Prader-Willi syndrome MedDRA version: 20.0 Level: PT Classification code 10036476 Term: Prader-Willi syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Fluimucil (acetylcysteine)
Product Name: Fluimucil
Product Code: R05CB01
Pharmaceutical Form: Effervescent tablet
Sponsors
Dutch Growth Research Foundation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: To be eligible to participate in this study, a subject must meet all the following criteria: - Prader-Willi syndrome (genetically proven) - Age between 6 - 24.99 years - Skin picking behaviour for at least one year, according the following DSM-5 criteria: 1. Repeated picking of the skin and damaging it. 2. Multiple attempts to stop the behaviour. 3. Picking of the skin provokes significant stress or limitations in social, professional functioning, or limits other important areas of functioning. 4. Picking of the skin is not attributed to the direct effect of drugs (e.g. cocaine) or to any other medical condition (e.g. scabies). 5. Picking of the skin is not explained by another mental disorder (e.g. psychosis or body dysmorphic disorder). Are the trial subjects under 18? yes Number of subjects for this age range: 15 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: A potential subject who meets any of the following criteria will be excluded from participation in this study: - Severe psychiatric problems. - Previous treatment with N-acetylcysteine in the last 3 months. - Current or recent (past 3 months) DSM-5 substance abuse or dependence. - Initiation of pharmacotherapy, psychotherapy, or behaviour therapy from a mental health perspective in the last 3 months. - Treatment with investigational medication or depot neuroleptics within 3 months. - Need for medication other than NAC with possible psychotropic effects or unfavourable interactions with NAC. - Asthma (given the possible worsening). - Active peptic ulcer considering the possible gastrointestinal- and vomiting side effects.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To investigate the effects of N-acetylcysteine treatment on skin picking behavior, measured by the change in total number and size of the skin picking laesions in children and young adults with PWS;Secondary Objective: To investigate the effects of N-acetylcysteine treatment in children and young adults with PWS on: - Other repetitive compulsive behaviour, e.g. nail biting and repeating or sorting and organizing behaviour - Cortisol levels, measured by hair cortisol levels - Quality of life ;Primary end point(s): The main study parameter will be the difference of the changes in number and size of the skin picking lesions, assessed by counting and measuring the lesions in cm. ;Timepoint(s) of evaluation of this end point: At start, 3 months, 6 months and 9 months. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Change in: - Skin picking behavior, measured with the Skin Picking Symptom Assessment Scale - Repetitive compulsive behaviour, measured by the Repetitive Behaviour Scale questionnaire - Hair cortisol levels - Quality of life, measured with the Pediatric Quality of Life Inventory - Safety parameters (laboratory parameters and medical assessments);Timepoint(s) of evaluation of this end point: At start, 3 months, 6 months and 9 months. | — |
Countries
Netherlands
Contacts
Public ContactProject leader Prader-Willi Studies
Dutch Growth Research Foundation
Outcome results
None listed