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Trial registered on ANZCTR


Registration number
ACTRN12611000334909
Ethics application status
Approved
Date submitted
25/03/2011
Date registered
30/03/2011
Date last updated
13/09/2013
Type of registration
Prospectively registered

Titles & IDs
Public title
A study on the satiety effect of Caralluma fimbriata extract for people with Prader - Willi syndrome (PWS).”
Scientific title
A study on children and adolescents with Prader-Willi syndrome, of Caralluma fimbriata extract and the placebo of maltodextrin/cabbage leaf, to determine any change in satiation on the markers of hyperphagia.
Secondary ID [1] 259861 0
Nil
Universal Trial Number (UTN)
U1111-1120-2917
Trial acronym
Nil
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Prader-Willi syndrome (PWS) PWS is caused by three types of abnormality in chromosome 15 at a prevalence rate of 1:11,000 to 1:22,000. The defining feature of PWS is food-seeking behaviour due to constant hunger. Hyperphagia and obesity are one of the main burdens in PWS and obesity is linked to increased morbidity and mortality. 265444 0
Condition category
Condition code
Human Genetics and Inherited Disorders 265601 265601 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
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.
Intervention code [1] 264283 0
Treatment: Drugs
Intervention code [2] 264284 0
Prevention
Comparator / control treatment
Maltodextrin and cabbage leaf. Dosage of the placebo 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. The Placebo capsules will be 200g Maltodextrin and 50g cabbage leaf, to appropriate the intervention colour and bitterness. This placebo is not active in satiation effect, it will be a 'sham' treatment.
Control group
Placebo

Outcomes
Primary outcome [1] 262391 0
This study is in respect to satiation, hyperphagia and food-seeking behaviour for people with Prader-Willi syndrome (PWS). The aim and objective of this double blind, randomized, cross-over trial will be to evidence the effect of an oral supplementation of Caralluma fimbriata extract(CFE) on the markers of satiation, for the proposed cohort of 16 individuals with PWS. Hyperphagia questionnaire: Scale hunger, food seeking behaviours, thoughts of food, the urge to eat, repetitive statements, temper tantrums, stealing food, and satiety (13 measures).
Timepoint [1] 262391 0
Parent/supervisors will rate the frequency and magnitude of their child's hyperphagia at the baseline clinical consultation, Pre, intervention/placebo and then at the 4 week post intervention/placebo point.
Primary outcome [2] 262405 0
Eight hyperphagia questionnaires: time points are at baseline and weekly during intervention or placebo.
Timepoint [2] 262405 0
Pre and post second treatment cross-over period,. (After two week wash-out.)
Secondary outcome [1] 273690 0
Nil
Timepoint [1] 273690 0
Nil

Eligibility
Key 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.
Minimum age
5 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
The 16 subjects (male and female) aged 5 - 17 years will be recruited via the PWS Associations (following association protocol), through the PWS clinic registration and within Joanne Griggs’ MIA Multiple Initiative Approach, contacts. Joanne has received letters of interest already because of her media events through a V.U. publicity representative in 2010. All proposed participants will go through informed consent procedures. All recruited subjects will be sent a study protocol by mail with a plain language statement as an expression of their interest. Those applying will be questioned by phone to ensure they fully understand the requirements of the study. Carers will be asked to speak with medical practitioners and any appropriate carer/family members and to consult with their children carefully. They will be encouraged to ask questions to clarify their role. When participants and carers understand the parameters of the study they will both sign the consent forms. Confidentiality protocol will be followed. All proposed participants will fill in a medical questionnaire to determine their eligibility for this pilot study. The details of the screening will include the inclusion criteria, medical history, medications, surgery/operations, family medical history and any health concerns/problems, school requirements, medical symptoms, current markers of hunger and satiety, behaviours, early intervention, current paediatrician and medical providers and supervision parameters. Proposed participants will be given an Inclusion/exclusion assessment score. Participant inclusion or exclusion in the pilot study will be respectfully advised and those included will be coded and randomized into group A or B by the principle investigator; Associate Professor Michael Mathai Victoria University. Dosage will be identified from recorded weight and age. Specified dose of either CFE 250mg or the placebo 250mg (up to 1000mg for adult weight ), will be assigned to coded containers; for 28 treatments over the four week period. Coded containers will be distributed and marked off the coded lists - on receipt, at the pre intervention clinical consultations conducted in the Victoria University Teaching (Nutritional Therapy) Clinic, Building 2, St Albans Campus, Melbourne, Victoria. New Zealand and interstate participants may gain anthropometric measurements from their medical practitioner .The cross-over intervention/placebo will follow the same process of distribution at the midline clinical consultation during wash-out. Student investigator and statistitian will be blinded to Intervention or placebo allocation. The vulnerability of this condition is apparent; it is a duty of care to keep supervisor/participant information, prior to, during or after this research project private. All information will be number coded and kept in a locked filing cabinet in the College of Health and Biomedicine, Victoria University. Participant’s names will be held in a different location, separate from their data. We will not disclose personal information at any time.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
As this is a small cohort (n=8 per group); the principle investigator will randomly pick names from the list blind to the student investigator and statistician.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Crossover
Other design features
2 week wash-out period.
Phase
Phase 1
Type of endpoint/s
Efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
24/06/2011
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
16
Accrual to date
Final
Recruitment outside Australia
Country [1] 5410 0
New Zealand
State/province [1] 5410 0

Funding & Sponsors
Funding source category [1] 264739 0
Self funded/Unfunded
Name [1] 264739 0
Victoria University, College of Health and Biomedicine.
Country [1] 264739 0
Australia
Primary sponsor type
University
Name
Victoria University
Address
College of Health and Biomedicine, Building 6 Victoria University, St Albans Campus, St Albans, Victoria, Australia 3021
Country
Australia
Secondary sponsor category [1] 263864 0
None
Name [1] 263864 0
Nil
Address [1] 263864 0
Nil
Country [1] 263864 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 266725 0
Human Research Ethics Committee
Ethics committee address [1] 266725 0
Human Research Ethics Committee
Office for Research
Victoria University
PO Box 14428,
Melbourne VIC 8001
Ethics committee country [1] 266725 0
Australia
Date submitted for ethics approval [1] 266725 0
30/03/2011
Approval date [1] 266725 0
26/08/2013
Ethics approval number [1] 266725 0
HRETH 11/71

Summary
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.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 32390 0
A/Prof Michael Mathai
Address 32390 0
College of Health and Biomedicine, Victoria University, St Albans Campus, St Albans, Victoria, Australia 3021
Country 32390 0
Australia
Phone 32390 0
+61 3 99192211
Fax 32390 0
Email 32390 0
[email protected]
Contact person for public queries
Name 15637 0
Joanne Griggs
Address 15637 0
124 Brougham St, Eltham Victoria, Australia 3095
Country 15637 0
Australia
Phone 15637 0
+61 0414800411
Fax 15637 0
+61 3 99192465
Email 15637 0
[email protected]
Contact person for scientific queries
Name 6565 0
Michael Mathai
Address 6565 0
College of Health and Biomedicine, Victoria University, St Albans Campus, St Albans, Victoria, Australia 3021
Country 6565 0
Australia
Phone 6565 0
+61 3 99192211
Fax 6565 0
+61 3 99192465
Email 6565 0
[email protected]

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