Phase 2
N=53
Safety, PK/PD (Pharmacokinetics/Pharmacodynamics) and Efficacy of ACP-001 Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)
Growth Hormone Deficiency (GHD)
Bottom Line
View on ClinicalTrials.gov: NCT01947907 ↗Enrolled (actual)
53
Serious AEs
1.9%
Results posted
Jan 2017
Primary outcome: Primary: Incidence of Anti-hGH Binding Antibody Formation — 1; 0; 0; 0 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- ACP-001 (Drug); Human Growth Hormone (Drug)
- Age
- Pediatric · 3+ yrs
- Sex
- All
- Sponsor
- Ascendis Pharma A/S
- Primary completion
- Jul 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Anti-hGH Binding Antibody Formation |
1; 0; 0; 0 | — |
| PRIMARY Incidence of Anti-hGH Neutralizing Antibody Formation |
0; 0; 0; 0 | — |
| PRIMARY Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator) |
7; 6; 6; 6 | — |
| PRIMARY Cmax of hGH |
12.558; 13.418; 31.8; 16.612 | — |
| PRIMARY AUC0-168h of hGH |
696.34; 787.41; 2167.43; 556.88 | — |
| PRIMARY E-Trough of IGF-1 |
97.17; 156; 167.83 | — |
| PRIMARY Emax of IGF-1 |
209.5; 276; 289.92 | — |
| PRIMARY AUEC0-168h of IGF-1 |
28526.19; 35591.94; 36066.01 | — |
| SECONDARY Annualized Height Velocity |
11.93; 12.89; 13.85; 11.64 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- Prepubertal children, Tanner stage 1
- Diagnosis of GHD, confirmed by two stimulation tests
- Bone age not greater than chronological age
- Impaired height and height velocity
- BMI within +/- 2 SD (standard deviations)
- Baseline IGF-1 (insulin-like growth factor)
- Normal fundoscopy
- Stable hormonal replacement therapy (other than hGH)
- Written Informed Consent
Exclusion Criteria
- Prior exposure to rhGH or IGF-I
- Past or present intracranial tumor; history or presence of malignant disease
- Small for gestational age (SGA)
- Malnutrition
- Psychosocial dwarfism
- Coeliac disease
- Anti-hGH antibodies
- Diabetes mellitus
- Chromosomal abnormalities (e.g. Turner syndrome, SHOX)
- Closed epiphyses
- Known or suspected HIV infection
Data sourced from ClinicalTrials.gov (NCT01947907). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.