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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03305016
Recruitment Status : Completed
First Posted : October 9, 2017
Last Update Posted : May 23, 2019
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S

Tracking Information
First Submitted Date  ICMJE October 4, 2017
First Posted Date  ICMJE October 9, 2017
Last Update Posted Date May 23, 2019
Actual Study Start Date  ICMJE November 13, 2017
Actual Primary Completion Date March 19, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 6, 2017)
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 26 weeks ]
Safety and tolerability of weekly TransCon hGH treatment
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03305016 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 6, 2017)
  • Annualized height velocity 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 ]
  • Change in height standard deviation scores 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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone Deficiency
Official Title  ICMJE 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
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Intervention Model Description:
All study participants will receive TransCon hGH
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Growth Hormone Deficiency, Pediatric
  • Endocrine System Diseases
  • Hormone Deficiency
  • Pituitary Diseases
Intervention  ICMJE Drug: TransCon hGH
Once weekly subcutaneous injection at a starting dose of 0.24 mg/kg/week
Study Arms  ICMJE Experimental: TransCon hGH
Once weekly subcutaneous injection of TransCon hGH
Intervention: Drug: TransCon hGH
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 27, 2018)
146
Original Estimated Enrollment  ICMJE
 (submitted: October 6, 2017)
150
Actual Study Completion Date  ICMJE March 19, 2019
Actual Primary Completion Date March 19, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Months to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Canada,   New Zealand,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03305016
Other Study ID Numbers  ICMJE TransCon hGH CT-302
U1111-1199-8218 ( Other Identifier: WHO UTN )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Ascendis Pharma A/S
Study Sponsor  ICMJE Ascendis Pharma A/S
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Aimee D Shu, MD Ascendis Pharma, Inc.
Study Director: David B Karpf, MD Ascendis Pharma, Inc.
PRS Account Ascendis Pharma A/S
Verification Date May 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP