Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT06295341 |
| Other study ID # |
01C312 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
May 10, 2023 |
| Est. completion date |
December 31, 2024 |
Study information
| Verified date |
February 2024 |
| Source |
Istituto Auxologico Italiano |
| Contact |
Alessandro Sartorio |
| Phone |
+390261911 |
| Email |
sartorio[@]auxologico.it |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
The first aim of the present study is to evaluate the psychological impact of the condition
of short stature (family) in a sample of Italian children, comparing them with subjects of
normal stature, measuring their levels of psychological well-being, psychological distress,
quality of health-related life and any behavioral issues. The secondary objective is to study
the psychological impact evaluated with the tests described below (see methods section) in
children with GH deficiency and the effects of replacement therapy (6 months) with GH from
recombinant DNA.
Description:
The observational study carried out at the Centre for growth and pubertal Disorders, Istituto
Auxologico Italiano, IRCCS, Milan, consists of two sub-projects that involve the recruitment
of:
- 70 children, aged between 6 and 14 years, of both sexes: 35 with familiar short stature
(height < 3rd centile according to the Italian reference standards) and 35 with normal
height (height > 25th centile), of normal weight, and their caregivers of reference.
Children with familial short stature will be characterized by: short stature in other
members of the family group, not necessarily the parents, harmonious appearance, without
particular clinical signs and normal pubertal development, parallel growth curve below
the 3rd centile, bone age corresponding to chronological age. Children and teenagers
with obesity (BMI > 97th centile) will be excluded
- 10 children, aged between 6 and 14 years, of both sexes, affected by isolated GH
deficiency according to the criteria established by AIFA note 39 for this pathology
(short stature: ≤ -3 SD or ≤ -2 SD and growth velocity/year ≤ -1.0 SD for age and sex
evaluated at least 6 months apart and peak GH at two different pharmacological stimulus
tests < 8 ng/ml). The exclusion criterion from the present study (and from treatment
with rhGH) will be the presence of organic pathologies at the hypothalamic-pituitary
level (assessed by performing brain MRI).
Children with GH deficiency will be evaluated in baseline conditions and after 6 months of
therapy with recombinant DNA GH (at a dose of 0.025-0.035 mg/kg of body weight per day (or
0.7-1.0 mg/m2 of body surface area per day).
The following variables will be investigated in the recruited subjects of the two subgroups:
- socio-demographic conditions (gender, age, provenance, level of education, family
composition)
- psychological well-being through the Psychological Well-Being Scales (PWB), a
self-administered questionnaire consisting of 18 items that investigates the degree of
psychological well-being by exploring six dimensions: self-acceptance, positive
relationships with others, autonomy, environmental control, personal growth, and purpose
of life. The questionnaire consists of 18 items on a 4-step Likert scale.
- psychological distress through the Depression Anxiety Stress Scale (DASS-21), a 21-item
self-report tool that measures various negative internal states: depression, anxiety,
and stress.
- quality of life through the Quality of Life in Short Stature Youth (QoLISSY), a 50-item
domain-specific questionnaire that measures health-related quality of life in children
with short stature.
- skills and problems through the Strengths and Difficulties Questionnaire (SDQ), a
questionnaire made up of 25 items able to assess emotional symptoms, behavioral
problems, hyperactivity/inattention, problematic relationships with peers, and prosocial
behavior.
- behavioral problems through the Child Behaviour Checklist for Children (CBCL), a
questionnaire to be administered to parents or caregivers of children who are able to
detect any behavioral problems. The instrument consists of 113 items that investigate
three general scales: total problems, internalizing problems, and externalizing problems
and 8 syndromic scales: social withdrawal, somatic complaints, anxiety, depression,
social problems, thinking problems, attention problems, antisocial behavior, and
aggressive behavior).
