A Multicenter, Open-Label, Extension Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Enrollment
- 220
- Locations
- 86
- Primary Endpoint
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and TEAE Leading to Study Discontinuation
Study Overview
Brief Summary
This was a phase 3 open-label multicenter extension study designed to evaluate the long-term safety and efficacy of Lonapegsomatropin administered once-weekly. The study participants were adults (males and females) with confirmed growth hormone deficiency (GHD) having completed the treatment period in study TCH-306 (foresiGHt; NCT04615273).
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 23 Years to 81 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Signing of the trial specific informed consent
- •Completion of the treatment period and Visit 7 assessments of trial TCH-306, including collection and upload of Visit 7 dual-X-ray-absorptiometry (DXA) scan
- •Fundoscopy at Visit 7 in trial TCH-306 without signs/symptoms of intracranial hypertension or diabetic retinopathy stage 2 / moderate or above
Exclusion Criteria
- •Diabetes mellitus if any of the following were met:
- •Poorly controlled diabetes, defined as HbA1C higher than 7.5% according to central laboratory at Visit 7 in trial TCH-306
- •Use of diabetes mellitus drugs other than metformin and/or dipeptidyl peptidase-4 (DPP-4) inhibitors
- •Active malignant disease or history of malignancy.
- •Known history of hypersensitivity and/or idiosyncrasy to the investigational product (somatropin or excipients)
- •Female who was pregnant, plans to become pregnant, or was breastfeeding
- •Female participant of childbearing potential (i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile) not willing throughout the trial to use contraceptives as required by local law or practice. Details included in Appendix 4/section 10.4 of the protocol
- •Male participant not willing throughout the trial to use contraceptives as required by local law or practice. Details included in Appendix 4/ section 10.4 of the protocol
- •Any disease or condition that, in the judgement of the investigator, may make the participant unlikely to comply with the requirements of the protocol or any condition that presents undue risk from the investigational product or trial procedures
Arms & Interventions
Lonapegsomatropin/Lonapegsomatropin
Participants who had completed treatment with lonapegsomatropin in TCH-306 study were enrolled in the extension study and received lonapegsomatropin administered once weekly by subcutaneous injection for a treatment period of up to 52 weeks.
Intervention: Lonapegsomatropin (Drug)
Placebo/Lonapegsomatropin
Participants who had completed treatment with placebo in TCH-306 study were enrolled in the extension study and received lonapegsomatropin administered once weekly by subcutaneous injection for a treatment period of up to 52 weeks.
Intervention: Lonapegsomatropin (Drug)
Somatropin/Lonapegsomatropin
Participants who had completed treatment with somatropin in TCH-306 study were enrolled in the extension study and received lonapegsomatropin administered once weekly by subcutaneous injection for a treatment period of up to 52 weeks.
Intervention: Lonapegsomatropin (Drug)
Outcomes
Primary Outcomes
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and TEAE Leading to Study Discontinuation
Time Frame: Up to 52 Weeks
An Adverse Event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and which did not necessarily have a causal relationship with the treatment. An AE was considered a TEAE if it occurred on or after the first dose of investigational product and was not present prior to the first dose, or it was present at the first dose but increased in severity during the trial. A serious AE was any untoward medical occurrence at any dose that met any of the following criteria: resulted in death; was life threatening; required or prolonged inpatient hospitalization; resulted in persistent or significant disability/incapacity; resulted in a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the trial drug or was considered a significant medical event by the investigator.
Secondary Outcomes
- Change From Baseline in Trunk Percent Fat at Week 52(Baseline main trial to week 52 (extension period))
- Change From Baseline in Trunk Fat Mass at Week 52(Baseline main trial to week 52 (extension period))
- Change From Baseline in Total Body Lean Mass at Week 52(Baseline main trial to week 52 (extension period))