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

Ascendis Pharma

Status and phase: Completed; Phase 3

Conditions: Hormone Deficiency; Adult Growth Hormone Deficiency; Endocrine System Diseases

Treatments: Drug: Lonapegsomatropin

Study type: Interventional

Funder types: Industry

Identifiers

NCT05171855

TCH-306EXT

Details and patient eligibility

About

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).

Enrollment: 220 patients

Sex: All

Ages: 23 to 81 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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220 participants in 3 patient groups

Lonapegsomatropin/Lonapegsomatropin

Experimental group

Description:

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.

Treatment:

Drug: Lonapegsomatropin

Placebo/Lonapegsomatropin

Experimental group

Description:

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.

Treatment:

Drug: Lonapegsomatropin

Somatropin/Lonapegsomatropin

Experimental group

Description:

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.

Treatment:

Drug: Lonapegsomatropin