Treatment of Uremic Pruritus With PA101B

Phase 2

Completed

• Conditions: Uremic Pruritus
• Interventions: Drug: PA101B; Drug: Placebo

• Registration Number: NCT02696499
• Lead Sponsor: Patara Pharma

Brief Summary

This is a randomized, double blind, placebo-controlled, parallel-arm, multi-center, Phase 2, proof-of-concept efficacy and safety study in patients with end-stage renal disease requiring hemodialysis.

The purpose of the study is to determine the efficacy and safety profile of PA101B delivered via eFlow high efficiency nebulizer in patients with uremic pruritus who are symptomatic despite using standard treatments.

Detailed Description

The symptom score for determining eligibility will be established during the Screening Period (up to 4 weeks) and eligible patients will be randomly allocated in a 2:1 ratio between the active and placebo arms at the baseline visit (Week 0). During the Treatment Period, clinic visits will occur at the end of Weeks 1, 3, 5 and 7. Following completion of the Treatment Period, patients will enter a 2-week Washout Period in both treatment arms.

Routine hemodialysis treatment will continue according to the dialysis unit's standard practice in all patients.

Patients will be allowed to continue to use the same daily doses of pre-randomization H1 antihistamines as well as the same daily doses of any other allowed medications throughout the study.

Blood samples will be collected for various biomarkers. In a subset of patients, additional blood samples will be collected for pharmacokinetic assessments. Clinical safety assessments will be performed at each clinic visit.

Study Timeline

• Study Start: 2016-02
• Study First Submitted: 2016-02-25
• Study First Submitted QC: 2016-02-25
• Study First Posted: 2016-03-02
• Primary Completion: 2016-08
• Study Completion: 2016-12
• Status Verified: 2018-04
• Disposition First Submitted: 2018-04-18
• Disposition First Submitted QC: 2018-04-18
• Last Update Submitted: 2018-04-18
• Disposition First Posted: 2018-04-20
• Last Update Posted: 2018-04-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 70

Inclusion Criteria

• Diagnosis of end-stage renal disease (ESRD) requiring hemodialysis for at least 3 months prior to the Screening Period
• Receiving conventional hemodialysis (i.e., not hemofiltration or hemodiafiltration)
• Pruritus present for at least 6 weeks of Screening
• Mean pruritus severity score on a numerical rating scale (NRS) > 4
• Patient-Assessed Disease Severity Scale Type B or C at Screening
• Documentation of a urea reduction ratio (URR) >65% or single-pooled Kt/V> 1.4 during Screening
• Willing and able to provide written informed consent

Exclusion Criteria

• 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
• Myocardial infarction within 6 months or unstable angina, acute coronary syndrome, or interventional coronary procedure within 2 months of Screening
• Upper or lower respiratory tract infection (including sinus infection) within 4 weeks of Screening
• 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)
• 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 Screening
• Hospitalization for any medical reason other than for a pre-planned procedure or dialysis access related procedure within the 2 weeks of Screening
• Malignancy requiring active treatment with a systemic drug
• Participation in any other investigation drug study within 4 weeks of Screening
• Current or anticipated use of baclofen, gabapentin, pregabalin and nalbuphine for the treatment of pruritus
• Current or anticipated use of glucocorticoids administered intravenously, orally, or transdermally
• Pregnant or breastfeeding females, or if of child-bearing potential unwilling to practice acceptable means of birth control or abstinence during the study

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
PA101B	PA101B	-
Placebo	Placebo	-

Primary Outcome Measures

Name	Time	Method
Itching intensity	7 weeks	Numerical rating scale (NRS)

Secondary Outcome Measures

Name	Time	Method
Pruritus-specific quality of life (QoL)	7 weeks	Skindex-10
Pruritus-specific sleep quality	7 weeks	Itch MOS
Assessment of depression	7 weeks	Beck Depression Inventory-II
Patient Global Impression of Change (PGIC)	7 weeks

Trial Locations

Locations (12)

US Renal Care; 🇺🇸 Gallup, New Mexico, United States

Renal Medicine Associates; 🇺🇸 Albuquerque, New Mexico, United States

Ridgewood Dialysis Center; 🇺🇸 Ridgewood, New York, United States

Genesis Clinical Research; 🇺🇸 Tampa, Florida, United States

Brookview Hills Research Associates LLC; 🇺🇸 Winston-Salem, North Carolina, United States

Arizona Kidney Disease and Hypertension Center; 🇺🇸 Phoenix, Arizona, United States

Pines Clinical Research, Inc.; 🇺🇸 Pembroke Pines, Florida, United States

North America Research Institute; 🇺🇸 San Dimas, California, United States

Valley Renal Medical Group; 🇺🇸 Northridge, California, United States

Boise Kidney and Hypertension Institute; 🇺🇸 Meridian, Idaho, United States

Fresenius Medical Care of Evergreen Park; 🇺🇸 Evergreen Park, Illinois, United States

Southeast Renal Research Institute; 🇺🇸 Chattanooga, Tennessee, United States