Pain Management in Children and Young Adults With Sickle Cell Disease
Condition: Sickle Cell Disease
MeSH terms: Anemia, Sickle Cell
Study type: Interventional
Phase: Phase 2

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.
    

Intervention name(s): Gabapentin; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): 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 900 mg.; 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.
Other intervention name(s): Neurontin(R)
Intervention MeSH terms: Gabapentin
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Pain control