A Phase 3 interventional study of Pregabalin in Pain, Neuropathic, Itch and Epidermolysis Bullosa, sponsored by The Hospital for Sick Children. Completed at 1 site in Canada. Open to participants aged 8 Years to 40 Years. Per ClinicalTrials.gov, last updated 2025-01-22.
Sponsored by The Hospital for Sick Children · Phase 3, Interventional, and Treatment
Recessive dystrophic epidermolysis bullosa (RDEB) patients' quality of life is severely affected by neuropathic pain and itch, which have recently been demonstrated to be secondary to skin small fiber neuropathy. To date, there is no evidence on what the best agent is to control these symptoms. Based on the anecdotal data and safety profile, the investigators believe that pregabalin is a therapeutic agent that will be effective and safe in this population. The investigators propose to conduct a blinded study, using pregabalin versus placebo in which each patient serves as its own control (cross-over design). This is a feasibility study that will provide preliminary data on efficacy and safety of pregabalin in RDEB patients with neuropathic pain and itch and gather much needed data (dosage, titration schedule, outcome measures, etc) to inform the design of a larger cohort, controlled, multicenter trial.
Neuropathic pain and itch are significant symptoms that affect RDEB patients' quality of life. To date, there is no evidence on what the best agent is to control these symptoms.
Based on the anecdotal data and the safety profile, the investigator believes that pregabalin is a therapeutic agent that will be effective and safe in this population.
This is a pilot, randomized, double-blinded, cross-over trial of pregabalin vs placebo for the treatment of RDEB associated neuropathic pain and itch. The study will run for 24 weeks and will have 4 separate phases with 6 overall visits: 1. Observation/wash out period (2 weeks); 2. ARM-1, ½ of the patients will receive pregabalin(A) and ½ the placebo (B) (10 weeks); 3. Wash out period (2 weeks) and 4. ARM-2 (patients who received placebo will receive pregabalin and pregabalin will receive placebo (10 weeks).
Patients will be recruited during the regular clinic visits or invited to participate via a letter followed by 2 phone calls.
Screening/Wash out period #1 (Visit 0)to assess eligibility criteria and the background pain and itch level (2 weeks), as well as the effectiveness of patients' "standard pain/itch interventions". Throughout this phase and the rest of the study, patients will receive pain and itch medications, except those that interfere with the pregabalin (see exclusion criteria).
ARM1 intervention study period (Visits 1 \& 2): ½ of the patients will receive the active intervention (Pregabalin) and ½ the placebo. This phase will be of 10 weeks duration and will consist of 4 weeks of escalating dose until the desired maximum, 4 weeks of active treatment and 2 weeks of titration down (see titration schedule in the Appendix 1)Patients will receive either active intervention (pregabalin) or placebo including instruction of how to administer them.
Visit 1: (Week 2)
Visit 2: (Week 10)
Wash out period #2 (2 weeks) Visit 3 (Week 14)
ARM2 intervention study period (Visits 4 \& 5 (Week 22 and 24): patients randomized to placebo will receive pregabalin and those on placebo will get the pregabalin conducted similarly as in ARM1
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Exclusion Criteria:
The study has a crossover design. Participants in this arm will receive pregabalin during the first study treatment period for ten weeks and placebo during their second ten-week treatment period . The dose will depend on the participant's weight and phase of treatment period. Each treatment period consists of 4 weeks of escalating dose until the desired maximum , 4 weeks of active treatment and 2 weeks of titrating down.
Drug: Pregabalin
Participants will receive placebo during the first treatment period of the study(10 weeks) followed by 10 weeks of pregabalin treatment . The dose will depend on the participant's weight and phase of the treatment period . Each treatment period consists of 3 phases: 4 weeks of escalating dose until the desired maximum, 4 weeks of active treatment and 2 weeks of titrating down.
Drug: Pregabalin
Pregabalin /placebo capsules will be taken by mouth and will be prescribed for the study participants in the doses, depending on their weight and treatment phase: Participant \< 25 kg at baseline will start with 50 mg per day, increasing the dose by 50 mg each week until their maximum dose 200 mg per day; Participants who are greater than or equal to 25 kg at baseline will start with 100 mg per day, increasing their dose by 50 mg each week until their target dose 300 mg per day is achieved. The study medication will be taken twice per day.The dose will be weaned down every 1-2 days by 25 mg in the last two weeks of each treatment period.
Also known as: Placebo
Difference in the mean pain scores between pregabalin and placebo group: VAS
It will be measured as difference in the mean pain values pre- and post-intervention for each group using the Visual Analog Scale (VAS).It is a 10 cm line with anchor statements on the left (no pain) and on the right (worst pain ever). The patient is asked to mark their pain level on the line.The pain is scored using the VAS by measuring the distance in centimeters (0-10) from the "no pain" anchoring point.
Time frame: 12 weeks
Proportion of patients on pregabalin achieving a ≥75% reduction in their mean pain intensity score across last 7 days of treatment compared to baseline/wash-out
Proportion of patients on pregabalin achieving a ≥75% reduction in their mean pain intensity score across last 7 days of treatment compared to baseline/wash-out
Time frame: 12 weeks
Proportion of patients on pregabalin achieving a ≥50% reduction in their mean pain intensity score across last 7 days of treatment compared to baseline/wash-out
Proportion of patients on pregabalin achieving a ≥50% reduction in their mean pain intensity score across last 7 days of treatment compared to baseline/wash-out
Time frame: 12 weeks
Difference in the average pain score assessed using VAS between first and second treatment periods (period effect)
Difference in the average pain score assessed using VAS, reported by the patient between first and second treatment periods. Visual Analog Scale (VAS) is a 10 cm line with anchor statements on the left (0 -no pain) and on the right (10- worst pain ever). The patient is asked to mark their pain level on the line. The pain is scored using the VAS by measuring the distance in centimeters (0-10) from the "no pain" anchoring point.
Time frame: 24 weeks
Proportion of patients on pregabalin achieving a ≥75% reduction in their mean itch intensity score across last 7 days of treatment compared to baseline/wash-out
Proportion of patients on pregabalin achieving a ≥75% reduction in their mean itch intensity score across last 7 days of treatment compared to baseline/wash-out.The intensity of itch is measured using the Visual Analog Scale for itch(VAS).It is a 10 cm line with anchor statements on the left (no itching) and on the right (worst possible itching). The patient is asked to mark the intensity of itch on the line. The itch is scored using the VAS by measuring the distance in centimeters (0-10)from the "no itch" anchoring point.
Time frame: 24 weeks
Proportion of patients on pregabalin achieving a ≥50% reduction in their mean itch intensity score across last 7 days of treatment compared to baseline/wash-out
Proportion of patients on pregabalin achieving a ≥50% reduction in their mean itch intensity score across last 7 days of treatment compared to baseline/wash-out
Time frame: 12 weerks
Difference in the average itch score as assessed using VAS between the first and the second treatment periods (period effect).
Difference in the average itch score as assessed using VAS( reported by the patient) between the first and the second treatment periods (period effect). The intensity of itch is measured using the Visual Analog Scale for itch (VAS).It is a 10 cm line with anchor statements on the left (0- no itching) and on the right (10 -worst possible itching). The patient is asked to mark the intensity of itch on the line. The itch is scored using the VAS by measuring the distance in centimeters (0-10) from the "no itch" anchoring point.
Time frame: 24 weeks
Changes in the Quality of Life in Epidermolysis Bullosa Questionnaire score(QOLEB) for patients >18yrs in the intervention versus placebo
Changes in the QOLEB for patients \>18yrs in the intervention versus placebo. QOLEB is a measurement tool containing 17questions.Each question has a score range from 0-3.Total score range is between 0 and 51. iscorEB is a measurement tool for evaluating the disease severity in EB patient.It evaluates the cutaneous,mucosal and other organ impact of EB and includes clinician and patient reported outcomes in a single instrument. Score ranges between 0-120 for both reported outcomes.
Time frame: 24 weeks
Changes in iscorEB (instrument for scoring clinical outcomes for research of EB), patient portion score in the intervention versus placebo
iscorEB is a measurement tool for evaluating the disease severity in EB patient. It evaluates the cutaneous, mucosal and other organ impact of EB and includes clinician and patient reported outcomes in a single instrument. Score ranges between 0-120 for both reported outcomes. Patient portion of this instrument contains 15 questions. Each question has a score range from 0-8.Total score range for iscorEB patient portion is between 0 and 120. Reduction in iscorEB scores indicates improvement. An increase in score indicates deterioration.
Time frame: 24 weeks
Correlation between pain score (as measured by VAS), iscorEB patient portion score and QOLEB (for patients over 18 yrs) total scores.
Correlation between pain score, as measured by Visual Analog Scale (VAS), iscorEB (instrument for scoring clinical outcomes for research of EB)- patient portion score, and Quality of Life in Epidermolysis Bullosa Questionnaire (QOLEB) score (for patients over 18 years). Please see full description of the mentioned instruments above.
Time frame: 24 weeks
Correlation between pain score( as measured by VAS) and iscorEB clinician portion score and Epidermolysis Bullosa Disease Activity and Scarring Index(EBDASI) score.
Correlation between pain score(as measured by Visual Analog Scale), iscorEB (instrument for scoring clinical outcomes for research of EB)- clinician portion score and EBDASI total score.Total EBDASI score ranges of 0-42, 43-106 and 107-506 corresponded to mild, moderate and severe disease respectively. Reduction in EBDASI activity scores of greater than 9 indicated clinically significant improvement. An increase of 3 in the activity score indicated deterioration. iscorEB is a measurement tool for evaluating the disease severity in EB patient. It evaluates the cutaneous, mucosal and other organ impact of EB and includes clinician and patient reported outcomes in a single instrument. Clinician score ranges between 0-120 between. Reduction in iscorEB scores indicates improvement. An increase in score indicates deterioration.
Time frame: 24 weeks
Proportion of patients experiencing adverse events, minor, severe and serious (life-threatening)
Proportion of patients experiencing adverse events
Time frame: 24 weeks
Proportion of patients that dropped out of the study as a result of an adverse event
Proportion of patients that dropped out of the study as a result of an adverse event
Time frame: 24 weeks
Correlation between itch score (as measured by VAS),iscorEB patient portion and QOLEB (for patients over 18 yrs) total scores.
Correlation between itch score( as measured by VAS),iscorEB patient portion, and QOLEB (for patients over 18 yrs.) total scores.
Time frame: 24 weeks
Correlation between itch score( as measured by VAS) and iscorEB clinician and Epidermolysis Bullosa Disease Activity and Scarring Index(EBDASI)
Correlation between itch score(as measured by VAS) and iscorEB clinician and EBDASI total scores. Total EBDASI score ranges of 0-42, 43-106 and 107-506 corresponded to mild, moderate and severe disease respectively. Reduction in EBDASI activity scores of greater than 9 indicated clinically significant improvement. An increase of 3 in the activity score indicated deterioration.
Time frame: 24 weeks
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