Clinical trial NCT03344458: 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
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
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: 2017-11-17
Enrollment: 298
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
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 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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability]
timeFrame: Up to Week 208
Secondary outcomes:
measure: Annualized Height Velocity by Visit
timeFrame: Up to Week 208
measure: IGF-1 SDS by Visit
timeFrame: Up to Week 208
measure: Height SDS - Change From Baseline
timeFrame: Up to Week 208
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Ascendis Pharma Investigational Site
state: Alabama
city: Los Angeles
country: United States
facility: Ascendis Pharma Investigational Site
state: California
city: Orange
country: United States
facility: Ascendis Pharma Investigational Site
state: California
city: Sacramento
country: United States
facility: Ascendis Pharma Investigational Site
state: California
city: Centennial
country: United States
facility: Ascendis Pharma Investigational Site
state: Colorado
city: Jacksonville
country: United States
facility: Ascendis Pharma Investigational Site
state: Florida
city: Orlando
country: United States
facility: Ascendis Pharma Investigational Site
state: Florida
city: Tallahassee
country: United States
facility: Ascendis Pharma Investigational Site
state: Florida
city: Saint Paul
country: United States
facility: Ascendis Pharma Investigational Site
state: Minnesota
city: Jackson
country: United States
facility: Ascendis Pharma Investigational Site
state: Mississippi
city: Lebanon
country: United States
facility: Ascendis Pharma Investigational Site
state: New Hampshire
city: Mineola
country: United States
facility: Ascendis Pharma Investigational Site
state: New York
city: New York
country: United States
facility: Ascendis Pharma Investigational Site
state: New York
city: Cleveland
country: United States
facility: Ascendis Pharma Investigational Site
state: Ohio
city: Oklahoma City
country: United States
facility: Ascendis Pharma Investigational Site
state: Oklahoma
city: Portland
country: United States
facility: Ascendis Pharma Investigational Site
state: Oregon
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: Fort Worth
country: United States
facility: Ascendis Pharma Investigational Site
state: Texas
city: Charlottesville
country: United States
facility: Ascendis Pharma Investigational Site
state: Virginia
city: Norfolk
country: United States
facility: Ascendis Pharma Investigational Site
state: Virginia
city: Tacoma
country: United States
facility: Ascendis Pharma Investigational Site
state: Washington
city: Yerevan
country: Armenia
facility: Ascendis Pharma Investigational Site
city: Clayton
country: Australia
facility: Ascendis Pharma Investigational Site
state: Victoria
city: Minsk
country: Belarus
facility: Ascendis Pharma Investigational Site
city: Varna
country: Bulgaria
facility: Ascendis Pharma Investigational Site
city: Tbilisi
country: Georgia
facility: Ascendis Pharma Investigational Site
city: Athens
country: Greece
facility: Ascendis Pharma Investigational Site
city: Auckland
country: New Zealand
facility: Ascendis Pharma Investigational Site
city: Gdańsk
country: Poland
facility: Ascendis Pharma Investigational Site
city: Warsaw
country: Poland
facility: Ascendis Pharma Investigational Site
city: Izhevsk
country: Russian Federation
facility: Ascendis Pharma Investigational Site
city: Krasnoyarsk
country: Russian Federation
facility: Ascendis Pharma Investigational Site
city: Moscow
country: Russian Federation
facility: Ascendis Pharma Investigational Site
city: Saint Petersburg
country: Russian Federation
facility: Ascendis Pharma Investigational Site
city: Saratov
country: Russian Federation
facility: Ascendis Pharma Investigational Site
city: Ufa
country: Russian Federation
facility: Ascendis Pharma Investigational Site
city: Vologda
country: Russian Federation
facility: Ascendis Pharma Investigational Site
city: Voronezh
country: Russian Federation
facility: Ascendis Pharma Investigational Site
city: Kharkiv
country: Ukraine
facility: Ascendis Pharma Investigational Site
city: Kyiv
country: Ukraine
facility: Ascendis Pharma Investigational Site
city: Odesa
country: Ukraine
facility: Ascendis Pharma Investigational Site
Study officials:
affiliation: Ascendis Pharma A/S
name: Michael Beckert, MD
role: STUDY_DIRECTOR
affiliation: Ascendis Pharma, Inc.
name: Aimee D Shu, MD
role: STUDY_DIRECTOR
References:
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.