Phase 2 Study With SNF472 in Calciphylaxis Patients

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

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).

Study Overview

• Status: Completed
• Conditions: Calciphylaxis; Calcific Uremic Arteriolopathy
• Intervention / Treatment: Drug: SNF472

• Study Type: Interventional
• Enrollment (Actual): 14
• Phase: Phase 2

Contacts and Locations

Study Locations

• United Kingdom
  • Salford, United Kingdom
    • Salford Royal Hospital
• United States
  • Massachusetts
    • Waltham, Massachusetts, United States, 02451
      • Fresenius Medical Services
  • Minnesota
    • Minneapolis, Minnesota, United States, 55404
      • DaVita Clinical Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. 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)
2. 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
3. Males or females aged ≥18
4. Patients on maintenance haemodialysis (HD)
5. 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)
6. 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:

1. Body weight above 150 kg
2. BMI >35 and central(abdominal) ulcers
3. History of bisphosphonate treatment within 12 months before entering into the study
4. Severely ill patients without reasonable expectation of survival for > 6 months according to the treating physician
5. Patients with scheduled parathyroidectomy during the run-in or study period
6. Female patients who are either intending to get pregnant or are undergoing treatment to get pregnant, as well as breast-feeding females
7. Participation in another clinical trial with an experimental drug within 90 days prior the inclusion
8. 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
9. Patients who, in the opinion of the Investigator, are considered unsuitable for any other reason

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: SNF472; SNF472 for calciphylaxis	Drug: SNF472; SNF472 for calciphylaxis

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Sanifit Therapeutics S. A.
• Investigators: Principal Investigator: Vincent Brandenburg, RWTH Aachen University

Publications and helpful links

Helpful Links

• USA FDA resources
• Drug Information available for: SNF472

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2016
• Primary Completion (Actual): November 15, 2017
• Study Completion (Actual): November 15, 2017

Study Registration Dates

• First Submitted: May 25, 2016
• First Submitted That Met QC Criteria: May 30, 2016
• First Posted (Estimate): June 3, 2016

Study Record Updates

• Last Update Posted (Actual): August 28, 2019
• Last Update Submitted That Met QC Criteria: August 27, 2019
• Last Verified: August 1, 2019

More Information

Terms related to this study

• Keywords: End Stage Renal Disease; Dialysis; Metabolic Diseases; Calcium Metabolism Disorders; Calciphylaxis; Calcific Uremic Arteriolopathy; Calcification; Calcinosis; Rare disease (ORPHA280062)
• Additional Relevant MeSH Terms: Metabolic Diseases; Calcium Metabolism Disorders; Calcinosis; Calciphylaxis
• Other Study ID Numbers: SNFCT2015_04