Prader-Willi SyndromeNCT00399893
Octreotide Therapy in Children and Young Adults With Prader-Willi Syndrome (PWS)
A study testing Octreotide in people with Prader-Willi Syndrome, run by Duke University at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 5 to 21 years
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- Octreotide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2006to Sep 2010
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Duke University Medical Center
Durham, North Carolina, 27710
About this study
Official 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
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).
Read the detailed description
Obesity continues to be a prevalent health concern affecting every race of the American population. According to data from the World Health Organization, 54% of U.S. adults are overweight (body mass index (BMI) >25 kg/m2 ) and 22% are obese (BMI >30 kg/m2) (1). In addition, 25% of U.S. children are overweight or obese (1). Studies show that obese children are likely to become obese adults (2-5). Also, recent studies report significant years of life lost due to the impact of being an obese adult (6, 7). Thus, insights into the pathogenesis of childhood obesity and preventative measures are needed to combat the inevitable increase in worldwide incidence of obesity and its associated co-morbidities. Recent studies have identified a new gastroenteric hormone, ghrelin, as a long-term regulator of energy balance in humans (12). Ghrelin is a 28 amino acid acylated peptide which is an endogenous ligand of the growth hormone secretagogue receptor (GHS-R), a hypothalamic G-protein-coupled receptor (13). Enteroendocrine cells (X/A-like cells) of the stomach are the major site of ghrelin synthesis, although a minor proportion of ghrelin synthesis occurs in other sites such as the hypothalamus, pituitary, duodenum, jejunum and lung (14) (15, 16).
The hypothesis that hyperghrelinemia causes some of the features of PWS predicts that this disorder will be ameliorated (partially or completely) by lowering ghrelin levels. We have recently shown that the somatostatin agonist, octreotide, suppresses ghrelin levels in humans. If octreotide remains effective in longer term studies, the drug may become an adjuvant therapy, in addition to growth hormone, to control the insatiable appetite and morbid obesity seen in this condition.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Octreotide, Placebo
- Keywords
- Childhood obesity, Prader-Willi Syndrome, Octreotide, Ghrelin, Weight loss, Body composition, Energy expenditure
- Medical subject headings
- Prader-Willi Syndrome, Pediatric Obesity, Weight Loss
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OctreotideDrug
Also called Sandostatin
Octreotide to be administered by subcutaneous injection three times daily
PlaceboDrug
Placebo to be administered by subcutaneous injection three times daily while on study
| Study group | Role | What they receive |
|---|---|---|
| Octreotide | Experimental | Octreotide to be administered by subcutaneous injection three times daily while on study Receives: Drug: Octreotide |
| Placebo | Placebo comparator | Placebo to be administered by subcutaneous injection three times daily while on study Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 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
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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
Measured: 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
Measured: 6 months
Secondary outcomes (2)
Number of Participants With Decreased Body Composition From Baseline to 6 Months by BOD POD®
Number of participants with decreased body-composition as Measured by BOD POD® body composition tracking system from baseline to 6 months of Octreotide or Placebo therapy
Measured: 6 months
Number of Participants With Decreased Body-composition From Baseline to 6 Months by DEXA
Number of participants with decreased body-composition as Measured by Dual Energy X-ray Absorptiometry (DEXA) scan from baseline to 6 months of Octreotide or Placebo therapy
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2006 (estimated)
- Study startDec 2006
- Primary completionApr 2009
- Study completionSep 2010
Why it stopped: Inadequate recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- More trials run by Duke UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Collaborators
- National Institutes of Health (NIH), National Center for Research Resources (NCRR), Novartis
- Responsible party
- Sponsor
- Investigators
- Andrea M Haqq, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00399893
- Sponsor's protocol ID
- Pro00005426
- Other IDs
- Protocol #00005426
- Status last verified
- Jun 2014
- First submitted
- 14 Nov 2006
- Last updated
- 24 Jul 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 12915638
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT00399893
- Acronym / sponsor ID
- Pro00005426
- Conditions
- Prader-Willi Syndrome
- Keywords
- Childhood obesity, Prader-Willi Syndrome, Octreotide, Ghrelin, Weight loss, Body composition, Energy expenditure
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Inadequate recruitment
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Collaborators
- National Institutes of Health (NIH), National Center for Research Resources (NCRR), Novartis
- Study start
- Dec 2006
- Primary completion
- Apr 2009 (actual)
- Study completion
- Sep 2010 (actual)
- First posted
- 15 Nov 2006 (estimated)
- Results
- Posted 24 Jul 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
Obesity continues to be a prevalent health concern affecting every race of the American population. According to data from the World Health Organization, 54% of U.S. adults are overweight (body mass index (BMI) >25 kg/m2 ) and 22% are obese (BMI >30 kg/m2) (1). In addition, 25% of U.S. children are overweight or obese (1). Studies show that obese children are likely to become obese adults (2-5). Also, recent studies report significant years of life lost due to the impact of being an obese adult (6, 7). Thus, insights into the pathogenesis of childhood obesity and preventative measures are needed to combat the inevitable increase in worldwide incidence of obesity and its associated co-morbidities. Recent studies have identified a new gastroenteric hormone, ghrelin, as a long-term regulator of energy balance in humans (12). Ghrelin is a 28 amino acid acylated peptide which is an endogenous ligand of the growth hormone secretagogue receptor (GHS-R), a hypothalamic G-protein-coupled receptor (13). Enteroendocrine cells (X/A-like cells) of the stomach are the major site of ghrelin synthesis, although a minor proportion of ghrelin synthesis occurs in other sites such as the hypothalamus, pituitary, duodenum, jejunum and lung (14) (15, 16).
The hypothesis that hyperghrelinemia causes some of the features of PWS predicts that this disorder will be ameliorated (partially or completely) by lowering ghrelin levels. We have recently shown that the somatostatin agonist, octreotide, suppresses ghrelin levels in humans. If octreotide remains effective in longer term studies, the drug may become an adjuvant therapy, in addition to growth hormone, to control the insatiable appetite and morbid obesity seen in this condition.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Octreotide | Experimental | Drug: OctreotideOctreotide to be administered by subcutaneous injection three times daily while on study |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo to be administered by subcutaneous injection three times daily while on study |
Who may qualify
- Ages
- 5 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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)
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OctreotideAlso: SandostatinOctreotide to be administered by subcutaneous injection three times daily | Drug | Octreotide |
| PlaceboPlacebo to be administered by subcutaneous injection three times daily while on study | Drug | Placebo |
Primary outcome
Number of Participants With Decrease in Fasting Total Ghrelin
Time frame: 6 months
Number of participants showing a decrease in Fasting total ghrelin from baseline to 6 months of treatment with Octreotide or placebo
Number of Participants With Decrease in Weight From Baseline to 6 Months
Time frame: 6 months
Number of participants who had a decrease in weight from baseline to 6 months of Octreotide or placebo therapy
Number of Participants With Decreased BMI Z-score From Baseline to 6 Months
Time frame: 6 months
Number of participants with decreased BMI z-score from baseline to 6 months of Octreotide or Placebo therapy
Number of Participants With Decreased Skin-fold Measurements From Baseline to 6 Months
Time frame: 6 months
Number of participants with decreased skin-fold measurements from baseline to 6 months of Octreotide or Placebo therapy
Number of Participants With Decrease in Hunger and Food Intake
Time frame: 6 months
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.
Number of Participants With Improved Insulin Regulation From Baseline to 6 Months
Time frame: 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.
Number of Participants With Improved Adiponectin Regulation From Baseline to 6 Months
Time frame: 6 months
Number of participants with improved Adiponectin regulation from baseline to 6 months of Octreotide or Placebo therapy
Number of Participants With Improved Leptin Regulation From Baseline to 6 Months
Time frame: 6 months
Number of participants with improved Leptin regulation from baseline to 6 months of Octreotide or Placebo therapy
Number of Participants With Improved Peptide YY (PYY) Regulation From Baseline to 6 Months
Time frame: 6 months
Number of participants with improved Peptide YY (PYY) regulation from baseline to 6 months of Octreotide or Placebo therapy
2 secondary outcomes
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 12915638Haqq 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrea M Haqq, MD principal investigator
Duke University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Octreotide in people with Prader-Willi Syndrome, run by Duke University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 24 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Jul 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Decrease in Fasting Total Ghrelin (time frame: 6 months). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00399893, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jul 2014.