None listed
Conditions
Brief summary
From one and a half years of age, (average 8yrs) most with PWS feel a chronic hunger without the capacity to feel satiated. Nutritional requirements must be established through minimal food intake and locked behavioural parameters. Independence is rare for people with PWS. It is hypothesised that ingestion of the cactus succulent Caralluma Fimbriata, may evidence apetite control on the satiety markers within this small cohort of 16 children/adolescents with PWS; utilizing a controlled, double- blind cross-over pilot study. This is the most sort after correction for those with PWS and their families.
Interventions
We propose to observe the capacity for the cactus succulent, Caralluma fimbriata extract (CFE) to relieve the sensation of persistent hunger for a small cohort of 16 children and adolescents with PWS. There will be two groups within the cross-over design. These will be called group A & group B, each comprising of 8 participants. The effect of the intervention/placebo, will be determined through daily oral supplementation with no invasive measures, over a period of four weeks, a wash out period o
We propose to observe the capacity for the cactus succulent, Caralluma fimbriata extract (CFE) to relieve the sensation of persistent hunger for a small cohort of 16 children and adolescents with PWS. There will be two groups within the cross-over design. These will be called group A & group B, each comprising of 8 participants. The effect of the intervention/placebo, will be determined through daily oral supplementation with no invasive measures, over a period of four weeks, a wash out period of two weeks will then take place, followed by the cross-over to the other intervention or placebo. Dosage of the CFE intervention will be delineated by using a dose of 1000mg per adult in 250mg capsules. Four 250mg capsules a day, daily for four weeks. Dosage will be estimated relative to each participant's weight, at 250mg per 10 kilogram of body weight. The recommended adult dose (1000mg/d) will not be exceeded. The estimated dose will be taken daily, for four weeks and will not increase during the trial period.
Sponsors
Victoria University
Study design
Allocation
Randomised controlled trial
Intervention model
Crossover
Primary purpose
Treatment
Masking
Blinded (masking used)
Eligibility
Sex/Gender
All
Age
5 Years to 17 Years
Healthy volunteers
Yes
Inclusion criteria
We will only include those with a confirmed diagnosis with any of the three types of deletion of PWS (listed above). PWS through FISH or DNA methylation analysis. All participants will be between the ages of 5 -17yrs This age group has been defined because typically the physical gross motor development has reached maturation and participants will be verbal and within the school system. This age allows for more constant parental supervision. Typical features within the inclusion criteria allow a child to function daily without medical intervention. therefore within healthy parameters. District: All recruited participants will be Australian and new Zealand citizens and will be willing to attend either the Melbourne clinical consultations at baseline, mid and post intervention period or define weight with a medical practitioner. New Zealand and interstate participants must gain paediatrition supervised anthropometric measures. Parent/Carers All participants will be under supervision. They must be within visual and auditory supervision in “all” environments; except within the parameters of natural toileting and sleep. This supervision will come form parents and carers within the home environment. They will need to speak English and be able to identify their child’s appetite behaviours. They may consult with outside carers: Teacher’s aides - if in the position of direct supervisor. Special school facilitators placed in a position of direct supervisor. Respite workers placed in a position of direct “one on one” supervision. Particpants We will include those people with PWS on Growth Hormone medication or natural therapies such as Coenzyme Q10, or fish oil. All participants will be healthy. Participants with PWS will still be considered healthy when they have any of the typical parameters defined below. Overweight within the 50 to 90 percentile Hypotonia (low muscle tone) and problems with strength, coordination and balance Developmental delay Growth Hormone (GH) deficiency Low sex steroids (testosterone and estrogen) Hypogonadism Sleep disturbances (not on a respirator) Temperature dysregulation High pain threshold Skin picking – cellulitis Hypopigmentation Dental problems and thick saliva Auditory difficulties or deafness. IQs within the range of 40 to 105 Those with learning issues. These may be difficulties in conceptual understanding, attention and short-term auditory memory. Autistic tendencies such as Obsessive Compulsive Disorder (OCD) behaviour and behavioural temper tantrums. Those with behaviour such as manipulation, possessive, stubbornness and stealing or lying related to food. Abnormalities in appetite regulating hormones ( eg. Ghrelin is elevated) Hyperphagia Minimal energy expenditure Slow metabolism. Participants from interstate throughout Australia and New Zealand are welcome.
Exclusion criteria
Those without a confirmed diagnosis of PWS. Those with PWS who do not speak English. Those with PWS and any of the conditions listed below: Impaired Glucose Tolerance IGT. Those people with PWS who have a prevalence of glucose intolerance or are considered to have an impaired glucose metabolism. Type 1 diabetes Type 2 diabetes. Chronic Asthma Metabolic syndrome Past indications of kidney dysfunction Liver damage Heart disease Bariatric surgery. Severe sleep-apnea. Those using night respiratory aids for sleeping. Those in psychiatric care. Those with a recorded history related to their inability to vomit. Those with a recorded history of reduced pain sensitivity. Those people with PWS who are not under supervision. Excluded medications Seizure medications. IE: phenabarbitone. Medications for ADHD. IE: Retelin. Psychiatric condition medications such as Antidepressants IE: Trycyclics. Testosterone Oestrogen (Note: Because of puberty related issues with PWS drugs such as testosterone or oestrogen may have been implemented. These may confound the results.) Carer exclusions Carer who do not speak English
Outcome results
None listed