Phase 3
N=146
A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone Deficiency
Growth Hormone Deficiency, Pediatric · Endocrine System Diseases · Hormone Deficiency · Pituitary Diseases
Bottom Line
View on ClinicalTrials.gov: NCT03305016 ↗Enrolled (actual)
146
Serious AEs
0.7%
Results posted
Jan 2022
Primary outcome: Primary: Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability] — 83; 6; 1; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- TransCon hGH (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Ascendis Pharma Endocrinology Division A/S
- Primary completion
- Mar 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability] |
83; 6; 1; 0 | — |
| SECONDARY Annualized Height Velocity (AHV) at 26 Weeks of Weekly Lonapegsomatropin Treatment |
8.72 | — |
| SECONDARY Number of Subjects With IGF-1 Standard Deviation Score (SDS) in the Range of 0.0 to +2.0 at 26 Weeks of Weekly Lonapegsomatropin Treatment |
74 | — |
| SECONDARY Change in Height Standard Deviation Scores (SDS) at 26 Weeks of Weekly Lonapegsomatropin Treatment |
0.25 | — |
| SECONDARY Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation |
4 | — |
Summary
A 26 week trial of TransCon hGH, a long-acting growth hormone product, administered once-a-week. Approximately 150 children (males and females) with growth hormone deficiency (GHD) will be included. All study participants will receive TransCon hGH. This is a global trial that will be conducted in, but not limited to, the United States, Canada, Australia, and New Zealand.
Eligibility Criteria
Inclusion Criteria
- Investigator-determined GHD diagnosis prior to the historical initiation of daily hGH therapy.
- 6 months to 17 years old, inclusive, at Visit 1
- If 3 to 17 years old, are taking daily hGH at a dose of ≥ 0.20 mg hGH/kg/week for at least 13 weeks but no more than 130 weeks prior to Visit 1
- If ≥ 6 months but 80 kg at Visit 1
- Females of child-bearing potential
- History of malignant disease
- Any clinically significant abnormality likely to affect growth or the ability to evaluate growth (eg, chronic diseases or conditions such as renal insufficiency, spinal cord irradiation, hypothyroidism, active celiac disease, malnutrition or psychosocial dwarfism)
- Poorly-controlled diabetes mellitus (HbA1c >8.0%) or diabetic complications
- Known neutralizing antibodies against hGH
- Major medical conditions, unless approved by Medical Monitor
- Pregnancy
- Presence of contraindications to hGH treatment
- Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver)
- Participation in any other trial of an investigational agent within 30 days prior to Visit 1
- Prior exposure to investigational hGH
Data sourced from ClinicalTrials.gov (NCT03305016). 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.