Calciphylaxis (Calcific uremic arteriolopathy [CUA]) MedDRA version: 21.0 Level: PT Classification code 10051714 Term: Calciphylaxis System Organ Class: 10027433 - Metabolism and nutrition disorders
Conditions
Sponsors
Sanifit Therapeutics S.A
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participants must meet the following criteria: 1. Above 18 years of age 2. Receiving maintenance dialysis in a clinical setting for at least 2 weeks prior to screening 3. Clinical diagnosis of CUA by the Investigator including more than 1 CUA lesion with ulceration. A central wound rating group will review wound images to confirm the primary wound is due to CUA. 4. CUA wound-related pain shown by a Pain VAS score =50 out of 100 5. Primary wound that can be clearly photographed for the purpose of protocol-specified wound healing assessments 6. Willing and able to understand and sign the informed consent form and willing to comply with all aspects of the protocol Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 36 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 30
Exclusion criteria
Exclusion criteria: Exclusion Criteria; 1. Subjects whose primary wound is due to causes other than CUA 2. History of treatment with bisphosphonates within 3 months of baseline (Week 1 Day 1) 3. Severely ill subjects without a reasonable expectation of survival for at least 6 months, based on the assessment of the Investigator 4. Subjects with a scheduled para-thyroidectomy during the study period 5. Expectation for kidney transplant within the next 6 months based on Investigator assessment or identification of a known living donor 6. 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 either completely avoid sexual intercourse with a person of the opposite sex or use a highly effective method of birth control from screening through at least 30 days after last dose of study drug 7. Significant noncompliance with dialysis treatment evidenced by repeated missed dialysis sessions (including if due to hospitalizations where dialysis treatment is unavailable) or significant noncompliance with medication regimen, in the judgment of the Investigator 8. Any history of active malignancy within the last year 9. Clinically significant illness other than CUA within 30 days prior to screening that, in the judgment of the Investigator, could interfere with interpretation of study results, impair compliance with study procedures, or impact the safety of the subject (e.g., unstable angina, unstable heart failure, stroke, uncontrolled hypertension, or other illness requiring hospitalization) 10. Participation in an investigational study and receipt of an investigational drug or investigational use of a licensed drug (with the exception of intravenous STS) within 30 days prior to screening. If participating in an investigational study of intravenous STS,all visits of that study must be completed prior to screening for this study. 11. Past or current participation in another clinical study with SNF472 12. History or presence of active alcoholism or drug abuse as determined by the Investigator within 6 months before screening or concurrent social conditions that, in the opinion of the Investigator, would potentially interfere with the subject's study compliance 13. Mental impairment or history of or current significant psychiatric disease that, in the opinion of the Investigator, may impair ability to provide informed consent or impact compliance with study procedures 14. Any other condition or circumstance that, in the opinion of the Investigator, may make the subject unlikely to complete the study or comply with study procedures and requirements, or may pose a risk to the subject's safety and well-being 15. Subjects whose CUA wounds exhibit significant improvement, in the opinion of the Investigator, between the first and second screening visit
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To evaluate the efficacy of SNF472 compared with placebo when added to background care for the treatment of CUA • To evaluate the safety and tolerability of SNF472 compared with placebo when added to background care for the treatment of CUA ;Secondary Objective: n/a;Primary end point(s): Efficacy Endpoints; Applies to part 1. These endpoints will compare the placebo and SNF472 groups as follows; Alternate Primary Efficacy Endpoints: • Absolute change from baseline to Week 12 in the BWAT-CUA score for the primary lesion • Absolute change from baseline to Week 12 in Pain VAS score Safety Endpoints; Applies throughout part 1 and part 2 • Proportion of subjects with AEs, SAEs, and deaths • Changes from baseline in the following: o Laboratory parameters o QTc interval and other ECG parameters o Holter monitoring results o Vital signs • Proportion of subjects with a CUA wound-related infection, sepsis, hospitalization, or any CUA wound-related complication;Timepoint(s) of evaluation of this end point: Efficacy (Part 1): Various time-points from baseline to week 12 Safety: | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Efficacy Endpoints (assessed hierarchically): • Absolute change from baseline to Week 12 in the Wound-QoL score • Absolute change from baseline to Week 12 in the BWAT total score for the primary lesion • Qualitative wound image evaluation for the primary lesion (worsened, equal to, or improved relative to baseline) at Week 12 • Rate of change in opioid use as measured in morphine milligram equivalents (MME) from baseline to Week 12;Timepoint(s) of evaluation of this end point: Various time-points from baseline to week 12 | — |
Countries
Belgium, France, Germany, Italy, Spain, United Kingdom, United States
Contacts
Public ContactSVP Development Operations
Sanifit Therapeutics S.A
Outcome results
None listed