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Completed

NCT Number: NCT03305016

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

A 26 week trial of TransCon hGH, a long-acting growth hormone product, administered once-a-week. Approximately 150 children (males and females) with growth hormone deficiency (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, Canada, Australia, and New Zealand.

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Key information

Conditions

Growth Hormone Deficiency, Pediatric Bone Diseases Bone Diseases, Developmental Bone Diseases, Endocrine Brain Diseases Central Nervous System Diseases Dwarfism Dwarfism, Pituitary Endocrine System Diseases Failure to Thrive Hormone Deficiency Hypopituitarism Hypothalamic Diseases Musculoskeletal Diseases Nervous System Diseases Pathological Conditions, Signs and Symptoms Pituitary Diseases Signs and Symptoms

Age range

6 month–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Monash Children's Hospital, Clayton, Victoria, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Investigator-determined GHD diagnosis prior to the historical initiation of daily hGH therapy.
  • 6 months to 17 years old, inclusive, at Visit 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
  • 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
  • Tanner stage < 5 at Visit 1
  • Open epiphyses (bone age ≤14.0 years for females or ≤16.0 years for males)
  • 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

  • Weight of < 5.5 kg or > 80 kg at Visit 1
  • Females of child-bearing potential
  • History of malignant disease
  • 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)
  • Poorly-controlled diabetes mellitus (HbA1c >8.0%) or diabetic complications
  • Known neutralizing antibodies against hGH
  • Major medical conditions, unless approved by Medical Monitor
  • Pregnancy
  • Presence of contraindications to hGH treatment
  • Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver)
  • Participation in any other trial of an investigational agent within 30 days prior to Visit 1
  • Prior exposure to investigational hGH

Treatment and study plan

TransCon hGH

Drug

Once weekly subcutaneous injection at a starting dose of 0.24 mg/kg/week

Primary outcomes

  1. Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]

    Time frame: 26 weeks

    Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment

Secondary outcomes

  1. Annualized Height Velocity (AHV) at 26 Weeks of Weekly Lonapegsomatropin Treatment

    Time frame: 26 weeks

    Annualized height velocity (AHV) at 26 weeks of weekly lonapegsomatropin (TransCon hGH) treatment. The AHV at each visit was modeled using ANCOVA adjusting for baseline age, peak GH levels (log transformed) at diagnosis, delta average-parental height SDS, prior GH dose level (log transformed), and prior GH dose duration (log transformed) as covariates and gender as a factor. Subjects who did not take prior GH treatment were not included in the model.

  2. Number of Subjects With IGF-1 Standard Deviation Score (SDS) in the Range of 0.0 to +2.0 at 26 Weeks of Weekly Lonapegsomatropin Treatment

    Time frame: 26 weeks

    IGF-1 Standard Deviation Score (SDS) is the number of standard deviations above or below the mean Insulin-like Growth Factor 1 (IGF-1) level for age and sex. IGF-1 SDS was derived using the LMS method as ((IGF-1/M)^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from Bidlingmaier et al. (2014). A Standard Deviation Score of 0 represents the population mean.

  3. Change in Height Standard Deviation Scores (SDS) at 26 Weeks of Weekly Lonapegsomatropin Treatment

    Time frame: Baseline and 26 weeks

    Height Standard Deviation Score (SDS) is the number of standard deviations above or below the mean height for age and sex. Height SDS was derived using the LMS method as ((Height/M)^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from 2000 CDC growth charts for the United States. A Standard Deviation Score of 0 represents the population mean. A higher change from baseline in Height SDS indicates a better outcome. The height SDS change from baseline at each visit was modeled using ANCOVA adjusting for baseline age, peak GH levels (log transformed) at diagnosis, delta average-parental height SDS, prior GH dose level (log transformed), and prior GH dose duration (log transformed) as covariates and gender as a factor. Subjects who did not take prior GH treatment were not included in the model.

  4. Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation

    Time frame: 26 weeks

    Number of participants with treatment emergent anti-hGH antibodies over 26 weeks of weekly lonapegsomatropin (TransCon hGH) treatment. All samples were negative for anti-hGH neutralizing antibodies.

Sponsors and collaborators

Lead sponsor

Ascendis Pharma Endocrinology Division A/S

Industry

Registry information

Official study 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

Important dates

Study start
2017
Primary completion
2019
Study completion
2019
First posted
Oct 9, 2017
Registry last updated
Jul 7, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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