk for 4 weeks
ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.04 mg hGH/kg/wk for 4 weeks
ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.08 mg hGH/kg/wk for 4 weeks
Human Growth Hormone: s.c., daily injection of Omnitrope equivalent to 0.04 mg hGH/kg/wk for 4 weeks
Overall Number of Participants Analyzed 10 8 9 8
Mean (Standard Deviation)
Unit of Measure: ng/mL
1.2  (0.8) 1.9  (0.9) 3.8  (2.0) 2.0  (1.1)
4.Secondary Outcome
Title Emax of IGF-I
Hide Description

As part of the following endpoint:

Pharmacodynamic (PD) response of serum Insulin-like Growth Factor-I (IGF-I) from ACP-001 treated dose groups compared to the PD response of IGF-I from the daily Omnitrope treated group.

Emax (maximum observed response) values at Week 4
Time Frame Days 22 to 29
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic and pharmacodynamic analysis was performed on all patients who had at least one measurement of the primary variable and who had attended the Day 28 study visit. Two patients in Cohort 2 (ACP-001, 0.04 mg hGH/kg/wk) were withdrawn from the study before Day 28 and were therefore excluded from the analysis.
Arm/Group Title ACP-001, 0.02 mg hGH/kg/wk ACP-001, 0.04 mg hGH/kg/wk ACP-001, 0.08 mg hGH/kg/wk Omnitrope, 0.04 mg hGH/kg/wk
Hide Arm/Group Description:
ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.02 mg hGH/kg/wk for 4 weeks
ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.04 mg hGH/kg/wk
ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.08 mg hGH/kg/wk
Human Growth Hormone: s.c., daily injection of Omnitrope equivalent to 0.04 mg hGH/kg/wk for 4 weeks
Overall Number of Participants Analyzed 10 8 9 8
Mean (Standard Deviation)
Unit of Measure: ng/mL
71.8  (26.9) 108.6  (91.7) 125.6  (70.1) 109.8  (37.1)
Time Frame Reported Adverse Events include events starting on or after Day 0 and ending on or before Day 42.
Adverse Event Reporting Description A subject with more than one finding in a specific category was only counted once.
 
Arm/Group Title ACP-001, 0.02 mg hGH/kg/wk ACP-001, 0.04 mg hGH/kg/wk ACP-001, 0.08 mg hGH/kg/wk Omnitrope, 0.04 mg hGH/kg/wk
Hide Arm/Group Description ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.02 mg hGH/kg/wk for 4 weeks ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.04 mg hGH/kg/wk for 4 weeks ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.08 mg hGH/kg/wk Human Growth Hormone: s.c., daily injection of Omnitrope equivalent to 0.04 mg hGH/kg/wk for 4 weeks
All-Cause Mortality
ACP-001, 0.02 mg hGH/kg/wk ACP-001, 0.04 mg hGH/kg/wk ACP-001, 0.08 mg hGH/kg/wk Omnitrope, 0.04 mg hGH/kg/wk
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/10 (0.00%)      0/10 (0.00%)      0/9 (0.00%)      0/8 (0.00%)    
Hide Serious Adverse Events
ACP-001, 0.02 mg hGH/kg/wk ACP-001, 0.04 mg hGH/kg/wk ACP-001, 0.08 mg hGH/kg/wk Omnitrope, 0.04 mg hGH/kg/wk
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/10 (10.00%)      0/10 (0.00%)      0/9 (0.00%)      0/8 (0.00%)    
Endocrine disorders         
Severe Adrenal Crisis † 1 [1]  1/10 (10.00%)  1 0/10 (0.00%)  0 0/9 (0.00%)  0 0/8 (0.00%)  0
Respiratory, thoracic and mediastinal disorders         
Pleuritic Pain † 1  1/10 (10.00%)  1 0/10 (0.00%)  0 0/9 (0.00%)  0 0/8 (0.00%)  0
1
Term from vocabulary, MedDRA 13.1
†
Indicates events were collected by systematic assessment
[1]
Patient had a history of adrenal crisis
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
ACP-001, 0.02 mg hGH/kg/wk ACP-001, 0.04 mg hGH/kg/wk ACP-001, 0.08 mg hGH/kg/wk Omnitrope, 0.04 mg hGH/kg/wk
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   6/10 (60.00%)      9/10 (90.00%)      7/9 (77.78%)      7/8 (87.50%)    
Gastrointestinal disorders         
Nausea † 1  0/10 (0.00%)  0 3/10 (30.00%)  3 2/9 (22.22%)  2 1/8 (12.50%)  3
Diarrhea † 1  1/10 (10.00%)  1 2/10 (20.00%)  2 0/9 (0.00%)  0 1/8 (12.50%)  1
Abdominal distension † 1  0/10 (0.00%)  0 1/10 (10.00%)  1 1/9 (11.11%)  3 1/8 (12.50%)  3
Dyspepsia † 1  1/10 (10.00%)  1 0/10 (0.00%)  0 1/9 (11.11%)  1 1/8 (12.50%)  1
Abdominal pain upper † 1  1/10 (10.00%)  1 1/10 (10.00%)  1 0/9 (0.00%)  0 0/8 (0.00%)  0
Toothache † 1  0/10 (0.00%)  0 1/10 (10.00%)  1 1/9 (11.11%)  1 0/8 (0.00%)  0
General disorders         
Fatigue † 1  3/10 (30.00%)  8 3/10 (30.00%)  4 1/9 (11.11%)  4 2/8 (25.00%)  12
Application site erythema † 1  2/10 (20.00%)  3 0/10 (0.00%)  0 0/9 (0.00%)  0 1/8 (12.50%)  1
Application site induration † 1  2/10 (20.00%)  3 0/10 (0.00%)  0 0/9 (0.00%)  0 1/8 (12.50%)  1
Edema peripheral † 1  0/10 (0.00%)  0 1/10 (10.00%)  1 1/9 (11.11%)  1 1/8 (12.50%)  2
Application site pruritus † 1  1/10 (10.00%)  2 0/10 (0.00%)  0 0/9 (0.00%)  0 1/8 (12.50%)  1
Asthenia † 1  2/10 (20.00%)  2 0/10 (0.00%)  0 0/9 (0.00%)  0 0/8 (0.00%)  0
Condition aggravated † 1  0/10 (0.00%)  0 1/10 (10.00%)  3 1/9 (11.11%)  3 0/8 (0.00%)  0
Influenza like illness † 1  0/10 (0.00%)  0 2/10 (20.00%)  2 0/9 (0.00%)  0 0/8 (0.00%)  0
Injection site pain † 1  1/10 (10.00%)  2 1/10 (10.00%)  4 0/9 (0.00%)  0 0/8 (0.00%)  0
Infections and infestations         
Nasopharyngitis † 1  0/10 (0.00%)  0 1/10 (10.00%)  2 2/9 (22.22%)  2 2/8 (25.00%)  2
Pharyngitis † 1  0/10 (0.00%)  0 1/10 (10.00%)  1 1/9 (11.11%)  2 1/8 (12.50%)  1
Bronchitis † 1  0/10 (0.00%)  0 1/10 (10.00%)  1 1/9 (11.11%)  2 0/8 (0.00%)  0
Musculoskeletal and connective tissue disorders         
Arthralgia † 1  2/10 (20.00%)  2 1/10 (10.00%)  2 1/9 (11.11%)  2 0/8 (0.00%)  0
Back pain † 1  1/10 (10.00%)  1 0/10 (0.00%)  0 2/9 (22.22%)  3 0/8 (0.00%)  0
Myalgia † 1  0/10 (0.00%)  0 1/10 (10.00%)  1 0/9 (0.00%)  0 1/8 (12.50%)  1
Neck pain † 1  0/10 (0.00%)  0 0/10 (0.00%)  0 1/9 (11.11%)  3 1/8 (12.50%)  1
Nervous system disorders         
Headache † 1  2/10 (20.00%)  2 3/10 (30.00%)  3 2/9 (22.22%)  7 2/8 (25.00%)  4
Dizziness † 1  0/10 (0.00%)  0 1/10 (10.00%)  1 1/9 (11.11%)  1 0/8 (0.00%)  0
Psychiatric disorders         
Affect lability † 1  2/10 (20.00%)  2 0/10 (0.00%)  0 0/9 (0.00%)  0 0/8 (0.00%)  0
Depressed mood † 1  2/10 (20.00%)  2 0/10 (0.00%)  0 0/9 (0.00%)  0 0/8 (0.00%)  0
Insomnia † 1  2/10 (20.00%)  3 0/10 (0.00%)  0 0/9 (0.00%)  0 0/8 (0.00%)  0
Stress † 1  0/10 (0.00%)  0 0/10 (0.00%)  0 0/9 (0.00%)  0 2/8 (25.00%)  3
Renal and urinary disorders         
Hematuria † 1  0/10 (0.00%)  0 0/10 (0.00%)  0 0/9 (0.00%)  0 2/8 (25.00%)  2
Respiratory, thoracic and mediastinal disorders         
Oropharyngeal pain † 1  1/10 (10.00%)  1 3/10 (30.00%)  4 2/9 (22.22%)  2 2/8 (25.00%)  2
Cough † 1  0/10 (0.00%)  0 3/10 (30.00%)  3 0/9 (0.00%)  0 0/8 (0.00%)  0
Rales † 1  1/10 (10.00%)  1 1/10 (10.00%)  1 1/9 (11.11%)  1 0/8 (0.00%)  0
Rhinorrhea † 1  1/10 (10.00%)  1 1/10 (10.00%)  1 1/9 (11.11%)  1 0/8 (0.00%)  0
Nasal congestion † 1  0/10 (0.00%)  0 1/10 (10.00%)  1 0/9 (0.00%)  0 1/8 (12.50%)  1
Skin and subcutaneous tissue disorders         
Dermatitis contact † 1  1/10 (10.00%)  2 0/10 (0.00%)  0 0/9 (0.00%)  0 1/8 (12.50%)  1
1
Term from vocabulary, MedDRA 13.1
†
Indicates events were collected by systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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.
 
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Michael Beckert, MD
Organization: Ascendis Pharma A/S
Phone: (+49) 172-155-2596
EMail: mb@ascendispharma.com
Layout table for additonal information
Responsible Party: Ascendis Pharma A/S
ClinicalTrials.gov Identifier: NCT01247675    
Other Study ID Numbers: ACP-001 CT-002
First Submitted: November 23, 2010
First Posted: November 24, 2010
Results First Submitted: November 3, 2016
Results First Posted: March 9, 2017
Last Update Posted: March 9, 2017