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Oxygen Versus Medical Air for Treatment of CSA in Prader Will Syndrome

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ClinicalTrials.gov Identifier: NCT03031626
Recruitment Status : Recruiting
First Posted : January 25, 2017
Last Update Posted : April 17, 2018
Sponsor:
Information provided by (Responsible Party):
Reshma Amin, The Hospital for Sick Children

Brief Summary:

The aim of this study is to determine if treatment with Medical Air (21% oxygen in room air) compared to supplemental oxygen (100% oxygen) will lead to similar improvements in the central apnea-hypopnea index (CAHI) for infants with Prader-Willi Syndrome.

Despite the vast amount of research investigating the cause of central sleep apnea, there remain gaps in knowledge, lending to further research efforts. The decision to compare oxygen to medical air is based on several theorized mechanisms. The first of which is the supposition that provision of medical air may act as an arousal stimulus for the hypothalamus, thereby preventing sleep disordered breathing. Secondly, the hypercapnic challenge performed by Livingston et al demonstrated a delayed hypercapneic arousal response in PWS subjects despite simultaneous hyperoxia, leading us to question if therapeutic oxygen really plays a significant role in treating CSA. Lastly, the delivery of medical air via nasal prongs may provide sufficient arousal to terminate the cycle of events leading to central apnea, as described by Urquhart et al.

A deeper understanding of central sleep apnea is essential to ameliorating its adverse sequelae, which include symptoms of ADHD, impaired attention, behavioral problems, and academic difficulties.


Condition or disease Intervention/treatment Phase
Sleep Apnea, Central Prader-Willi Syndrome Biological: Medical Air vs Oxygen Phase 4

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description:

The screening visit will occur during a regularly scheduled clinic visit. The patients that have consented for the study will all undergo the baseline PSG. Only patients with clinically significant CSA after Visit 1 will undergo randomization and complete Visit 2.

Those who have a CAHI greater or equal to 5 will be randomly assigned to Arm A or Arm B. Randomization will be performed using a computer with random numbers drawn. Arm A will receive Medical Air followed by Oxygen and Arm B will receive Oxygen followed by Medical Air.

Masking: Triple (Care Provider, Investigator, Outcomes Assessor)
Masking Description: The split-night polysomnograms (Arm A, Arm B) will be scored by two independent sleep technologists Three independent sleep physicians will be involved in each case; 1 to perform the initial consult in the sleep clinic, a 2nd to interpret the baseline PSG, and a 3rd to interpret the split-night PSG (with oxygen and medical air). Arm A and Arm B will be analyzed blindly.
Primary Purpose: Supportive Care
Official Title: Comparison of Therapeutic Oxygen Versus Medical Air for the Treatment of Central Sleep Apnea in Infants and Children With Prader Willi Syndrome: A Proof of Concept Study
Actual Study Start Date : September 1, 2016
Estimated Primary Completion Date : September 1, 2018
Estimated Study Completion Date : December 15, 2018


Arm Intervention/treatment
Experimental: Arm A: Medical air followed by oxygen Biological: Medical Air vs Oxygen
Medical Air/Oxygen will be given

Experimental: Arm B: Oxygen followed by medical air Biological: Medical Air vs Oxygen
Medical Air/Oxygen will be given




Primary Outcome Measures :
  1. Delta CAHI1 [ Time Frame: 2 years ]
    Difference in CAHI at baseline compared to supplemental oxygen Delta CAHI1: CAHIoxygen - CAHIbaseline

  2. Delta CAHI2 [ Time Frame: 2 years ]
    Difference in CAHI at baseline compared to medical air Delta CAHI2: CAHImedical air - CAHIbaseline

  3. Difference in CAHI1 and CAHI2 [ Time Frame: 2 years ]
    A comparison of Delta CAHI1 and Delta CAHI2 DeltaCAHI1: DeltaCAHI2


Secondary Outcome Measures :
  1. Arousal Index1 [ Time Frame: 2 years ]
    Difference in Arousal Index at baseline compared to medical air Delta Arousal Index: Arousal Indexmedical air - Arousal Indexbaseline

  2. Arousal Index2 [ Time Frame: 2 years ]
    Difference in Arousal Index at baseline compared to Supplemental oxygen Delta Arousal Index: Arousal Indexoxygen - Arousal Indexbaseline

  3. Desaturation Index1 [ Time Frame: 2 years ]
    Difference in Desaturation Index at baseline compared to medical air Delta Arousal Index: Desaturation Indexmedical air - Desaturation Indexbaseline

  4. Desaturation Index2 [ Time Frame: 2 years ]
    Difference in Desaturation Index at baseline compared to supplemental oxygen Delta Arousal Index: Desaturation Indexoxygen - Desaturation Indexbaseline



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Ages Eligible for Study:   up to 2 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. infants under age two with genetically confirmed Prader-Willi Syndrome
  2. referred to HSC sleep clinic for evaluation with polysomnogram prior to initiation of growth hormone
  3. infants found to have clinically significant central sleep apnea, defined as an apnea-hypopnea index (AHI) equal to or greater than 5

Exclusion Criteria:

  1. infants delivered prematurely (less than 37 weeks gestational age)
  2. term infants with a history of hypoxic-ischemic encephalopathy or stroke
  3. any concurrent diagnoses that may cause sleep-disordered breathing (ie. craniofacial abnormalities, arnold-chiari malformation, etc)
  4. infants with a need for daytime supplemental oxygen (ie. cardiac anomalies)
  5. infants found to have low baseline oxygen saturations on PSG

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): NCT03031626


Locations
Canada, Ontario
The Hospital for Sick Children Recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Reshma Amin, MD    416-813-6346 ext 206346    reshma.amin@sickkids.ca   
Contact: weeda zabih    416-813-7654 ext 328361    weeda.zabih@sickkids.ca   
Principal Investigator: Reshma Amin, MD         
Sub-Investigator: Suhail Al-Saleh, MD         
Sub-Investigator: Indra Narang, MD         
Sub-Investigator: Haley Fishman, MD         
Sub-Investigator: Adele Baker, RRT         
Sub-Investigator: Allison Zweerink, RNP         
The Hospital for Sick Children Recruiting
Toronto, Ontario, Canada, M5G0A4
Contact: Reshma Amin, MD    416-813-6346 ext 206346    reshma.amin@sickkids.ca   
Contact: Weeda Zabih    416-648-3516 ext 328361    weeda.zabih@sickkids.ca   
Sponsors and Collaborators
The Hospital for Sick Children

Responsible Party: Reshma Amin, Respirologist, Clinician Investigator, The Hospital for Sick Children
ClinicalTrials.gov Identifier: NCT03031626     History of Changes
Other Study ID Numbers: 1000053577
First Posted: January 25, 2017    Key Record Dates
Last Update Posted: April 17, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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

Additional relevant MeSH terms:
Intellectual Disability
Syndrome
Sleep Apnea Syndromes
Prader-Willi Syndrome
Hyperphagia
Sleep Apnea, Central
Disease
Pathologic Processes
Apnea
Respiration Disorders
Respiratory Tract Diseases
Sleep Disorders, Intrinsic
Dyssomnias
Sleep Wake Disorders
Nervous System Diseases
Neurobehavioral Manifestations
Neurologic Manifestations
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Signs and Symptoms, Digestive
Signs and Symptoms