Clinical trial NCT03831880: Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD
Official title: A PHASE 3, RANDOMIZED, MULTICENTER, OPEN-LABEL, CROSSOVER STUDY ASSESSING SUBJECT PERCEPTION OF TREATMENT BURDEN WITH USE OF WEEKLY GROWTH HORMONE (SOMATROGON) VERSUS DAILY GROWTH HORMONE (GENOTROPIN (REGISTERED)) INJECTIONS IN CHILDREN WITH GROWTH HORMONE DEFICIENCY
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Growth Hormone Deficiency
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2019-02-06
Enrollment: 87
Sex: ALL
Minimum age (years): 3
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: OTHER

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.

Detailed description: 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.

Eligibility criteria: 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).

Primary outcomes:
measure: Total Score Related to Overall Life Interference Assessed at Baseline, Using Dyad Clinical Outcomes Assessment 1 (DCOA 1) Questionnaire
timeFrame: Baseline
measure: Total Score Related to Overall Life Interference Assessed at Week 12, Using DCOA 1 Questionnaire
timeFrame: Week 12
measure: Total Score Related to Overall Life Interference Assessed at Week 24, Using DCOA 1 Questionnaire
timeFrame: Week 24
measure: Total Score Related to Overall Life Interference by Treatment in Overall Study, Using DCOA 1 Questionnaire
timeFrame: Baseline up to Week 24
Secondary outcomes:
measure: Total Score Related to Pen Ease of Use Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
timeFrame: Baseline, Week 12, Week 24
measure: Total Score Related to Pen Ease of Use by Treatment in Overall Study, Using DCOA 1 Questionnaire
timeFrame: Baseline up to Week 24
measure: Total Score Related to Ease of the Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
timeFrame: Baseline, Week 12, Week 24
measure: Total Score Related to Ease of the Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire
timeFrame: Baseline up to Week 24
measure: Total Score Related to Convenience of the Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
timeFrame: Baseline, Week 12, Week 24
measure: Total Score Related to Convenience of the Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire
timeFrame: Baseline up to Week 24
measure: Total Score Related to Satisfaction With Overall Treatment Experience Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
timeFrame: Baseline, Week 12, Week 24
measure: Total Score Related to Satisfaction With Overall Treatment Experience by Treatment in Overall Study, Using DCOA 1 Questionnaire
timeFrame: Baseline up to Week 24
measure: Total Scores Related to Willingness to Continue Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
timeFrame: Baseline, Week 12, Week 24
measure: Total Scores Related to Willingness to Continue Injection Schedule by Treatment in Overall Study, Using DCOA 1 Questionnaire
timeFrame: Baseline up to Week 24
measure: Total Scores Related to Injection Signs and Symptoms for Participants Aged 8 Years and Above Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
timeFrame: Baseline, Week 12, Week 24
measure: Total Scores Related to Injection Signs and Symptoms for Participants Aged 8 Years and Above by Treatment in Overall Study, Using DCOA 1 Questionnaire
timeFrame: Baseline up to Week 24
measure: Total Scores Related to Assessment of Signs, Completed by Caregiver for Children Aged <8 Years Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
timeFrame: Baseline, Week 12, Week 24
measure: Total Scores Related to Assessment of Signs, Completed by Caregiver for Children Aged <8 Years by Treatment in Overall Study, Using DCOA 1 Questionnaire
timeFrame: Baseline up to Week 24
measure: Total Scores Related to Caregiver Life Interference, Including Family Life Interference Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
timeFrame: Baseline, Week 12, Week 24
measure: Total Scores Related to Caregiver Life Interference, Including Family Life Interference by Treatment in Overall Study, Using DCOA 1 Questionnaire
timeFrame: Baseline up to Week 24
measure: Total Scores Related to Missed Injections Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
timeFrame: Baseline, Week 12, Week 24
measure: Total Scores Related to Missed Injections by Treatment in Overall Study, Using DCOA 1 Questionnaire
timeFrame: Baseline up to Week 24
measure: Number of Participants as Per Responses to Choice of Injection Pen Assessed at Week 24, Using DCOA 2 Questionnaire
timeFrame: Week 24
measure: Number of Participants as Per Responses to Preferred Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire
timeFrame: Week 24
measure: Number of Participants as Per Responses to Convenience of the Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire
timeFrame: Week 24
measure: Number of Participants as Per Responses to Ease of Following Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire
timeFrame: Week 24
measure: Number of Participants as Per Responses to Pen Ease of Use Assessed at Week 24, Using DCOA 2 Questionnaire
timeFrame: Week 24
measure: Number of Participants as Per Responses to Participant Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire
timeFrame: Week 24
measure: Number of Participants as Per Responses to Caregiver Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire
timeFrame: Week 24
measure: Number of Participants as Per Responses to Family Life Interference Assessed at Week 24, Using DCOA 2 Questionnaire
timeFrame: Week 24
measure: Number of Participants as Per Response to Benefit Relating to the Injection Schedule Assessed at Week 24, Using DCOA 2 Questionnaire
timeFrame: Week 24
measure: Number of Participants as Per Responses to Intention to Comply Assessed at Week 24, Using DCOA 2 Questionnaire
timeFrame: Week 24
measure: Patient Global Impression Severity-Impact on Daily Activities (PGIS-IDA) Score Assessed at Baseline, Week 12 and Week 24
timeFrame: Baseline, Week 12, Week 24
measure: Patient Global Impression Severity-Impact on Daily Activities (PGIS-IDA) Score by Treatment in Overall Study
timeFrame: Baseline up to Week 24
Other outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), Treatment-Emergent Treatment Related AEs and SAEs
timeFrame: Baseline up to 35 days after last dose of study drug (up to 29 Weeks)
measure: Number of Participants With Adverse Events According to Severity
timeFrame: Baseline up to 35 days after last dose of study drug (up to 29 Weeks)
measure: Number of Participants With Discontinuation Due to Adverse Events (AEs)
timeFrame: Baseline up to 35 days after last dose of study drug (up to 29 Weeks)
measure: Number of Participants With Laboratory Abnormalities
timeFrame: Week 1 to Week 12, Week 13 to Week 24
measure: Number of Participants With Positive Anti-Recombinant Human Growth Hormone (rhGH) Antibodies and Neutralizing Antibodies (NAb)
timeFrame: Baseline, Week 12, Week 24
measure: Number of Participants With Positive Anti-Somatrogon Antibodies and Neutralizing Antibodies (NAb)
timeFrame: Baseline, Week 12, Week 24
Locations:
city: Sacramento
country: United States
facility: Center of Excellence in Diabetes and Endocrinology
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Centennial
country: United States
facility: Rocky Mountain Pediatric Endocrinology
state: Colorado
city: Greenwood Village
country: United States
facility: Pediatric Endocrine Associates, PC
state: Colorado
city: Jacksonville
country: United States
facility: Nemours Children's Health System
state: Florida
city: Jacksonville
country: United States
facility: Nemours Children's Specialty Care
state: Florida
city: Orlando
country: United States
facility: Nemours Biomedical Research
state: Florida
city: Orlando
country: United States
facility: Nemours Children's Hospital
state: Florida
city: Pensacola
country: United States
facility: Nemours Children's Clinic
state: Florida
city: Tampa
country: United States
facility: Shriners Hospitals for Children
state: Florida
city: Atlanta
country: United States
facility: Emory Children's Center
state: Georgia
city: Indianapolis
country: United States
facility: IU Health Pharmacy
state: Indiana
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Kansas City
country: United States
facility: Children's Mercy Hospital and Clinics
state: Missouri
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
city: Tacoma
country: United States
facility: MultiCare Health System - Mary Bridge Children's Health Center
state: Washington
city: Tacoma
country: United States
facility: MultiCare Institute for Research & Innovation
state: Washington
city: Varna
country: Bulgaria
facility: First Pediatric Clinic UMHAT "St. Marina" EAD
city: Brno
country: Czechia
facility: Fakultni nemocnice Brno, Pediatricka Klinika
city: Brno
country: Czechia
facility: Nemocnicni lekarna FN Brno
city: Praha 5
country: Czechia
facility: Fakultni nemocnice v Motole, Pediatricka klinika 2.LF UK a FN Motol
city: Praha 5
country: Czechia
facility: Nemocnicni lekarna FN Motol
city: Bratislava
country: Slovakia
facility: Nemocnicna Lekaren Nudch
city: Bratislava
country: Slovakia
facility: Národný ústav detských chorôb, Detská klinika
city: Kosice
country: Slovakia
facility: Detská fakultná nemocnica Košice Klinika detí a dorastu LF UPJŠ a DFN
city: Kosice
country: Slovakia
facility: Nemocnica lekaren DFN Kosice, Klinika deti a dorastu
city: London
country: United Kingdom
facility: St. Georges University Hospitals NHS Foundation Trust
city: London
country: United Kingdom
facility: St. Georges University Hospital NHS Foundation Trust
city: London
country: United Kingdom
facility: The Institute of Child Health, University College London
city: London
country: United Kingdom
facility: Great Ormond Street Hospital
city: London
country: United Kingdom
facility: NIHR Clinical Research Facility, Great Ormond Street Hospital for Children NHS Trust
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: