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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
298

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Ascendis Pharma A/S

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 19, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 21, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 21, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 14, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 15, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 17, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 8, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 12, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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