NCT03344458|CompletedPhase 3ResultsDMCFDA Drug

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

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

Ascendis Pharma A/S ClinicalTrials.gov

Compare

2 other identifiers

TransCon hGH CT-301EXT2017-003410-20

Study Type

interventional

Target

298

Locations

11 countries

Sites

Timeline

RegisteredNov 2017

StartedDec 2017

CompletionFeb 2023

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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

298

participants targeted

Target at P50-P75 for phase_3

Timeline

Completed

Started Dec 2017

Longer than P75 for phase_3

Geographic Reach

11 countries

42 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

5.2 years study duration

First Submitted

Initial submission to the registry

November 14, 2017

Completed

3 days until next milestone

First Posted

Study publicly available on registry

November 17, 2017

Completed

1 month until next milestone

Study Start

First participant enrolled

December 19, 2017

Completed

5.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 21, 2023

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 21, 2023

Completed

1.2 years until next milestone

Results Posted

Study results publicly available

May 8, 2024

Completed

Last Updated

May 8, 2024

Status Verified

April 1, 2024

Enrollment Period

5.2 years

First QC Date

November 14, 2017

Results QC Date

February 21, 2024

Last Update Submit

April 12, 2024

Conditions

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

Keywords

Human Growth HormonehGHGHDrhGHPediatric Growth Hormone DeficiencyLong Acting Growth HormoneSomatropinProdrugGrowth FailureGrowth Hormone Replacement TherapySustained Release Growth HormoneGrowth Hormone DeficiencyTransCon GH

Outcome Measures

Primary Outcomes (1)

Number of Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability]
Long-term safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment
Up to Week 208

Secondary Outcomes (3)

Annualized Height Velocity by Visit
Up to Week 208
IGF-1 SDS by Visit
Up to Week 208
Height SDS - Change From Baseline
Up to Week 208

Study Arms (1)

TransCon hGH

EXPERIMENTAL

Once weekly subcutaneous injection of TransCon hGH

Drug: TransCon hGH

Interventions

TransCon hGHDRUG

Once weekly subcutaneous injection of TransCon hGH

TransCon hGH

Eligibility Criteria

Age1 Year - 18 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

Children who have completed a prior phase 3 TransCon hGH trial
Children who have not permanently discontinued study drug in the prior trial
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

You may not qualify if:

Poorly-controlled diabetes mellitus (HbA1c ≥ 8.0%) or diabetic complications
Evidence of closed epiphyses, defined as bone age \> 14.0 years for females or \> 16.0 years for males
Major medical conditions unless approved by Medical Expert
Known hypersensitivity to the components of the trial medication
Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver)
Pregnancy
Any other reason that in the opinion of the investigator would prevent the subject from completing participation or following the trial schedule

Contact the study team to confirm eligibility.