Pain Management in Children and Young Adults With Sickle Cell Disease | RxWiki

Pain Management in Children and Young Adults With Sickle Cell Disease

Overview[ - collapse ][ - ]

Purpose	This is a phase II double-blind placebo-controlled clinical trial evaluating the effect of gabapentin when added to standard pain management for patients with sickle cell disease experiencing acute pain crisis in the ambulatory care setting. Sickle cell pain is different for every patient. Some patients get complete relief from routine pain medicines, and others need more time or more doses of pain medicines before the pain goes away completely. It is known that humans have many types of pain, including something called neuropathic pain. Neuropathic pain in other conditions (such as diabetes) has been treated successfully with a medicine called gabapentin. The investigators in this study suspect that some sickle cell pain is a combination of pain types. They would like to see if adding gabapentin to the usual pain medicines makes pain go away faster or more completely. Primary Objective: - To assess the analgesic efficacy of gabapentin vs. placebo for pain during vaso-occlusive crisis (VOC) in participants with sickle cell disease (SCD). A response to study drug will be defined by a decrease in pain score of ≥ 33% between presentation to the acute care setting and assessment at 3 hours post administration of study drug. Secondary Objective: - To compare the total morphine equivalent dose (mg/kg) used to control pain during VOC between presentation to the acute care setting and assessment at 3 hours post administration of study drug in the gabapentin vs. placebo groups.
Condition	Sickle Cell Disease
Intervention	Drug: Gabapentin Drug: Placebo
Phase	Phase 2
Sponsor	St. Jude Children's Research Hospital
Responsible Party	St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier	NCT01954927
First Received	September 27, 2013
Last Updated	April 4, 2014
Last verified	April 2014

Tracking Information[ + expand ][ + ]

First Received Date	September 27, 2013
Last Updated Date	April 4, 2014
Start Date	October 2013
Estimated Primary Completion Date	October 2017
Current Primary Outcome Measures	Number and proportion of participants with successful pain interventions by arm [Time Frame: Baseline and 3 hours post administration of study drug] [Designated as safety issue: No]Pain scales used are the numerical rating system, the Faces Pain Scale, and the FLACC pain scale (for patients 7 years or older, ages 4-6 years, or less than 4 years, respectively). For each patient, if the reduction of the pain scores (0=no pain and 10=worst possible pain) between presentation to the acute care setting and 3 hours post administration of study drug is 33% or greater, then this patient will be defined as having a successful intervention. The proportions of successful interventions in the gabapentin and placebo groups will be estimated and compared using Z-test.
Current Secondary Outcome Measures	Total dose (mg) of opioid analgesic administered [Time Frame: All doses given between baseline and 3 hours post administration of study drug] [Designated as safety issue: No]Summary statistics of the total morphine dose or morphine equivalent (mg/kg) used to control pain during VOC between presentation to the acute care setting and 3 hours post administration of study drug, in the gabapentin and placebo groups will be provided. Test of normality such as Shapiro-Wilk test will be applied to the total morphine dose or morphine equivalent (mg/kg) to examine their deviation from the normal distribution. A two-sample t-test or Wilcoxon rank sum test will be used to compare the total morphine dose or morphine equivalent (mg/kg) for the gabapentin vs. placebo groups depending on whether the normality assumption of the data holds.

Descriptive Information[ + expand ][ + ]

Brief Title	Pain Management in Children and Young Adults With Sickle Cell Disease
Official Title	Pain Management of Vaso-Occlusive Crisis in Children and Young Adults With Sickle Cell Disease
Brief Summary	This is a phase II double-blind placebo-controlled clinical trial evaluating the effect of gabapentin when added to standard pain management for patients with sickle cell disease experiencing acute pain crisis in the ambulatory care setting. Sickle cell pain is different for every patient. Some patients get complete relief from routine pain medicines, and others need more time or more doses of pain medicines before the pain goes away completely. It is known that humans have many types of pain, including something called neuropathic pain. Neuropathic pain in other conditions (such as diabetes) has been treated successfully with a medicine called gabapentin. The investigators in this study suspect that some sickle cell pain is a combination of pain types. They would like to see if adding gabapentin to the usual pain medicines makes pain go away faster or more completely. Primary Objective: - To assess the analgesic efficacy of gabapentin vs. placebo for pain during vaso-occlusive crisis (VOC) in participants with sickle cell disease (SCD). A response to study drug will be defined by a decrease in pain score of ≥ 33% between presentation to the acute care setting and assessment at 3 hours post administration of study drug. Secondary Objective: - To compare the total morphine equivalent dose (mg/kg) used to control pain during VOC between presentation to the acute care setting and assessment at 3 hours post administration of study drug in the gabapentin vs. placebo groups.
Detailed Description	Upon participant enrollment, study staff will randomize the participant to one of 2 possible treatment arms: a single dose of gabapentin or a single dose of placebo. Morphine or other opioid and non-steroidal anti-inflammatory drugs will be available to both groups as needed for pain and will be administered according to the current standard of care for pain in VOC from the Department of Hematology at St. Jude Children's Research Hospital (SJCRH). Randomization will be performed in the SJCRH pharmacy by a pharmacist. The randomization will be stratified by three age categories (1-3 years of age, 4-6 years, and 7 years or older) for which distinct pain assessment tools are applied and for 2 pain score categories at assessment at presentation (4-6 and 7-10, respectively). A block randomization with block sizes varying randomly between 4 and 6 will be used in each stratum. Pain scores will be obtained at presentation to the acute care setting and 3 hours post administration of study drug. One to three days after taking the study drug, a member of the study team will contact the participant to see if there have been any side effects. After this follow up conversation, participation in the study is complete.
Study Type	Interventional
Study Phase	Phase 2
Study Design	Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Condition	Sickle Cell Disease
Intervention	Drug: Gabapentin Gabapentin is supplied as an oral suspension. Patients randomized to the gabapentin arm will receive a single dose of gabapentin as soon after enrollment as feasible. The gabapentin dose will be given orally and will be approximately 15 mg/kg with a maximum dose of 900mg. Other Names: Neurontin(R)Drug: Placebo Placebo will be prepared by the SJCRH pharmacy and will be similar in appearance, quantity and taste to the gabapentin drug. Patients randomized to the placebo arm will receive a single dose of placebo as soon after enrollment as feasible. The placebo dose will be given orally and will be approximately 15 mg/kg with a maximum dose of 900mg.
Study Arm (s)	Active Comparator: Gabapentin Patients will be randomized to receive one dose of gabapentin. Placebo Comparator: Placebo Patients will be randomized to receive one dose of placebo.

Recruitment Information[ + expand ][ + ]

Recruitment Status	Recruiting
Estimated Enrollment	190
Estimated Completion Date	October 2017
Estimated Primary Completion Date	October 2017
Eligibility Criteria	Inclusion Criteria: - Participant must have sickle cell disease (any genotype) documented in the St. Jude medical record. - Participant must be seeking care for acute vaso-occlusive pain at St. Jude Children's Research Hospital. - Participant age must be ≥1 year and <21 years. Exclusion Criteria: - Prior randomization in this study. - Mild pain (score <4) or pain for which treatment with opioid is not indicated. - Pregnant or lactating female. - Decreased glomerular filtration rate (GFT) (<60ml/min/1.73m^2) as estimated by the revised Schwartz equation. - Current treatment with gabapentinoid drugs (gabapentin or pregabalin). - Known seizure disorder. - Current treatment with antiepileptic agents. - Pain in combination with other clinical symptoms that require additional interventions, including fever with focus, acute chest syndrome, acute injury, or splenic sequestration. - Allergy to gabapentin. - Current participation in another research study with an investigational new drug/device (IND/IDE) agent. - Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
Gender	Both
Ages	1 Year
Accepts Healthy Volunteers	No
Contacts	Contact: Doralina Anghelescu, MD 866-278-5833 info@stjude.org
Location Countries	United States

Administrative Information[ + expand ][ + ]

NCT Number	NCT01954927
Other Study ID Numbers	PMVOC
Has Data Monitoring Committee	No
Information Provided By	St. Jude Children's Research Hospital
Study Sponsor	St. Jude Children's Research Hospital
Collaborators	Scan / Design Foundation
Investigators	Principal Investigator: Doralina Anghelescu, MD St. Jude Children's Research Hospital
Verification Date	April 2014

Locations[ + expand ][ + ]