Clinical trial NCT04195906: Phase 3 Study of SNF472 for Calciphylaxis
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of SNF472 When Added to Background Care for the Treatment of Calciphylaxis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Calciphylaxis
Calcific Uremic Arteriolopathy
Keywords: Calciphylaxis
Calcific Uremic Arteriolopathy
Wound
ESRD
Lead sponsor: Sanifit Therapeutics S. A.
Sponsor class: OTHER
First posted: 2019-12-12
Enrollment: 71
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The primary objectives are to assess the efficacy, safety, and tolerability of SNF472 compared to placebo when added to background care for the treatment of calciphylaxis (CUA).

Detailed description: The formation and growth of calcified deposits in arterioles and other small blood vessels appears to be fundamental to the development of CUA especially in end stage renal disease patients. This phase 3 double-blind, randomized, placebo-controlled study is designed to assess the effect of SNF472 when added to background care to improve wound healing, as evaluated using Bates-Jensen Wound Assessment Tool (BWAT) scoring and pain as reported by the subject using a VAS scale. The study consists of a double-blind, randomized, placebo controlled period of 12 weeks followed by an open-label period of 12 weeks.. .

Eligibility criteria: Inclusion Criteria:

* Female or male subjects, 18 years of age or older
* Receiving maintenance HD in a clinical setting for at least 2 weeks prior to screening
* Clinical diagnosis of CUA by the Investigator including ≥1 CUA lesion with ulceration of the epithelial surface
* CUA wound-related pain shown by a Pain VAS score ≥50 out of 100
* Primary lesion that can be clearly photographed for the purpose of protocol-specified wound healing assessments.
* Willing and able to understand and sign the informed consent form and willing to comply with all aspects of the protocol

Exclusion Criteria:

* History of treatment with bisphosphonates within 3 months of baseline
* Severely ill subjects without a reasonable expectation of survival for at least 6 months
* Subjects with a scheduled parathyroidectomy during the study period
* Expectation for kidney transplant within the next 6 months based on Investigator assessment or identification of a known living donor
* Pregnant or trying to become pregnant, currently breastfeeding, or of childbearing potential (including perimenopausal women who have had a menstrual period within one year) and not willing to comply with protocol required contraception criteria
* Significant noncompliance with dialysis
* History of active malignancy within the last year with the exception of localized basal cell or squamous cell carcinoma
* Clinically significant illness other than CUA within 30 days
* Participation in an investigational study and receipt of an investigational drug or investigational use of a licensed drug within 30 days prior to screening.
* History or presence of active alcoholism or drug abuse as determined by the Investigator within 6 months
* Mental impairment, current significant psychiatric disease, or other conditions or circumstances that would make the subject unlikely to complete the study or comply with the study procedures.
* Subjects whose CUA lesions exhibit significant improvement, in the opinion of the Investigator, between the first and second screening visit

Primary outcomes:
measure: Absolute Change in the BWAT - CUA Score for the Primary Lesion
timeFrame: from Baseline to Week 12
measure: Absolute Change in Pain Visual Analog Score
timeFrame: from Baseline to Week 12
Secondary outcomes:
measure: Absolute Change in the Wound-Quality of Life Score
timeFrame: from Baseline to Week 12
measure: Absolute Change in the BWAT Total Score for the Primary Lesion
timeFrame: from Baseline to Week 12
measure: Qualitative Wound Image Evaluation for the Primary Lesion
timeFrame: at Week 12
measure: Rate of Change in Opioid Use as Measured in Morphine Milligram Equivalents (MME)
timeFrame: from Baseline to Week 12
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: AKDHC Medical Research Services
state: Arizona
city: El Centro
country: United States
facility: California Institute of Renal Research
state: California
city: Escondido
country: United States
facility: California Institute of Renal Research
state: California
city: Glendale
country: United States
facility: Kidney Disease Medical Group
state: California
city: Lynwood
country: United States
facility: DaVita Clinical Research
state: California
city: Riverside
country: United States
facility: Apex Research of Riverside
state: California
city: San Diego
country: United States
facility: Fresenius Kidney Care
state: California
city: San Dimas
country: United States
facility: North America Research Institute
state: California
city: Vacaville
country: United States
facility: Amicis Research Center
state: California
city: Denver
country: United States
facility: Colorado Kidney Care
state: Colorado
city: Boca Raton
country: United States
facility: Boca Nephrology, PA
state: Florida
city: Hollywood
country: United States
facility: DaVita Clinical Research
state: Florida
city: Spring Hill
country: United States
facility: Novel Outcomes Research
state: Florida
city: Tampa
country: United States
facility: DaVita Clinical Research
state: Florida
city: Tampa
country: United States
facility: Fresenius Kidney Care
state: Florida
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Pontiac
country: United States
facility: Michigan Kidney Consultants
state: Michigan
city: Roseville
country: United States
facility: DaVita Clinical Research
state: Michigan
city: Minneapolis
country: United States
facility: DaVita Clinical Research
state: Minnesota
city: Brookhaven
country: United States
facility: Fresenius Kidney Care
state: Mississippi
city: Kansas City
country: United States
facility: DaVita Clinical Research
state: Missouri
city: Las Vegas
country: United States
facility: DaVita Clinical Research
state: Nevada
city: Reno
country: United States
facility: Fresenius Kidney Care
state: Nevada
city: Bronx
country: United States
facility: DaVita Clinical Research
state: New York
city: Asheville
country: United States
facility: DaVita Clinical Research
state: North Carolina
city: Durham
country: United States
facility: Fresenius Kidney Care
state: North Carolina
city: Winston-Salem
country: United States
facility: Piedmont Dialysis Center
state: North Carolina
city: Columbus
country: United States
facility: Hypertension Nephrology Consultants, Inc
state: Ohio
city: Bethlehem
country: United States
facility: Fresenius Kidney Care
state: Pennsylvania
city: Chester
country: United States
facility: DaVita Clinical Research
state: Pennsylvania
city: Columbia
country: United States
facility: Fresenius Kidney Care
state: South Carolina
city: Knoxville
country: United States
facility: Knoxville Kidney Center
state: Tennessee
city: Houston
country: United States
facility: DaVita Clinical Research
state: Texas
city: San Antonio
country: United States
facility: Clinical Advancement Center
state: Texas
city: Chesapeake
country: United States
facility: DaVita Clinical Research
state: Virginia
city: Norfolk
country: United States
facility: DaVita Clinical Research
state: Virginia
city: Roanoke
country: United States
facility: Fresenius Kidney Care
state: Virginia
city: Wauwatosa
country: United States
facility: DaVita Clinical Research
state: Wisconsin
city: Bruxelles
country: Belgium
facility: Clinques Universitaries de Bruxelles Hopital
city: Leuven
country: Belgium
facility: UZ Leuven
city: Roeselare
country: Belgium
facility: AZ Delta
city: Villingen-Schwenningen
country: Germany
facility: Nephrologischen Zentrum Villingen-Schwenningen
state: Baden Wuerttemberg
city: Berlin
country: Germany
facility: Charite Universitaetsmedizin Berlin - Campus Charite Mitte
city: Düsseldorf
country: Germany
facility: DaVita Deutschland AG
city: Bydgoszcz
country: Poland
facility: Centrum Dializ Fresenius, Ośrodek Dializ nr 10 w Bydgoszczy 85-826
city: Krakow
country: Poland
facility: Centrum Dializ Fresenius, Ośrodek Dializ nr 18 w Krakowie..
city: Miechów
country: Poland
facility: DaVita Sp. z o.o., Stacja Dializ w Miechowie
city: Radom
country: Poland
facility: Centrum Dializ Fresenius Ośrodek Dializ nr 32 w Radomiu 26-617 .
city: Łódź
country: Poland
facility: Uniwersytecki Szpital Kliniczny nr 1 im. N. Barlickiego w Lodzi Stacja Dializ,
city: Barcelona
country: Spain
facility: Fundacio Puigvert
city: Barcelona
country: Spain
facility: University of Barcelona Hospital Clinic
city: Córdoba
country: Spain
facility: Hospital Universitario Reina Sofia
city: Exeter
country: United Kingdom
facility: Royal Devon and Exeter Hospital (Wonford)
state: Devon
city: Salford
country: United Kingdom
facility: Salford Royal NHS Foundation Trust
state: Greater Manchester
city: Leicester
country: United Kingdom
facility: Leicester General Hospital
state: Leicestershire
city: Glasgow
country: United Kingdom
facility: Queen Elizabeth University Hospital Campus
state: Strathclyde
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital
state: West Midlands
city: London
country: United Kingdom
facility: Kings College Hospital
Study officials:
affiliation: Sanifit Therapeutics
name: Alex Gold, MD
role: STUDY_DIRECTOR
References: