Trial Outcomes & Findings for Phase 2 Study With SNF472 in Calciphylaxis Patients (NCT NCT02790073)

NCT ID: NCT02790073

Last Updated: 2019-08-28

Results Overview

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).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

14 participants

Primary outcome timeframe

12 weeks

Results posted on

2019-08-28

Participant Flow

Participant milestones

Participant milestones
Measure
SNF472
SNF472 for Calciphylaxis
Overall Study
STARTED
14
Overall Study
COMPLETED
11
Overall Study
NOT COMPLETED
3

Reasons for withdrawal

Reasons for withdrawal
Measure
SNF472
SNF472 for Calciphylaxis
Overall Study
Death
1
Overall Study
Withdrawal from HD
1
Overall Study
Withdrawal by Subject
1

Baseline Characteristics

Phase 2 Study With SNF472 in Calciphylaxis Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
SNF472
n=14 Participants
SNF472 for calciphylaxis
Age, Continuous
60.5 years
STANDARD_DEVIATION 14.1 • n=99 Participants
Sex: Female, Male
Female
11 Participants
n=99 Participants
Sex: Female, Male
Male
3 Participants
n=99 Participants
Race (NIH/OMB)
American Indian or Alaska Native
2 Participants
n=99 Participants
Race (NIH/OMB)
Asian
0 Participants
n=99 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
Race (NIH/OMB)
Black or African American
2 Participants
n=99 Participants
Race (NIH/OMB)
White
10 Participants
n=99 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
Region of Enrollment
United States
13 participants
n=99 Participants
Region of Enrollment
United Kingdom
1 participants
n=99 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: Subjects who received at least 1 dose of SNF472 and had at least 1 postbaseline efficacy measurement

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).

Outcome measures

Outcome measures
Measure
SNF472
n=14 Participants
SNF472 for calciphylaxis
Wound Healing
-8.1 units on a scale
Standard Deviation 8.5

SECONDARY outcome

Timeframe: 12 weeks

Population: Subjects who received at least 1 dose of SNF472 and had at least 1 postbaseline efficacy measurement

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).

Outcome measures

Outcome measures
Measure
SNF472
n=14 Participants
SNF472 for calciphylaxis
Wound Pain
-23.6 units on a scale
Standard Deviation 30.0

SECONDARY outcome

Timeframe: 12 weeks

Population: Subjects who received at least 1 dose of SNF472 and had at least 1 postbaseline efficacy measurement

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.

Outcome measures

Outcome measures
Measure
SNF472
n=14 Participants
SNF472 for calciphylaxis
Wound-QoL Global Score
-0.9 units on a scale
Standard Deviation 0.87

Adverse Events

SNF472

Serious events: 7 serious events
Other events: 13 other events
Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure
SNF472
n=14 participants at risk
SNF472 for Calciphylaxis
Cardiac disorders
Cardio-respiratory arrest
7.1%
1/14 • Number of events 1 • 12 weeks
Cardiac disorders
Cardiogenic shock
7.1%
1/14 • Number of events 1 • 12 weeks
Gastrointestinal disorders
Haematemesis
7.1%
1/14 • Number of events 1 • 12 weeks
Infections and infestations
Cellulitis
7.1%
1/14 • Number of events 1 • 12 weeks
Infections and infestations
Gangrene
7.1%
1/14 • Number of events 1 • 12 weeks
Infections and infestations
Infection
7.1%
1/14 • Number of events 1 • 12 weeks
Infections and infestations
Sepsis
7.1%
1/14 • Number of events 1 • 12 weeks
Infections and infestations
Urinary tract infection
7.1%
1/14 • Number of events 1 • 12 weeks
Injury, poisoning and procedural complications
Abdominal wound dehiscence
7.1%
1/14 • Number of events 1 • 12 weeks
Metabolism and nutrition disorders
Fluid overload
7.1%
1/14 • Number of events 1 • 12 weeks
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
7.1%
1/14 • Number of events 1 • 12 weeks
Vascular disorders
Dry gangrene
7.1%
1/14 • Number of events 1 • 12 weeks
Vascular disorders
Hypertensive emergency
7.1%
1/14 • Number of events 1 • 12 weeks

Other adverse events

Other adverse events
Measure
SNF472
n=14 participants at risk
SNF472 for Calciphylaxis
Cardiac disorders
Tachycardia
7.1%
1/14 • Number of events 1 • 12 weeks
Ear and labyrinth disorders
Vertigo
7.1%
1/14 • Number of events 1 • 12 weeks
Gastrointestinal disorders
Diarrhoea
14.3%
2/14 • Number of events 2 • 12 weeks
Gastrointestinal disorders
Dyspepsia
7.1%
1/14 • Number of events 1 • 12 weeks
Gastrointestinal disorders
Gastrooesophageal reflux disease
7.1%
1/14 • Number of events 1 • 12 weeks
Gastrointestinal disorders
Nausea
14.3%
2/14 • Number of events 2 • 12 weeks
Gastrointestinal disorders
Rectal haemorrhage
7.1%
1/14 • Number of events 1 • 12 weeks
Gastrointestinal disorders
Vomiting
7.1%
1/14 • Number of events 1 • 12 weeks
General disorders
Asthenia
7.1%
1/14 • Number of events 1 • 12 weeks
General disorders
Chest discomfort
7.1%
1/14 • Number of events 2 • 12 weeks
General disorders
Peripheral swelling
7.1%
1/14 • Number of events 1 • 12 weeks
Infections and infestations
Anal abscess
7.1%
1/14 • Number of events 1 • 12 weeks
Infections and infestations
Cellulitis
7.1%
1/14 • Number of events 1 • 12 weeks
Infections and infestations
Conjunctivitis
7.1%
1/14 • Number of events 1 • 12 weeks
Infections and infestations
Pneumonia
7.1%
1/14 • Number of events 1 • 12 weeks
Infections and infestations
Upper respiratory tract infection
7.1%
1/14 • Number of events 1 • 12 weeks
Injury, poisoning and procedural complications
Arteriovenous fistula thrombosis
14.3%
2/14 • Number of events 2 • 12 weeks
Injury, poisoning and procedural complications
Fall
7.1%
1/14 • Number of events 1 • 12 weeks
Injury, poisoning and procedural complications
Laceration
7.1%
1/14 • Number of events 1 • 12 weeks
Injury, poisoning and procedural complications
Pubis fracture
7.1%
1/14 • Number of events 1 • 12 weeks
Injury, poisoning and procedural complications
Rib fracture
7.1%
1/14 • Number of events 1 • 12 weeks
Injury, poisoning and procedural complications
Stress fracture
7.1%
1/14 • Number of events 1 • 12 weeks
Injury, poisoning and procedural complications
Wound complication
7.1%
1/14 • Number of events 6 • 12 weeks
Investigations
Electrocardiogram QT prolonged
14.3%
2/14 • Number of events 2 • 12 weeks
Investigations
Haemoglobin decreased
7.1%
1/14 • Number of events 1 • 12 weeks
Investigations
Oxygen saturation decreased
7.1%
1/14 • Number of events 1 • 12 weeks
Metabolism and nutrition disorders
Hyperkalaemia
7.1%
1/14 • Number of events 1 • 12 weeks
Metabolism and nutrition disorders
Hypernatraemia
7.1%
1/14 • Number of events 1 • 12 weeks
Musculoskeletal and connective tissue disorders
Muscle spasms
7.1%
1/14 • Number of events 4 • 12 weeks
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
7.1%
1/14 • Number of events 1 • 12 weeks
General disorders
Peripheral oedema
7.1%
1/14 • Number of events 1 • 12 weeks
Investigations
Cellulitis
7.1%
1/14 • Number of events 1 • 12 weeks
Metabolism and nutrition disorders
Fluid overload
7.1%
1/14 • Number of events 1 • 12 weeks
Musculoskeletal and connective tissue disorders
Pain in extremities
7.1%
1/14 • Number of events 1 • 12 weeks
Nervous system disorders
Dyskinesia
7.1%
1/14 • Number of events 1 • 12 weeks
Nervous system disorders
Hypoaesthesia
14.3%
2/14 • Number of events 5 • 12 weeks
Skin and subcutaneous tissue disorders
Decubitis ulcer
7.1%
1/14 • Number of events 2 • 12 weeks
Skin and subcutaneous tissue disorders
Skin lesion
14.3%
2/14 • Number of events 2 • 12 weeks
Skin and subcutaneous tissue disorders
Skin ulcer
7.1%
1/14 • Number of events 2 • 12 weeks
Vascular disorders
Hypertension
14.3%
2/14 • Number of events 3 • 12 weeks

Additional Information

Rekha Garg MD MS

Sanifit

Phone: 18582815978

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: LTE60