Pain Management in Children and Young Adults With Sickle Cell Disease

NCT01954927 | Completed Phase 2 Results FDA Drug

• 1 other identifier: PMVOC
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 1

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.

Conditions

Sickle Cell Disease

Keywords

Pain control

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Successful Pain Interventions by Arm Between Presentation and 3 Hours Post Administration of Study Drug

  Pain scales used are the numerical rating system, the Faces Pain Scale, and the Faces, Legs, Arms, Cry and Consolability (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.

  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 (1)

• Morphine Equivalent Doses Administered From Presentation to 3-hours Post Treatment With Gabapentin/Placebo

  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 (5)

• Number of Participants With Successful Pain Interventions by Arm Between Presentation and Point of Decision for Either Hospital Admission or Discharge to Home

  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.

• Morphine Equivalent Doses Administered From Presentation to the Point of Decision for Either Admission or Discharge to Home

  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.

• Hospital Admission

  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.

Study Arms (2)

Gabapentin

ACTIVE COMPARATOR

Patients will be randomized to receive one dose of gabapentin.

Drug: Gabapentin

Placebo

PLACEBO COMPARATOR

Patients will be randomized to receive one dose of placebo.

Drug: Placebo

Interventions

Gabapentin DRUG

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.

Also known as: Neurontin(R)

Gabapentin

Placebo DRUG

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.

Placebo

Eligibility Criteria

• Age: 1 Year - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead
• Scan | Design Foundation: collaborator

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

MeSH Terms

Conditions

Anemia, Sickle Cell; Agnosia

Interventions

Gabapentin

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Perceptual Disorders; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Amines; Organic Chemicals; gamma-Aminobutyric Acid; Aminobutyrates; Butyrates; Acids, Acyclic; Carboxylic Acids; Cyclohexanecarboxylic Acids; Acids, Carbocyclic; Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Hydrocarbons; Amino Acids; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Doralina Anghelescu, MD
• Organization: St. Jude Children's Research Hospital

doralina.anghelescu@stjude.org

(901) 595-4034

Study Officials

• Doralina Anghelescu, MD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 27, 2013
• First Posted: October 7, 2013
• Study Start: October 7, 2013
• Primary Completion: January 3, 2018
• Study Completion: January 3, 2018
• Last Updated: April 2, 2019
• Results First Posted: April 2, 2019

Record last verified: 2018-07

Locations

US (1)