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Trial · Interventional

CompletedPhase 3Results postedRegistry data

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

NCTNCT03305016 (opens ClinicalTrials.gov in a new tab)Other idTransCon hGH CT-302Other idU1111-1199-8218
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 3
Enrollment
146 participants (actual)
Sponsor
Ascendis Pharma Endocrinology Division A/S · Industry
Design
Allocation not applicable · Open label (no masking)
Countries
4 countries

United States, Australia, Canada, New Zealand

Start
13 Nov 2017
Primary completion
19 Mar 2019
Completion
19 Mar 2019
First posted
9 Oct 2017
Last update posted
7 Jul 2026
Conditions
Growth Hormone Deficiency, Pediatric · Endocrine System Diseases · Hormone Deficiency · Pituitary Diseases

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2017201820192020
  1. Ph 3

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

    NCT03305016

    13 Nov 2017 to 19 Mar 2019

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • TransCon hGHDrug

Arms1

  • Experimental

    TransCon hGH

    Once weekly subcutaneous injection of TransCon hGH

Primary outcomes1

Outcomes the sponsor listed as primary.

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

    Time frame · 26 weeks

    Registry description

    Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment

Secondary outcomes(4)

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

    Time frame · 26 weeks

    Registry description

    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

    Registry description

    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

    Registry description

    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

    Registry description

    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.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeCount of participants· 26 weeks

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

GroupValue (Participants)
Lonapegsomatropin83
Secondary and other outcome results(4)
Secondary outcomeLeast squares mean· 26 weeks

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

GroupValue (cm/year)Spread
Lonapegsomatropin8.720.24
Secondary outcomeCount of participants· 26 weeks

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

GroupValue (Participants)
Lonapegsomatropin74
Secondary outcomeLeast squares mean· Baseline and 26 weeks

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

GroupValue (standard deviation score)Spread
Lonapegsomatropin0.250.02
Secondary outcomeCount of participants· 26 weeks

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

GroupValue (Participants)
Lonapegsomatropin4

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
LonapegsomatropinOnce weekly subcutaneous injection of lonapegsomatropin (TransCon hGH) at a starting dose of 0.24 mg/kg/week0 / 1461 / 14665 / 146

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 6 Months up to 17 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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

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