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

NCT03831880 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: C03110022018-000918-38
• Study Type: interventional
• Target: 87
• Locations: 5 countries
• Sites: 32

Brief Summary

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.

Conditions

Growth Hormone Deficiency

Outcome Measures

Primary Outcomes (4)

• Total Score Related to Overall Life Interference Assessed at Baseline, Using Dyad Clinical Outcomes Assessment 1 (DCOA 1) Questionnaire

  Participants were assessed for their treatment burden using DCOA 1 questionnaire completed by participant/caregiver dyads. The participant life interference questionnaire component of the DCOA 1 had 7 questions (life interference \[5 questions\]: a measure of life interference \[daily activities/social activities/leisure/night away from home/travel\]; life interference-changes to life routine \[1 question\]: a measure of how often changes are made to life routine; and life interference-bother of growth hormone \[GH\] injections \[1 question\]: a measure of how often the growth hormone injections cause bother) and all questions used a 5-point scale: 1= never, 2= rarely, 3= sometimes, 4= often, 5= always. The overall life interference total score was sum of all 7 questions, scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant less life interference (better outcome).

  Baseline

• Total Score Related to Overall Life Interference Assessed at Week 12, Using DCOA 1 Questionnaire

  Participants were assessed for their treatment burden using DCOA 1 questionnaire completed by participant/caregiver dyads. The participant life interference questionnaire component of the DCOA 1 had 7 questions (life interference \[5 questions\]: a measure of life interference \[daily activities/social activities/leisure/night away from home/travel\]; life interference-changes to life routine \[1 question\]: a measure of how often changes are made to life routine; and life interference-bother of growth hormone \[GH\] injections \[1 question\]: a measure of how often the growth hormone injections cause bother) and all questions used a 5-point scale: 1= never, 2= rarely, 3= sometimes, 4= often, 5= always. The overall life interference total score was sum of all 7 questions, scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant less life interference (better outcome).

  Week 12

• Total Score Related to Overall Life Interference Assessed at Week 24, Using DCOA 1 Questionnaire

  Participants were assessed for their treatment burden using DCOA 1 questionnaire completed by participant/caregiver dyads. The participant life interference questionnaire component of the DCOA 1 had 7 questions (life interference \[5 questions\]: a measure of life interference \[daily activities/social activities/leisure/night away from home/travel\]; life interference-changes to life routine \[1 question\]: a measure of how often changes are made to life routine; and life interference-bother of growth hormone \[GH\] injections \[1 question\]: a measure of how often the growth hormone injections cause bother) and all questions used a 5-point scale: 1= never, 2= rarely, 3= sometimes, 4= often, 5= always. The overall life interference total score was sum of all 7 questions, scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant less life interference (better outcome).

  Week 24

• Total Score Related to Overall Life Interference by Treatment in Overall Study, Using DCOA 1 Questionnaire

  Participants were assessed for their treatment burden using DCOA 1 questionnaire completed by participant/caregiver dyads. The participant life interference questionnaire component of the DCOA 1 had 7 questions (life interference \[5 questions\]: a measure of life interference \[daily activities/social activities/leisure/night away from home/travel\]; life interference-changes to life routine \[1 question\]: a measure of how often changes are made to life routine; and life interference-bother of growth hormone \[GH\] injections \[1 question\]: a measure of how often the growth hormone injections cause bother) and all questions used a 5-point scale: 1= never, 2= rarely, 3= sometimes, 4= often, 5= always. The overall life interference total score was sum of all 7 questions, scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant less life interference (better outcome).

  Baseline up to Week 24

Secondary Outcomes (30)

• Total Score Related to Pen Ease of Use Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire

  Baseline, Week 12, Week 24

• Total Score Related to Pen Ease of Use by Treatment in Overall Study, Using DCOA 1 Questionnaire

  Baseline up to Week 24

• Total Score Related to Ease of the Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire

  Baseline, Week 12, Week 24

• Total Score Related to Ease of the Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire

  Baseline up to Week 24

• Total Score Related to Convenience of the Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire

  Baseline, Week 12, Week 24

Other Outcomes (6)

• Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), Treatment-Emergent Treatment Related AEs and SAEs

  Baseline up to 35 days after last dose of study drug (up to 29 Weeks)

• Number of Participants With Adverse Events According to Severity

  Baseline up to 35 days after last dose of study drug (up to 29 Weeks)

• Number of Participants With Discontinuation Due to Adverse Events (AEs)

  Baseline up to 35 days after last dose of study drug (up to 29 Weeks)

Study Arms (2)

Daily to Weekly

OTHER

Genotropin to somatrogon

Drug: Genotropin; Drug: somatrogon

Weekly to Daily

OTHER

somatrogon to Genotropin

Drug: Genotropin; Drug: somatrogon

Interventions

Genotropin DRUG

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

Daily to Weekly; Weekly to Daily

somatrogon DRUG

0.66 mg/kg/week given subcutaneously once weekly

Daily to Weekly; Weekly to Daily

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Children aged 3 years old and \<18 years with either isolated GHD, or GH insufficiency.
• 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.
• IGF I SDS \< 2.
• 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.

You may not qualify if:

• History of leukemia, lymphoma, sarcoma or any other cancer.
• History of radiation therapy or chemotherapy.
• Children with psychosocial dwarfism.
• Children born small for gestational age (SGA) - birth weight and/or birth length \< 2 SDS for gestational age.
• Other causes of short stature such as uncontrolled primary hypothyroidism and rickets.
• 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.
• Treatment with regularly scheduled daily or weekly injectable medications other than Genotropin® Pen, Genotropin GoQuick®, HumatroPen® (USA only), or Omnitrope® Pen (USA only).
• Diabetes Mellitus.
• Current treatment with Genotropin MiniQuick.
• History of any exposure to a long acting hGH preparation.
• Known or suspected human immunodeficiency virus (HIV) positive patient, or patient with advanced diseases such as acquired immunodeficiency syndrome (AIDS) or tuberculosis.
• Drug, substance, or alcohol abuse.
• Known hypersensitivity to the components of the medication.
• 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.
• 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.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (32)

Center of Excellence in Diabetes and Endocrinology

Sacramento, California, 95821, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Rocky Mountain Pediatric Endocrinology

Centennial, Colorado, 80112, United States

Location

Pediatric Endocrine Associates, PC

Greenwood Village, Colorado, 80111, United States

Location

Nemours Children's Health System

Jacksonville, Florida, 32207, United States

Location

Nemours Children's Specialty Care

Jacksonville, Florida, 32207, United States

Location

Nemours Biomedical Research

Orlando, Florida, 32827, United States

Location

Nemours Children's Hospital

Orlando, Florida, 32827, United States

Location

Nemours Children's Clinic

Pensacola, Florida, 32514, United States

Location

Shriners Hospitals for Children

Tampa, Florida, 33612, United States

Location

Emory Children's Center

Atlanta, Georgia, 30322, United States

Location

IU Health Pharmacy

Indianapolis, Indiana, 46202, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Children's Mercy Hospital and Clinics

Kansas City, Missouri, 64111, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

MultiCare Health System - Mary Bridge Children's Health Center

Tacoma, Washington, 98405, United States

Location

MultiCare Institute for Research & Innovation

Tacoma, Washington, 98405, United States

Location

First Pediatric Clinic UMHAT "St. Marina" EAD

Varna, 9010, Bulgaria

Location

Fakultni nemocnice Brno, Pediatricka Klinika

Brno, 61300, Czechia

Location

Nemocnicni lekarna FN Brno

Brno, 61300, Czechia

Location

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

Prague, 150 06, Czechia

Location

Nemocnicni lekarna FN Motol

Prague, 150 06, Czechia

Location

Nemocnicna Lekaren Nudch

Bratislava, 833 40, Slovakia

Location

Národný ústav detských chorôb, Detská klinika

Bratislava, 833 40, Slovakia

Location

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

Košice, 040 11, Slovakia

Location

Nemocnica lekaren DFN Kosice, Klinika deti a dorastu

Košice, 040 11, Slovakia

Location

St. Georges University Hospitals NHS Foundation Trust

London, SW17 0QT, United Kingdom

Location

St. Georges University Hospital NHS Foundation Trust

London, SW17 0RE, United Kingdom

Location

The Institute of Child Health, University College London

London, WC1N 1EH, United Kingdom

Location

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

Location

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

London, WC1N 3JH, United Kingdom

Location

Related Links

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

MeSH Terms

Conditions

Dwarfism, Pituitary

Interventions

Human Growth Hormone; somatrogon

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2018
• First Posted: February 6, 2019
• Study Start: February 7, 2019
• Primary Completion: August 28, 2020
• Study Completion: August 28, 2020
• Last Updated: October 14, 2021
• Results First Posted: October 14, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will share

Locations

SL (4); GB (5); BU (1); CZ (4); US (18)