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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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: NCT03344458 |
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Recruitment Status :
Active, not recruiting
First Posted : November 17, 2017
Last Update Posted : October 14, 2021
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Sponsor:
Ascendis Pharma A/S
Information provided by (Responsible Party):
Ascendis Pharma A/S
- Study Details
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- No Results Posted
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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 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 300 participants |
| Allocation: | N/A |
| 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 2026 |
| Estimated Study Completion Date : | December 2026 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Metatropic dysplasia
Isolated growth hormone deficiency
Pseudoachondroplasia
MedlinePlus related topics:
Hormones
| Arm | Intervention/treatment |
|---|---|
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Experimental: TransCon hGH
Once weekly subcutaneous injection of TransCon hGH
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Drug: TransCon hGH
Once weekly subcutaneous injection of TransCon hGH |
Primary Outcome Measures :
- 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 :
- Annualized height velocity (HV) with long-term dosing of weekly TransCon hGH treatment [ Time Frame: Up to 4 years ]
- 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 ]
- Change in height standard deviation scores with long-term dosing of weekly TransCon hGH treatment [ Time Frame: Up to 4 years ]
- Incidence of antibodies against TransCon hGH with long-term dosing of weekly TransCon hGH treatment [ Time Frame: Up to 4 years ]
Information from the National Library of Medicine
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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:
- Children who have completed a prior phase 3 TransCon hGH trial
- Children who have not permanently discontinued study drug in the prior trial
- 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:
- Poorly-controlled diabetes mellitus (HbA1c ≥ 8.0%) or diabetic complications
- Evidence of closed epiphyses, defined as bone age > 14.0 years for females or > 16.0 years for males
- Major medical conditions unless approved by Medical Expert
- Known hypersensitivity to the components of the trial medication
- Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver)
- Pregnancy
- 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
Locations
| United States, Alabama | |
| Ascendis Pharma Investigational Site | |
| Birmingham, Alabama, United States, 35233 | |
| United States, California | |
| Ascendis Pharma Investigational Site | |
| Los Angeles, California, United States, 90048 | |
| Ascendis Pharma Investigational Site | |
| Orange, California, United States, 92868 | |
| Ascendis Pharma Investigational Site | |
| Sacramento, California, United States, 95821 | |
| United States, Colorado | |
| Ascendis Pharma Investigational Site | |
| Centennial, Colorado, United States, 80112 | |
| United States, Florida | |
| Ascendis Pharma Investigational Site | |
| Jacksonville, Florida, United States, 32207 | |
| Ascendis Pharma Investigational Site | |
| Orlando, Florida, United States, 32806 | |
| Ascendis Pharma Investigational Site | |
| Tallahassee, Florida, United States, 32308 | |
| United States, Minnesota | |
| Ascendis Pharma Investigational Site | |
| Saint Paul, Minnesota, United States, 55102 | |
| United States, Mississippi | |
| Ascendis Pharma Investigational Site | |
| Jackson, Mississippi, United States, 39216 | |
| United States, New Hampshire | |
| Ascendis Pharma Investigational Site | |
| Lebanon, New Hampshire, United States, 03756 | |
| United States, New York | |
| Ascendis Pharma Investigational Site | |
| Mineola, New York, United States, 11501 | |
| Ascendis Pharma Investigational Site | |
| New York, New York, United States, 10029 | |
| United States, Ohio | |
| Ascendis Pharma Investigational Site | |
| Cleveland, Ohio, United States, 44195 | |
| United States, Oklahoma | |
| Ascendis Pharma Investigational Site | |
| Oklahoma City, Oklahoma, United States, 73104 | |
| United States, Oregon | |
| Ascendis Pharma Investigational Site | |
| Portland, Oregon, United States, 97227 | |
| Ascendis Pharma Investigational Site | |
| Portland, Oregon, United States, 97239 | |
| United States, Texas | |
| Ascendis Pharma Investigational Site | |
| Dallas, Texas, United States, 75390 | |
| Ascendis Pharma Investigational Site | |
| Fort Worth, Texas, United States, 76104 | |
| United States, Virginia | |
| Ascendis Pharma Investigational Site | |
| Charlottesville, Virginia, United States, 22908 | |
| Ascendis Pharma Investigational Site | |
| Norfolk, Virginia, United States, 23507 | |
| United States, Washington | |
| Ascendis Pharma Investigational Site | |
| Tacoma, Washington, United States, 98405 | |
| Armenia | |
| Ascendis Pharma Investigational Site | |
| Yerevan, Armenia, 0075 | |
| Australia, Victoria | |
| Ascendis Pharma Investigational Site | |
| Clayton, Victoria, Australia, 3168 | |
| Belarus | |
| Ascendis Pharma Investigational Site | |
| Minsk, Belarus, 220020 | |
| Bulgaria | |
| Ascendis Pharma Investigational Site | |
| Varna, Bulgaria, 9010 | |
| Georgia | |
| Ascendis Pharma Investigational Site | |
| Tbilisi, Georgia, 0159 | |
| Greece | |
| Ascendis Pharma Investigational Site | |
| Athens, Greece, 11527 | |
| New Zealand | |
| Ascendis Pharma Investigational Site | |
| Auckland, New Zealand, 1010 | |
| Poland | |
| Ascendis Pharma Investigational Site | |
| Gdańsk, Poland, 80-952 | |
| Ascendis Pharma Investigational Site | |
| Warsaw, Poland, 02-691 | |
| Russian Federation | |
| Ascendis Pharma Investigational Site | |
| 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 | |
| Ukraine | |
| Ascendis Pharma Investigational Site | |
| Kharkiv, Ukraine, 61093 | |
| Ascendis Pharma Investigational Site | |
| Kyiv, Ukraine, 01021 | |
| Ascendis Pharma Investigational Site | |
| Odesa, Ukraine, 65031 | |
Sponsors and Collaborators
Ascendis Pharma A/S
Investigators
| Study Director: | Michael Beckert, MD | Ascendis Pharma A/S | |
| Study Director: | Aimee D Shu, MD | Ascendis Pharma, Inc. |
| Responsible Party: | Ascendis Pharma A/S |
| ClinicalTrials.gov Identifier: | NCT03344458 |
| Other Study ID Numbers: |
TransCon hGH CT-301EXT 2017-003410-20 ( EudraCT Number ) |
| First Posted: | November 17, 2017 Key Record Dates |
| Last Update Posted: | October 14, 2021 |
| Last Verified: | October 2021 |
| 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:
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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 |