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

NCTNCT01947907Other idACP-001_CT-004

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Overview

Status: Completed
Phase: Phase 2
Enrollment: 53 participants (actual)
Sponsor: Ascendis Pharma A/S · Industry
Design: Randomized · Open label (no masking)
Countries: 14 countries
Belarus, Bulgaria, Czechia, Egypt, France, Germany, Greece, Hungary, Poland, Romania, Russia, Slovenia, Turkey (Türkiye), Ukraine
Start: Jul 2013
Primary completion: Jul 2015
Completion: Sept 2015
First posted: 23 Sept 2013 (estimated)
Last update posted: 8 Jul 2026
Conditions: Growth Hormone Deficiency (GHD)

Timeline

1 trial · 1 completed · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

2013201420152016

Ph 2
Safety, PK/PD (Pharmacokinetics/Pharmacodynamics) and Efficacy of ACP-001 Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)
NCT01947907
Jul 2013 to Sept 2015

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

A six month study of ACP-001, a long-acting growth hormone product, versus standard human growth hormone therapy. ACP-001 will be given once-a-week, standard human growth hormone (hGH) will be given on a daily basis. The primary aim is to demonstrate safety, pharmacokinetics and pharmacodynamics over a period of six months. A secondary objective is the comparison of height velocity (HV) of the ACP-001 treated groups to the daily hGH treatment group.

Interventions

ACP-001Drug
Human Growth HormoneDrug

Arms4

Experimental
ACP-001, dose-level 1
Once weekly subcutaneous injection of ACP-001
Experimental
ACP-001, dose-level 2
Once weekly subcutaneous injection of ACP-001
Experimental
ACP-001, dose-level 3
Once weekly subcutaneous injection of ACP-001
Active comparator
Human Growth Hormone
Once daily subcutaneous injection of human Growth Hormone (rhGH)

Primary outcomes8

Outcomes the sponsor listed as primary.

Incidence of Anti-hGH Binding Antibody Formation
Time frame · Visit 2 - Visit 5
Registry description
Number of subjects with positive results for anti-hGH binding antibodies at two consecutive post-dose visits
Incidence of Anti-hGH Neutralizing Antibody Formation
Time frame · Visit 2 - Visit 5
Registry description
Number of subjects with positive results for anti-hGH neutralizing antibodies at two consecutive post-dose visits
Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)
Time frame · Start of study treatment through Visit 5 (Week 27)
Registry description
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.
Cmax of hGH
Time frame · 0 hours to 168 hours at Visit 3 (Week 13)
Registry description
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)
AUC0-168h of hGH
Time frame · 0 hours to 168 hours at Visit 3 (Week 13)
Registry description
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)
E-Trough of IGF-1
Time frame · 0 hours to 168 hours at Visit 3 (Week 13)
Registry description
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
Emax of IGF-1
Time frame · 0 hours to 168 hours at Visit 3 (Week 13)
Registry description
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
AUEC0-168h of IGF-1
Time frame · 0 hours to 168 hours at Visit 3 (Week 13)
Registry description
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

Secondary outcomes(1)

Annualized Height Velocity
Time frame · Baseline to 6 months (Visit 5)
Registry description
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

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeNumber· Visit 2 - Visit 5

Incidence of Anti-hGH Binding Antibody Formation

Group	Value (participants)
ACP-001, Dose-level 1	1
ACP-001, Dose-level 2	0
ACP-001, Dose-level 3	0
Human Growth Hormone	0

Primary outcomeNumber· Visit 2 - Visit 5

Incidence of Anti-hGH Neutralizing Antibody Formation

Group	Value (participants)
ACP-001, Dose-level 1	0
ACP-001, Dose-level 2	0
ACP-001, Dose-level 3	0
Human Growth Hormone	0

Primary outcomeNumber· Start of study treatment through Visit 5 (Week 27)