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临床试验/NL-OMON48505
NL-OMON48505尚未招募3 期

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 个研究点目标入组 64 人开始时间: 待定最近更新:

试验速览

阶段
3 期
状态
尚未招募
发起方
入组人数
64

研究概览

简要总结

暂无简介。

研究设计

研究类型
Interventional

入排标准

年龄范围
2 至 17(—)

入选标准

  • - 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.

排除标准

  • - 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

研究者

发起方
Stichting Kind en Groei

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