A Phase 1 interventional study of Dronabinol 2.5 MG and Microcrystalline cellulose in Sickle Cell Disease, sponsored by Yale University. Terminated at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-01-26.
Sponsored by Yale University · Phase 1, Interventional, and Treatment
This study is designed to address the feasibility of a randomized, double masked, cross-over study of dronabinol as a palliative agent in the treatment of pain, inflammation, and other complications of sickle cell disease (SCD).
The Primary Hypothesis is that such a study will be feasible as defined by subject adherence to study medication and study procedures and avoidance of other cannabinoid containing substances during the trial period as well as by ability to mask subjects and investigators to treatment assignment
Secondary hypotheses are:
Dronabinol will:
Reduce patient-reported pain interference Reduce patient-reported pain scores and change patient-reported pain quality. Reduce use of opioid pain medications. Improve patient-reported stiffness, nausea and vomiting, sleep, mood, anxiety, and social functioning.
Reduce markers of inflammation.
1,103 studies on the registry are indexed under Anemia, Sickle Cell; 235 are open to participants now.
This study's enrollment of 6 is below the median of 40 across 750 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →Yale University is the lead sponsor of 1,724 studies on the registry; 298 are open to participants now.
Of its 210 completed or terminated interventional studies of FDA-regulated products, 126 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Participants will take 2.5 mg of Dronabinol for 2 weeks, 1 week washout and then take 2 weeks of placebo (microcrystalline cellulos). Subjects will take up to 8 capsules daily of the treatment daily during each phase.
Drug: Dronabinol 2.5 MG · Drug: Microcrystalline cellulose
Participants will take placebo (microcrystalline cellulos) for 2 weeks, 1 week washout and then take 2.5 mg of Dronabinol for 2 weeks. Subjects will take up to 8 capsules daily of the treatment daily during each phase.
Drug: Dronabinol 2.5 MG · Drug: Microcrystalline cellulose
Subjects will take dronabinol daily during 2 week dosing period separated by a one week washout period.
Subjects will take placebo daily during 2 week dosing period separated by a one week washout period.
Also known as: Placebo
Feasibility will be defined by ability to mask patients and investigators to treatment assignment and adherence
Feasibility will be defined by ability to mask patients and investigators to treatment assignment and adherence which will be defined in 3 ways.
Time frame: 1 year
Adherence
Adherence to study drug will be assessed with weekly pill counts and urine toxicology.
Time frame: 1 year
Avoidance
Avoidance of other cannabinoid containing substances will also be defined using urine toxicology once at the end of each treatment period and once at the end of the wash out period.
Time frame: 7 weeks
Adherence to other study proceedures
Adherence to other study procedures will be defined as percent of visits attended, percent of urine and serum studies collected and percent of patient reported outcomes and daily pain severity and pain unpleasentness journals returned.
Time frame: 7 weeks
Patient reported 7-day pain interference
The primary endpoint for this study will be second treatment week 7-day pain impact as measured by the Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) 7-day pain impact domain. ASCQ-Me domains are validated to measure pain and other quality of life outcomes in adults with Sickle Cell Disease (SCD) of all genotypes. The published mean for the validated scores is 50 and the standard deviation is 10.56 Lower scores represent more severe symptoms. When the validation cohort was placed into tertiles of severity, the tertile with most severe disease had a mean pain score of 53, the middle tertile had a score of 49, and the least severe disease tertile had a mean score of 46. This suggests that a change of 4 has clinical significance and a change of 7 has even more clinical significance. We have chosen a priori to define a difference in pain impact score of 5 points as showing superiority.
Time frame: 7 days
Patient Pain Severity
Daily reports of pain severity on a numeric rating scale of 0-10 with 0 representing the least severe pain (no pain) and 10 representing the most severe pain.
Time frame: end of 2nd week
Patient Pain Unpleasantness
Daily reports of pain unpleasantness on a numeric rating scale of 0-10 with 0 representing the least pain unpleasantness (no unpleasantness) and 10 representing the most unpleasant pain.
Time frame: end of 2nd week
PROMIS (Patient Reported Outcomes Measurement Information System) Nociceptive Pain Severity
Nociceptive pain quality as defined by the patient report outcome measurement information system (PROMIS) nociceptive pain quality 5a scale. All PROMIS scores will be presented as T scores. The T score is a standardized score with a mean of 50 and a standard deviation of 10. The value of 50 represents the score of the average field test respondent.
Time frame: end of 2nd week
PROMIS Neuropathic Pain Severity
Neuropathic pain quality as defined by the patient report outcome measurement information system (PROMIS) neuropathic pain quality 5a scale.All PROMIS scores will be presented as T scores. The T score is a standardized score with a mean of 50 and a standard deviation of 10. The value of 50 represents the score of the average field test respondent.
Time frame: end of 2nd week
PROMIS Gastrointestinal Nausea short form measure
PROMIS short form Gastrointestinal Nausea and vomiting 4a. All PROMIS scores will be presented as T scores. The T score is a standardized score with a mean of 50 and a standard deviation of 10. The value of 50 represents the score of the average field test respondent.
Time frame: end of 2nd week
PROMIS short form for emotional distress anxiety 8a.
PROMIS short form Gastrointestinal Nausea and vomiting 4a. All PROMIS scores will be presented as T scores. The T score is a standardized score with a mean of 50 and a standard deviation of 10. The value of 50 represents the score of the average field test respondent.
Time frame: end of 2nd week
Adult Sickle Cell Quality (ASCQ)-Me short form measures of emotional impact
Each ASCQ-Me score is reported on the same standardized scale with a mean of 50 and a standard deviation of 10. The value of 50 represents the health score of the average field test respondent.
Time frame: end of 2nd week
Adult Sickle Cell Quality (ASCQ)-Me short form measure of sleep impact
Each ASCQ-Me score is reported on the same standardized scale with a mean of 50 and a standard deviation of 10. The value of 50 represents the health score of the average field test respondent.
Time frame: end of 2nd week
Adult Sickle Cell Quality (ASCQ)-Me short form measure of stiffness impact
Each ASCQ-Me score is reported on the same standardized scale with a mean of 50 and a standard deviation of 10. The value of 50 represents the health score of the average field test respondent.
Time frame: end of 2nd week
Adult Sickle Cell Quality (ASCQ)-Me short form measure of social functioning impact
Each ASCQ-Me score is reported on the same standardized scale with a mean of 50 and a standard deviation of 10. The value of 50 represents the health score of the average field test respondent.
Time frame: end of 2nd week
Opioid Utilization
Secondary comparisons will be made between subjects on dronabinol to themselves on placebo for patient reported outcomes for opioid use. Opioid utilization will be based on reports of opioids dispensed obtained from the Connecticut Prescription Monitoring Program. Reports will be in the form of average daily opioids used in oral morphine equivalents.
Time frame: end of 2nd week
Markers of Inflammation Concentration of white blood cell count differential
Secondary comparisons will be made between subjects on dronabinol to themselves on placebo for differential white blood cell count at the end of the second week of the treatment period.
Time frame: end of 2nd week
Markers of Inflammation C reactive protein
Secondary comparisons will be made between subjects on dronabinol to themselves on placebo of C reactive protein at the end of the second week of the treatment period.
Time frame: end of 2nd week
Markers of Inflammation serum tryptase
Secondary comparisons will be made between subjects on dronabinol to themselves on placebo for serum tryptase at the end of the second week of the treatment period.
Time frame: end of 2nd week
Serum pro-inflammatory cytokines
Secondary comparisons will be made between subjects on dronabinol to themselves on placebo for pro-inflammatory cytokines at the end of the second week of the treatment period.
Time frame: end of 2nd week
Serum measure of Substance P
Secondary comparisons will be made between subjects on dronabinol to themselves on placebo for Substance P at the end of the second week of the treatment period.
Time frame: end of 2nd week
This study is terminated, as verified in Jan 2021. You cannot join it, but the record below documents what was studied.
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