Prader-Willi-like (PWL) study

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

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-004729-97-NL

Enrollment

Registered

2019-06-06

Start date

2019-08-16

Completion date

Unknown

Last updated

2019-12-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Prader-Willi-like phenotype MedDRA version: 20.0 Level: LLT Classification code 10018747 Term: Growth hormone System Organ Class: 100000004848

Interventions

Trade Name: GENOTROPIN (Somatropin) Product Name: Genotropin Product Code: H01AC01 Pharmaceutical Form: Powder and solution for solution for injection

Eligibility

Sex/Gender

All

Inclusion criteria

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 region). - 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 region. - Age: 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 AND • Developmental/psychomotor delay (IQ=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion 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

Design outcomes

Primary

Measure	Time frame
Main Objective: To investigate the effects and safety of GH treatment on body composition, motor development, growth, glucose metabolism, serum lipids, cognition, behaviour, bone mineral density, and quality of life in children with PWL. To investigate the baseline clinical characteristics and underlying (epi)genetic causes of children with PWL and their relation to the effects and safety of GH treatment.;Secondary Objective: To investigate the effects and safety of GH treatment on motor development, growth, glucose metabolism, serum lipids, cognition, behaviour, bone mineral density, and quality of life in children with PWL. To investigate the baseline clinical characteristics and underlying (epi)genetic causes of children with PWL and their relation to the effects and safety of GH treatment.;Primary end point(s): The main study parameter will be the difference of the change in body fat percentage between the treatment group and control group during the first six months after the start of the study, as assessed by the DXA scan.;Timepoint(s) of evaluation of this end point: Six months, twelve months, 24 months.	—

Measure

Time frame

Main Objective: To investigate the effects and safety of GH treatment on body composition, motor development, growth, glucose metabolism, serum lipids, cognition, behaviour, bone mineral density, and quality of life in children with PWL. To investigate the baseline clinical characteristics and underlying (epi)genetic causes of children with PWL and their relation to the effects and safety of GH treatment.;Secondary Objective: To investigate the effects and safety of GH treatment on motor development, growth, glucose metabolism, serum lipids, cognition, behaviour, bone mineral density, and quality of life in children with PWL. To investigate the baseline clinical characteristics and underlying (epi)genetic causes of children with PWL and their relation to the effects and safety of GH treatment.;Primary end point(s): The main study parameter will be the difference of the change in body fat percentage between the treatment group and control group during the first six months after the start of the study, as assessed by the DXA scan.;Timepoint(s) of evaluation of this end point: Six months, twelve months, 24 months.

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Secondary

Measure	Time frame
Secondary end point(s): At baseline: - Clinical characteristics of PWL - (Epi)genetic abnormalities found in the subjects with PWL - Baseline serum IGF-1 levels and GH peak levels during a GH stimulation test (clonidine test). A difference between the change during the first six months of the study in the treatment versus the control group, in: - Height, weight, head circumference - Resting Energy Expenditure - Lean body mass - Laboratory parameters: glucose metabolism, serum lipids, thyroid hormone levels, IGF-I and IGF binding proteins, ghrelin. - Blood pressure - Sleep-related breathing disorders. - Cognition, behaviour and social emotional development A difference between measurements at the start of the GH treatment and measurements after twelve and 24 months in all subjects: - Body fat percentage - Height, weight, head circumference, sitting height, arm span, length of foot, tibia and hand - Resting Energy Expenditure - Lean body mass - Laboratory parameters: glucose metabolism, serum lipids, thyroid hormone levels, IGF-I and IGF binding proteins, ghrelin. - Blood pressure - Sleep-related breathing disorders. - Cognition, behaviour and social emotional development;Timepoint(s) of evaluation of this end point: Six months, twelve months, 24 months.	—

Measure

Time frame

Secondary end point(s): At baseline: - Clinical characteristics of PWL - (Epi)genetic abnormalities found in the subjects with PWL - Baseline serum IGF-1 levels and GH peak levels during a GH stimulation test (clonidine test). A difference between the change during the first six months of the study in the treatment versus the control group, in: - Height, weight, head circumference - Resting Energy Expenditure - Lean body mass - Laboratory parameters: glucose metabolism, serum lipids, thyroid hormone levels, IGF-I and IGF binding proteins, ghrelin. - Blood pressure - Sleep-related breathing disorders. - Cognition, behaviour and social emotional development A difference between measurements at the start of the GH treatment and measurements after twelve and 24 months in all subjects: - Body fat percentage - Height, weight, head circumference, sitting height, arm span, length of foot, tibia and hand - Resting Energy Expenditure - Lean body mass - Laboratory parameters: glucose metabolism, serum lipids, thyroid hormone levels, IGF-I and IGF binding proteins, ghrelin. - Blood pressure - Sleep-related breathing disorders. - Cognition, behaviour and social emotional development;Timepoint(s) of evaluation of this end point: Six months, twelve months, 24 months.

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Countries

Netherlands

Contacts

Public ContactProject leader Prader-Willi-like

Dutch Growth Research Foundation

praderwilli@kindengroei.nl+31102251533

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026