NL-OMON48505Not yet recruitingPhase 3
Randomized, controlled growth hormone study in children and adolescents with a Prader-Willi-like phenotype, followed by a two year open-label growth hormone study - effects on body composition, growth, psychosocial development and quality of life - Prader-Willi-like (PWL) study
Stichting Kind en Groei0 sites64 target enrollmentStarted: TBDLast updated:
Trial Snapshot
- Phase
- Phase 3
- Status
- Not yet recruiting
- Sponsor
- Enrollment
- 64
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional
Eligibility Criteria
- Ages
- 2 to 17 (—)
Inclusion Criteria
- •- Prader-Willi-like phenotype, according to the PWL criteria (see below), or
- •with a proven molecular diagnosis of a uniparental disomy of chromosome 14
- •(mUPD14), or a mutation or duplication in the 15q11.2-q13 region (PWS-critical
- •- Absence of a molecular diagnosis of PWS. This includes a type 1 (from
- •breakpoint 1 to breakpoint 4) or type 2 (from breakpoint 2 to breakpoint 4)
- •deletion, a uniparental disomy or imprinting center defect of the 15q11.2-q13
- •o Boys: 4 to 16 years
- •o Girls: 4 to 14 years.
- •- Written informed consent. , PWL criteria:
- •The PWL phenotype is present in case of the following symptoms:
- •Hyperphagia and/or rapid weight gain between the ages of 1 and 8 years
- •Developmental/psychomotor delay (IQ<85 and/or enrolment in a school for
- •children with special needs)
- •One or more of the following symptoms:
- •o Infantile central hypotonia
- •o Behavioural problems (temper tantrums, (auto)aggression, autistic and/or
- •obsessive compulsive behaviour
- •o Poor speech
- •o Vision abnormalities (e.g. myopia, esotropia)
- •o Short stature (as defined by < -2SD-score, or -2SD relative to the target
- •o Small hands and feet for height and/or age.
- •N.B. Patients diagnosed with UPD14 or a mutation/duplication in the 15q11.2
- •(Prader-Willi critical) region do not have to meet the PWL-criteria.
Exclusion Criteria
- •- Non-cooperative behavior
- •- Extremely low dietary intake of less than the minimally required intake
- •according to WHO
- •- Use of medication to reduce weight
- •- BMI > +4SD
- •- Height velocity <1 cm per year and/or closed epiphyseal growth plates, as
- •determined by an x-ray of the hand
- •- Pregnancy
Investigators
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