Phase 2 Study With SNF472 in Calciphylaxis Patients

Study Overview
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Brief Summary
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To evaluate the effect of SNF472 on top of standard of care on promoting wound healing and other parameters of therapeutic response in haemodialysis patients with calciphylaxis (calcific uraemic arteriolopathy, CUA).

Official Title
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Phase 2 Open Label Single Arm Repeat Dose Study to Assess the Effect of SNF472 on Wound Healing in Uraemic Calciphylaxis Patients

Conditions
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Calciphylaxis, Calcific Uremic Arteriolopathy

Intervention / Treatment
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* Drug: SNF472


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with either newly diagnosed CUA OR recurrent CUA that has been dormant with no skin lesion involvement for at least 90 days from study start (new or recurrent diagnosis must be made within 5 weeks of study start) Patients who signed the written informed consent to participate in this clinical trial (prior to any clinical trial-related procedures being performed), after reading the Patient Information Sheet and Informed Consent Form (ICF), and who had the opportunity to discuss the clinical trial with the Investigator or designee Males or females aged ≥18 Patients on maintenance haemodialysis (HD) Patients with at least a minimum level of pain on Visual Analog Scale (VAS) scale or on pain-killers stronger than non-steroidal anti-inflammatory drugs (NSAIDs) Females of child-bearing potential should use a highly effective contraceptive measure throughout the study and have a negative serum pregnancy test at entry. Male patients having sexual relationship in which pregnancy can occur should take adequate contraceptive precautions (wear a condom) Exclusion Criteria: Body weight above 150 kg BMI >35 and central(abdominal) ulcers History of bisphosphonate treatment within 12 months before entering into the study Severely ill patients without reasonable expectation of survival for > 6 months according to the treating physician Patients with scheduled parathyroidectomy during the run-in or study period Female patients who are either intending to get pregnant or are undergoing treatment to get pregnant, as well as breast-feeding females Participation in another clinical trial with an experimental drug within 90 days prior the inclusion Any psychological, emotional problems, any disorders or resultant therapy that is likely to invalidate informed consent, or limit the ability of the patient to comply with the Clinical Trial Protocol requirements Patients who, in the opinion of the Investigator, are considered unsuitable for any other reason

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: SNF472<br>SNF472 for calciphylaxis | Drug: SNF472<br>* SNF472 for calciphylaxis<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Wound Healing | Absolute change in Bates-Jensen Wound Assessment (BWAT) total score between baseline (Week 1) and Week 12 for the primary lesion (the largest one). The Bates-Jensen Wound Assessment (BWAT) is a standardized tool for quantitative assessment of wound healing that includes the 13 items listed below. Size Depth Edges Undermining or pockets Necrotic tissue type Necrotic tissue amount Exudate type Exudate amount Surrounding skin color Peripheral tissue edema Peripheral tissue induration Granulation tissue Epithelialization Each item was rated on a scale of 1 (best) to 5 (worst). The Bates-Jensen Wound Assessment (BWAT) total score is the sum of the individual items with a possible range of 13 (best) to 65 (worst). | 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Wound Pain | Absolute change from baseline (Week 1) and Week 12 in the Pain Visual Analogue Scale (VAS) Score. The Pain Visual Analogue Scale (VAS) Score is a horizontal line, 100 mm in length, anchored by word descriptors at each end. The subject marked the point on the line that represented his/her perception of his/her current pain status. The Pain Visual Analogue Scale (VAS) Score was determined by measuring in millimeters from the left hand end of the line (no pain) to the point that the subject marked. The Pain Visual Analogue Scale (VAS) Score range 0 (best) to 100 (Worst). | 12 weeks | 
| Wound-QoL Global Score | Absolute change from baseline (Week 1) and Week 12 in the Wound-QoL global score. The Wound-QoL questionnaire measures the disease-specific, health related QoL of patients with chronic wounds. It consists of 17 items on impairments that are assessed in retrospect to the preceding 7 days and rated on a 0 (best) to 4 (worse) scale with possible responses from not at all to very much. The total score is the average of the 17 responses. | 12 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Calciphylaxis, Calcific Uremic Arteriolopathy, Dialysis, End Stage Renal Disease, Rare disease (ORPHA280062), Calcification, Calcium Metabolism Disorders, Calcinosis, Metabolic Diseases