A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)

NCT ID: NCT02781727

Last Updated: 2022-01-04

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

162 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-12-13

Study Completion Date

2019-01-17

Brief Summary

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A 52 week trial of TransCon hGH, a long-acting growth hormone product, versus human growth hormone therapy. TransCon hGH will be given once-a-week, human growth hormone (hGH) will be given daily. Approximately 150 prepubertal, hGH-treatment naïve children (males and females) with GHD will be included. Randomization will occur in a 2:1 ratio (TransCon hGH : Genotropin). This is a global trial that will be conducted in Armenia, Australia, Belarus, Bulgaria, Georgia, Greece, Italy, New Zealand, Poland, Romania, Russia, Turkey, Ukraine, and the United States.

Detailed Description

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Conditions

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Growth Hormone Deficiency, Pediatric hGH (Human Growth Hormone) Endocrine System Diseases Hormones Pituitary Diseases

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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TransCon hGH

Once weekly subcutaneous injection of TransCon hGH

Group Type EXPERIMENTAL

Once weekly subcutaneous injection of TransCon hGH

Intervention Type DRUG

Once weekly subcutaneous injection

human growth hormone (Genotropin)

Once daily subcutaneous injection of Genotropin

Group Type ACTIVE_COMPARATOR

Once daily subcutaneous injection of Genotropin

Intervention Type DRUG

Once daily subcutaneous injection

Interventions

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Once weekly subcutaneous injection of TransCon hGH

Once weekly subcutaneous injection

Intervention Type DRUG

Once daily subcutaneous injection of Genotropin

Once daily subcutaneous injection

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Prepubertal children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage 1 (Tanner 1982) aged:

* Boys: 3-12 years, inclusive
* Girls: 3-11 years, inclusive
* Impaired height (HT) defined as at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS ≤ -2.0) according to the 2000 CDC Growth Charts for the United States Methods and Development, available at http://www.cdc.gov/growthcharts/
* Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH level of ≤10 ng/mL, determined with a validated assay
* Bone age (BA) at least 6 months less than chronological age
* Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1)
* Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject (if the subject is able to read, understand, and sign)

Exclusion Criteria

* Children with a body weight below 12 kg
* Prior exposure to recombinant hGH or IGF-1 therapy
* Children with past or present intracranial tumor growth as confirmed by a sellar MRI scan (with contrast) at Screening (MRI results from up to 6 months prior to Screening may be accepted)
* Children with psychosocial dwarfism
* Children with idiopathic short stature
* History or presence of malignant disease; any evidence of present tumor growth
* Closed epiphyses
* Major medical conditions and/or presence of contraindication to hGH treatment
* Participation in any other trial of an investigational agent within 3 months prior to Screening
Minimum Eligible Age

3 Years

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ascendis Pharma Endocrinology Division A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Michael Beckert, MD

Role: STUDY_DIRECTOR

Ascendis Pharma A/S

Aimee D Shu, MD

Role: STUDY_DIRECTOR

Ascendis Pharma, Inc.

Locations

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Ascendis Pharma Investigational Site

Birmingham, Alabama, United States

Site Status

Ascendis Pharma Investigational Site

Little Rock, Arkansas, United States

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Ascendis Pharma Investigational Site

Los Angeles, California, United States

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Orange, California, United States

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Centennial, Colorado, United States

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Ascendis Pharma Investigational Site

Jacksonville, Florida, United States

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Orlando, Florida, United States

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Saint Paul, Minnesota, United States

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Jackson, Mississippi, United States

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Lebanon, New Hampshire, United States

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Mineola, New York, United States

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Oklahoma City, Oklahoma, United States

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Portland, Oregon, United States

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Dallas, Texas, United States

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Fort Worth, Texas, United States

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Tacoma, Washington, United States

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Yerevan, , Armenia

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Ascendis Pharma Investigational Site

Clayton, , Australia

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Minsk, , Belarus

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Varna, , Bulgaria

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Tbilisi, , Georgia

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Tbilisi, , Georgia

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Tbilisi, , Georgia

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Athens, , Greece

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Milan, , Italy

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Roma, , Italy

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Grafton, , New Zealand

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Gdansk, , Poland

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Warsaw, , Poland

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Iași, , Romania

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Izhevsk, , Russia

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Kazan', , Russia

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Krasnoyarsk, , Russia

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Moscow, , Russia

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Moscow, , Russia

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Nizhny Novgorod, , Russia

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Novosibirsk, , Russia

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Omsk, , Russia

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Saint Petersburg, , Russia

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Saint Petersburg, , Russia

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Samara, , Russia

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Saratov, , Russia

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Tomsk, , Russia

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Ufa, , Russia

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Vologda, , Russia

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Voronezh, , Russia

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Ascendis Pharma Investigational Site

Izmir, , Turkey (Türkiye)

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Ascendis Pharma Investigational Site

Melikgazi, , Turkey (Türkiye)

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Ascendis Pharma Investigational Site

Trabzon, , Turkey (Türkiye)

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Ascendis Pharma Investigational Site

Kharkiv, , Ukraine

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Ascendis Pharma Investigational Site

Kyiv, , Ukraine

Site Status

Ascendis Pharma Investigational Site

Kyiv, , Ukraine

Site Status

Ascendis Pharma Investigational Site

Odesa, , Ukraine

Site Status

Countries

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United States Armenia Australia Belarus Bulgaria Georgia Greece Italy New Zealand Poland Romania Russia Turkey (Türkiye) Ukraine

References

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Thornton PS, Maniatis AK, Aghajanova E, Chertok E, Vlachopapadopoulou E, Lin Z, Song W, Christoffersen ED, Breinholt VM, Kovalenko T, Giorgadze E, Korpal-Szczyrska M, Hofman PL, Karpf DB, Shu AD, Beckert M. Weekly Lonapegsomatropin in Treatment-Naive Children With Growth Hormone Deficiency: The Phase 3 heiGHt Trial. J Clin Endocrinol Metab. 2021 Oct 21;106(11):3184-3195. doi: 10.1210/clinem/dgab529.

Reference Type DERIVED
PMID: 34272849 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2016-001145-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

TransCon hGH CT-301

Identifier Type: -

Identifier Source: org_study_id

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