Octreotide Therapy in Children and Young Adults With Prader-Willi Syndrome (PWS)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00399893
• Date of registration: 14/11/2006
• Prospective Registration: Yes
• Primary sponsor: Duke University
• Public title: Octreotide Therapy in Children and Young Adults With Prader-Willi Syndrome (PWS)
• Scientific title: Investigation of the Developmental, Nutritional and Hormonal Regulation of Ghrelin in Children and Young Adults With Prader-Willi Syndrome (PWS): Octreotide Intervention Sub-study
• Date of first enrolment: December 2006
• Target sample size: 5
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00399893
• Study type: Interventional
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: Andrea M Haqq, MD
• Affiliation: Duke University

Key inclusion & exclusion criteria

Inclusion Criteria:

- Diagnosis of PWS confirmed by chromosome analysis

- Ages 5 years to 21 years

- BMI for age = (greater-than or equal to)85th percentile

- Written informed consent and assent obtained and willingness to comply with the study
schedule and procedures

- Free T4, Thyroid stimulating hormone (TSH) values in the normal range (either
endogenous or with thyroxine replacement)

Exclusion Criteria:

- Patients with any other clinically significant disease that would have an impact on
body composition, including diabetes mellitus, chronic inflammatory bowel disease,
chronic severe liver or kidney disease or neurologic disorders

- Concomitant use of an investigational drug or Octreotide in the past year

- Use of steroids for longer than 7 days within the past 30 days



Age minimum: 5 Years
Age maximum: 21 Years
Gender: All

Health Condition(s) or Problem(s) studied

Prader-Willi Syndrome

Intervention(s)

Drug: Octreotide

Drug: Placebo

Primary Outcome(s)

Number of Participants With Decrease in Fasting Total Ghrelin [Time Frame: 6 months]

Number of Participants With Improved Leptin Regulation From Baseline to 6 Months [Time Frame: 6 months]

Number of Participants With Improved Peptide YY (PYY) Regulation From Baseline to 6 Months [Time Frame: 6 months]

Number of Participants With Decreased Skin-fold Measurements From Baseline to 6 Months [Time Frame: 6 months]

Number of Participants With Improved Insulin Regulation From Baseline to 6 Months [Time Frame: 6 months]

Number of Participants With Decreased BMI Z-score From Baseline to 6 Months [Time Frame: 6 months]

Number of Participants With Decrease in Weight From Baseline to 6 Months [Time Frame: 6 months]

Number of Participants With Improved Adiponectin Regulation From Baseline to 6 Months [Time Frame: 6 months]

Number of Participants With Decrease in Hunger and Food Intake [Time Frame: 6 months]

Secondary Outcome(s)

Number of Participants With Decreased Body Composition From Baseline to 6 Months by BOD POD® [Time Frame: 6 months]

Number of Participants With Decreased Body-composition From Baseline to 6 Months by DEXA [Time Frame: 6 months]

Secondary ID(s)

Pro00005426

Protocol #00005426

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Center for Research Resources (NCRR)

National Institutes of Health (NIH)

Novartis

Results

• Results available: Yes
• Date Posted: 24/07/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00399893