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A Long-Term Trial Investigating Safety and Efficacy of TransCon hGH in Children With Growth Hormone Deficiency Who Have Completed a Prior TransCon hGH Clinical Trial (enliGHten)

Primary Purpose

Growth Hormone Deficiency, Pediatric, Endocrine System Diseases, Hormone Deficiency

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
TransCon hGH
Sponsored by
Ascendis Pharma A/S
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Growth Hormone Deficiency, Pediatric focused on measuring Human Growth Hormone, hGH, GHD, rhGH, Pediatric Growth Hormone Deficiency, Long Acting Growth Hormone, Somatropin, Prodrug, Growth Failure, Growth Hormone Replacement Therapy, Sustained Release Growth Hormone, Growth Hormone Deficiency, TransCon GH

Eligibility Criteria

1 Year - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Children who have completed a prior phase 3 TransCon hGH trial
  2. Children who have not permanently discontinued study drug in the prior trial
  3. Written, signed, informed consent of the parent or legal guardian of the subject and written assent of the subject as required by the IRB/HREC/IEC

Exclusion Criteria:

  1. Poorly-controlled diabetes mellitus (HbA1c ≥ 8.0%) or diabetic complications
  2. Evidence of closed epiphyses, defined as bone age > 14.0 years for females or > 16.0 years for males
  3. Major medical conditions unless approved by Medical Expert
  4. Known hypersensitivity to the components of the trial medication
  5. Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver)
  6. Pregnancy
  7. Any other reason that in the opinion of the investigator would prevent the subject from completing participation or following the trial schedule

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
  • 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
  • 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
  • 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

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

TransCon hGH

Arm Description

Once weekly subcutaneous injection of TransCon hGH

Outcomes

Primary Outcome Measures

Incidence of Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability]
Long-term safety and tolerability of weekly TransCon hGH treatment

Secondary Outcome Measures

Annualized height velocity (HV) with long-term dosing of weekly TransCon hGH treatment
Proportion of subjects with IGF-1 standard deviation score (SDS) in the normal range of 0.0 to +2.0 with long-term dosing of weekly TransCon hGH treatment
Change in height standard deviation scores with long-term dosing of weekly TransCon hGH treatment
Incidence of antibodies against TransCon hGH with long-term dosing of weekly TransCon hGH treatment

Full Information

First Posted
November 14, 2017
Last Updated
October 10, 2023
Sponsor
Ascendis Pharma A/S
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1. Study Identification

Unique Protocol Identification Number
NCT03344458
Brief Title
A Long-Term Trial Investigating Safety and Efficacy of TransCon hGH in Children With Growth Hormone Deficiency Who Have Completed a Prior TransCon hGH Clinical Trial
Acronym
enliGHten
Official Title
enliGHten: A Multicenter, Phase 3, Long-Term, Open-Label Trial Investigating Safety and Efficacy of TransCon hGH Administered Once-Weekly in Children With Growth Hormone Deficiency (GHD) Who Have Completed a Prior TransCon hGH Clinical Trial
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Completed
Study Start Date
December 19, 2017 (Actual)
Primary Completion Date
February 21, 2023 (Actual)
Study Completion Date
February 21, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ascendis Pharma 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
Yes

5. Study Description

Brief Summary
A multicenter, phase 3, long-term extension trial of TransCon hGH administered once-weekly in children with growth hormone deficiency (GHD) who previously participated in a phase 3 TransCon hGH trial. Approximately 300 children (males and females) with GHD will be included. All study participants will receive TransCon hGH. This is a global trial that will be conducted in, but not limited to, the United States, Poland, Bulgaria, Ukraine, Armenia, Russia and Australia.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Growth Hormone Deficiency, Pediatric, Endocrine System Diseases, Hormone Deficiency, Pituitary Diseases
Keywords
Human Growth Hormone, hGH, GHD, rhGH, Pediatric Growth Hormone Deficiency, Long Acting Growth Hormone, Somatropin, Prodrug, Growth Failure, Growth Hormone Replacement Therapy, Sustained Release Growth Hormone, Growth Hormone Deficiency, TransCon GH

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
All study participants will receive TransCon hGH
Masking
None (Open Label)
Allocation
N/A
Enrollment
298 (Actual)

8. Arms, Groups, and Interventions

Arm Title
TransCon hGH
Arm Type
Experimental
Arm Description
Once weekly subcutaneous injection of TransCon hGH
Intervention Type
Drug
Intervention Name(s)
TransCon hGH
Intervention Description
Once weekly subcutaneous injection of TransCon hGH
Primary Outcome Measure Information:
Title
Incidence of Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability]
Description
Long-term safety and tolerability of weekly TransCon hGH treatment
Time Frame
Up to 4 years
Secondary Outcome Measure Information:
Title
Annualized height velocity (HV) with long-term dosing of weekly TransCon hGH treatment
Time Frame
Up to 4 years
Title
Proportion of subjects with IGF-1 standard deviation score (SDS) in the normal range of 0.0 to +2.0 with long-term dosing of weekly TransCon hGH treatment
Time Frame
Up to 4 years
Title
Change in height standard deviation scores with long-term dosing of weekly TransCon hGH treatment
Time Frame
Up to 4 years
Title
Incidence of antibodies against TransCon hGH with long-term dosing of weekly TransCon hGH treatment
Time Frame
Up to 4 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Children who have completed a prior phase 3 TransCon hGH trial Children who have not permanently discontinued study drug in the prior trial Written, signed, informed consent of the parent or legal guardian of the subject and written assent of the subject as required by the IRB/HREC/IEC Exclusion Criteria: Poorly-controlled diabetes mellitus (HbA1c ≥ 8.0%) or diabetic complications Evidence of closed epiphyses, defined as bone age > 14.0 years for females or > 16.0 years for males Major medical conditions unless approved by Medical Expert Known hypersensitivity to the components of the trial medication Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver) Pregnancy Any other reason that in the opinion of the investigator would prevent the subject from completing participation or following the trial schedule
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michael Beckert, MD
Organizational Affiliation
Ascendis Pharma A/S
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Aimee D Shu, MD
Organizational Affiliation
Ascendis Pharma, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Ascendis Pharma Investigational Site
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Sacramento
State/Province
California
ZIP/Postal Code
95821
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Centennial
State/Province
Colorado
ZIP/Postal Code
80112
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32207
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Tallahassee
State/Province
Florida
ZIP/Postal Code
32308
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55102
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Lebanon
State/Province
New Hampshire
ZIP/Postal Code
03756
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Mineola
State/Province
New York
ZIP/Postal Code
11501
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Portland
State/Province
Oregon
ZIP/Postal Code
97227
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
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23507
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98405
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Yerevan
ZIP/Postal Code
0075
Country
Armenia
Facility Name
Ascendis Pharma Investigational Site
City
Clayton
State/Province
Victoria
ZIP/Postal Code
3168
Country
Australia
Facility Name
Ascendis Pharma Investigational Site
City
Minsk
ZIP/Postal Code
220020
Country
Belarus
Facility Name
Ascendis Pharma Investigational Site
City
Varna
ZIP/Postal Code
9010
Country
Bulgaria
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
Auckland
ZIP/Postal Code
1010
Country
New Zealand
Facility Name
Ascendis Pharma Investigational Site
City
Gdańsk
ZIP/Postal Code
80-952
Country
Poland
Facility Name
Ascendis Pharma Investigational Site
City
Warsaw
ZIP/Postal Code
02-691
Country
Poland
Facility Name
Ascendis Pharma Investigational Site
City
Izhevsk
ZIP/Postal Code
426009
Country
Russian Federation
Facility Name
Ascendis Pharma Investigational Site
City
Krasnoyarsk
ZIP/Postal Code
660022
Country
Russian Federation
Facility Name
Ascendis Pharma Investigational Site
City
Moscow
ZIP/Postal Code
117036
Country
Russian Federation
Facility Name
Ascendis Pharma Investigational Site
City
Saint Petersburg
ZIP/Postal Code
194100
Country
Russian Federation
Facility Name
Ascendis Pharma Investigational Site
City
Saratov
ZIP/Postal Code
410054
Country
Russian Federation
Facility Name
Ascendis Pharma Investigational Site
City
Ufa
ZIP/Postal Code
450008
Country
Russian Federation
Facility Name
Ascendis Pharma Investigational Site
City
Vologda
ZIP/Postal Code
160022
Country
Russian Federation
Facility Name
Ascendis Pharma Investigational Site
City
Voronezh
ZIP/Postal Code
394024
Country
Russian Federation
Facility Name
Ascendis Pharma Investigational Site
City
Kharkiv
ZIP/Postal Code
61093
Country
Ukraine
Facility Name
Ascendis Pharma Investigational Site
City
Kyiv
ZIP/Postal Code
01021
Country
Ukraine
Facility Name
Ascendis Pharma Investigational Site
City
Odesa
ZIP/Postal Code
65031
Country
Ukraine

12. IPD Sharing Statement

Citations:
PubMed Identifier
35428884
Citation
Maniatis AK, Casella SJ, Nadgir UM, Hofman PL, Saenger P, Chertock ED, Aghajanova EM, Korpal-Szczyrska M, Vlachopapadopoulou E, Malievskiy O, Chaychenko T, Cappa M, Song W, Mao M, Mygind PH, Smith AR, Chessler SD, Komirenko AS, Beckert M, Shu AD, Thornton PS. Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results. J Clin Endocrinol Metab. 2022 Jun 16;107(7):e2680-e2689. doi: 10.1210/clinem/dgac217.
Results Reference
derived

Learn more about this trial

A Long-Term Trial Investigating Safety and Efficacy of TransCon hGH in Children With Growth Hormone Deficiency Who Have Completed a Prior TransCon hGH Clinical Trial

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