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

NCT03031626 | Completed Phase 4 DMC

• 1 other identifier: 1000053577
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

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.

Conditions

Sleep Apnea, Central; Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (3)

• Delta CAHI1

  Difference in CAHI at baseline compared to supplemental oxygen Delta CAHI1: CAHIoxygen - CAHIbaseline

  2 years

• Delta CAHI2

  Difference in CAHI at baseline compared to medical air Delta CAHI2: CAHImedical air - CAHIbaseline

  2 years

• Difference in CAHI1 and CAHI2

  A comparison of change in CAHI1 and change in CAHI2 DeltaCAHI1: DeltaCAHI2

  2 years

Secondary Outcomes (4)

• Arousal Index1

  2 years

• Arousal Index2

  2 years

• Desaturation Index1

  2 years

• Desaturation Index2

  2 years

Study Arms (2)

Arm A: Medical air followed by oxygen

EXPERIMENTAL

Biological: Medical Air vs Oxygen

Arm B: Oxygen followed by medical air

EXPERIMENTAL

Biological: Medical Air vs Oxygen

Interventions

Medical Air vs Oxygen BIOLOGICAL

Medical Air/Oxygen will be given

Arm A: Medical air followed by oxygen; Arm B: Oxygen followed by medical air

Eligibility Criteria

• Age: Up to 2 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• The Hospital for Sick Children: lead

Study Sites (1)

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

MeSH Terms

Conditions

Sleep Apnea, Central; Prader-Willi Syndrome

Condition Hierarchy (Ancestors)

Sleep Apnea Syndromes; Apnea; Respiration Disorders; Respiratory Tract Diseases; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Nervous System Diseases; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Study Officials

• Reshma Amin, MD

  The Hospital for Sick Children

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: 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).
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Respirologist, Clinician Investigator

Model Details: 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.

Study Record Dates

• First Submitted: January 20, 2017
• First Posted: January 25, 2017
• Study Start: September 1, 2016
• Primary Completion: March 15, 2025
• Study Completion: March 15, 2025
• Last Updated: June 8, 2025

Record last verified: 2025-06

Locations

CA (1)