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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

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.

Condition or disease Intervention/treatment Phase
Growth Hormone Deficiency, Pediatric Endocrine System Diseases Hormone Deficiency Pituitary Diseases Drug: TransCon hGH Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 146 participants
Intervention Model: Single Group Assignment
Intervention Model Description: All study participants will receive TransCon hGH
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : November 13, 2017
Actual Primary Completion Date : March 19, 2019
Actual Study Completion Date : March 19, 2019


Arm Intervention/treatment
Experimental: TransCon hGH
Once weekly subcutaneous injection of TransCon hGH
Drug: TransCon hGH
Once weekly subcutaneous injection at a starting dose of 0.24 mg/kg/week




Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 26 weeks ]
    Safety and tolerability of weekly TransCon hGH treatment


Secondary Outcome Measures :
  1. Annualized height velocity at 26 weeks of weekly TransCon hGH treatment [ Time Frame: 26 weeks ]
  2. 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 ]
  3. Change in height standard deviation scores at 26 weeks of weekly TransCon hGH treatment [ Time Frame: 26 weeks ]
  4. Incidence of antibodies against TransCon hGH over 26 weeks of weekly TransCon hGH treatment [ Time Frame: 26 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03305016


  Show 24 Study Locations
Sponsors and Collaborators
Ascendis Pharma A/S
Investigators
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Study Director: Aimee D Shu, MD Ascendis Pharma, Inc.
Study Director: David B Karpf, MD Ascendis Pharma, Inc.

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Responsible Party: Ascendis Pharma A/S
ClinicalTrials.gov Identifier: NCT03305016     History of Changes
Other Study ID Numbers: TransCon hGH CT-302
U1111-1199-8218 ( Other Identifier: WHO UTN )
First Posted: October 9, 2017    Key Record Dates
Last Update Posted: May 23, 2019
Last Verified: May 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ascendis Pharma A/S:
Human Growth Hormone
hGH
GHD
rhGH
Pediatric Growth Hormone Deficiency
Long Acting Growth Hormone
Somatropin
Prodrug
Growth Failure
Growth Hormone Replacement Therapy
Sustained Release Growth Hormone
Growth Hormone Deficiency
TransCon GH
Additional relevant MeSH terms:
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Dwarfism, Pituitary
Pituitary Diseases
Endocrine System Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs