Clinical trial NCT01954927: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sickle Cell Disease
Keywords: Pain control
Lead sponsor: St. Jude Children's Research Hospital
Sponsor class: OTHER
Collaborators: name: Scan | Design Foundation
First posted: 2013-10-07
Enrollment: 90
Sex: ALL
Minimum age (years): 1
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 (± 15 minutes) post administration of study drug. Participants who were discharged will be contacted by study staff between 24 and 72 hours following administration of study drug to see if there have been any side effects. Patients who were admitted after administration of the study drug will be monitored through hospital record to determine if any unexpected events occurred. After this follow up, participation in the study is complete.

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.

Primary outcomes:
measure: Number of Participants With Successful Pain Interventions by Arm Between Presentation and 3 Hours Post Administration of Study Drug
timeFrame: Baseline and 3 hours (±30 minutes) post administration of study drug. The 3-hour pain assessment time-period was extended for subjects that were sleep until the first available measurement.
Secondary outcomes:
measure: Morphine Equivalent Doses Administered From Presentation to 3-hours Post Treatment With Gabapentin/Placebo
timeFrame: The 3-hour pain assessment was the pain assessment closest in time to the 3-hour time and was typically within 30 minutes of target. The time period was extended for 12 patients that were sleeping.
Other outcomes:
measure: Number of Participants With Successful Pain Interventions by Arm Between Presentation and Point of Decision for Either Hospital Admission or Discharge to Home
timeFrame: From time of presentation to the acute care setting until time of either discharge to home or admission to the hospital, up to 8 hours.
measure: Morphine Equivalent Doses Administered From Presentation to the Point of Decision for Either Admission or Discharge to Home
timeFrame: From time of presentation to the acute care setting until time of either discharge to home or admission to the hospital, up to 8 hours.
measure: Hospital Admission
timeFrame: From time of presentation to the acute care setting until time of either discharge to home or admission to the hospital, up to 8 hours.
measure: Absolute Change in Pain From Study Drug to 3 Hours Post Administration of Study Drug
timeFrame: Study drug administration to 3-hours post study drug administration
measure: Absolute Change in Pain, Study Drug to Hospital Discharge Decision
timeFrame: From time of presentation to the acute care setting until time of either discharge to home or admission to the hospital, up to 8 hours.
Locations:
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
Study officials:
affiliation: St. Jude Children's Research Hospital
name: Doralina Anghelescu, MD
role: PRINCIPAL_INVESTIGATOR
References: