A long-term extension trial of TransCon hGH, a sustained-release recombinant human growth hormone product, for treatment of Growth Hormone Deficiency in prepubertal children

• Register: EUCTR
• Last refreshed on: 9 January 2023
• Main ID: EUCTR2017-003410-20-BG
• Date of registration: 09/01/2018
• Prospective Registration: Yes
• Primary sponsor: Ascendis Pharma Endocrinology Division A/S
• Public title: A long-term extension trial of TransCon hGH, a sustained-release recombinant human growth hormone product, for treatment of Growth Hormone Deficiency in prepubertal children
• 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 - enliGHten
• Date of first enrolment: 20/04/2018
• Target sample size: 300
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-003410-20
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Armenia, Australia, Belarus, Bulgaria, Canada, Georgia, Greece, Italy, New Zealand, Poland, Romania, Russian Federation, Turkey, Ukraine, United States

Contacts

• Name: Clinical Trial Information Desk
• Affiliation: Ascendis Pharma A/S

• Name: Clinical Trial Information Desk
• Affiliation: Ascendis Pharma A/S

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 investigational product 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 institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC)
Are the trial subjects under 18? yes
Number of subjects for this age range: 300
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 regard 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:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Growth hormone deficiency (GHD) in prepubertal children
MedDRA version: 20.0 Level: PT Classification code 10056438 Term: Growth hormone deficiency System Organ Class: 10014698 - Endocrine disorders

Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Intervention(s)

Product Name: TransCon hGH (ACP-011)
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: lonapegsomatropin
Other descriptive name: TRANSCON PEG40 HGH
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 12.1-

Product Name: TransCon hGH (ACP-011)
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: lonapegsomatropin
Other descriptive name: TRANSCON PEG40 HGH
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 24.2-

Primary Outcome(s)

Main Objective: To assess long-term safety of weekly TransCon hGH in children with GHD previously treated in a phase 3 TransCon hGH trial

Timepoint(s) of evaluation of this end point: Throughout the trial

Primary end point(s): Safety Endpoints
The safety endpoints as measured throughout the long-term dosing of weekly TransCon hGH treatment include the following:
? Incidence of AEs
? Incidence of antibodies against hGH, including neutralizing antibodies
? Incidence of antibodies against PEG
? Incidence of antibodies against TransCon hGH
? Incidence of IGF-1 SDS > 2.0, > 3.0
? Parameters of HbA1c and lipids
? Hormone levels, including thyroid status and morning cortisol
? All other hematology and chemistry parameters
? Vital sign measurements

Secondary Objective: - To assess annualized height velocity (HV) with long-term dosing of weekly TransCon hGH treatment
- To assess the 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
- To evaluate the change in height standard deviation scores (?HSDS) with long-term dosing of weekly TransCon hGH treatment
- To determine the incidence of antibodies against TransCon hGH (anti-hGH and anti-PEG) with long-term dosing of weekly TransCon hGH treatment
- To assess the preference for weekly TransCon hGH or daily Genotropin
- To assess the treatment satisfaction of weekly TransCon hGH over time

Secondary Outcome(s)

Secondary end point(s): Efficacy Endpoints
The efficacy endpoints of long-term weekly TransCon hGH treatment include the following:
? Annualized HV
? ?HSDS
? Proportion of subjects with IGF-1 SDS of 0 to +2.0. Additionally, cut points of -2.0 to +2.0 and -1.0 to +2.0 will be assessed.
? IGF-1 SDS
? IGFBP-3 SDS
Pharmacodynamic Endpoint
? Serum IGF-1 SDS at 5 days ±1 day post-dose
Other Endpoints
? Preference for weekly TransCon hGH or daily Genotropin treatment
? Satisfaction with weekly TransCon hGH

Timepoint(s) of evaluation of this end point: Throughout the trial

Secondary ID(s)

TransCon_hGH_CT-301EXT

Source(s) of Monetary Support

Ascendis Pharma Endocrinology Division A/S

Ethics review

Status: Approved
Approval date: 20/04/2018
Contact: