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A Long-Term Trial Investigating Safety and Efficacy of TransCon hGH in Children With Growth Hormone Deficiency Who Have Completed a Prior TransCon hGH Clinical Trial (enliGHten)

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ClinicalTrials.gov Identifier: NCT03344458
Recruitment Status : Recruiting
First Posted : November 17, 2017
Last Update Posted : January 10, 2019
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S

Brief Summary:
A multicenter, phase 3, long-term extension trial of TransCon hGH administered once-weekly in children with growth hormone deficiency (GHD) who previously participated in a phase 3 TransCon hGH trial. Approximately 300 children (males and females) with 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, Poland, Bulgaria, Ukraine, Armenia, Russia and Australia.

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)
Estimated Enrollment : 300 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: enliGHten: A Multicenter, Phase 3, Long-Term, Open-Label Trial Investigating Safety and Efficacy of TransCon hGH Administered Once-Weekly in Children With Growth Hormone Deficiency (GHD) Who Have Completed a Prior TransCon hGH Clinical Trial
Actual Study Start Date : December 19, 2017
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : December 2021


Arm Intervention/treatment
Experimental: TransCon hGH
Once weekly subcutaneous injection of TransCon hGH
Drug: TransCon hGH
Once weekly subcutaneous injection of TransCon hGH




Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability] [ Time Frame: Up to 4 years ]
    Long-term safety and tolerability of weekly TransCon hGH treatment


Secondary Outcome Measures :
  1. Annualized height velocity (HV) with long-term dosing of weekly TransCon hGH treatment [ Time Frame: Up to 4 years ]
  2. Proportion of subjects with IGF-1 standard deviation score (SDS) in the normal range of 0.0 to +2.0 with long-term dosing of weekly TransCon hGH treatment [ Time Frame: Up to 4 years ]
  3. Change in height standard deviation scores with long-term dosing of weekly TransCon hGH treatment [ Time Frame: Up to 4 years ]
  4. Incidence of antibodies against TransCon hGH with long-term dosing of weekly TransCon hGH treatment [ Time Frame: Up to 4 years ]


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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Children who have completed a prior phase 3 TransCon hGH trial
  2. Children who have not permanently discontinued study drug in the prior trial
  3. 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. Poorly-controlled diabetes mellitus (HbA1c ≥ 8.0%) or diabetic complications
  2. Evidence of closed epiphyses, defined as bone age > 14.0 years for females or > 16.0 years for males
  3. Major medical conditions unless approved by Medical Expert
  4. Known hypersensitivity to the components of the trial medication
  5. Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver)
  6. Pregnancy
  7. Any other reason that in the opinion of the investigator would prevent the subject from completing participation or following the trial schedule

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): NCT03344458


Contacts
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Contact: Michael Beckert, MD +49 172-155-2596 mb@ascendispharma.com
Contact: Aimee D Shu, MD +1 650-461-4835 ads@ascendispharma.com

  Show 42 Study Locations
Sponsors and Collaborators
Ascendis Pharma A/S
Investigators
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Study Director: Michael Beckert, MD Ascendis Pharma A/S
Study Director: Aimee D Shu, MD Ascendis Pharma, Inc.

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Responsible Party: Ascendis Pharma A/S
ClinicalTrials.gov Identifier: NCT03344458     History of Changes
Other Study ID Numbers: TransCon hGH CT-301EXT
2017-003410-20 ( EudraCT Number )
First Posted: November 17, 2017    Key Record Dates
Last Update Posted: January 10, 2019
Last Verified: January 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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Endocrine System Diseases
Dwarfism, Pituitary
Pituitary Diseases
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs