Clinical trial NCT05171855: A Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency
Official title: A Multicenter, Open-Label, Extension Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Adult Growth Hormone Deficiency
Endocrine System Diseases
Hormone Deficiency
Keywords: 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
Lead sponsor: Ascendis Pharma A/S
Sponsor class: INDUSTRY
First posted: 2021-12-29
Enrollment: 233
Sex: ALL
Minimum age (years): 23
Maximum age (years): 81
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open label treatment with weekly Lonapegsomatropin
Primary purpose: TREATMENT

Brief 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).


Eligibility criteria: 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

Primary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events
timeFrame: Throughout the 52 week treatment period
Secondary outcomes:
measure: Change from Baseline in Trunk Percent Fat
timeFrame: Week 52
measure: Change from Baseline in Trunk Fat Mass
timeFrame: Week 52
measure: Change from Baseline in Total Body Lean Mass
timeFrame: Week 52
measure: Evaluate serum IGF-1 and IGF-1 SDS
timeFrame: Week 52
measure: Evaluate serum hGH, lonapegsomatropin, and mPEG levels
timeFrame: Week 52
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Ascendis Pharma Investigational Site
state: Arizona
city: Torrance
country: United States
facility: Ascendis Pharma Investigational Site
state: California
city: Chicago
country: United States
facility: Ascendis Pharma Investigational Site
state: Illinois
city: Indianapolis
country: United States
facility: Ascendis Pharma Investigational Site
state: Indiana
city: Boston
country: United States
facility: Ascendis Pharma Investigational Site
state: Massachusetts
city: Dearborn
country: United States
facility: Ascendis Pharma Investigational Site
state: Michigan
city: Rochester
country: United States
facility: Ascendis Pharma Investigational Site
state: Minnesota
city: Saint Louis
country: United States
facility: Ascendis Pharma Investigational Site
state: Missouri
city: Las Vegas
country: United States
facility: Ascendis Pharma Investigational Site
state: Nevada
city: Reno
country: United States
facility: Ascendis Pharma Investigational Site
state: Nevada
city: New York
country: United States
facility: Ascendis Pharma Investigational Site
state: New York
city: Portland
country: United States
facility: Ascendis Pharma Investigational Site
state: Oregon
city: Dallas
country: United States
facility: Ascendis Pharma Investigational Site
state: Texas
city: San Antonio
country: United States
facility: Ascendis Pharma Investigational Site
state: Texas
city: Seattle
country: United States
facility: Ascendis Pharma Investigational Site
state: Washington
city: Yerevan
country: Armenia
facility: Ascendis Pharma Investigational Site
city: Saint Leonards
country: Australia
facility: Ascendis Pharma Investigational Site
state: New South Wales
city: Sydney
country: Australia
facility: Ascendis Pharma Investigational Site
state: New South Wales
city: Box Hill
country: Australia
facility: Ascendis Pharma Investigational Site
state: Victoria
city: Fitzroy
country: Australia
facility: Ascendis Pharma Investigational Site
state: Victoria
city: Parkville
country: Australia
facility: Ascendis Pharma Investigational Site
state: Victoria
city: Perth
country: Australia
facility: Ascendis Pharma Investigational Site
state: Western Australia
city: Halifax
country: Canada
facility: Ascendis Pharma Investigational Site
state: Nova Scotia
city: Lyon
country: France
facility: Ascendis Pharma Investigational Site
city: Marseille
country: France
facility: Ascendis Pharma Investigational Site
city: Nantes
country: France
facility: Ascendis Pharma Investigational Site
city: Tbilisi
country: Georgia
facility: Ascendis Pharma Investigational Site
city: Tbilisi
country: Georgia
facility: Ascendis Pharma Investigational Site
city: München
country: Germany
facility: Ascendis Pharma Investigational Site
city: Athens
country: Greece
facility: Ascendis Pharma Investigational Site
city: Thessaloníki
country: Greece
facility: Ascendis Pharma Investigational Site
city: Haifa
country: Israel
facility: Ascendis Pharma Investigational Site
city: Petah tikva
country: Israel
facility: Ascendis Pharma Investigational Site
city: Tel Aviv
country: Israel
facility: Ascendis Pharma Investigational Site
city: Genova
country: Italy
facility: Ascendis Pharma Investigational Site
city: Rome
country: Italy
facility: Ascendis Pharma Investigational Site
city: Rome
country: Italy
facility: Ascendis Pharma Investigational Site
city: Kobe
country: Japan
facility: Ascendis Pharma Investigational Site
state: Hyogo
city: Kawasaki
country: Japan
facility: Ascendis Pharma Investigational Site
state: Kanagawa
city: Yokohama
country: Japan
facility: Ascendis Pharma Investigational Site
state: Kanagawa
city: Chiba
country: Japan
facility: Ascendis Pharma Investigational Site
city: Fukuoka
country: Japan
facility: Ascendis Pharma Investigational Site
city: Ishikawa
country: Japan
facility: Ascendis Pharma Investigational Site
city: Kagoshima
country: Japan
facility: Ascendis Pharma Investigational Site
city: Kawasaki
country: Japan
facility: Ascendis Pharma Investigational Site
city: Kawasaki
country: Japan
facility: Ascendis Pharma Investigational Site
city: Kitakyushu
country: Japan
facility: Ascendis Pharma Investigational Site
city: Matsumoto
country: Japan
facility: Ascendis Pharma Investigational Site
city: Miyakojima
country: Japan
facility: Ascendis Pharma Investigational Site
city: Nagakute
country: Japan
facility: Ascendis Pharma Investigational Site
city: Nara
country: Japan
facility: Ascendis Pharma Investigational Site
city: Okayama
country: Japan
facility: Ascendis Pharma Investigational Site
city: Osaka
country: Japan
facility: Ascendis Pharma Investigational Site
city: Shizuoka
country: Japan
facility: Ascendis Pharma Investigational Site
city: Suita
country: Japan
facility: Ascendis Pharma Investigational Site
city: Yamagata
country: Japan
facility: Ascendis Pharma Investigational Site
city: Yokohama
country: Japan
facility: Ascendis Pharma Investigational Site
city: Soeul
country: Korea, Republic of
facility: Ascendis Pharma Investigational Site
city: George Town
country: Malaysia
facility: Ascendis Pharma Investigational Site
city: Melaka
country: Malaysia
facility: Ascendis Pharma Investigational Site
city: Putrajaya
country: Malaysia
facility: Ascendis Pharma Investigational Site
city: Kraków
country: Poland
facility: Ascendis Pharma Investigational Site
city: Warszawa
country: Poland
facility: Ascendis Pharma Investigational Site
city: Wrocław
country: Poland
facility: Ascendis Pharma Investigational Site
city: Łódź
country: Poland
facility: Ascendis Pharma Investigational Site
city: Bucharest
country: Romania
facility: Ascendis Pharma Investigational Site
city: Iaşi
country: Romania
facility: Ascendis Pharma Investigational Site
city: Belgrade
country: Serbia
facility: Ascendis Pharma Investigational Site
city: Bratislava
country: Slovakia
facility: Ascendis Pharma Investigational Site
city: Ľubochňa
country: Slovakia
facility: Ascendis Pharma Investigational Site
city: Alicante
country: Spain
facility: Ascendis Pharma Investigational Site
city: Barcelona
country: Spain
facility: Ascendis Pharma Investigational Site
city: Barcelona
country: Spain
facility: Ascendis Pharma Investigational Site
city: Sevilla
country: Spain
facility: Ascendis Pharma Investigational Site
city: Ankara
country: Turkey
facility: Ascendis Pharma Investigational Site
city: Antalya
country: Turkey
facility: Ascendis Pharma Investigational Site
city: Aydın
country: Turkey
facility: Ascendis Pharma Investigational Site
city: İzmit
country: Turkey
facility: Ascendis Pharma Investigational Site
city: Kayseri
country: Turkey
facility: Ascendis Pharma Investigational Site
city: Ivano-Frankivs'k
country: Ukraine
facility: Ascendis Pharma Investigational Site
city: Kyiv
country: Ukraine
facility: Ascendis Pharma Investigational Site
city: Kyiv
country: Ukraine
facility: Ascendis Pharma Investigational Site
city: Kyiv
country: Ukraine
facility: Ascendis Pharma Investigational Site
city: Vinnytsia
country: Ukraine
facility: Ascendis Pharma Investigational Site
city: Leeds
country: United Kingdom
facility: Ascendis Pharma Investigational Site
Study officials:
References: