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

Endocrine System Diseases Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05171855
• Other study ID #: TCH-306EXT
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: December 16, 2021
• Est. completion date: January 2025

Study information

• Verified date: June 2024
• Source: Ascendis Pharma A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 233
• Est. completion date: January 2025
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 23 Years to 81 Years

Eligibility

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:

1. Poorly controlled diabetes, defined as HbA1C higher than 7.5% according to central laboratory at Visit 6 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. Exceptions are:

1. Resection of in situ carcinoma of the cervix uteri

2. 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

Related Conditions & MeSH terms

• Adult Growth Hormone Deficiency
• Dwarfism, Pituitary
• Endocrine System Diseases
• Hormone Deficiency

Intervention

Drug:
• Lonapegsomatropin
Study participants are individually dosed with subcutaneous injection of Lonapegsomatropin once-weekly for 52 weeks

Locations

Country	Name	City	State
Armenia	Ascendis Pharma Investigational Site	Yerevan
Australia	Ascendis Pharma Investigational Site	Box Hill	Victoria
Australia	Ascendis Pharma Investigational Site	Fitzroy	Victoria
Australia	Ascendis Pharma Investigational Site	Parkville	Victoria
Australia	Ascendis Pharma Investigational Site	Perth	Western Australia
Australia	Ascendis Pharma Investigational Site	Saint Leonards	New South Wales
Australia	Ascendis Pharma Investigational Site	Sydney	New South Wales
Canada	Ascendis Pharma Investigational Site	Halifax	Nova Scotia
France	Ascendis Pharma Investigational Site	Lyon
France	Ascendis Pharma Investigational Site	Marseille
France	Ascendis Pharma Investigational Site	Nantes
Georgia	Ascendis Pharma Investigational Site	Tbilisi
Georgia	Ascendis Pharma Investigational Site	Tbilisi
Germany	Ascendis Pharma Investigational Site	München
Greece	Ascendis Pharma Investigational Site	Athens
Greece	Ascendis Pharma Investigational Site	Thessaloníki
Israel	Ascendis Pharma Investigational Site	Haifa
Israel	Ascendis Pharma Investigational Site	Petah tikva
Israel	Ascendis Pharma Investigational Site	Tel Aviv
Italy	Ascendis Pharma Investigational Site	Genova
Italy	Ascendis Pharma Investigational Site	Rome
Italy	Ascendis Pharma Investigational Site	Rome
Japan	Ascendis Pharma Investigational Site	Chiba
Japan	Ascendis Pharma Investigational Site	Fukuoka
Japan	Ascendis Pharma Investigational Site	Ishikawa
Japan	Ascendis Pharma Investigational Site	Kagoshima
Japan	Ascendis Pharma Investigational Site	Kawasaki
Japan	Ascendis Pharma Investigational Site	Kawasaki
Japan	Ascendis Pharma Investigational Site	Kawasaki	Kanagawa
Japan	Ascendis Pharma Investigational Site	Kitakyushu
Japan	Ascendis Pharma Investigational Site	Kobe	Hyogo
Japan	Ascendis Pharma Investigational Site	Matsumoto
Japan	Ascendis Pharma Investigational Site	Miyakojima
Japan	Ascendis Pharma Investigational Site	Nagakute
Japan	Ascendis Pharma Investigational Site	Nara
Japan	Ascendis Pharma Investigational Site	Okayama
Japan	Ascendis Pharma Investigational Site	Osaka
Japan	Ascendis Pharma Investigational Site	Shizuoka
Japan	Ascendis Pharma Investigational Site	Suita
Japan	Ascendis Pharma Investigational Site	Yamagata
Japan	Ascendis Pharma Investigational Site	Yokohama
Japan	Ascendis Pharma Investigational Site	Yokohama	Kanagawa
Korea, Republic of	Ascendis Pharma Investigational Site	Soeul
Malaysia	Ascendis Pharma Investigational Site	George Town
Malaysia	Ascendis Pharma Investigational Site	Melaka
Malaysia	Ascendis Pharma Investigational Site	Putrajaya
Poland	Ascendis Pharma Investigational Site	Kraków
Poland	Ascendis Pharma Investigational Site	Lódz
Poland	Ascendis Pharma Investigational Site	Warszawa
Poland	Ascendis Pharma Investigational Site	Wroclaw
Romania	Ascendis Pharma Investigational Site	Bucharest
Romania	Ascendis Pharma Investigational Site	Iasi
Serbia	Ascendis Pharma Investigational Site	Belgrade
Slovakia	Ascendis Pharma Investigational Site	Bratislava
Slovakia	Ascendis Pharma Investigational Site	Lubochna
Spain	Ascendis Pharma Investigational Site	Alicante
Spain	Ascendis Pharma Investigational Site	Barcelona
Spain	Ascendis Pharma Investigational Site	Barcelona
Spain	Ascendis Pharma Investigational Site	Sevilla
Turkey	Ascendis Pharma Investigational Site	Ankara
Turkey	Ascendis Pharma Investigational Site	Antalya
Turkey	Ascendis Pharma Investigational Site	Aydin
Turkey	Ascendis Pharma Investigational Site	Izmit
Turkey	Ascendis Pharma Investigational Site	Kayseri
Ukraine	Ascendis Pharma Investigational Site	Ivano-Frankivs'k
Ukraine	Ascendis Pharma Investigational Site	Kyiv
Ukraine	Ascendis Pharma Investigational Site	Kyiv
Ukraine	Ascendis Pharma Investigational Site	Kyiv
Ukraine	Ascendis Pharma Investigational Site	Vinnytsia
United Kingdom	Ascendis Pharma Investigational Site	Leeds
United States	Ascendis Pharma Investigational Site	Boston	Massachusetts
United States	Ascendis Pharma Investigational Site	Chicago	Illinois
United States	Ascendis Pharma Investigational Site	Dallas	Texas
United States	Ascendis Pharma Investigational Site	Dearborn	Michigan
United States	Ascendis Pharma Investigational Site	Indianapolis	Indiana
United States	Ascendis Pharma Investigational Site	Las Vegas	Nevada
United States	Ascendis Pharma Investigational Site	New York	New York
United States	Ascendis Pharma Investigational Site	Phoenix	Arizona
United States	Ascendis Pharma Investigational Site	Portland	Oregon
United States	Ascendis Pharma Investigational Site	Reno	Nevada
United States	Ascendis Pharma Investigational Site	Rochester	Minnesota
United States	Ascendis Pharma Investigational Site	Saint Louis	Missouri
United States	Ascendis Pharma Investigational Site	San Antonio	Texas
United States	Ascendis Pharma Investigational Site	Seattle	Washington
United States	Ascendis Pharma Investigational Site	Torrance	California

Sponsors (1)

Lead Sponsor	Collaborator
Ascendis Pharma Endocrinology Division A/S

Countries where clinical trial is conducted

United States,  Armenia,  Australia,  Canada,  France,  Georgia,  Germany,  Greece,  Israel,  Italy,  Japan,  Korea, Republic of,  Malaysia,  Poland,  Romania,  Serbia,  Slovakia,  Spain,  Turkey,  Ukraine,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Evaluate serum hGH, lonapegsomatropin, and mPEG levels	To evaluate the pharmacokinetics (PK) of once-weekly lonapegsomatropin in adults with growth hormone deficiency	Week 52