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

Primary Purpose

Adult Growth Hormone Deficiency, Endocrine System Diseases, Hormone Deficiency

Status
Enrolling by invitation
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Lonapegsomatropin
Sponsored by
Ascendis Pharma Endocrinology Division A/S
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Adult Growth Hormone Deficiency focused on measuring 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

Eligibility Criteria

23 Years - 81 Years (Adult, Older Adult)All SexesDoes not accept 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 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

Sites / Locations

  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
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  • Ascendis Pharma Investigational Site
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  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
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  • Ascendis Pharma Investigational Site
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  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
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  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
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Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Lonapegsomatropin

Arm Description

Lonapegsomatropin administered once-weekly by subcutaneous injection

Outcomes

Primary Outcome Measures

Incidence of Treatment-Emergent Adverse Events
To evaluate the safety and tolerability of once-weekly lonapegsomatropin in adults with growth hormone deficiency

Secondary Outcome Measures

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
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
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
Evaluate serum IGF-1 and IGF-1 SDS
To evaluate the pharmacodynamics (PD) of once-weekly lonapegsomatropin in adults with growth hormone deficiency
Evaluate serum hGH, lonapegsomatropin, and mPEG levels
To evaluate the pharmacokinetics (PK) of once-weekly lonapegsomatropin in adults with growth hormone deficiency

Full Information

First Posted
September 24, 2021
Last Updated
October 18, 2023
Sponsor
Ascendis Pharma Endocrinology Division A/S
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1. Study Identification

Unique Protocol Identification Number
NCT05171855
Brief Title
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

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
December 16, 2021 (Actual)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
January 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ascendis Pharma Endocrinology Division A/S

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
Open label treatment with weekly Lonapegsomatropin
Masking
None (Open Label)
Allocation
N/A
Enrollment
240 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Lonapegsomatropin
Arm Type
Experimental
Arm Description
Lonapegsomatropin administered once-weekly by subcutaneous injection
Intervention Type
Drug
Intervention Name(s)
Lonapegsomatropin
Other Intervention Name(s)
ACP-011
Intervention Description
Study participants are individually dosed with subcutaneous injection of Lonapegsomatropin once-weekly for 52 weeks
Primary Outcome Measure Information:
Title
Incidence of Treatment-Emergent Adverse Events
Description
To evaluate the safety and tolerability of once-weekly lonapegsomatropin in adults with growth hormone deficiency
Time Frame
Throughout the 52 week treatment period
Secondary Outcome Measure Information:
Title
Change from Baseline in Trunk Percent Fat
Description
Change from baseline in trunk percent fat (as assessed by dual-energy x ray absorptiometry [DXA]) at Week 52
Time Frame
Week 52
Title
Change from Baseline in Trunk Fat Mass
Description
Change from baseline in trunk fat mass (as assessed by dual-energy x ray absorptiometry [DXA]) at Week 52
Time Frame
Week 52
Title
Change from Baseline in Total Body Lean Mass
Description
Change from baseline in total body lean mass (as assessed by dual-energy x ray absorptiometry [DXA]) at Week 52
Time Frame
Week 52
Title
Evaluate serum IGF-1 and IGF-1 SDS
Description
To evaluate the pharmacodynamics (PD) of once-weekly lonapegsomatropin in adults with growth hormone deficiency
Time Frame
Week 52
Title
Evaluate serum hGH, lonapegsomatropin, and mPEG levels
Description
To evaluate the pharmacokinetics (PK) of once-weekly lonapegsomatropin in adults with growth hormone deficiency
Time Frame
Week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
23 Years
Maximum Age & Unit of Time
81 Years
Accepts Healthy Volunteers
No
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: 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
Facility Information:
Facility Name
Ascendis Pharma Investigational Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85048
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Torrance
State/Province
California
ZIP/Postal Code
90509
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Dearborn
State/Province
Michigan
ZIP/Postal Code
48126
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89148
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Reno
State/Province
Nevada
ZIP/Postal Code
89511
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
New York
State/Province
New York
ZIP/Postal Code
10017
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78232
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Seattle
State/Province
Washington
ZIP/Postal Code
98108
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Yerevan
ZIP/Postal Code
0075
Country
Armenia
Facility Name
Ascendis Pharma Investigational Site
City
Saint Leonards
State/Province
New South Wales
ZIP/Postal Code
2065
Country
Australia
Facility Name
Ascendis Pharma Investigational Site
City
Sydney
State/Province
New South Wales
ZIP/Postal Code
2109
Country
Australia
Facility Name
Ascendis Pharma Investigational Site
City
Box Hill
State/Province
Victoria
ZIP/Postal Code
3128
Country
Australia
Facility Name
Ascendis Pharma Investigational Site
City
Fitzroy
State/Province
Victoria
ZIP/Postal Code
3065
Country
Australia
Facility Name
Ascendis Pharma Investigational Site
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Name
Ascendis Pharma Investigational Site
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Facility Name
Ascendis Pharma Investigational Site
City
Halifax
State/Province
Nova Scotia
ZIP/Postal Code
B3H 1V7
Country
Canada
Facility Name
Ascendis Pharma Investigational Site
City
Nantes
ZIP/Postal Code
44093
Country
France
Facility Name
Ascendis Pharma Investigational Site
City
Tbilisi
ZIP/Postal Code
0144
Country
Georgia
Facility Name
Ascendis Pharma Investigational Site
City
Tbilisi
ZIP/Postal Code
0159
Country
Georgia
Facility Name
Ascendis Pharma Investigational Site
City
Athens
ZIP/Postal Code
11527
Country
Greece
Facility Name
Ascendis Pharma Investigational Site
City
Thessaloníki
ZIP/Postal Code
54636
Country
Greece
Facility Name
Ascendis Pharma Investigational Site
City
Genova
ZIP/Postal Code
16132
Country
Italy
Facility Name
Ascendis Pharma Investigational Site
City
Kobe
State/Province
Hyogo
ZIP/Postal Code
650-0047
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Kawasaki
State/Province
Kanagawa
ZIP/Postal Code
216-8511
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Yokohama
State/Province
Kanagawa
ZIP/Postal Code
222-0036
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Chiba
ZIP/Postal Code
260-8677
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Fukuoka
ZIP/Postal Code
812-8582
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Ishikawa
ZIP/Postal Code
920-0293
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Kagoshima
ZIP/Postal Code
890-8520
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Kawasaki
ZIP/Postal Code
210-0024
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Kawasaki
ZIP/Postal Code
211-8533
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Kitakyushu
ZIP/Postal Code
807-8555
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Matsumoto
ZIP/Postal Code
390-8510
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Miyakojima
ZIP/Postal Code
534-0021
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Nagakute
ZIP/Postal Code
480-1195
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Nara
ZIP/Postal Code
634-8522
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Okayama
ZIP/Postal Code
700-8558
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Osaka
ZIP/Postal Code
550-0006
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Shizuoka
ZIP/Postal Code
422-8527
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Suita
ZIP/Postal Code
565-0871
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Yamagata
ZIP/Postal Code
990-9585
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Yokohama
ZIP/Postal Code
236-0004
Country
Japan
Facility Name
Ascendis Pharma Investigational Site
City
Kraków
ZIP/Postal Code
31-501
Country
Poland
Facility Name
Ascendis Pharma Investigational Site
City
Warszawa
ZIP/Postal Code
03-242
Country
Poland
Facility Name
Ascendis Pharma Investigational Site
City
Wrocław
ZIP/Postal Code
50-367
Country
Poland
Facility Name
Ascendis Pharma Investigational Site
City
Łódź
ZIP/Postal Code
93-338
Country
Poland
Facility Name
Ascendis Pharma Investigational Site
City
Bucharest
ZIP/Postal Code
11868
Country
Romania
Facility Name
Ascendis Pharma Investigational Site
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
Facility Name
Ascendis Pharma Investigational Site
City
Bratislava
ZIP/Postal Code
82606
Country
Slovakia
Facility Name
Ascendis Pharma Investigational Site
City
Ľubochňa
ZIP/Postal Code
3491
Country
Slovakia
Facility Name
Ascendis Pharma Investigational Site
City
Alicante
ZIP/Postal Code
3010
Country
Spain
Facility Name
Ascendis Pharma Investigational Site
City
Barcelona
ZIP/Postal Code
8035
Country
Spain
Facility Name
Ascendis Pharma Investigational Site
City
Barcelona
ZIP/Postal Code
8041
Country
Spain
Facility Name
Ascendis Pharma Investigational Site
City
Aydın
ZIP/Postal Code
09010
Country
Turkey
Facility Name
Ascendis Pharma Investigational Site
City
İzmit
ZIP/Postal Code
41001
Country
Turkey
Facility Name
Ascendis Pharma Investigational Site
City
Ivano-Frankivs'k
ZIP/Postal Code
76008
Country
Ukraine
Facility Name
Ascendis Pharma Investigational Site
City
Kyiv
ZIP/Postal Code
03115
Country
Ukraine
Facility Name
Ascendis Pharma Investigational Site
City
Kyiv
ZIP/Postal Code
04001
Country
Ukraine
Facility Name
Ascendis Pharma Investigational Site
City
Kyiv
ZIP/Postal Code
04114
Country
Ukraine
Facility Name
Ascendis Pharma Investigational Site
City
Vinnytsia
ZIP/Postal Code
21010
Country
Ukraine

12. IPD Sharing Statement

Learn more about this trial

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

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