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Safety, PK/PD (Pharmacokinetics/Pharmacodynamics) and Efficacy of ACP-001 Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)

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ClinicalTrials.gov Identifier: NCT01947907
Recruitment Status : Completed
First Posted : September 23, 2013
Results First Posted : January 19, 2017
Last Update Posted : January 19, 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: Growth Hormone Deficiency (GHD)
Interventions: Drug: ACP-001
Drug: Human Growth Hormone

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
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


Participant Flow:   Overall Study
    ACP-001, Dose-level 1   ACP-001, Dose-level 2   ACP-001, Dose-level 3   Human Growth Hormone
STARTED   12   14   14   13 
COMPLETED   12   14   14   13 
NOT COMPLETED   0   0   0   0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
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

Total Total of all reporting groups

Baseline Measures
   ACP-001, Dose-level 1   ACP-001, Dose-level 2   ACP-001, Dose-level 3   Human Growth Hormone   Total 
Overall Participants Analyzed 
[Units: Participants]
 12   14   14   13   53 
Age 
[Units: Participants]
Count of Participants
         
<=18 years      12 100.0%      14 100.0%      14 100.0%      13 100.0%      53 100.0% 
Between 18 and 65 years      0   0.0%      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
>=65 years      0   0.0%      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 7.98  (2.889)   8.24  (2.13)   7.32  (2.784)   7.53  (2.483)   7.77  (2.57) 
Gender 
[Units: Participants]
Count of Participants
         
Female      3  25.0%      4  28.6%      5  35.7%      3  23.1%      15  28.3% 
Male      9  75.0%      10  71.4%      9  64.3%      10  76.9%      38  71.7% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
         
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Asian      0   0.0%      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Black or African American      0   0.0%      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
White      12 100.0%      14 100.0%      14 100.0%      13 100.0%      53 100.0% 
More than one race      0   0.0%      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Region of Enrollment 
[Units: Participants]
         
Russian Federation   4   4   7   6   21 
Romania   1   0   0   1   2 
Hungary   0   0   1   0   1 
Egypt   2   4   0   1   7 
Ukraine   1   4   1   1   7 
Belarus   2   2   2   1   7 
Greece   1   0   2   0   3 
Turkey   1   0   0   2   3 
Poland   0   0   1   0   1 
Bulgaria   0   0   0   1   1 
Baseline IGF-1 SDS (standard deviation score) 
[Units: Standard Deviation Score]
Mean (Standard Deviation)
 -2.034  (0.7429)   -2.017  (0.7713)   -2.19  (0.7169)   -2.502  (0.896)   -2.19  (0.782) 
Baseline Height SDS 
[Units: Standard Deviation Score]
Mean (Standard Deviation)
 -3.05  (1.127)   -2.75  (0.383)   -3.17  (1.04)   -3.27  (1.077)   -3.06  (0.905) 
Screening Peak GH Values 
[Units: ng/mL]
Mean (Standard Deviation)
 5.09  (3.169)   5.16  (2.598)   4.44  (2.770)   5.15  (3.068)   4.95  (2.829) 


  Outcome Measures

1.  Primary:   Incidence of Anti-hGH Binding Antibody Formation   [ Time Frame: Visit 2 - Visit 5 ]

2.  Primary:   Incidence of Anti-hGH Neutralizing Antibody Formation   [ Time Frame: Visit 2 - Visit 5 ]

3.  Primary:   Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)   [ Time Frame: Start of study treatment through Visit 5 (Week 27) ]

4.  Primary:   Cmax of hGH   [ Time Frame: 0 hours to 168 hours at Visit 3 (Week 13) ]

5.  Primary:   AUC0-168h of hGH   [ Time Frame: 0 hours to 168 hours at Visit 3 (Week 13) ]

6.  Primary:   E-Trough of IGF-1   [ Time Frame: 0 hours to 168 hours at Visit 3 (Week 13) ]

7.  Primary:   Emax of IGF-1   [ Time Frame: 0 hours to 168 hours at Visit 3 (Week 13) ]

8.  Primary:   AUEC0-168h of IGF-1   [ Time Frame: 0 hours to 168 hours at Visit 3 (Week 13) ]

9.  Secondary:   Annualized Height Velocity   [ Time Frame: Baseline to 6 months (Visit 5) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  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.


Results Point of Contact:  
Name/Title: VP Clinical Development
Organization: Ascendis Pharma A/S
phone: +49 172 155 2596
e-mail: mb@ascendispharma.com


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Ascendis Pharma A/S
ClinicalTrials.gov Identifier: NCT01947907     History of Changes
Other Study ID Numbers: ACP-001_CT-004
First Submitted: June 3, 2013
First Posted: September 23, 2013
Results First Submitted: October 3, 2016
Results First Posted: January 19, 2017
Last Update Posted: January 19, 2017