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| Name | Class |
|---|---|
| Scan | Design Foundation | UNKNOWN |
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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:
Secondary Objective:
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Gabapentin | Active Comparator | Patients will be randomized to receive one dose of gabapentin. |
|
| Placebo | Placebo Comparator | Patients will be randomized to receive one dose of placebo. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Morphine Equivalent Doses Administered From Presentation to 3-hours Post Treatment With Gabapentin/Placebo | The equivalent dose of morphine in mg | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Successful Pain Interventions by Arm Between Presentation and Point of Decision for Either Hospital Admission or Discharge to Home | 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 Point of decision for either hospital admission or discharge to home is 33% or greater, then this patient will be defined as having a successful intervention. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Doralina Anghelescu, MD | St. Jude Children's Research Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | United States |
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| Label | URL |
|---|---|
| St. Jude Children's Research Hospital | View source |
| Clinical Trials Open at St. Jude | View source |
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The study was stopped early due to slow accrual when there were 82 patients with evaluable pain assessments available for the primary objective. Patients asleep at pain assessment times led to missing pain assessments.
Participants meeting eligibility criteria were enrolled between 10/7/2013 to 1/3/2018. They were randomized to either gabapentin or placebo.
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| ID | Title | Description |
|---|---|---|
| FG000 | Gabapentin | Participants were randomized to receive one dose of gabapentin. Gabapentin: Gabapentin is supplied as an oral suspension. Patients randomized to the gabapentin arm received a single dose of gabapentin as soon after enrollment as feasible, given orally, approximately 15 milligram/kilogram (mg/kg) with a maximum dose of 900 milligram (mg). |
| FG001 | Placebo | Participants were randomized to receive one dose of placebo. Placebo: Placebo was prepared by the SJCRH pharmacy, similar in appearance, quantity and taste to the gabapentin drug. Patients randomized to the placebo arm received a single dose of placebo as soon after enrollment as feasible, given orally, in a volume that matched the active medication arm. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Gabapentin | Participants were randomized to receive one dose of gabapentin. Gabapentin: Gabapentin is supplied as an oral suspension. Patients randomized to the gabapentin arm received a single dose of gabapentin as soon after enrollment as feasible, given orally, approximately 15 milligram/kilogram (mg/kg) with a maximum dose of 900 milligram (mg). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Patients with Sickle cell disease (any genotype) ages ≥1 year and <21 years seeking care for acute vaso-occlusive pain at St Jude Children's Hospital. A total of 4 subjects were missing the pain assessment at 3 hours (2 in each arm). The resulting sample sizes were n=40 (Gabapentin arm) and n=42 (placebo arm), for a total sample size of n=82. | Posted | Count of Participants | Participants | 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. |
Adverse events were evaluated by one phone contact within the following 72 hours.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Gabapentin | Participants were randomized to receive one dose of gabapentin. Gabapentin: Gabapentin is supplied as an oral suspension. Patients randomized to the gabapentin arm received a single dose of gabapentin as soon after enrollment as feasible, given orally, approximately 15 mg/kg with a maximum dose of 900 mg. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dizziness | Nervous system disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Doralina Anghelescu, MD | St. Jude Children's Research Hospital | (901) 595-4034 | doralina.anghelescu@stjude.org |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jul 2, 2018 | Jan 3, 2019 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D000377 | Agnosia |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D000077206 | Gabapentin |
| ID | Term |
|---|---|
| D000588 | Amines |
| D009930 | Organic Chemicals |
| D005680 | gamma-Aminobutyric Acid |
| D000613 | Aminobutyrates |
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|
|
| 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. |
|
| 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 | To compare the total morphine equivalent dose (mg/kg) used to control pain during VOC between presentation to the acute care setting and the point of decision for either admission or discharge to home, in the gabapentin and placebo groups. | 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 | To compare the rate of admission related to pain management, in the gabapentin vs. placebo groups. (Outcome: binary response - admitted or discharged) | 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. |
| Absolute Change in Pain From Study Drug to 3 Hours Post Administration of Study Drug | To compare the change in pain score from time of administration of study drug to assessment at 3 hours post administration of study drug in the gabapentin vs. placebo groups. (0=no pain and 10=worst possible pain) | Study drug administration to 3-hours post study drug administration |
| Absolute Change in Pain, Study Drug to Hospital Discharge Decision | To compare the change in pain score from time of administration of study drug to the point of decision for either admission or discharge to home, in the gabapentin and placebo groups. (0=no pain and 10=worst possible pain) | 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. |
| BG001 |
| Placebo |
Participants were randomized to receive one dose of placebo. Placebo: Placebo was prepared by the SJCRH pharmacy, similar in appearance, quantity and taste to the gabapentin drug. Patients randomized to the placebo arm received a single dose of placebo as soon after enrollment as feasible, given orally, in a volume that matched the active medication arm. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Median | Inter-Quartile Range | years |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Sickle cell genotype | Count of Participants | Participants |
|
| Pain score category at presentation | (0=no pain and 10=worst possible pain) | Count of Participants | Participants |
|
| Pain at presentation (before randomization) | (0=no pain and 10=worst possible pain) | Mean | Standard Deviation | units on a scale |
|
| Pain at presentation (before randomization) | (0=no pain and 10=worst possible pain) | Median | Inter-Quartile Range | units on a scale |
|
| ID | Title | Description |
|---|---|---|
| OG000 | Gabapentin | Participants were randomized to receive one dose of gabapentin. Gabapentin: Gabapentin is supplied as an oral suspension. Patients randomized to the gabapentin arm received a single dose of gabapentin as soon after enrollment as feasible, given orally, approximately 15 mg/kg with a maximum dose of 900 mg. |
| OG001 | Placebo | Participants were randomized to receive one dose of placebo. Placebo: Placebo was prepared by the SJCRH pharmacy, similar in appearance, quantity and taste to the gabapentin drug. Patients randomized to the placebo arm received a single dose of placebo as soon after enrollment as feasible, given orally, in a volume that matched the active medication arm. |
|
|
|
| Secondary | Morphine Equivalent Doses Administered From Presentation to 3-hours Post Treatment With Gabapentin/Placebo | The equivalent dose of morphine in mg | Patients with Sickle cell disease (any genotype) ages ≥1 year and <21 years seeking care for acute vaso-occlusive pain at St Jude Children's Hospital. | Posted | Median | Inter-Quartile Range | mg/kg | 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 Pre-specified | Number of Participants With Successful Pain Interventions by Arm Between Presentation and Point of Decision for Either Hospital Admission or Discharge to Home | 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 Point of decision for either hospital admission or discharge to home is 33% or greater, then this patient will be defined as having a successful intervention. | Patients with Sickle cell disease (any genotype) ages ≥1 year and <21 years seeking care for acute vaso-occlusive pain at St Jude Children's Hospital. There were 2 subjects in the Gabapentin group that did not have a pain assessment admit/discharge decision. N=84 patients had assessments at both presentation and admit/discharge. | Posted | Count of Participants | Participants | 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. |
|
|
|
|
| Other Pre-specified | Morphine Equivalent Doses Administered From Presentation to the Point of Decision for Either Admission or Discharge to Home | To compare the total morphine equivalent dose (mg/kg) used to control pain during VOC between presentation to the acute care setting and the point of decision for either admission or discharge to home, in the gabapentin and placebo groups. | Patients with Sickle cell disease (any genotype) ages ≥1 year and <21 years seeking care for acute vaso-occlusive pain at St Jude Children's Hospital. | Posted | Median | Inter-Quartile Range | mg/kg | 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. |
|
|
|
|
| Other Pre-specified | Hospital Admission | To compare the rate of admission related to pain management, in the gabapentin vs. placebo groups. (Outcome: binary response - admitted or discharged) | Patients with Sickle cell disease (any genotype) ages ≥1 year and <21 years seeking care for acute vaso-occlusive pain at St Jude Children's Hospital. | Posted | Count of Participants | Participants | 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. |
|
|
|
|
| Other Pre-specified | Absolute Change in Pain From Study Drug to 3 Hours Post Administration of Study Drug | To compare the change in pain score from time of administration of study drug to assessment at 3 hours post administration of study drug in the gabapentin vs. placebo groups. (0=no pain and 10=worst possible pain) | Patients with Sickle cell disease (any genotype) ages ≥1 year and <21 years seeking care for acute vaso-occlusive pain at St Jude Children's Hospital. Placebo arm: 6 subjects missed pain assessments at treatment; 2 missed pain assessment at 3h. Gabapentin arm: 7 missed pain assessment at treatment; 2 of the 7 also missed the assessment at 3h. | Posted | Median | Inter-Quartile Range | score on a scale | Study drug administration to 3-hours post study drug administration |
|
|
|
|
| Other Pre-specified | Absolute Change in Pain, Study Drug to Hospital Discharge Decision | To compare the change in pain score from time of administration of study drug to the point of decision for either admission or discharge to home, in the gabapentin and placebo groups. (0=no pain and 10=worst possible pain) | Patients with Sickle cell disease (any genotype) ages ≥1 year and <21 years seeking care for acute vaso-occlusive pain at St Jude Children's Hospital. Placebo arm: 6 subjects missed pain assessments at treatment. Gabapentin arm: 7 subjects missed pain assessment at treatment; 2 of the 7 missed the assessment at hospital discharge decision. | Posted | Median | Inter-Quartile Range | score on a scale | 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. |
|
|
|
|
| 0 |
| 42 |
| 0 |
| 42 |
| 5 |
| 42 |
| EG001 | Placebo | Participants were randomized to receive one dose of placebo. Placebo: Placebo was prepared by the SJCRH pharmacy, similar in appearance, quantity and taste to the gabapentin drug. Patients randomized to the placebo arm received a single dose of placebo as soon after enrollment as feasible, given orally, in a volume that matched the active medication arm. | 0 | 44 | 0 | 44 | 2 | 44 |
| Fatigue | General disorders | Non-systematic Assessment |
|
| Headache | General disorders | Non-systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Respiratory, thoracic and mediastinal disorders - Other | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | Non-systematic Assessment |
|
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| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D010468 | Perceptual Disorders |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D002087 |
| Butyrates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D003509 | Cyclohexanecarboxylic Acids |
| D000146 | Acids, Carbocyclic |
| D003510 | Cyclohexanes |
| D003516 | Cycloparaffins |
| D006840 | Hydrocarbons, Alicyclic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |