Moderate to severe pruritus significantly impairs the quality of life in peritoneal dialysis patients, and effective treatment options remain limited. κ-opioid receptor agonists may alleviate itching by modulating neural signaling pathways. This study is a multicenter, prospective, single-arm clinical trial, planning to enroll 93 patients. It aims to test the hypothesis that nalfurafine hydrochloride orally disintegrating tablets compared to baseline, with acceptable safety.
This is a multicenter, prospective, single-arm clinical study designed to evaluate the efficacy and safety of nalfurafine hydrochloride orally disintegrating tablets in treating moderate-to-severe pruritus in peritoneal dialysis patients. The study consists of three phases: a screening period (1-2 weeks), a treatment period (4 weeks), and a follow-up period (1 week). During the screening phase, baseline pruritus levels are established using the Visual Analog Scale (VAS) and the Shichuan-Kawashima Pruritus Severity Score. The treatment period begins with an initial dose of 2.5 μg/day, which may be adjusted to 5 μg/day after 2 weeks based on symptom response; the follow-up phase assesses pruritus improvement and safety. The study plans to enroll 93 patients (including a 20% dropout rate), with the primary endpoint being the change in VAS score from baseline to week 4 of treatment. Secondary endpoints include VAS score changes at different stages, quality-of-life improvements, and adverse event incidence. Eligible patients are aged 18-85 years, undergoing regular peritoneal dialysis for ≥3 months, and meeting baseline VAS criteria for moderate-to-severe pruritus. The primary efficacy measure is the mean change in daily maximum VAS scores (baseline vs. week 4), while secondary measures include pruritus severity scores, sleep quality, dose adjustment rates, and laboratory safety data. Statistical analyses will follow intention-to-treat (ITT) and per-protocol (PP) principles. Based on preliminary data (mean 28.4 mm, standard deviation 21.82 mm), the sample size calculation ensures 95% power to validate efficacy hypotheses. The intervention involves monotherapy with nalfurafine hydrochloride orally disintegrating tablets, starting at 2.5 μg/day, with a potential increase to 5 μg/day after 2 weeks. Standardized scales assess pruritus, quality of life, and sleep improvements, alongside monitoring of vital signs, electrocardiograms, and laboratory parameters for safety evaluation. Enrollment criteria require ≥5 days of recorded morning/evening VAS scores during the baseline period (average ≥50 mm) and ≥2 days with Shichuan-Kawashima pruritus scores ≥3 (moderate severity). The study anticipates a significant reduction in VAS scores as the primary endpoint, with secondary endpoints including dose adjustments, safety events, and patient-reported outcomes. This single-arm self-controlled study validates drug efficacy while strictly controlling dropout rates and data integrity to ensure result reliability.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
93
Nalfurafine 2.5μg daily for 2 weeks. Afterwards, the dose can be increased to 5μg daily if necessary (maximum dose not exceeding 5μg daily), continued for another 2 weeks.
Guangdong Provincial People's Hospital
Guangzhou, Guangdong, China
RECRUITINGChange in Visual Analog Scale (VAS) score for Itch from baseline to Week4 of treatment period
Calculation: Change = (Average of daily maximum VAS scores during baseline period) - (Average of daily maximum VAS scores during the 4th week of treatment period). Only dates with both daytime and nighttime VAS recordings were included in the assessment. Daily maximum VAS score: Defined as the higher value between the VAS measured at waking and bedtime each day. VAS scores range from 0 to 10 points.0 = "No itch" ,10 = "Worst itch". Higher scores indicate worse itch severity.
Time frame: This study includes a screening period (1-2 weeks, baseline assessment), a 4-week treatment phase (starting with nalfurafine 2.5μg/day, potentially increased to 5μg), and a 1-week follow-up period (to evaluate changes in itching severity).
Change in Visual Analog Scale(VAS) for Itch scores at Weeks 1, 2, 4 of treatment and follow-up period
Change from baseline in daily maximum Visual Analog Scale for Itch (VAS) scores at each assessment time point (Weeks 1, 2, 4 of treatment and follow-up period).VAS scores range from 0 to 10 points.0 = "No itch" ,10 = "Worst itch". Higher scores indicate worse itch severity.
Time frame: Baseline, Week 1, Week 2, Week 4, and Follow-up
Change in Xie-Kawashima Itch Scale scores
Change from baseline in daily maximum Xie-Kawashima Itch Scale scores at each assessment time point (Weeks 1, 2, 4 of treatment and follow-up period). Xie-Kawashima Itch Scale scores range from 0 to 4 points.0 = "No itch" ,4 = "Worst itch". Higher scores indicate worse itch severity.
Time frame: Baseline, Week 1, Week 2, Week 4, and Follow-up
The degree of change in Visual Analog Scale (VAS) scores from Baseline to Week 2,4 of treatment period
Change of itch intensity,assessed by the Visual Analog Scale for Itch from baseline to Week 2,4 of treatment period. Calculated as: average of baseline VAS score minus average of VAS score during Week 2,4 of treatment period. Response Criteria: Clinically Significant Improvement:Mean VAS score \< 20 mm during treatment period OR reduction ≥40 mm from baseline Improvement:Reduction ≥20 mm but \<40 mm from baseline (excluding Clinically Significant Improvement) No Clinically Meaningful Improvement:Neither Category 1 nor Category 2 criteria met.
Time frame: Baseline to Week 2,4 of treatment period
Change in Health-Related Quality of Life Scores
Kidney Disease Quality of Life-36 (KDQOL-36) Scale score Range: 36-185 points.Higher scores indicate better quality of life. Skindex-16 Dermatology Life Quality Index Scale score Range: 0-96 points.Higher scores indicate worse quality of life.
Time frame: Baseline to Week 4 of treatment period
Change in Pittsburgh Sleep Quality Index (PSQI) total score
Change from baseline to Week 4 of treatment in sleep quality measured by Pittsburgh Sleep Quality Index (PSQI). Total Score Range: 0 to 21 points.Higher scores indicate worse sleep quality.
Time frame: Baseline to Week 4 of treatment period
Proportion of participants escalating dosage of nalfurafine hydrochloride
Proportion of participants escalating dosage from 2.5μg QD to 5μg QD during 4-week treatment period. Drug Formulation: Nalfurafine hydrochloride orally disintegrating tablets. Calculation: Numerator: Patients with dose escalation Denominator: Total treated patients
Time frame: Day 1 to Week 4 of treatment period
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