Clinical trial NCT03305016: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: 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
Lead sponsor: Ascendis Pharma A/S
Sponsor class: INDUSTRY
First posted: 2017-10-09
Enrollment: 146
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: All study participants will receive TransCon hGH
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
timeFrame: 26 weeks
Secondary outcomes:
measure: Annualized Height Velocity (AHV) at 26 Weeks of Weekly Lonapegsomatropin Treatment
timeFrame: 26 weeks
measure: 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
timeFrame: 26 weeks
measure: Change in Height Standard Deviation Scores (SDS) at 26 Weeks of Weekly Lonapegsomatropin Treatment
timeFrame: Baseline and 26 weeks
measure: Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation
timeFrame: 26 weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama
state: Alabama
city: Los Angeles
country: United States
facility: Neufeld Medical Group Inc.
state: California
city: Sacramento
country: United States
facility: Center of Excellence in Diabetes and Endocrinology
state: California
city: Centennial
country: United States
facility: Rocky Mountain Pediatric Endocrinology
state: Colorado
city: Jacksonville
country: United States
facility: Nemours Children's Health System
state: Florida
city: Orlando
country: United States
facility: Orlando Health Inc.
state: Florida
city: Tallahassee
country: United States
facility: Tallahassee Memorial Hospital
state: Florida
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Saint Paul
country: United States
facility: Children's Minnesota
state: Minnesota
city: Jackson
country: United States
facility: University of Mississippi Medical Center
state: Mississippi
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: Mineola
country: United States
facility: NYU Winthrop Hospital
state: New York
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Oklahoma City
country: United States
facility: University of Oklahoma Health Sciences Center
state: Oklahoma
city: Portland
country: United States
facility: Children's Diabetes and Endocrine Center
state: Oregon
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Dallas
country: United States
facility: Children's Medical Center
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: Charlottesville
country: United States
facility: University of Virginia Children's Hospital
state: Virginia
city: Norfolk
country: United States
facility: Children's Hospital of The King's Daughters
state: Virginia
city: Clayton
country: Australia
facility: Monash Children's Hospital
state: Victoria
city: Edmonton
country: Canada
facility: Stollery Children's Hospital
state: Alberta
city: Grafton
country: New Zealand
facility: The Liggins Institute, The University of Auckland
state: Auckland
Study officials:
affiliation: Ascendis Pharma, Inc.
name: Aimee D Shu, MD
role: STUDY_DIRECTOR
affiliation: Ascendis Pharma, Inc.
name: David B Karpf, MD
role: STUDY_DIRECTOR
References: