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

Ascendis Pharma Endocrinology Division A/S220 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

220

Conditions

Adult Growth Hormone Deficiency Endocrine System Diseases Hormone Deficiency

Interventions

LonapegsomatropinDRUG

Study participants were individually dosed with subcutaneous injection of Lonapegsomatropin once-weekly for 52 weeks.

Eligibility

Sex: ALLMin age: 23 YearsMax age: 81 Years

Medical Language ↔ Plain English

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: 1. Poorly controlled diabetes, defined as HbA1C higher than 7.5% according to central laboratory at Visit 7 in trial TCH-306 2. 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

Locations (85)

Outcomes

Primary Outcomes

Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and TEAE Leading to Study Discontinuation

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.

Time frame: Up to 52 Weeks

Secondary Outcomes

Change From Baseline in Trunk Percent Fat at Week 52

Trunk percent fat was assessed by dual-energy X-ray absorptiometry.

Time frame: Baseline main trial to week 52 (extension period)

Change From Baseline in Trunk Fat Mass at Week 52

Trunk fat mass was assessed by dual-energy X-ray absorptiometry.

Time frame: Baseline main trial to week 52 (extension period)

Change From Baseline in Total Body Lean Mass at Week 52

Total body lean mass was assessed by dual-energy X-ray absorptiometry.

Time frame: Baseline main trial to week 52 (extension period)

Data from ClinicalTrials.gov

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