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

Endocrine System Diseases Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03344458
• Other study ID #: TransCon hGH CT-301EXT
• Secondary ID: 2017-003410-20
• Status: Completed
• Phase: Phase 3
• Start date: December 19, 2017
• Est. completion date: February 21, 2023

Study information

• Verified date: October 2023
• Source: Ascendis Pharma A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 298
• Est. completion date: February 21, 2023
• Est. primary completion date: February 21, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Endocrine System Diseases
• Growth Hormone Deficiency, Pediatric
• Hormone Deficiency
• Pituitary Diseases

Intervention

Drug:
• TransCon hGH
Once weekly subcutaneous injection of TransCon hGH

Locations

Country	Name	City	State
Armenia	Ascendis Pharma Investigational Site	Yerevan
Australia	Ascendis Pharma Investigational Site	Clayton	Victoria
Belarus	Ascendis Pharma Investigational Site	Minsk
Bulgaria	Ascendis Pharma Investigational Site	Varna
Georgia	Ascendis Pharma Investigational Site	Tbilisi
Greece	Ascendis Pharma Investigational Site	Athens
New Zealand	Ascendis Pharma Investigational Site	Auckland
Poland	Ascendis Pharma Investigational Site	Gdansk
Poland	Ascendis Pharma Investigational Site	Warsaw
Russian Federation	Ascendis Pharma Investigational Site	Izhevsk
Russian Federation	Ascendis Pharma Investigational Site	Krasnoyarsk
Russian Federation	Ascendis Pharma Investigational Site	Moscow
Russian Federation	Ascendis Pharma Investigational Site	Saint Petersburg
Russian Federation	Ascendis Pharma Investigational Site	Saratov
Russian Federation	Ascendis Pharma Investigational Site	Ufa
Russian Federation	Ascendis Pharma Investigational Site	Vologda
Russian Federation	Ascendis Pharma Investigational Site	Voronezh
Ukraine	Ascendis Pharma Investigational Site	Kharkiv
Ukraine	Ascendis Pharma Investigational Site	Kyiv
Ukraine	Ascendis Pharma Investigational Site	Odesa
United States	Ascendis Pharma Investigational Site	Birmingham	Alabama
United States	Ascendis Pharma Investigational Site	Centennial	Colorado
United States	Ascendis Pharma Investigational Site	Charlottesville	Virginia
United States	Ascendis Pharma Investigational Site	Cleveland	Ohio
United States	Ascendis Pharma Investigational Site	Dallas	Texas
United States	Ascendis Pharma Investigational Site	Fort Worth	Texas
United States	Ascendis Pharma Investigational Site	Jackson	Mississippi
United States	Ascendis Pharma Investigational Site	Jacksonville	Florida
United States	Ascendis Pharma Investigational Site	Lebanon	New Hampshire
United States	Ascendis Pharma Investigational Site	Los Angeles	California
United States	Ascendis Pharma Investigational Site	Mineola	New York
United States	Ascendis Pharma Investigational Site	New York	New York
United States	Ascendis Pharma Investigational Site	Norfolk	Virginia
United States	Ascendis Pharma Investigational Site	Oklahoma City	Oklahoma
United States	Ascendis Pharma Investigational Site	Orange	California
United States	Ascendis Pharma Investigational Site	Orlando	Florida
United States	Ascendis Pharma Investigational Site	Portland	Oregon
United States	Ascendis Pharma Investigational Site	Portland	Oregon
United States	Ascendis Pharma Investigational Site	Sacramento	California
United States	Ascendis Pharma Investigational Site	Saint Paul	Minnesota
United States	Ascendis Pharma Investigational Site	Tacoma	Washington
United States	Ascendis Pharma Investigational Site	Tallahassee	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Ascendis Pharma A/S

Countries where clinical trial is conducted

United States,  Armenia,  Australia,  Belarus,  Bulgaria,  Georgia,  Greece,  New Zealand,  Poland,  Russian Federation,  Ukraine, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability]	Long-term safety and tolerability of weekly TransCon hGH treatment	Up to 4 years
Secondary	Annualized height velocity (HV) with long-term dosing of weekly TransCon hGH treatment		Up to 4 years
Secondary	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		Up to 4 years
Secondary	Change in height standard deviation scores with long-term dosing of weekly TransCon hGH treatment		Up to 4 years
Secondary	Incidence of antibodies against TransCon hGH with long-term dosing of weekly TransCon hGH treatment		Up to 4 years