Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ACP-001 · 3 trials · 3 indications
Number of subjects with positive results for anti-hGH binding antibodies at two consecutive post-dose visits
Number of subjects with positive results for anti-hGH neutralizing antibodies at two consecutive post-dose visits
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.
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)
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)
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
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
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
Number of subjects with treatment emergent anti-hGH binding antibodies
| Arm | Type | Description |
|---|---|---|
| ACP-001, dose-level 1 | EXPERIMENTAL | Once weekly subcutaneous injection of ACP-001 |
| ACP-001, dose-level 2 | EXPERIMENTAL | Once weekly subcutaneous injection of ACP-001 |
| ACP-001, dose-level 3 | EXPERIMENTAL | Once weekly subcutaneous injection of ACP-001 |
| Human Growth Hormone | ACTIVE_COMPARATOR | Once daily subcutaneous injection of human Growth Hormone (rhGH) |
| ACP-001, 0.02 mg hGH/kg/wk | EXPERIMENTAL | Once weekly subcutaneous injection of ACP-001 equivalent to 0.02 mg hGH/kg/week for 4 weeks |
| ACP-001, 0.04 mg hGH/kg/wk | EXPERIMENTAL | Once weekly subcutaneous injection of ACP-001 equivalent to 0.04 mg hGH/kg/week for 4 weeks |
| ACP-001, 0.08 mg hGH/kg/wk | EXPERIMENTAL | Once weekly subcutaneous injection of ACP-001 equivalent to 0.08 mg hGH/kg/week for 4 weeks |
| Omnitrope, 0.04 mg hGH/kg/wk | ACTIVE_COMPARATOR | Once daily subcutaneous injection of human Growth Hormone (Omnitrope) equivalent to 0.04 mg hGH/kg/week for 4 weeks |
| ACP-001, dose-level 4 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| ACP-001 | DRUG | Once weekly subcutaneous injection |
| Human Growth Hormone | DRUG | Once daily subcutaneous injection of human Growth Hormone |
| ACP-001 (TransCon hGH) | DRUG | s.c., weekly injection |
| Omnitrope | DRUG | s.c., daily injection |
| ACP-001 (TransCon PEG hGH) | DRUG | ACP-001, dose-level 1, s.c., single-dose |
| Placebo | DRUG | Placebo, s.c., single-dose |
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 fu...
ACP-001 is an investigational small molecule being developed for growth hormone deficiency (GHD), including adult growth hormone deficiency. It has been studied in healthy volunteers and in children with growth hormone deficiency. The drug is in clinical development and is not approved by the FDA.
ACP-001 is a long-acting growth hormone product, designed to provide sustained exposure to growth hormone. It is being studied for its pharmacokinetics, pharmacodynamics, safety, and efficacy in growth hormone deficiency. The drug is administered weekly, compared to daily human growth hormone.
ACP-001 is being developed by Ascendis Pharma A/S, a biopharmaceutical company listed on the NASDAQ under the ticker ASND. The company is conducting clinical trials to evaluate the drug's safety and efficacy in growth hormone deficiency.
ACP-001 has completed Phase 2 clinical trials. It has been studied in Phase 1 and Phase 2 trials, including a Phase 2 study in adults with growth hormone deficiency and a Phase 2 study in children with growth hormone deficiency. The drug is investigational and not yet approved.
ACP-001 has been studied in three completed clinical trials: NCT01010425, a Phase 1 study in healthy volunteers; NCT01247675, a Phase 2 study in adults with growth hormone deficiency; and NCT01947907, a Phase 2 study in children with growth hormone deficiency. All trials are completed.
ACP-001 is also known as TransCon hGH, as indicated in the clinical trial titles. The drug is a long-acting growth hormone formulation being developed for growth hormone deficiency. It is administered weekly and has been studied in both adult and pediatric populations.