Observational Approach Versus Surgical Intervention for Stones

Children's Hospital of Philadelphia1,658 enrolled

Overview

The aims of the Observational Approach versus Surgical Intervention for Asymptomatic Stone (OASIS) trial are: 1) to determine whether observation compared to upfront surgery results in less healthcare-related life disruption due to kidney stones among children and adults with asymptomatic kidney stones; 2) to identify the groups benefiting the most from each strategy; and 3) to determine the preferences and values informing the choice between observation and upfront surgery.

A critical barrier to improving the health of the nearly 40 million Americans with kidney stone disease is the weak evidence base supporting the management of individuals with asymptomatic kidney stones. The proposed Observational Approach versus Surgical Intervention for Asymptomatic Stone (OASIS) randomized clinical trial answers the research question of whether observation or upfront surgery results in less life disruption from asymptomatic kidney stones for children and adults. The management options for patients with asymptomatic stones are 1) observation or 2) upfront surgical treatment, both of which are considered safe, part of usual care, widely accessible, and covered by insurance. OASIS responds to the charge for well-designed Randomized Controlled Trials (RCT) from the Agency for Healthcare Research and Quality (AHRQ) systematic review on kidney stone disease management and ensures patient centeredness by examining outcomes that patients have identified as meaningful. Intervention Randomization to either: 1. Observation is the surveillance of asymptomatic stones with periodic CT, ultrasound, or x-ray, and clinic visits. 2. Upfront surgery is the planned removal of asymptomatic stones and includes ureteroscopy (URS) and shockwave lithotripsy (SWL), which are routinely used in standard clinical care. Observational Cohort (Non-Randomized Cohort): Up to 300 eligible subjects who are willing to consent to data collection but refuse randomization will be enrolled and followed for two years. Study Design OASIS is a pragmatic randomized controlled superiority trial conducted at approximately 30 sites that participate in Patient Centered Outcomes Research Network (PCORnet). The trial will include an additional observational cohort for eligible subjects who decline randomization.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

Conditions

Nephrolithiasis Kidney Stone

Interventions

SurgeryPROCEDURE

Upfront surgery is the planned removal of asymptomatic stones and includes ureteroscopy and shockwave lithotripsy, which are routinely used in standard clinical care.

ObservationOTHER

Observation is the surveillance of asymptomatic stones with periodic CT, ultrasound, or x-ray, and clinic visits.

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Individuals age 6 or older 2. Diagnosis of kidney stone disease (nephrolithiasis) 3. Asymptomatic, non-obstructive kidney stones measuring 4 to 10 mm (inclusive) * Asymptomatic is defined as the absence of abdominal pain, flank pain, gross hematuria, and nausea and/or vomiting in the last 3 months that in the judgement of the site PI could be attributable to kidney stones. * Non-obstructive is defined as a stone in a renal calyx or stones in multiple renal calyces on CT, ultrasound, and/or x-ray obtained within 6 months of screening. 4. Informed consent and if appropriate, child assent. 5. Willingness to be randomized to surgery or observation Note: subjects otherwise eligible who decline randomization may be enrolled in a parallel observational cohort. 6. Fluent in English or Spanish 7. Willingness to be audio- and/or video- recorded during interviews Exclusion Criteria: 1. Untreated hydronephrosis or caliectasis 2. Pain (e.g., renal colic, abdominal pain) or gross hematuria attributed to kidney stones. 3. Recurrent symptomatic urinary tract infections (≥3 in 12 months or ≥2 in 6 months). A symptomatic urinary tract infection is defined as having at least one bladder or voiding symptom (e.g., frequency, urgency, dysuria) with bacteriuria (≥105 colony forming units/mL of no more than 2 urinary tract pathogens). 4. Stone in the renal pelvis, ureter, bladder or urethra 5. Pregnant females 6. Solitary kidney, transplant kidney, urinary diversion, medullary sponge kidney 7. Anatomic abnormality of the kidney (e.g., horseshoe kidney) 8. Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures

Locations (2)

University of Minnesota

Minneapolis, Minnesota, United States

NOT_YET_RECRUITING

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

RECRUITING

Outcomes

Primary Outcomes

Healthcare-related life disruption

Composite of: Stone-specific emergency department (ED)/urgent care visits, stone-specific hospitalizations and surgery for stones for new symptoms (Shockwave Lithotripsy (SWL) and Ureteroscopy (URS)) as reported via Patient Centered Outcomes Research Network (PCORnet) Common Data Model (CDM) query and participant self-report on REDCap.

Time frame: Every 3 months (+/- 14 days) from date of consent (observational cohort) or randomization (randomized cohort) until 24 months.

Secondary Outcomes

Patient reported physical and emotional health (Pain Interference)

Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Interference (PI) is a standardized, self-reported assessment tool developed by the National Institutes of Health (NIH). It measures how much pain hinders a person's physical, mental, social, and recreational activities, as well as their sleep and enjoyment of life. It uses a standardized T-score (average is 50; standard deviation is 10) to reflect the level of disruption. Higher numbers mean more pain interference (pain is having a greater negative impact on your daily life, making it harder to participate in work, social, and physical activities). The study team is using the validated versions for adults (18 years and older at time of consent), children (ages 8-17 years at time of consent), and parent-proxy versions for caregivers of children ages (6-7 years at time of consent). This information will be captured via patient report in REDCap.

Time frame: Every 3 months (+/- 14 days) from date of consent (observational cohort) or randomization (randomized cohort) until 24 months.

Patient reported physical and emotional health (Anxiety)

Patient-Reported Outcomes Measurement Information System (PROMIS) Anxiety is a standardized, self-reported assessment tool developed by the National Institutes of Health (NIH). It measures the severity of fear, worry, dread, hyperarousal, and physical stress symptoms over the past seven days on a 5-point scale (ranging from never to always). Raw scores are converted into a standardized T-score with an average of 50 and a standard deviation of 10. A higher T-score indicates a higher severity of anxiety. The study team is using validated versions for adults (18 years and older at time of consent), children (ages 8-17 years at time of consent), and parent-proxy versions for caregivers of children ages (6-7 years at time of consent). This information will be captured via patient report in REDCap.

Central Contacts

Rebecca D McCune, BS, MS

CONTACT

267-909-6030 mccuner@chop.edu

Brian J Augelli, BS

CONTACT

609-504-4908 augellib@chop.edu

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.