To evaluate the effects of N-acetylcysteine (NAC; Fluimucil® in Dutch) on skin picking behaviour in children and young adults with PWS.
ID
Source
Brief title
Condition
- Chromosomal abnormalities, gene alterations and gene variants
- Hypothalamus and pituitary gland disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Change in number and size of the skin picking lesions.
Secondary outcome
Change in repetitive/compulsive behaviour, measured by:
• Repetitive Behaviour Scale (RBS)
• Skin Picking-Symptom Assessment Scale (SP-SAS)
• Clinical Global Impression Scale (CGI)
Change in quality of life, measured by:
• Pediatric Quality of Life Inventory (PedsQL)
Change in cortisol concentration in hair samples.
Change in safety parameters (laboratory parameters and medical assessments).
Background summary
Patients with PWS suffer from behavioural problems in addition to their
physical abnormalities. The onset of these behavioural problems is during
childhood. Children with PWS might present with significant maladaptive
behavioural and emotional characteristics including temper tantrums,
inappropriate social behaviour and self-injurious behaviour. Skin picking is
the most common form of self-injurious behaviour in patients with PWS and can
lead to severe wounds and infections. Several studies demonstrated that
N-acetylcysteine is a promising treatment for patients with problems in
obsessive- compulsive spectrum. N-acetylcysteine restores the extracellular
glutamate concentration in the nucleus accumbens and this mechanism might play
a role in the prevention of compulsive behaviours. An open label pilot study
showed that N-acetylcysteine treatment in patients with PWS showed a reduction
in skin-picking behaviour. Therefore, the aim of this study is to investigate
the effect of N-acetylcysteine versus placebo in children and young adults with
PWS who are suffering from skin picking behaviour.
Study objective
To evaluate the effects of N-acetylcysteine (NAC; Fluimucil® in Dutch) on skin
picking behaviour in children and young adults with PWS.
Study design
A randomized, doubleblind, placebo-controlled, cross-over trial: daily dose of
n-acetylcysteine versus placebo for three months each. With a washout period of
three months between cross-over.
Intervention
Daily oral administration of acetylcysteine or placebo:
<30kg:
4 weeks: 600mg
8 weeks: 1200mg
>30kg < 59.99 kg:
4 weeks 600mg
4 weeks 1200mg
4 weeks 1800mg
>60kg:
4 weeks 600mg
4 weeks 1200mg
4 weeks 2400mg
Study burden and risks
Burden: once a day oral intake of tablet(s) for two periods of 3 months. During
this time there will be maximal 5 outpatient hospital visits for the
measurements of the skin picking lesions and the body locations of the lesions.
Four of the five hospital visits will include blood sample draw, all the visits
include blood pressure and heart rate measurement.
Risks: Based on literature data, there are no severe side effects or adverse
events expected. The most common side effects of NAC, which appear in 0,01-1%
of patients are hypersensitivity reactions to NAC, like itching, skin rash,
headache and mild gastrointestinal complaints like nausea, cramps, diarrhoea,
flatulence. The patients and their parents are expected to be very motivated to
participate in this study, considering the negative impact of their
skin-picking behaviour on the quality of life of the patients and family.
Westzeedijk 106
Rotterdam 3016AH
NL
Westzeedijk 106
Rotterdam 3016AH
NL
Listed location countries
Age
Inclusion criteria
- Prader-Willi syndrome (genetically proven)
- Age between 6 - 25 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 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).
Exclusion criteria
- Severe psychiatric problems, as active psychosis or behavioural problems that
hinder study participation.
- 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
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2019-000735-61-NL |
| CCMO | NL74197.078.20 |