Uremic Pruritus in patients with end-stage renal disease requiring hemodialysis MedDRA version: 18.1 Level: LLT Classification code 10060884 Term: Uremic pruritus System Organ Class: 100000004858
Conditions
Interventions
Product Name: Disodium Cromoglycate
Product Code: PA101B
Pharmaceutical Form: Nebuliser solution
INN or Proposed INN: not available
CAS Number: 15826-37-6
Current Sponsor code: PA101B
Other descriptiv
Sponsors
Patara Pharma, LLC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female patients age 18 through 80 years, inclusive. 2. Diagnosis of end-stage renal disease (ESRD) receiving hemodialysis for at least 3 months prior to the Screening Period 3. Able to receive conventional hemodialysis (i.e., not hemofiltration or hemodiafiltration) during the study 4. Pruritus present for at least 6 weeks 5. Mean pruritus severity score on a numerical rating scale (NRS) > 4 from the Screening Period (based on a minimum of 8 scores) 6. Patient-Assessed Disease Severity Scale Type B or C at the Screening Period 7. If receiving H1 antihistamines for pruritus treatment, the drug and dose has been stable during the 2 weeks prior to Screening and is expected to remain stable throughout the study 8. Hemoglobin > 8 g/dL at the Screening Period 9. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) 65% or single-pooled Kt/V >1.4 during the Screening Period 11. Sufficient manual dexterity to use the hand-held nebulizer or a caregiver who can reliably assist with assembly and use of the nebulizer 12. Adequate venous access for blood drawing, unless the dialysis access will be used for this purpose 13. Willingness and ability to provide written informed consent Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 45 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 15
Exclusion criteria
Exclusion criteria: 1. Current or recent history of clinically significant medical condition, laboratory abnormality, or illness that could put the patient at risk or compromise the quality of the study data as determined by the investigator 2. Myocardial infarction within 6 months or unstable angina, acute coronary syndrome, or interventional coronary procedure within 2 months prior to the Screening Visit 3. An upper or lower respiratory tract infection (including sinus infection) within 4 weeks prior to the Screening Visit 4. Severely symptomatic cardiopulmonary disease defined by the use of home oxygen treatment, dyspnea at rest or with minimal exertion, uncontrolled arrhythmias (e.g. atrial fibrillation with inadequate rate control), or history of life-threatening arrhythmias (e.g. cardiac arrest or syncope related to arrhythmia) 5. Acute exacerbation of asthma or chronic obstructive pulmonary disease resulting in hospitalization or visit to an emergency department or urgent care clinic within 6 months of the Screening Visit 6. Hospitalization for any medical reason other than for a pre-planned procedure or dialysis access related procedure within the 2 weeks prior to the Baseline Visit 7. Known malignancy for which the patient is receiving active treatment with a systemic drug 8. Participation in any other investigation drug study within 4 weeks prior to the Screening Visit 9. Use of cromolyn sodium within 2 weeks of the Screening Visit 10. Current (i.e., within 2 weeks prior to Screening) or anticipated use of baclofen, gabapentin, pregabalin and nalbuphine for the treatment of pruritus during the study (Note: Use of these medications for indications other than pruritus is permitted if the dose has been stable during the 2 weeks prior to Screening and is anticipated to remain stable during the study) 11. Current (i.e., within 2 weeks prior to Screening) or anticipated use during the study of glucocorticoids administered intravenous, oral, or transdermally (Note: Use of inhaled, intranasal, intrarticular, otic, and ophthalmic glucocorticoids is permitted during the study) 12. Females who are pregnant or breastfeeding, or if of child-bearing potential unwilling to practice acceptable means of birth control or abstinence during the study (e.g., abstinence, combination barrier and spermicide, or hormonal) 13. History of hypersensitivity or intolerance to cromolyn sodium 14. Anticipated changes in the dialysis regimen or modality during the trial (e.g., change in type of filter, increase or decrease in prescribed dialysis duration by 1 hour, change or in prescribed blood flow by >100 mL/minute that is expected to significantly affect dialysis clearance as determined by the investigator) or any such changes during the 2 weeks prior to the Screening Period
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To assess the effectiveness of inhaled PA101B delivered via eFlow high efficiency nebulizer in treating uremic pruritus in patients with end-stage renal disease (ESRD) requiring hemodialysis. Improvements in pruritus will be assessed by measuring the change from baseline in worst itching intensity using a numerical rating scale (NRS) as a patient-reported outcome measure • To evaluate the safety and tolerability of inhaled PA101B in patients with ESRD requiring hemodialysis • To assess biomarker levels prior to and following PA101B administration;Secondary Objective: Not applicable;Primary end point(s): The change from Baseline (i.e., mean NRS of all available scores from the Placebo Run-in Period) in severity of pruritus at Week 7 (i.e., mean of all available NRS scores during Weeks 6 – 7);Timepoint(s) of evaluation of this end point: Week 7 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The change from Baseline (i.e., the Placebo Run-in Period) in itch-specific quality of life (the total Skindex-10) score at Week 7 (i.e., mean score during Weeks 6 – 7);Timepoint(s) of evaluation of this end point: Week 7 | — |
Countries
Poland, United States
Contacts
Public ContactRod Saponjic
Patara Pharma, LLC
Outcome results
None listed