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enliGHten: 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

Sponsor
Ascendis Pharma A/S (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT03344458
Collaborator
(none)
300
Enrollment
42
Locations
1
Arm
107.4
Anticipated Duration (Months)
7.1
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

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
  • Drug: TransCon hGH
 Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
300 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Intervention Model Description:
All study participants will receive TransCon hGHAll 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 :
Dec 19, 2017
Anticipated Primary Completion Date :
Dec 1, 2026
Anticipated Study Completion Date :
Dec 1, 2026

Arms and Interventions

Arm Intervention/Treatment
Experimental: TransCon hGH

Once weekly subcutaneous injection of TransCon hGH

Drug: TransCon hGH
Once weekly subcutaneous injection of TransCon hGH

Outcome Measures

Primary Outcome Measures

  1. Incidence of Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability] [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 [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 [Up to 4 years]

  3. Change in height standard deviation scores with long-term dosing of weekly TransCon hGH treatment [Up to 4 years]

  4. Incidence of antibodies against TransCon hGH with long-term dosing of weekly TransCon hGH treatment [Up to 4 years]

Eligibility Criteria

Criteria

Ages Eligible for Study:
1 Year to 18 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Ascendis Pharma Investigational Site Birmingham Alabama United States 35233
2 Ascendis Pharma Investigational Site Los Angeles California United States 90048
3 Ascendis Pharma Investigational Site Orange California United States 92868
4 Ascendis Pharma Investigational Site Sacramento California United States 95821
5 Ascendis Pharma Investigational Site Centennial Colorado United States 80112
6 Ascendis Pharma Investigational Site Jacksonville Florida United States 32207
7 Ascendis Pharma Investigational Site Orlando Florida United States 32806
8 Ascendis Pharma Investigational Site Tallahassee Florida United States 32308
9 Ascendis Pharma Investigational Site Saint Paul Minnesota United States 55102
10 Ascendis Pharma Investigational Site Jackson Mississippi United States 39216
11 Ascendis Pharma Investigational Site Lebanon New Hampshire United States 03756
12 Ascendis Pharma Investigational Site Mineola New York United States 11501
13 Ascendis Pharma Investigational Site New York New York United States 10029
14 Ascendis Pharma Investigational Site Cleveland Ohio United States 44195
15 Ascendis Pharma Investigational Site Oklahoma City Oklahoma United States 73104
16 Ascendis Pharma Investigational Site Portland Oregon United States 97227
17 Ascendis Pharma Investigational Site Portland Oregon United States 97239
18 Ascendis Pharma Investigational Site Dallas Texas United States 75390
19 Ascendis Pharma Investigational Site Fort Worth Texas United States 76104
20 Ascendis Pharma Investigational Site Charlottesville Virginia United States 22908
21 Ascendis Pharma Investigational Site Norfolk Virginia United States 23507
22 Ascendis Pharma Investigational Site Tacoma Washington United States 98405
23 Ascendis Pharma Investigational Site Yerevan Armenia 0075
24 Ascendis Pharma Investigational Site Clayton Victoria Australia 3168
25 Ascendis Pharma Investigational Site Minsk Belarus 220020
26 Ascendis Pharma Investigational Site Varna Bulgaria 9010
27 Ascendis Pharma Investigational Site Tbilisi Georgia 0159
28 Ascendis Pharma Investigational Site Athens Greece 11527
29 Ascendis Pharma Investigational Site Auckland New Zealand 1010
30 Ascendis Pharma Investigational Site Gdańsk Poland 80-952
31 Ascendis Pharma Investigational Site Warsaw Poland 02-691
32 Ascendis Pharma Investigational Site Izhevsk Russian Federation 426009
33 Ascendis Pharma Investigational Site Krasnoyarsk Russian Federation 660022
34 Ascendis Pharma Investigational Site Moscow Russian Federation 117036
35 Ascendis Pharma Investigational Site Saint Petersburg Russian Federation 194100
36 Ascendis Pharma Investigational Site Saratov Russian Federation 410054
37 Ascendis Pharma Investigational Site Ufa Russian Federation 450008
38 Ascendis Pharma Investigational Site Vologda Russian Federation 160022
39 Ascendis Pharma Investigational Site Voronezh Russian Federation 394024
40 Ascendis Pharma Investigational Site Kharkiv Ukraine 61093
41 Ascendis Pharma Investigational Site Kyiv Ukraine 01021
42 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.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Ascendis Pharma A/S
ClinicalTrials.gov Identifier:
NCT03344458
Other Study ID Numbers:
  • TransCon hGH CT-301EXT
  • 2017-003410-20
First Posted:
Nov 17, 2017
Last Update Posted:
Oct 14, 2021
Last Verified:
Oct 1, 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
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:
Dwarfism, Pituitary
Pituitary Diseases
Endocrine System Diseases

Study Results

No Results Posted as of Oct 14, 2021