Clinical trial NCT03672617: Validation of a Questionnaire That Identifies the Reasons for Non-adherence to Existing Growth Hormone Therapy
Official title: Validation of a Patient-reported Outcome (PRO) Measure That Assesses Reasons for Non-adherence to Growth Hormone Therapy (GHT)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Growth Hormone Deficiency in Children
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2018-09-14
Enrollment: 321
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Three hundred (300) participants at about 30 sites for paediatric endocrinology in Germany will be included in this validation study.

Brief summary: The aim of the study is to examine the suitability, the so-called validation, of a questionnaire, with which one can grasp the reasons why injections of growth hormones are omitted by patients ("non-adherence"). Participants are treated with growth hormone and are therefore eligible to take part in the study. Study doctor will ask participants to answer questionnaires. two times within 14 days. The first time participants answer during the routine visit to the practice / clinic and the second time at home. There are no risks associated with participating in the study as it does not affect participant's medical treatment.


Eligibility criteria: Inclusion Criteria: - Children/adolescents, male or female, aged 8 to 18 years who self-inject GH as well as their parents/legal guardians or Parents/Legal guardians who administer GH to a child/adolescent, male or female, from birth to 18 years - The child/adolescent is on prescribed daily GHT due to idiopathic growth hormone deficiency (GHD), multiple pituitary hormone deficiency (MPHD/organic GHD), small for gestational age (SGA) or Turner syndrome (TS) for at least 6 months before screening visit - Signed informed consent obtained by parent/legal guardian and child/adolescent before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol) Exclusion Criteria: - Mental incapacity, unwillingness or language barrier of participant precluding adequate understanding or cooperation - Previous participation of participant in this study. Participation is defined as having given informed consent in this study - Treatment of child/adolescent with any investigational drug within 30 days prior to enrolment into the study

Primary outcomes:
measure: Validity of the Growth Hormone Compliance Questionnaire child/adolescent version (GHCQ-CA)
timeFrame: Day 1
measure: Validity of the GHCQ-CA
timeFrame: Day 14
measure: Reliability of the GHCQ-CA
timeFrame: Day 1
measure: Reliability of the GHCQ-CA
timeFrame: Day 14
measure: Validity of the Growth Hormone Compliance Questionnaire parent version (GHCQ-P)
timeFrame: Day 1
measure: Validity of the GHCQ-P
timeFrame: Day 14
measure: Reliability of the GHCQ-P
timeFrame: Day 1
measure: Reliability of the GHCQ-P
timeFrame: Day 14
Secondary outcomes:
Other outcomes:
Locations:
city: Mainz
country: Germany
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Reporting Anchor and Disclosure (1452)
role: STUDY_DIRECTOR
References:
citation: de Zwaan M, Fischer-Jacobs J, Wabitsch M, Reinehr T, Meckes-Ferber S, Crosby RD. Development and Psychometric Evaluation of an Instrument Assessing Barriers to Growth Hormone Treatment (BAR-GHT). Front Endocrinol (Lausanne). 2020 Feb 25;11:84. doi: 10.3389/fendo.2020.00084. eCollection 2020.