Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD

NCT ID: NCT03831880

Last Updated: 2021-10-14

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

87 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-02-07

Study Completion Date

2020-08-28

Brief Summary

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This is an open label randomized 24 week crossover trial assessing the treatment burden of a weekly growth hormone injection regimen (somatrogon) compared to a daily growth hormone injection regimen (Genotropin). Approximately 90 children with growth hormone deficiency who have been stable on treatment with daily Genotropin will be enrolled.

Detailed Description

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Subjects will be randomized to one of two sequences, either 12 weeks of continued treatment with daily Genotropin followed by 12 weeks of treatment with weekly somatrogon, or 12 weeks of treatment with weekly somatrogon followed by 12 weeks of treatment with daily Genotropin. Subjects will have study visits at Baseline, Weeks 6, 12, 18, and 24. Subjects will also be followed up by phone 8 to 12 days after each treatment period begins (Week 1 and Week 13). Subjects and caregivers (as a Dyad) will complete questionnaires assessing treatment burden at baseline and at the end of each 12 week treatment period. All subjects/caregivers will receive a follow up phone call at Week 28.

Conditions

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Growth Hormone Deficiency

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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Daily to Weekly

Genotropin to somatrogon

Group Type OTHER

Genotropin

Intervention Type DRUG

Genotropin (dose \[mg\] at time of enrollment) given subcutaneously once daily

somatrogon

Intervention Type DRUG

0.66 mg/kg/week given subcutaneously once weekly

Weekly to Daily

somatrogon to Genotropin

Group Type OTHER

Genotropin

Intervention Type DRUG

Genotropin (dose \[mg\] at time of enrollment) given subcutaneously once daily

somatrogon

Intervention Type DRUG

0.66 mg/kg/week given subcutaneously once weekly

Interventions

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Genotropin

Genotropin (dose \[mg\] at time of enrollment) given subcutaneously once daily

Intervention Type DRUG

somatrogon

0.66 mg/kg/week given subcutaneously once weekly

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Children aged 3 years old and \<18 years with either isolated GHD, or GH insufficiency.
2. Currently on treatment with either Genotropin Pen®, Genotropin GoQuick Pen®, HumatroPen® (United States of America \[USA\] only), or Omnitrope® Pen (USA only) ≥3 months and have been compliant on a stable dose (±10%) for at least 3 months prior to screening.
3. IGF I SDS \< 2.
4. Subjects on hormonal replacement therapy for other hypothalamic pituitary axis (HPA) hormonal deficiencies and/or diabetes insipidus must be on an optimized and stable treatment regimen, as determined by the Investigator, for at least 3 months prior to screening.

Exclusion Criteria

1. History of leukemia, lymphoma, sarcoma or any other cancer.
2. History of radiation therapy or chemotherapy.
3. Children with psychosocial dwarfism.
4. Children born small for gestational age (SGA) - birth weight and/or birth length \< 2 SDS for gestational age.
5. Other causes of short stature such as uncontrolled primary hypothyroidism and rickets.
6. Chromosomal abnormalities including Turner's syndrome, Laron syndrome, Noonan syndrome, Prader Willi syndrome, Russell Silver syndrome, short stature homeobox (SHOX) mutations/deletions or skeletal dysplasias.
7. Treatment with regularly scheduled daily or weekly injectable medications other than Genotropin® Pen, Genotropin GoQuick®, HumatroPen® (USA only), or Omnitrope® Pen (USA only).
8. Diabetes Mellitus.
9. Current treatment with Genotropin MiniQuick.
10. History of any exposure to a long acting hGH preparation.
11. Known or suspected human immunodeficiency virus (HIV) positive patient, or patient with advanced diseases such as acquired immunodeficiency syndrome (AIDS) or tuberculosis.
12. Drug, substance, or alcohol abuse.
13. Known hypersensitivity to the components of the medication.
14. Pregnant female subjects; breastfeeding female subjects; fertile male subjects and female subjects of childbearing potential who are unwilling or unable to use a highly effective method of contraception as outlined in this protocol for the duration of the study and for at least 28 days after the last dose of investigational product.
15. Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.
16. Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or subjects who are Pfizer employees, including their family members, directly involved in the conduct of the study.
17. Participation in other studies involving investigational drug(s) within 30 days prior to study entry and/or during study participation.
18. Patient and/or the parent/legal guardian are likely to be non-compliant with respect to study conduct.
19. Subject and/or the parent/legal guardian are unable to understand written and/or verbal instructions on the proper use of growth hormone injection devices.
20. Children with closed epiphyses (this determination can be based on available existing clinical data).
Minimum Eligible Age

3 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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Center of Excellence in Diabetes and Endocrinology

Sacramento, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Rocky Mountain Pediatric Endocrinology

Centennial, Colorado, United States

Site Status

Pediatric Endocrine Associates, PC

Greenwood Village, Colorado, United States

Site Status

Nemours Children's Health System

Jacksonville, Florida, United States

Site Status

Nemours Children's Specialty Care

Jacksonville, Florida, United States

Site Status

Nemours Biomedical Research

Orlando, Florida, United States

Site Status

Nemours Children's Hospital

Orlando, Florida, United States

Site Status

Nemours Children's Clinic

Pensacola, Florida, United States

Site Status

Shriners Hospitals for Children

Tampa, Florida, United States

Site Status

Emory Children's Center

Atlanta, Georgia, United States

Site Status

IU Health Pharmacy

Indianapolis, Indiana, United States

Site Status

Riley Hospital for Children

Indianapolis, Indiana, United States

Site Status

Children's Mercy Hospital and Clinics

Kansas City, Missouri, United States

Site Status

Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

MultiCare Health System - Mary Bridge Children's Health Center

Tacoma, Washington, United States

Site Status

MultiCare Institute for Research & Innovation

Tacoma, Washington, United States

Site Status

First Pediatric Clinic UMHAT "St. Marina" EAD

Varna, , Bulgaria

Site Status

Fakultni nemocnice Brno, Pediatricka Klinika

Brno, , Czechia

Site Status

Nemocnicni lekarna FN Brno

Brno, , Czechia

Site Status

Fakultni nemocnice v Motole, Pediatricka klinika 2.LF UK a FN Motol

Prague, , Czechia

Site Status

Nemocnicni lekarna FN Motol

Prague, , Czechia

Site Status

Nemocnicna Lekaren Nudch

Bratislava, , Slovakia

Site Status

Národný ústav detských chorôb, Detská klinika

Bratislava, , Slovakia

Site Status

Detská fakultná nemocnica Košice Klinika detí a dorastu LF UPJŠ a DFN

Košice, , Slovakia

Site Status

Nemocnica lekaren DFN Kosice, Klinika deti a dorastu

Košice, , Slovakia

Site Status

St. Georges University Hospitals NHS Foundation Trust

London, , United Kingdom

Site Status

St. Georges University Hospital NHS Foundation Trust

London, , United Kingdom

Site Status

The Institute of Child Health, University College London

London, , United Kingdom

Site Status

Great Ormond Street Hospital

London, , United Kingdom

Site Status

NIHR Clinical Research Facility, Great Ormond Street Hospital for Children NHS Trust

London, , United Kingdom

Site Status

Countries

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United States Bulgaria Czechia Slovakia United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://pmiform.com/clinical-trial-info-request?StudyID=C0311002

To obtain contact information for a study center near you, click here.

Other Identifiers

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2018-000918-38

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

C0311002

Identifier Type: -

Identifier Source: org_study_id