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A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone Deficiency

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 Endocrinology Division 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

6 Months - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Investigator-determined GHD diagnosis prior to the historical initiation of daily hGH therapy.

  2. 6 months to 17 years old, inclusive, at Visit 1

    1. If 3 to 17 years old, are taking daily hGH at a dose of ≥ 0.20 mg hGH/kg/week for at least 13 weeks but no more than 130 weeks prior to Visit 1
    2. If ≥ 6 months but < 3 years old, are either hGH treatment-naïve or are taking daily hGH at a dose of ≥ 0.20mg hGH/kg/week for no more than 130 weeks prior to Visit 1
  3. Tanner stage < 5 at Visit 1
  4. Open epiphyses (bone age ≤14.0 years for females or ≤16.0 years for males)
  5. 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. Weight of < 5.5 kg or > 80 kg at Visit 1
  2. Females of child-bearing potential
  3. History of malignant disease
  4. Any clinically significant abnormality likely to affect growth or the ability to evaluate growth (eg, chronic diseases or conditions such as renal insufficiency, spinal cord irradiation, hypothyroidism, active celiac disease, malnutrition or psychosocial dwarfism)
  5. Poorly-controlled diabetes mellitus (HbA1c >8.0%) or diabetic complications
  6. Known neutralizing antibodies against hGH
  7. Major medical conditions, unless approved by Medical Monitor
  8. Pregnancy
  9. Presence of contraindications to hGH treatment
  10. Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver)
  11. Participation in any other trial of an investigational agent within 30 days prior to Visit 1
  12. Prior exposure to investigational hGH

Sites / Locations

  • University of Alabama
  • Neufeld Medical Group Inc.
  • Center of Excellence in Diabetes and Endocrinology
  • Rocky Mountain Pediatric Endocrinology
  • Nemours Children's Health System
  • Orlando Health Inc.
  • Tallahassee Memorial Hospital
  • University of Iowa
  • Children's Minnesota
  • University of Mississippi Medical Center
  • Dartmouth Hitchcock Medical Center
  • NYU Winthrop Hospital
  • Icahn School of Medicine at Mount Sinai
  • Cleveland Clinic Foundation
  • University of Oklahoma Health Sciences Center
  • Children's Diabetes and Endocrine Center
  • Oregon Health & Science University
  • Children's Medical Center
  • Cook Children's Medical Center
  • University of Virginia Children's Hospital
  • Children's Hospital of The King's Daughters
  • Monash Children's Hospital
  • Stollery Children's Hospital
  • The Liggins Institute, The University of Auckland

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

Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment

Secondary Outcome Measures

Annualized Height Velocity (AHV) at 26 Weeks of Weekly Lonapegsomatropin Treatment
Annualized height velocity (AHV) at 26 weeks of weekly lonapegsomatropin (TransCon hGH) treatment. The AHV at each visit was modeled using ANCOVA adjusting for baseline age, peak GH levels (log transformed) at diagnosis, delta average-parental height SDS, prior GH dose level (log transformed), and prior GH dose duration (log transformed) as covariates and gender as a factor. Subjects who did not take prior GH treatment were not included in the model.
Number of Subjects With IGF-1 Standard Deviation Score (SDS) in the Range of 0.0 to +2.0 at 26 Weeks of Weekly Lonapegsomatropin Treatment
IGF-1 Standard Deviation Score (SDS) is the number of standard deviations above or below the mean Insulin-like Growth Factor 1 (IGF-1) level for age and sex. IGF-1 SDS was derived using the LMS method as ((IGF-1/M)^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from Bidlingmaier et al. (2014). A Standard Deviation Score of 0 represents the population mean.
Change in Height Standard Deviation Scores (SDS) at 26 Weeks of Weekly Lonapegsomatropin Treatment
Height Standard Deviation Score (SDS) is the number of standard deviations above or below the mean height for age and sex. Height SDS was derived using the LMS method as ((Height/M)^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from 2000 CDC growth charts for the United States. A Standard Deviation Score of 0 represents the population mean. A higher change from baseline in Height SDS indicates a better outcome. The height SDS change from baseline at each visit was modeled using ANCOVA adjusting for baseline age, peak GH levels (log transformed) at diagnosis, delta average-parental height SDS, prior GH dose level (log transformed), and prior GH dose duration (log transformed) as covariates and gender as a factor. Subjects who did not take prior GH treatment were not included in the model.
Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation
Number of participants with treatment emergent anti-hGH antibodies over 26 weeks of weekly lonapegsomatropin (TransCon hGH) treatment. All samples were negative for anti-hGH neutralizing antibodies.

Full Information

First Posted
October 4, 2017
Last Updated
December 7, 2021
Sponsor
Ascendis Pharma Endocrinology Division A/S
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1. Study Identification

Unique Protocol Identification Number
NCT03305016
Brief Title
A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone Deficiency
Official Title
fliGHt: A Multicenter, Phase 3, Open-Label, 26-Week Trial Investigating the Safety, Tolerability and Efficacy of TransCon hGH Administered Once Weekly in Children With GHD
Study Type
Interventional

2. Study Status

Record Verification Date
December 2021
Overall Recruitment Status
Completed
Study Start Date
November 13, 2017 (Actual)
Primary Completion Date
March 19, 2019 (Actual)
Study Completion Date
March 19, 2019 (Actual)

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
Yes

5. Study Description

Brief Summary
A 26 week trial of TransCon hGH, a long-acting growth hormone product, administered once-a-week. Approximately 150 children (males and females) with growth hormone deficiency (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, Canada, Australia, and New Zealand.

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
146 (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 at a starting dose of 0.24 mg/kg/week
Primary Outcome Measure Information:
Title
Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
Description
Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment
Time Frame
26 weeks
Secondary Outcome Measure Information:
Title
Annualized Height Velocity (AHV) at 26 Weeks of Weekly Lonapegsomatropin Treatment
Description
Annualized height velocity (AHV) at 26 weeks of weekly lonapegsomatropin (TransCon hGH) treatment. The AHV at each visit was modeled using ANCOVA adjusting for baseline age, peak GH levels (log transformed) at diagnosis, delta average-parental height SDS, prior GH dose level (log transformed), and prior GH dose duration (log transformed) as covariates and gender as a factor. Subjects who did not take prior GH treatment were not included in the model.
Time Frame
26 weeks
Title
Number of Subjects With IGF-1 Standard Deviation Score (SDS) in the Range of 0.0 to +2.0 at 26 Weeks of Weekly Lonapegsomatropin Treatment
Description
IGF-1 Standard Deviation Score (SDS) is the number of standard deviations above or below the mean Insulin-like Growth Factor 1 (IGF-1) level for age and sex. IGF-1 SDS was derived using the LMS method as ((IGF-1/M)^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from Bidlingmaier et al. (2014). A Standard Deviation Score of 0 represents the population mean.
Time Frame
26 weeks
Title
Change in Height Standard Deviation Scores (SDS) at 26 Weeks of Weekly Lonapegsomatropin Treatment
Description
Height Standard Deviation Score (SDS) is the number of standard deviations above or below the mean height for age and sex. Height SDS was derived using the LMS method as ((Height/M)^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from 2000 CDC growth charts for the United States. A Standard Deviation Score of 0 represents the population mean. A higher change from baseline in Height SDS indicates a better outcome. The height SDS change from baseline at each visit was modeled using ANCOVA adjusting for baseline age, peak GH levels (log transformed) at diagnosis, delta average-parental height SDS, prior GH dose level (log transformed), and prior GH dose duration (log transformed) as covariates and gender as a factor. Subjects who did not take prior GH treatment were not included in the model.
Time Frame
Baseline and 26 weeks
Title
Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation
Description
Number of participants with treatment emergent anti-hGH antibodies over 26 weeks of weekly lonapegsomatropin (TransCon hGH) treatment. All samples were negative for anti-hGH neutralizing antibodies.
Time Frame
26 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Investigator-determined GHD diagnosis prior to the historical initiation of daily hGH therapy. 6 months to 17 years old, inclusive, at Visit 1 If 3 to 17 years old, are taking daily hGH at a dose of ≥ 0.20 mg hGH/kg/week for at least 13 weeks but no more than 130 weeks prior to Visit 1 If ≥ 6 months but < 3 years old, are either hGH treatment-naïve or are taking daily hGH at a dose of ≥ 0.20mg hGH/kg/week for no more than 130 weeks prior to Visit 1 Tanner stage < 5 at Visit 1 Open epiphyses (bone age ≤14.0 years for females or ≤16.0 years for males) 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: Weight of < 5.5 kg or > 80 kg at Visit 1 Females of child-bearing potential History of malignant disease Any clinically significant abnormality likely to affect growth or the ability to evaluate growth (eg, chronic diseases or conditions such as renal insufficiency, spinal cord irradiation, hypothyroidism, active celiac disease, malnutrition or psychosocial dwarfism) Poorly-controlled diabetes mellitus (HbA1c >8.0%) or diabetic complications Known neutralizing antibodies against hGH Major medical conditions, unless approved by Medical Monitor Pregnancy Presence of contraindications to hGH treatment Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver) Participation in any other trial of an investigational agent within 30 days prior to Visit 1 Prior exposure to investigational hGH
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Aimee D Shu, MD
Organizational Affiliation
Ascendis Pharma, Inc.
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
David B Karpf, MD
Organizational Affiliation
Ascendis Pharma, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
University of Alabama
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Neufeld Medical Group Inc.
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
Center of Excellence in Diabetes and Endocrinology
City
Sacramento
State/Province
California
ZIP/Postal Code
95821
Country
United States
Facility Name
Rocky Mountain Pediatric Endocrinology
City
Centennial
State/Province
Colorado
ZIP/Postal Code
80112
Country
United States
Facility Name
Nemours Children's Health System
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32207
Country
United States
Facility Name
Orlando Health Inc.
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Facility Name
Tallahassee Memorial Hospital
City
Tallahassee
State/Province
Florida
ZIP/Postal Code
32308
Country
United States
Facility Name
University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Children's Minnesota
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55102
Country
United States
Facility Name
University of Mississippi Medical Center
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Facility Name
Dartmouth Hitchcock Medical Center
City
Lebanon
State/Province
New Hampshire
ZIP/Postal Code
03756
Country
United States
Facility Name
NYU Winthrop Hospital
City
Mineola
State/Province
New York
ZIP/Postal Code
11501
Country
United States
Facility Name
Icahn School of Medicine at Mount Sinai
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
University of Oklahoma Health Sciences Center
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Children's Diabetes and Endocrine Center
City
Portland
State/Province
Oregon
ZIP/Postal Code
97227
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Children's Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
Cook Children's Medical Center
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
University of Virginia Children's Hospital
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22903
Country
United States
Facility Name
Children's Hospital of The King's Daughters
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23507
Country
United States
Facility Name
Monash Children's Hospital
City
Clayton
State/Province
Victoria
ZIP/Postal Code
3168
Country
Australia
Facility Name
Stollery Children's Hospital
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2B7
Country
Canada
Facility Name
The Liggins Institute, The University of Auckland
City
Grafton
State/Province
Auckland
ZIP/Postal Code
1023
Country
New Zealand

12. IPD Sharing Statement

Learn more about this trial

A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone Deficiency

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