ClinicalTrials.gov
ClinicalTrials.gov Menu

Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03197662
Recruitment Status : Recruiting
First Posted : June 23, 2017
Last Update Posted : December 4, 2018
Sponsor:
Information provided by (Responsible Party):
Eric Hollander, Montefiore Medical Center

Brief Summary:

The investigators propose a phase 2 randomized double blind 8-week treatment trial of intranasal OXT vs. placebo in 50 subjects aged 5 to 17 years with PWS in order to assess IN-OXT's affect on measurements of (1) eating behaviors (2) repetitive behaviors (3) weight and body composition (4) quality of life (5) salivary OXT and hormone levels (including ghrelin, pancreatic polypeptide, peptide YY, GLP-1, insulin, glucagon, testosterone, and estrogen). If superior to placebo, this data will add to the current knowledge that OXT is an effective treatment for hyperphagia as well as other symptoms of PWS.

Funding Source- FDA OOPD


Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Hyperphagia Drug: Intranasal Oxytocin (IN-OXT) Drug: Matched Placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Phase 2 Study: Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
Actual Study Start Date : April 11, 2018
Estimated Primary Completion Date : February 2020
Estimated Study Completion Date : August 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Oxytocin

Arm Intervention/treatment
Experimental: Experimental: Intranasal Oxytocin (IN-OXT)
Syntocinon (synthetic oxytocin) will be used in this protocol. Each subject will receive a dose of 16 IU QD and will be instructed to inhale 2 puffs per nostril (4 IU each).
Drug: Intranasal Oxytocin (IN-OXT)
Each subject will receive a dose of 16 IU QD, and will be instructed to inhale 2 puffs per nostril every day (4 IU each). If needed, treatment will be titrated due to side effects or non-response.
Other Name: Syntocinon

Placebo Comparator: Placebo Comparator: Matched Placebo
Each subject will receive a dose of 16 IU QD, and will be instructed to inhale 2 puffs per nostril (4 IU each).
Drug: Matched Placebo
Each subject will receive a dose of 16 IU QD, and will be instructed to inhale 2 puffs per nostril every day (4 IU each). If needed, treatment will be titrated due to side effects or non-response.




Primary Outcome Measures :
  1. Hyperphagia Questionnaire for Clinical Trials [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
    Assesses Eating Behaviors and Hyperphagia in PWS. Repeated Measures Analysis.


Secondary Outcome Measures :
  1. Repetitive Behavior Scale Revised (RBS-R) [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
    Change in Repetitive Behavior Scale (RBS-R) from baseline to endpoint

  2. BMI [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
    Change in BMI from baseline to endpoint

  3. Body Composition [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
    Change in Body Composition (measured via bioelectrical impedance analysis) from baseline to endpoint

  4. World Health Organization Quality of Life Questionnaire (WHOQOL) [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
    Change in World Health Organization Quality of Life Questionnaire from baseline to endpoint

  5. Abberant Behavior Checklist [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
    Change in Aberrant Behavior Checklist (ABC) from baseline to Endpoint.

  6. Salivary Oxytocin Concentration [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
    Change in Salivary Oxytocin Concentration from baseline to endpoint

  7. Caregiver Strain Questionnaire [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
    Change in Caregiver Strain Questionnaire from baseline to endpoint

  8. Montefiore-Einstein Rigidity Scale-Revised PWS (MERS-R-PWS): [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
    Change in three domains of rigid behavior from baseline to endpoint


Other Outcome Measures:
  1. Automated, Self-Administered, 24 Hour Recall Diary System [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
    Change in ASA 24: Automated, Self-Administered, 24 Hour Recall Diary System from baseline to endpoint

  2. Relationship between weight-based dosing and hyperphagia treatment response [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
  3. Hormone levels (Ghrelin, pancreatic polypeptide, peptide YY, GLP-1, insulin, glucagon, testosterone, and estrogen) [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
    Change in hormone levels (ghrelin, pancreatic polypeptide, peptide YY, GLP-1, insulin, glucagon, testosterone, and estrogen) from baseline to endpoint.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   5 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female pediatric outpatients aged 5 to 17 years
  2. Must be in PWS nutritional phase 2b or 3 as determined by PI
  3. Must be on growth hormone treatment and have been receiving stable doses of growth hormone treatment for at least one year prior to screening date. Treatment cannot have been interrupted for more than one week within 3 months of screening.
  4. Diagnosis of PWS confirmed by exam, genetic testing and patient medical records.
  5. A score of at least moderate severity on the Hyperphagia Questionnaire for Clinical Trials at both screening and baseline visits.
  6. Stable dosages of hormone treatments (including testosterone and estrogen supplements) for 4 weeks prior to randomization and for the duration of the study.
  7. Stable dosages of metabolic treatments that could affect appetite (including metformin) for 4 weeks prior to randomization and for the duration of the study.
  8. Physical exam and laboratory results that are within the normal range for individuals with PWS.
  9. Presence of a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the child's development and behavior improvement throughout the study.

Exclusion Criteria:

  1. Exposure to any investigational agent in the 30 days prior to randomization.
  2. Child not receiving growth hormone treatment
  3. Children weighing less than 40 lbs
  4. Children with unstable Type 2 Diabetes confirmed by Hemoglobin A1C levels at screening
  5. Children with unstable medical co-morbidities at baseline.
  6. Children with active upper respiratory infections at screening.
  7. A primary psychiatric diagnosis other than ASD, including bipolar disorder, psychosis, schizophrenia, PTSD or MDD. These patients will be excluded due to potential confounding results.
  8. Pregnant or lactating patients or patients who will not agree to use a double barrier method of contraception. IN-OXT has not been studied in pregnant or lactating women.
  9. Females using an estrogen-based contraceptive. As an alternative to an estrogen based contraceptive, subjects will be counseled to use progesterone-based contraceptives; cervical cap; cervical sponges; or spermicidal foam in combination with a condom. Subjects will need to use a double barrier method to be in the study.
  10. A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger the subject's well-being.
  11. A known diagnosis of Rett's Syndrome of Childhood Disintegrative Disorder or marked sensory impairment such as deafness or blindness.
  12. Subjects who have changes in allied health therapies, behavioral or educational interventions within four weeks prior to randomization other than those associated with school holidays.
  13. Subjects who have had changes in medications or medication doses of risperidone, apriprazole, other antipsychotic medications, clonidine, guanfacine, stimulants or anti-convulsants within four weeks of randomization.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03197662


Contacts
Contact: Bonnie Taylor, PhD 718-839-7530 botaylor@montefiore.org
Contact: Danya Schlussel 718-839-7501 dschluss@montefiore.org

Locations
United States, New York
Montefiore Medical Center, Albert Einstein College of Medicine Recruiting
Bronx, New York, United States, 10461
Contact: Danya Schlussel, BA    718-839-7501    dschluss@montefiore.org   
Contact: Bonnie Taylor, PhD    718-839-7530    botaylor@montefiore.org   
Principal Investigator: Eric Hollander, MD         
Sponsors and Collaborators
Montefiore Medical Center

Responsible Party: Eric Hollander, Director, Autism and Obsessive Compulsive Spectrum Program, Anxiety and Depression Program, Montefiore Medical Center
ClinicalTrials.gov Identifier: NCT03197662     History of Changes
Other Study ID Numbers: 2017-8076
FD-R-05106-01 ( Other Grant/Funding Number: FDA-OOPD )
First Posted: June 23, 2017    Key Record Dates
Last Update Posted: December 4, 2018
Last Verified: December 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Eric Hollander, Montefiore Medical Center:
Prader-Willi Syndrome
Hyperphagia

Additional relevant MeSH terms:
Syndrome
Prader-Willi Syndrome
Hyperphagia
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Signs and Symptoms, Digestive
Signs and Symptoms
Oxytocin
Oxytocics
Reproductive Control Agents
Physiological Effects of Drugs