A Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency

Study Overview
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Brief Summary
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This is 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 are adults (males and females) with confirmed growth hormone deficiency (GHD) having completed the treatment period in study TCH-306 (foresiGHt).

Official Title
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A Multicenter, Open-Label, Extension Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency

Conditions
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Adult Growth Hormone Deficiency, Endocrine System Diseases, Hormone Deficiency

Intervention / Treatment
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* Drug: Lonapegsomatropin


Participation Criteria
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Eligibility Criteria
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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 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 are met: Poorly controlled diabetes, defined as HbA1C higher than 7.5% according to central laboratory at Visit 6 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. Exceptions are: Resection of in situ carcinoma of the cervix uteri Complete eradication of squamous cell or basal cell carcinoma of the skin Known history of hypersensitivity and/or idiosyncrasy to the investigational product (somatropin or excipients) Female who is pregnant, plans to become pregnant, or is 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

Ages Eligible for Study
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Minimum Age: 23 Years
Maximum Age: 81 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Open label treatment with weekly Lonapegsomatropin
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Lonapegsomatropin<br>Lonapegsomatropin administered once-weekly by subcutaneous injection | Drug: Lonapegsomatropin<br>* Study participants are individually dosed with subcutaneous injection of Lonapegsomatropin once-weekly for 52 weeks<br>* Other names: ACP-011;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment-Emergent Adverse Events | To evaluate the safety and tolerability of once-weekly lonapegsomatropin in adults with growth hormone deficiency | Throughout the 52 week treatment period | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from Baseline in Trunk Percent Fat | Change from baseline in trunk percent fat (as assessed by dual-energy x ray absorptiometry [DXA]) at Week 52 | Week 52 | 
| Change from Baseline in Trunk Fat Mass | Change from baseline in trunk fat mass (as assessed by dual-energy x ray absorptiometry [DXA]) at Week 52 | Week 52 | 
| Change from Baseline in Total Body Lean Mass | Change from baseline in total body lean mass (as assessed by dual-energy x ray absorptiometry [DXA]) at Week 52 | Week 52 | 
| Evaluate serum IGF-1 and IGF-1 SDS | To evaluate the pharmacodynamics (PD) of once-weekly lonapegsomatropin in adults with growth hormone deficiency | Week 52 | 
| Evaluate serum hGH, lonapegsomatropin, and mPEG levels | To evaluate the pharmacokinetics (PK) of once-weekly lonapegsomatropin in adults with growth hormone deficiency | Week 52 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Human Growth Hormone, hGH, rhGH, GHD, Adult Growth Hormone Deficiency, Long Acting Growth Hormone, Lonapegsomatropin, Prodrug, Growth Hormone Replacement Therapy, Sustained Release Growth Hormone, Growth Hormone Deficiency, TransCon hGH, Skytrofa