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

• Register: ClinicalTrials.gov
• Last refreshed on: 23 October 2023
• Main ID: NCT03344458
• Date of registration: 14/11/2017
• Prospective Registration: Yes
• Primary sponsor: Ascendis Pharma A/S
• Public 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 enliGHten
• Scientific 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
• Date of first enrolment: December 19, 2017
• Target sample size: 298
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03344458
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

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

Contacts

• Name: Michael Beckert, MD
• Affiliation: Ascendis Pharma A/S

• Name: Aimee D Shu, MD
• Affiliation: Ascendis Pharma, Inc.

Key inclusion & exclusion 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



Age minimum: 1 Year
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Growth Hormone Deficiency, Pediatric

Endocrine System Diseases

Hormone Deficiency

Pituitary Diseases

Intervention(s)

Drug: TransCon hGH

Primary Outcome(s)

Incidence of Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability] [Time Frame: Up to 4 years]

Secondary Outcome(s)

Annualized height velocity (HV) with long-term dosing of weekly TransCon hGH treatment [Time Frame: Up to 4 years]

Incidence of antibodies against TransCon hGH with long-term dosing of weekly TransCon hGH treatment [Time Frame: Up to 4 years]

Change in height standard deviation scores with long-term dosing of weekly TransCon hGH treatment [Time Frame: Up to 4 years]

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]

Secondary ID(s)

TransCon hGH CT-301EXT

2017-003410-20

Source(s) of Monetary Support

Please refer to primary and secondary sponsors