Prader-Willi Syndrome Macronutrient Study

NCT ID: NCT02011360

Last Updated: 2016-12-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-05-31

Study Completion Date

2016-05-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The overall objective is to explore the mechanisms by which macronutrients regulate food intake and weight gain in Prader Willi Syndrome (PWS).

Previous studies from the investigators' labs suggest that the increased appetite of PWS may be triggered or maintained by an increase in the levels of ghrelin, an appetite-stimulating hormone produced primarily by the stomach. This study will compare the effects of low carbohydrate diet versus low fat diet on levels of ghrelin, appetite suppressing hormones and markers of insulin sensitivity in patients with PWS.

The investigators hypothesize that the low carbohydrate diet will suppress plasma active ghrelin and increase appetite-suppressing hormones to a greater degree and for longer duration than the low fat diet and will thereby reduce hyperphagia and increase satiety. The investigators also hypothesize that the low carb diet will improve hormonal and metabolic markers (fatty acids, amino acids and organic acids) of insulin sensitivity and inflammatory cytokine profiles of children with PWS.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Prader Willi Syndrome Syndromic Obesity Childhood Obesity

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Prader Willi syndrome childhood obesity ghrelin appetite regulation

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

CROSSOVER

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Low carbohydrate diet

Low carbohydrate diet: 15%carb; 65%fat; 20% protein. This will be administered over 72 hour hospital stay.

Group Type OTHER

Low Fat diet

Intervention Type OTHER

Low Fat diet: 65%carb; 15%fat; 20% protein

Low Fat diet

Low fat diet: 65%carb; 15%fat; 20% protein. This will be administered over a 72 hour hospital stay.

Group Type OTHER

Low Carbohydrate diet

Intervention Type OTHER

Low carbohydrate diet: 15%carb; 65%fat; 20% protein

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Low Carbohydrate diet

Low carbohydrate diet: 15%carb; 65%fat; 20% protein

Intervention Type OTHER

Low Fat diet

Low Fat diet: 65%carb; 15%fat; 20% protein

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* diagnosis of Prader Willi syndrome confirmed by chromosome analysis (ie. interstitial deletion of paternally-derived chromosome 15q, uniparental maternal disomy or other chromosome 15 abnormalities)
* age 5 years to 17 years
* written informed consent and assent obtained and willingness to comply with the study schedule and procedures
* free T4, TSH values in the normal range (either endogenous or with thyroxine replacement)
* weight stable (BMI percentile fluctuation of \<5 percentiles) over the preceding 2 months prior to the study

Exclusion Criteria

* presence of other clinically significant disease that would impact body composition including diabetes mellitus, chronic inflammatory bowel disease, chronic severe liver or kidney disease or neurologic disorders
* concomitant use of medications known to affect body weight or investigational drug in the past year
Minimum Eligible Age

5 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Foundation for Prader-Willi Research

OTHER

Sponsor Role collaborator

Duke University

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Michael Freemark, MD

Role: PRINCIPAL_INVESTIGATOR

Duke University

Andrea M Haqq, MD

Role: PRINCIPAL_INVESTIGATOR

University of Alberta

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Duke University Medical Center

Durham, North Carolina, United States

Site Status

University of Alberta

Edmonton, Alberta, Canada

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Canada

References

Explore related publications, articles, or registry entries linked to this study.

Dykens EM, Maxwell MA, Pantino E, Kossler R, Roof E. Assessment of hyperphagia in Prader-Willi syndrome. Obesity (Silver Spring). 2007 Jul;15(7):1816-26. doi: 10.1038/oby.2007.216.

Reference Type BACKGROUND
PMID: 17636101 (View on PubMed)

Haqq AM, Muehlbauer M, Svetkey LP, Newgard CB, Purnell JQ, Grambow SC, Freemark MS. Altered distribution of adiponectin isoforms in children with Prader-Willi syndrome (PWS): association with insulin sensitivity and circulating satiety peptide hormones. Clin Endocrinol (Oxf). 2007 Dec;67(6):944-51. doi: 10.1111/j.1365-2265.2007.02991.x. Epub 2007 Jul 30.

Reference Type BACKGROUND
PMID: 17666087 (View on PubMed)

Haqq AM, Muehlbauer MJ, Newgard CB, Grambow S, Freemark M. The metabolic phenotype of Prader-Willi syndrome (PWS) in childhood: heightened insulin sensitivity relative to body mass index. J Clin Endocrinol Metab. 2011 Jan;96(1):E225-32. doi: 10.1210/jc.2010-1733. Epub 2010 Oct 20.

Reference Type BACKGROUND
PMID: 20962018 (View on PubMed)

Haqq AM, Farooqi IS, O'Rahilly S, Stadler DD, Rosenfeld RG, Pratt KL, LaFranchi SH, Purnell JQ. Serum ghrelin levels are inversely correlated with body mass index, age, and insulin concentrations in normal children and are markedly increased in Prader-Willi syndrome. J Clin Endocrinol Metab. 2003 Jan;88(1):174-8. doi: 10.1210/jc.2002-021052.

Reference Type BACKGROUND
PMID: 12519848 (View on PubMed)

Cummings DE, Clement K, Purnell JQ, Vaisse C, Foster KE, Frayo RS, Schwartz MW, Basdevant A, Weigle DS. Elevated plasma ghrelin levels in Prader Willi syndrome. Nat Med. 2002 Jul;8(7):643-4. doi: 10.1038/nm0702-643. No abstract available.

Reference Type BACKGROUND
PMID: 12091883 (View on PubMed)

Haqq AM, Grambow SC, Muehlbauer M, Newgard CB, Svetkey LP, Carrel AL, Yanovski JA, Purnell JQ, Freemark M. Ghrelin concentrations in Prader-Willi syndrome (PWS) infants and children: changes during development. Clin Endocrinol (Oxf). 2008 Dec;69(6):911-20. doi: 10.1111/j.1365-2265.2008.03385.x. Epub 2008 Aug 15.

Reference Type BACKGROUND
PMID: 18710462 (View on PubMed)

Newgard CB, An J, Bain JR, Muehlbauer MJ, Stevens RD, Lien LF, Haqq AM, Shah SH, Arlotto M, Slentz CA, Rochon J, Gallup D, Ilkayeva O, Wenner BR, Yancy WS Jr, Eisenson H, Musante G, Surwit RS, Millington DS, Butler MD, Svetkey LP. A branched-chain amino acid-related metabolic signature that differentiates obese and lean humans and contributes to insulin resistance. Cell Metab. 2009 Apr;9(4):311-26. doi: 10.1016/j.cmet.2009.02.002.

Reference Type BACKGROUND
PMID: 19356713 (View on PubMed)

Laferrere B, Reilly D, Arias S, Swerdlow N, Gorroochurn P, Bawa B, Bose M, Teixeira J, Stevens RD, Wenner BR, Bain JR, Muehlbauer MJ, Haqq A, Lien L, Shah SH, Svetkey LP, Newgard CB. Differential metabolic impact of gastric bypass surgery versus dietary intervention in obese diabetic subjects despite identical weight loss. Sci Transl Med. 2011 Apr 27;3(80):80re2. doi: 10.1126/scitranslmed.3002043.

Reference Type BACKGROUND
PMID: 21525399 (View on PubMed)

Wang TJ, Larson MG, Vasan RS, Cheng S, Rhee EP, McCabe E, Lewis GD, Fox CS, Jacques PF, Fernandez C, O'Donnell CJ, Carr SA, Mootha VK, Florez JC, Souza A, Melander O, Clish CB, Gerszten RE. Metabolite profiles and the risk of developing diabetes. Nat Med. 2011 Apr;17(4):448-53. doi: 10.1038/nm.2307. Epub 2011 Mar 20.

Reference Type BACKGROUND
PMID: 21423183 (View on PubMed)

Sondike SB, Copperman N, Jacobson MS. Effects of a low-carbohydrate diet on weight loss and cardiovascular risk factor in overweight adolescents. J Pediatr. 2003 Mar;142(3):253-8. doi: 10.1067/mpd.2003.4.

Reference Type BACKGROUND
PMID: 12640371 (View on PubMed)

Foster GD, Wyatt HR, Hill JO, McGuckin BG, Brill C, Mohammed BS, Szapary PO, Rader DJ, Edman JS, Klein S. A randomized trial of a low-carbohydrate diet for obesity. N Engl J Med. 2003 May 22;348(21):2082-90. doi: 10.1056/NEJMoa022207.

Reference Type BACKGROUND
PMID: 12761365 (View on PubMed)

Samaha FF, Iqbal N, Seshadri P, Chicano KL, Daily DA, McGrory J, Williams T, Williams M, Gracely EJ, Stern L. A low-carbohydrate as compared with a low-fat diet in severe obesity. N Engl J Med. 2003 May 22;348(21):2074-81. doi: 10.1056/NEJMoa022637.

Reference Type BACKGROUND
PMID: 12761364 (View on PubMed)

Stern L, Iqbal N, Seshadri P, Chicano KL, Daily DA, McGrory J, Williams M, Gracely EJ, Samaha FF. The effects of low-carbohydrate versus conventional weight loss diets in severely obese adults: one-year follow-up of a randomized trial. Ann Intern Med. 2004 May 18;140(10):778-85. doi: 10.7326/0003-4819-140-10-200405180-00007.

Reference Type BACKGROUND
PMID: 15148064 (View on PubMed)

Brehm BJ, Seeley RJ, Daniels SR, D'Alessio DA. A randomized trial comparing a very low carbohydrate diet and a calorie-restricted low fat diet on body weight and cardiovascular risk factors in healthy women. J Clin Endocrinol Metab. 2003 Apr;88(4):1617-23. doi: 10.1210/jc.2002-021480.

Reference Type BACKGROUND
PMID: 12679447 (View on PubMed)

Yancy WS Jr, Olsen MK, Guyton JR, Bakst RP, Westman EC. A low-carbohydrate, ketogenic diet versus a low-fat diet to treat obesity and hyperlipidemia: a randomized, controlled trial. Ann Intern Med. 2004 May 18;140(10):769-77. doi: 10.7326/0003-4819-140-10-200405180-00006.

Reference Type BACKGROUND
PMID: 15148063 (View on PubMed)

Dansinger ML, Gleason JA, Griffith JL, Selker HP, Schaefer EJ. Comparison of the Atkins, Ornish, Weight Watchers, and Zone diets for weight loss and heart disease risk reduction: a randomized trial. JAMA. 2005 Jan 5;293(1):43-53. doi: 10.1001/jama.293.1.43.

Reference Type BACKGROUND
PMID: 15632335 (View on PubMed)

Bistrian BR, Blackburn GL, Stanbury JB. Metabolic aspects of a protein-sparing modified fast in the dietary management of Prader-Willi obesity. N Engl J Med. 1977 Apr 7;296(14):774-9. doi: 10.1056/NEJM197704072961402.

Reference Type BACKGROUND
PMID: 840278 (View on PubMed)

Fields DA, Higgins PB, Radley D. Air-displacement plethysmography: here to stay. Curr Opin Clin Nutr Metab Care. 2005 Nov;8(6):624-9. doi: 10.1097/01.mco.0000171127.44525.07.

Reference Type BACKGROUND
PMID: 16205463 (View on PubMed)

Day N, McKeown N, Wong M, Welch A, Bingham S. Epidemiological assessment of diet: a comparison of a 7-day diary with a food frequency questionnaire using urinary markers of nitrogen, potassium and sodium. Int J Epidemiol. 2001 Apr;30(2):309-17. doi: 10.1093/ije/30.2.309.

Reference Type BACKGROUND
PMID: 11369735 (View on PubMed)

Stubbs RJ, Hughes DA, Johnstone AM, Rowley E, Reid C, Elia M, Stratton R, Delargy H, King N, Blundell JE. The use of visual analogue scales to assess motivation to eat in human subjects: a review of their reliability and validity with an evaluation of new hand-held computerized systems for temporal tracking of appetite ratings. Br J Nutr. 2000 Oct;84(4):405-15. doi: 10.1017/s0007114500001719.

Reference Type BACKGROUND
PMID: 11103211 (View on PubMed)

Liu J, Prudom CE, Nass R, Pezzoli SS, Oliveri MC, Johnson ML, Veldhuis P, Gordon DA, Howard AD, Witcher DR, Geysen HM, Gaylinn BD, Thorner MO. Novel ghrelin assays provide evidence for independent regulation of ghrelin acylation and secretion in healthy young men. J Clin Endocrinol Metab. 2008 May;93(5):1980-7. doi: 10.1210/jc.2007-2235. Epub 2008 Mar 18.

Reference Type BACKGROUND
PMID: 18349056 (View on PubMed)

Haqq AM, Lien LF, Boan J, Arlotto M, Slentz CA, Muehlbauer MJ, Rochon J, Gallup D, McMahon RL, Bain JR, Stevens R, Millington D, Butler MD, Newgard CB, Svetkey LP. The Study of the Effects of Diet on Metabolism and Nutrition (STEDMAN) weight loss project: Rationale and design. Contemp Clin Trials. 2005 Dec;26(6):616-25. doi: 10.1016/j.cct.2005.09.003. Epub 2005 Oct 18.

Reference Type BACKGROUND
PMID: 16239128 (View on PubMed)

Nass R, Liu J, Hellmann P, Coschigano KT, Gaylinn B, Berryman DE, Kopchick JJ, Thorner MO. Chronic changes in peripheral growth hormone levels do not affect ghrelin stomach mRNA expression and serum ghrelin levels in three transgenic mouse models. J Neuroendocrinol. 2004 Aug;16(8):669-75. doi: 10.1111/j.1365-2826.2004.01220.x.

Reference Type BACKGROUND
PMID: 15271059 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

SFR2382

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

Pro00049226

Identifier Type: -

Identifier Source: org_study_id