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)

Ascendis Pharma

Status and phase: Completed; Phase 3

Conditions: Pituitary Diseases; Hormone Deficiency; Growth Hormone Deficiency, Pediatric; Endocrine System Diseases

Treatments: Drug: TransCon hGH

Study type: Interventional

Funder types: Industry

Identifiers

NCT03344458

2017-003410-20 (EudraCT Number)

TransCon hGH CT-301EXT

Details and patient eligibility

About

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.

Enrollment: 298 patients

Sex: All

Ages: 1 to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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298 participants in 1 patient group

TransCon hGH

Experimental group

Description:

Once weekly subcutaneous injection of TransCon hGH

Treatment:

Drug: TransCon hGH