Phase 3 Study of SNF472 for Calciphylaxis

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

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).

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.. .

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