A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone Deficiency

• Register: ClinicalTrials.gov
• Last refreshed on: 13 September 2021
• Main ID: NCT03305016
• Date of registration: 04/10/2017
• Prospective Registration: Yes
• Primary sponsor: Ascendis Pharma Endocrinology Division A/S
• Public title: A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone Deficiency
• Scientific title: fliGHt: A Multicenter, Phase 3, Open-Label, 26-Week Trial Investigating the Safety, Tolerability and Efficacy of TransCon hGH Administered Once Weekly in Children With GHD
• Date of first enrolment: November 13, 2017
• Target sample size: 146
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03305016
• 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

Australia, Canada, New Zealand, United States

Contacts

• Name: David B Karpf, MD
• Affiliation: Ascendis Pharma, Inc.

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

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Investigator-determined GHD diagnosis prior to the historical initiation of daily hGH
therapy.

2. 6 months to 17 years old, inclusive, at Visit 1

1. If 3 to 17 years old, are taking daily hGH at a dose of = 0.20 mg hGH/kg/week for
at least 13 weeks but no more than 130 weeks prior to Visit 1

2. If = 6 months but < 3 years old, are either hGH treatment-naïve or are taking
daily hGH at a dose of = 0.20mg hGH/kg/week for no more than 130 weeks prior to
Visit 1

3. Tanner stage < 5 at Visit 1

4. Open epiphyses (bone age =14.0 years for females or =16.0 years for males)

5. 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. Weight of < 5.5 kg or > 80 kg at Visit 1

2. Females of child-bearing potential

3. History of malignant disease

4. Any clinically significant abnormality likely to affect growth or the ability to
evaluate growth (eg, chronic diseases or conditions such as renal insufficiency,
spinal cord irradiation, hypothyroidism, active celiac disease, malnutrition or
psychosocial dwarfism)

5. Poorly-controlled diabetes mellitus (HbA1c >8.0%) or diabetic complications

6. Known neutralizing antibodies against hGH

7. Major medical conditions, unless approved by Medical Monitor

8. Pregnancy

9. Presence of contraindications to hGH treatment

10. Likely to be non-compliant with respect to trial conduct (in regards to the subject
and/or the parent/legal guardian/caregiver)

11. Participation in any other trial of an investigational agent within 30 days prior to
Visit 1

12. Prior exposure to investigational hGH



Age minimum: 6 Months
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pituitary Diseases

Hormone Deficiency

Endocrine System Diseases

Growth Hormone Deficiency, Pediatric

Intervention(s)

Drug: TransCon hGH

Primary Outcome(s)

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [Time Frame: 26 weeks]

Secondary Outcome(s)

Change in height standard deviation scores at 26 weeks of weekly TransCon hGH treatment [Time Frame: 26 weeks]

Proportion of subjects with IGF-1 standard deviation score (SDS) in the normal range of 0.0 to +2.0 at 26 weeks of weekly TransCon hGH treatment [Time Frame: 26 weeks]

Incidence of antibodies against TransCon hGH over 26 weeks of weekly TransCon hGH treatment [Time Frame: 26 weeks]

Annualized height velocity at 26 weeks of weekly TransCon hGH treatment [Time Frame: 26 weeks]

Secondary ID(s)

TransCon hGH CT-302

U1111-1199-8218

Source(s) of Monetary Support

Please refer to primary and secondary sponsors