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)

April 12, 2024 updated by: Ascendis Pharma A/S

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

298

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Armenia
      • Yerevan, Armenia, 0075
        • Ascendis Pharma Investigational Site
  • Australia
    • Victoria
      • Clayton, Victoria, Australia, 3168
        • Ascendis Pharma Investigational Site
  • Belarus
      • Minsk, Belarus, 220020
        • Ascendis Pharma Investigational Site
  • Bulgaria
      • Varna, Bulgaria, 9010
        • Ascendis Pharma Investigational Site
  • Georgia
      • Tbilisi, Georgia, 0159
        • Ascendis Pharma Investigational Site
  • Greece
      • Athens, Greece, 11527
        • Ascendis Pharma Investigational Site
  • New Zealand
      • Auckland, New Zealand, 1010
        • Ascendis Pharma Investigational Site
  • Poland
      • Gdańsk, Poland, 80-952
        • Ascendis Pharma Investigational Site
      • Warsaw, Poland, 02-691
        • Ascendis Pharma Investigational Site
  • Russian Federation
      • Izhevsk, Russian Federation, 426009
        • Ascendis Pharma Investigational Site
      • Krasnoyarsk, Russian Federation, 660022
        • Ascendis Pharma Investigational Site
      • Moscow, Russian Federation, 117036
        • Ascendis Pharma Investigational Site
      • Saint Petersburg, Russian Federation, 194100
        • Ascendis Pharma Investigational Site
      • Saratov, Russian Federation, 410054
        • Ascendis Pharma Investigational Site
      • Ufa, Russian Federation, 450008
        • Ascendis Pharma Investigational Site
      • Vologda, Russian Federation, 160022
        • Ascendis Pharma Investigational Site
      • Voronezh, Russian Federation, 394024
        • Ascendis Pharma Investigational Site
  • Ukraine
      • Kharkiv, Ukraine, 61093
        • Ascendis Pharma Investigational Site
      • Kyiv, Ukraine, 01021
        • Ascendis Pharma Investigational Site
      • Odesa, Ukraine, 65031
        • Ascendis Pharma Investigational Site
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Ascendis Pharma Investigational Site
    • California
      • Los Angeles, California, United States, 90048
        • Ascendis Pharma Investigational Site
      • Orange, California, United States, 92868
        • Ascendis Pharma Investigational Site
      • Sacramento, California, United States, 95821
        • Ascendis Pharma Investigational Site
    • Colorado
      • Centennial, Colorado, United States, 80112
        • Ascendis Pharma Investigational Site
    • Florida
      • Jacksonville, Florida, United States, 32207
        • Ascendis Pharma Investigational Site
      • Orlando, Florida, United States, 32806
        • Ascendis Pharma Investigational Site
      • Tallahassee, Florida, United States, 32308
        • Ascendis Pharma Investigational Site
    • Minnesota
      • Saint Paul, Minnesota, United States, 55102
        • Ascendis Pharma Investigational Site
    • Mississippi
      • Jackson, Mississippi, United States, 39216
        • Ascendis Pharma Investigational Site
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Ascendis Pharma Investigational Site
    • New York
      • Mineola, New York, United States, 11501
        • Ascendis Pharma Investigational Site
      • New York, New York, United States, 10029
        • Ascendis Pharma Investigational Site
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Ascendis Pharma Investigational Site
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Ascendis Pharma Investigational Site
    • Oregon
      • Portland, Oregon, United States, 97239
        • Ascendis Pharma Investigational Site
      • Portland, Oregon, United States, 97227
        • Ascendis Pharma Investigational Site
    • Texas
      • Dallas, Texas, United States, 75390
        • Ascendis Pharma Investigational Site
      • Fort Worth, Texas, United States, 76104
        • Ascendis Pharma Investigational Site
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • Ascendis Pharma Investigational Site
      • Norfolk, Virginia, United States, 23507
        • Ascendis Pharma Investigational Site
    • Washington
      • Tacoma, Washington, United States, 98405
        • Ascendis Pharma Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: TransCon hGH
Once weekly subcutaneous injection of TransCon hGH
Drug: TransCon hGH
Once weekly subcutaneous injection of TransCon hGH

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability]
Time Frame: Up to Week 208
Long-term safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment
Up to Week 208

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized Height Velocity by Visit
Time Frame: Up to Week 208
Annualized height velocity (AHV) by visit with long-term dosing of weekly lonapegsomatropin treatment
Up to Week 208
IGF-1 SDS by Visit
Time Frame: Up to Week 208
Insulin-like Growth Factor-1 (IGF-1) standard deviation score (SDS) by visit with long-term dosing of weekly lonapegsomatropin treatment at Week 13, Week 52, Week 104, Week 156, and Week 208. IGF-1 SDS is the number of standard deviations above or below the mean IGF-1 level for a child of the same age and sex. The target range for IGF-1 SDS was 0 to +2. An IGF-1 SDS of 0 represents the population mean. If the IGF-1 SDS is < 0, then the negative score indicates an IGF-1 below the mean IGF-1 for a child of the same age and sex. If the IGF-1 SDS is > 0, then the positive score indicates an IGF-1 above the mean IGF-1 for a child of the same age and sex. A positive score of IGF-1 SDS indicates a better outcome.
Up to Week 208
Height SDS - Change From Baseline
Time Frame: Up to Week 208
Change in Height Standard Deviation Score (SDS) from baseline with long-term dosing of weekly lonapegsomatropin treatment at Week 13, Week 52, Week 104, Week 156, and Week 208. Height SDS is the number of standard deviations above or below the mean height for age and sex. A Standard Deviation Score of 0 equals the population mean. If the Height SDS is < 0, then the negative score indicates a height below the mean height for a child of the same age and sex. If the Height SDS is > 0, then the positive score indicates a height above the mean height for a child of the same age and sex. A positive change from baseline in Height SDS indicates improved outcomes.
Up to Week 208

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ascendis Pharma A/S

Investigators

  • Study Director: Michael Beckert, MD, Ascendis Pharma A/S
  • Study Director: Aimee D Shu, MD, Ascendis Pharma, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 19, 2017

Primary Completion (Actual)

February 21, 2023

Study Completion (Actual)

February 21, 2023

Study Registration Dates

First Submitted

November 14, 2017

First Submitted That Met QC Criteria

November 15, 2017

First Posted (Actual)

November 17, 2017

Study Record Updates

Last Update Posted (Actual)

May 8, 2024

Last Update Submitted That Met QC Criteria

April 12, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TransCon hGH CT-301EXT
  • 2017-003410-20 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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