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CompletedNCT02790073Updated Aug 28, 2019Results posted

Phase 2 Study With SNF472 in Calciphylaxis Patients

A Phase 2 interventional study of SNF472 in Calciphylaxis and Calcific Uremic Arteriolopathy, sponsored by Sanifit Therapeutics S. A.. Completed at 3 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2019-08-28.

Sponsored by Sanifit Therapeutics S. A. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
14
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study summary

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).

02

Conditions studied

  • Calciphylaxis
  • Calcific Uremic Arteriolopathy

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Keywords

  • Calciphylaxis
  • Calcific Uremic Arteriolopathy
  • Dialysis
  • End Stage Renal Disease
  • Rare disease (ORPHA280062)
  • Calcification
  • Calcium Metabolism Disorders
  • Calcinosis
  • Metabolic Diseases
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
14 participants (actual)

Study arms

  • Other
    SNF472

    SNF472 for calciphylaxis

    Drug: SNF472

Interventions

  • DrugSNF472

    SNF472 for calciphylaxis

05

What researchers measure

Primary outcomes

  1. 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).

    Time frame: 12 weeks

Secondary outcomes

  1. 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).

    Time frame: 12 weeks

  2. 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.

    Time frame: 12 weeks

06

Results

Posted Aug 28, 2019

Participant flow

Participant flow — Overall Study
MilestoneSNF472
Started14
Completed11
Not completed3
Withdrew: Death1
Withdrew: Withdrawal from hd1
Withdrew: Withdrawal by subject1

Outcome measures

PrimaryWound 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).

Time frame:
12 weeks
Reported as:
Mean · units on a scale
Wound Healing
units on a scaleSNF472
Wound Healing-8.1 ± 8.5
Statistical analysis
  • SNF472 · t-test, 2 sided · p = <0.001
SecondaryWound 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).

Time frame:
12 weeks
Reported as:
Mean · units on a scale
Wound Pain
units on a scaleSNF472
Wound Pain-23.6 ± 30.0
Statistical analysis
  • SNF472 · t-test, 2 sided · p = 0.015
SecondaryWound-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.

Time frame:
12 weeks
Reported as:
Mean · units on a scale
Wound-QoL Global Score
units on a scaleSNF472
Wound-QoL Global Score-0.9 ± 0.87
Statistical analysis
  • SNF472 · t-test, 2 sided · p = 0.003

Adverse events

Collected over 12 weeks. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
SNF4722/14 (14.3%)7/14 (50%)13/14 (92.9%)
Most frequent serious events
Showing 10 of 13
Most frequent serious events
EventSNF472
Cardio-respiratory arrestCardiac disorders1/14
Cardiogenic shockCardiac disorders1/14
HaematemesisGastrointestinal disorders1/14
CellulitisInfections and infestations1/14
GangreneInfections and infestations1/14
InfectionInfections and infestations1/14
SepsisInfections and infestations1/14
Urinary tract infectionInfections and infestations1/14
Abdominal wound dehiscenceInjury, poisoning and procedural complications1/14
Fluid overloadMetabolism and nutrition disorders1/14
Most frequent other events
Showing 10 of 40
Most frequent other events
EventSNF472
DiarrhoeaGastrointestinal disorders2/14
NauseaGastrointestinal disorders2/14
Arteriovenous fistula thrombosisInjury, poisoning and procedural complications2/14
Electrocardiogram QT prolongedInvestigations2/14
HypoaesthesiaNervous system disorders2/14
Skin lesionSkin and subcutaneous tissue disorders2/14
HypertensionVascular disorders2/14
TachycardiaCardiac disorders1/14
VertigoEar and labyrinth disorders1/14
DyspepsiaGastrointestinal disorders1/14

Baseline characteristics

Age, Continuous
Age, Continuous(years)SNF472
Mean60.5 ± 14.1
Sex: Female, Male
Sex: Female, Male(Participants)SNF472
Female11
Male3
Race (NIH/OMB)
Race (NIH/OMB)(Participants)SNF472
American Indian or Alaska Native2
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American2
White10
More than one race0
Unknown or Not Reported0
Region of Enrollment
Region of Enrollment(participants)SNF472
United States13
United Kingdom1
07

Study locations

3 sites
  • Fresenius Medical Services
    Waltham, Massachusetts 02451, United States
  • Davita Clinical Research
    Minneapolis, Minnesota 55404, United States
  • Salford Royal Hospital
    Salford, United Kingdom
08

References and documents

Study documents

  • Study protocol · Apr 11, 2017
  • Statistical analysis plan · Jan 31, 2018

Documents are hosted by the registry — open the source record to download them.

09

Registry details

Key details

Study ID
NCT02790073
Lead sponsor
Sanifit Therapeutics S. A.
Responsible party
Sponsor
First posted
Jun 3, 2016
Start date
May 2016
Primary completion
Nov 15, 2017
Completion
Nov 15, 2017
Results posted
Aug 28, 2019
Last update
Aug 28, 2019

Study contacts

Vincent Brandenburg
principal investigator · RWTH Aachen University
View the source record on ClinicalTrials.gov ↗

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