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.
Who can participate
Age range
3 Years – 12 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of Anti-hGH Binding Antibody Formation
Timeframe: Visit 2 - Visit 5
2
Incidence of Anti-hGH Neutralizing Antibody Formation
Timeframe: Visit 2 - Visit 5
3
Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)
Timeframe: Start of study treatment through Visit 5 (Week 27)
4
Cmax of hGH
Timeframe: 0 hours to 168 hours at Visit 3 (Week 13)
5
AUC0-168h of hGH
Timeframe: 0 hours to 168 hours at Visit 3 (Week 13)
6
E-Trough of IGF-1
Timeframe: 0 hours to 168 hours at Visit 3 (Week 13)
7
Emax of IGF-1
Timeframe: 0 hours to 168 hours at Visit 3 (Week 13)