Oxygen Versus Medical Air for Treatment of CSA in Prader Will Syndrome
Primary Purpose
Sleep Apnea, Central, Prader-Willi Syndrome
Status
Recruiting
Phase
Phase 4
Locations
Canada
Study Type
Interventional
Intervention
Medical Air vs Oxygen
Sponsored by
About this trial
This is an interventional treatment trial for Sleep Apnea, Central
Eligibility Criteria
Inclusion Criteria:
- infants under age two with genetically confirmed Prader-Willi Syndrome
- referred to HSC sleep clinic for evaluation with polysomnogram prior to initiation of growth hormone
- infants found to have clinically significant central sleep apnea, defined as an apnea-hypopnea index (AHI) equal to or greater than 5
Exclusion Criteria:
- infants delivered prematurely (less than 37 weeks gestational age)
- term infants with a history of hypoxic-ischemic encephalopathy or stroke
- any concurrent diagnoses that may cause sleep-disordered breathing (ie. craniofacial abnormalities, arnold-chiari malformation, etc)
- infants with a need for daytime supplemental oxygen (ie. cardiac anomalies)
- infants found to have low baseline oxygen saturations on PSG
Sites / Locations
- The Hospital for Sick ChildrenRecruiting
- The Hospital for Sick ChildrenRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Arm A: Medical air followed by oxygen
Arm B: Oxygen followed by medical air
Arm Description
Outcomes
Primary Outcome Measures
Delta CAHI1
Difference in CAHI at baseline compared to supplemental oxygen Delta CAHI1: CAHIoxygen - CAHIbaseline
Delta CAHI2
Difference in CAHI at baseline compared to medical air Delta CAHI2: CAHImedical air - CAHIbaseline
Difference in CAHI1 and CAHI2
A comparison of change in CAHI1 and change in CAHI2 DeltaCAHI1: DeltaCAHI2
Secondary Outcome Measures
Arousal Index1
Difference in Arousal Index at baseline compared to medical air Delta Arousal Index: Arousal Indexmedical air - Arousal Indexbaseline
Arousal Index2
Difference in Arousal Index at baseline compared to Supplemental oxygen Delta Arousal Index: Arousal Indexoxygen - Arousal Indexbaseline
Desaturation Index1
Difference in Desaturation Index at baseline compared to medical air Delta Arousal Index: Desaturation Indexmedical air - Desaturation Indexbaseline
Desaturation Index2
Difference in Desaturation Index at baseline compared to supplemental oxygen Delta Arousal Index: Desaturation Indexoxygen - Desaturation Indexbaseline
Full Information
NCT ID
NCT03031626
First Posted
January 20, 2017
Last Updated
October 24, 2022
Sponsor
The Hospital for Sick Children
1. Study Identification
Unique Protocol Identification Number
NCT03031626
Brief Title
Oxygen Versus Medical Air for Treatment of CSA in Prader Will Syndrome
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
Study Type
Interventional
2. Study Status
Record Verification Date
October 2022
Overall Recruitment Status
Recruiting
Study Start Date
September 1, 2016 (Actual)
Primary Completion Date
August 15, 2023 (Anticipated)
Study Completion Date
December 15, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
The Hospital for Sick Children
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sleep Apnea, Central, Prader-Willi Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Crossover Assignment
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
Care ProviderInvestigatorOutcomes Assessor
Masking Description
The split-night polysomnograms (Arm A, Arm B) will be scored by a blinded independent sleep technologist. A Sleep Physician will report the sleep study and be blinded as to the gas assignment (ie Arm A or Arm B).
Allocation
Randomized
Enrollment
10 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Arm A: Medical air followed by oxygen
Arm Type
Experimental
Arm Title
Arm B: Oxygen followed by medical air
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Medical Air vs Oxygen
Intervention Description
Medical Air/Oxygen will be given
Primary Outcome Measure Information:
Title
Delta CAHI1
Description
Difference in CAHI at baseline compared to supplemental oxygen Delta CAHI1: CAHIoxygen - CAHIbaseline
Time Frame
2 years
Title
Delta CAHI2
Description
Difference in CAHI at baseline compared to medical air Delta CAHI2: CAHImedical air - CAHIbaseline
Time Frame
2 years
Title
Difference in CAHI1 and CAHI2
Description
A comparison of change in CAHI1 and change in CAHI2 DeltaCAHI1: DeltaCAHI2
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Arousal Index1
Description
Difference in Arousal Index at baseline compared to medical air Delta Arousal Index: Arousal Indexmedical air - Arousal Indexbaseline
Time Frame
2 years
Title
Arousal Index2
Description
Difference in Arousal Index at baseline compared to Supplemental oxygen Delta Arousal Index: Arousal Indexoxygen - Arousal Indexbaseline
Time Frame
2 years
Title
Desaturation Index1
Description
Difference in Desaturation Index at baseline compared to medical air Delta Arousal Index: Desaturation Indexmedical air - Desaturation Indexbaseline
Time Frame
2 years
Title
Desaturation Index2
Description
Difference in Desaturation Index at baseline compared to supplemental oxygen Delta Arousal Index: Desaturation Indexoxygen - Desaturation Indexbaseline
Time Frame
2 years
10. Eligibility
Sex
All
Maximum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
infants under age two with genetically confirmed Prader-Willi Syndrome
referred to HSC sleep clinic for evaluation with polysomnogram prior to initiation of growth hormone
infants found to have clinically significant central sleep apnea, defined as an apnea-hypopnea index (AHI) equal to or greater than 5
Exclusion Criteria:
infants delivered prematurely (less than 37 weeks gestational age)
term infants with a history of hypoxic-ischemic encephalopathy or stroke
any concurrent diagnoses that may cause sleep-disordered breathing (ie. craniofacial abnormalities, arnold-chiari malformation, etc)
infants with a need for daytime supplemental oxygen (ie. cardiac anomalies)
infants found to have low baseline oxygen saturations on PSG
Facility Information:
Facility Name
The Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Reshma Amin, MD
Phone
416-813-6346
Ext
206346
Email
reshma.amin@sickkids.ca
First Name & Middle Initial & Last Name & Degree
Munazzah Ambreen
Email
munazzah.ambreen@sickkids.ca
First Name & Middle Initial & Last Name & Degree
Reshma Amin, MD
First Name & Middle Initial & Last Name & Degree
Suhail Al-Saleh, MD
First Name & Middle Initial & Last Name & Degree
Haley Fishman, MD
Facility Name
The Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Reshma Amin, MD
Phone
416-813-6346
Ext
206346
Email
reshma.amin@sickkids.ca
First Name & Middle Initial & Last Name & Degree
Munazzah Ambreen
Email
munazzah.ambreen@sickkids.ca
12. IPD Sharing Statement
Plan to Share IPD
Undecided
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Oxygen Versus Medical Air for Treatment of CSA in Prader Will Syndrome
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