NCT00399893

Brief Summary

The purpose of this study is to investigate over a 6 month period the effect of octreotide therapy on food intake, sense of hunger, body weight, body composition, efficiency of burning calories, biomarkers of weight regulation and growth hormone markers in children and young Adults with Prader-Willi Syndrome(PWS).

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
5

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Dec 2006

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 14, 2006

Completed
1 day until next milestone

First Posted

Study publicly available on registry

November 15, 2006

Completed
16 days until next milestone

Study Start

First participant enrolled

December 1, 2006

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2009

Completed
1.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2010

Completed
3.9 years until next milestone

Results Posted

Study results publicly available

July 24, 2014

Completed
Last Updated

July 24, 2014

Status Verified

June 1, 2014

Enrollment Period

2.3 years

First QC Date

November 14, 2006

Results QC Date

January 28, 2013

Last Update Submit

June 25, 2014

Conditions

Prader-Willi Syndrome

Keywords

Childhood obesityPrader-Willi SyndromeOctreotideGhrelinWeight lossBody compositionEnergy expenditure

Outcome Measures

Primary Outcomes (9)

  • Number of Participants With Decrease in Fasting Total Ghrelin

    Number of participants showing a decrease in Fasting total ghrelin from baseline to 6 months of treatment with Octreotide or placebo

    6 months

  • Number of Participants With Decrease in Weight From Baseline to 6 Months

    Number of participants who had a decrease in weight from baseline to 6 months of Octreotide or placebo therapy

    6 months

  • Number of Participants With Decreased BMI Z-score From Baseline to 6 Months

    Number of participants with decreased BMI z-score from baseline to 6 months of Octreotide or Placebo therapy

    6 months

  • Number of Participants With Decreased Skin-fold Measurements From Baseline to 6 Months

    Number of participants with decreased skin-fold measurements from baseline to 6 months of Octreotide or Placebo therapy

    6 months

  • Number of Participants With Decrease in Hunger and Food Intake

    Measured by hunger and hyperphagia by questionnaires and parent-reported 72-hour food recall from baseline to 6 months. Multiple questionnaires consisting of a battery of free text answer questions and food diaries are combined in order to make a behavioral assessment of the participants food state of hunger and food intake. There is no defined scale for this assessment. Each participants responses and parent responses are combined.

    6 months

  • Number of Participants With Improved Insulin Regulation From Baseline to 6 Months

    Number of participants with improved Insulin regulation from baseline to 6 months of Octreotide or Placebo therapy. Insulin regulation was measured by immunochemiluminescent assay.

    6 months

  • Number of Participants With Improved Adiponectin Regulation From Baseline to 6 Months

    Number of participants with improved Adiponectin regulation from baseline to 6 months of Octreotide or Placebo therapy

    6 months

  • Number of Participants With Improved Leptin Regulation From Baseline to 6 Months

    Number of participants with improved Leptin regulation from baseline to 6 months of Octreotide or Placebo therapy

    6 months

  • Number of Participants With Improved Peptide YY (PYY) Regulation From Baseline to 6 Months

    Number of participants with improved Peptide YY (PYY) regulation from baseline to 6 months of Octreotide or Placebo therapy

    6 months

Secondary Outcomes (2)

  • Number of Participants With Decreased Body Composition From Baseline to 6 Months by BOD POD®

    6 months

  • Number of Participants With Decreased Body-composition From Baseline to 6 Months by DEXA

    6 months

Study Arms (2)

Octreotide

EXPERIMENTAL

Octreotide to be administered by subcutaneous injection three times daily while on study

Drug: Octreotide

Placebo

PLACEBO COMPARATOR

Placebo to be administered by subcutaneous injection three times daily while on study

Drug: Placebo

Interventions

OctreotideDRUG

Octreotide to be administered by subcutaneous injection three times daily

Also known as: Sandostatin
Octreotide
PlaceboDRUG

Placebo to be administered by subcutaneous injection three times daily while on study

Placebo

Eligibility Criteria

Age5 Years - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Related Publications (1)

  • Haqq AM, Stadler DD, Rosenfeld RG, Pratt KL, Weigle DS, Frayo RS, LaFranchi SH, Cummings DE, Purnell JQ. Circulating ghrelin levels are suppressed by meals and octreotide therapy in children with Prader-Willi syndrome. J Clin Endocrinol Metab. 2003 Aug;88(8):3573-6. doi: 10.1210/jc.2003-030205.

    PMID: 12915638BACKGROUND

MeSH Terms

Conditions

Prader-Willi SyndromePediatric ObesityWeight Loss

Interventions

Octreotide

Condition Hierarchy (Ancestors)

Intellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesChromosome DisordersGenetic Diseases, InbornImprinting DisordersObesityOverweightOvernutritionNutrition DisordersNutritional and Metabolic DiseasesBody WeightSigns and SymptomsPathological Conditions, Signs and SymptomsBody Weight Changes

Intervention Hierarchy (Ancestors)

Peptides, CyclicMacrocyclic CompoundsPolycyclic CompoundsPeptidesAmino Acids, Peptides, and Proteins

Results Point of Contact

Title
Michael Freemark, MD
Organization
Duke University Medical Center
andrea.haqq@albertahealthservices.ca
919-684-5091

Study Officials

  • Andrea M Haqq, MD

    Duke University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 14, 2006

First Posted

November 15, 2006

Study Start

December 1, 2006

Primary Completion

April 1, 2009

Study Completion

September 1, 2010

Last Updated

July 24, 2014

Results First Posted

July 24, 2014

Record last verified: 2014-06

Locations

US(1)