Clinical Trial Results:
Phase 2 Open Label Single Arm Repeat Dose Study to Assess the Effect of SNF472 on Wound Healing in Uraemic Calciphylaxis Patients
|
Summary
|
|
EudraCT number |
2015-004313-25 |
Trial protocol |
ES GB |
Global end of trial date |
14 Nov 2017
|
|
Results information
|
|
Results version number |
v1(current) |
This version publication date |
24 May 2019
|
First version publication date |
24 May 2019
|
Other versions |
|
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
|
|||
|
Trial identification
|
|||
Sponsor protocol code |
SNFCT2015_04
|
||
|
Additional study identifiers
|
|||
ISRCTN number |
- | ||
US NCT number |
NCT02790073 | ||
WHO universal trial number (UTN) |
- | ||
|
Sponsors
|
|||
Sponsor organisation name |
Laboratoris Sanifit
|
||
Sponsor organisation address |
Parc Bit, Europa Building, Ctra. Valldemossa km 7.4, Palma de Mallorca, Spain, 07121
|
||
Public contact |
R&D Department, Laboratoris Sanifit, +34 971439925, ana-zeralda.canals@sanifit.com
|
||
Scientific contact |
R&D Department, Laboratoris Sanifit, +34 971439925, ana-zeralda.canals@sanifit.com
|
||
|
Paediatric regulatory details
|
|||
Is trial part of an agreed paediatric investigation plan (PIP) |
No
|
||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
|
||
Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
|
||
|
Results analysis stage
|
|||
Analysis stage |
Final
|
||
Date of interim/final analysis |
23 Nov 2018
|
||
Is this the analysis of the primary completion data? |
Yes
|
||
Primary completion date |
14 Nov 2017
|
||
Global end of trial reached? |
Yes
|
||
Global end of trial date |
14 Nov 2017
|
||
Was the trial ended prematurely? |
No
|
||
|
General information about the trial
|
|||
Main objective of the trial |
The objective of this study was to evaluate the effect of SNF472 on top of standard of care on promoting wound healing and other parameters of therapeutic response in hemodialysis (HD) patients with calciphylaxis (calcific uremic arteriolopathy [CUA]).
|
||
Protection of trial subjects |
Written informed consent was obtained from each subject prior to evaluations being performed for eligibility. Subjects were given adequate time to review the information in the informed consent and were allowed to ask, and have answered, questions concerning all portions of the conduct of the study. Through the informed consent process each subject was made aware of the purpose of the study, the procedures, the benefits and risks of the study, the discomforts and the precautions taken. Any side effects or other health issues occurring during the study were followed up by the study doctor. Subjects were able to stop taking part in the study at any time without giving any reason.
|
||
Background therapy |
Standard of care for CUA was in accordance with each site’s standard procedures. | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
01 Jan 2016
|
||
Long term follow-up planned |
No
|
||
Independent data monitoring committee (IDMC) involvement? |
No
|
||
|
Population of trial subjects
|
|||
Number of subjects enrolled per country |
|||
Country: Number of subjects enrolled |
United Kingdom: 2
|
||
Country: Number of subjects enrolled |
United States: 12
|
||
Worldwide total number of subjects |
14
|
||
EEA total number of subjects |
2
|
||
Number of subjects enrolled per age group |
|||
In utero |
0
|
||
Preterm newborn - gestational age < 37 wk |
0
|
||
Newborns (0-27 days) |
0
|
||
Infants and toddlers (28 days-23 months) |
0
|
||
Children (2-11 years) |
0
|
||
Adolescents (12-17 years) |
0
|
||
Adults (18-64 years) |
8
|
||
From 65 to 84 years |
5
|
||
85 years and over |
1
|
||
|
|||||||||||||||
|
Recruitment
|
|||||||||||||||
Recruitment details |
For this study, both non-hospitalised and hospitalised calciphylaxis patients were recruited after obtaining written informed consent, with a preference for non-hospitalised patients, wherever possible. | ||||||||||||||
|
Pre-assignment
|
|||||||||||||||
Screening details |
Potential patients for the inclusion in the clinical trial attended the Screening Visit within 14 days before receiving the first dose of IMP. | ||||||||||||||
|
Period 1
|
|||||||||||||||
Period 1 title |
Overall Study (overall period)
|
||||||||||||||
Is this the baseline period? |
Yes | ||||||||||||||
Allocation method |
Non-randomised - controlled
|
||||||||||||||
Blinding used |
Not blinded | ||||||||||||||
|
Arms
|
|||||||||||||||
|
Arm title
|
SNF472 | ||||||||||||||
Arm description |
SNF472 30mg/ml or 90 mg/ml solution for infusion | ||||||||||||||
Arm type |
Experimental | ||||||||||||||
Investigational medicinal product name |
SNF472
|
||||||||||||||
Investigational medicinal product code |
|||||||||||||||
Other name |
|||||||||||||||
Pharmaceutical forms |
Solution for infusion
|
||||||||||||||
Routes of administration |
Intravenous use
|
||||||||||||||
Dosage and administration details |
SNF472, was provided as 10 or 5 mL of sterile liquid in transparent glass vials, containing either 300 mg or 450 mg of SNF472, respectively, for a concentration of 30 mg/mL or 90 mg/mL. The SNF472 solution was diluted in a 100-mL saline bag prior to administration. SNF472 was administered TIW to the subject by slow infusion (2.5 to 4 hours) during the subject’s regular HD session. The dose of SNF472 administered at each session was 400 to 900 mg, depending on the subject’s body weight category at screening, resulting in per kilogram doses ranging from 5.6 to 8.6 mg/kg.
|
||||||||||||||
|
|||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||
|
Baseline characteristics reporting groups
|
|||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Overall Study
|
||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Intention to treat | ||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||
|
|||
|
End points reporting groups
|
|||
Reporting group title |
SNF472
|
||
Reporting group description |
SNF472 30mg/ml or 90 mg/ml solution for infusion | ||
Subject analysis set title |
Intention to Treat
|
||
Subject analysis set type |
Intention-to-treat | ||
Subject analysis set description |
The primary analysis population for efficacy analyses was the Intention-to-Treat Population (ITT), which included subjects who received at least 1 dose of SNF472 and had at least 1 postbaseline efficacy measurement
|
||
|
|||||||||
End point title |
Wound Healing [1] | ||||||||
End point description |
The BWAT is a standardized tool for quantitative assessment of wound healing that includes the 13 items (size, depth, edges, undermining, necrotic tissue type and amount, exudate type and amount, surrounding skin color, peripheral tissue edema and induration, granulation tissue and epithelialization. Each item is rated on a scale of 1 (best) to 5 (worst). The BWAT total is the sum of the individual items with a possible range of 13 (best) to 65 (worst).
|
||||||||
End point type |
Primary
|
||||||||
End point timeframe |
Absolute change in Bates-Jensen Wound Assessment Tool (BWAT) total score between baseline (week1) and week 12 for the primary lesion (largest lesion).
|
||||||||
| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Statistical analysis of the primary efficacy endpoints, change in BWAT total score between baseline and Week 12 for the primary lesion was summarized descriptively and analysed using a paired Student’t t-test for the ITT population. The same analysis was conducted for the secondary efficacy endpoints of VAS and Wound Quality of Life. |
|||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Wound Pain | ||||||||
End point description |
The Pain Visual Analogue Scale (VAS) is a horisontal line, 100 mm in length, anchored by word descriptors at each end. The subject marks the point on the line that represents his/her perception of his/her current pain status. The VAS was determined by measuring in millimeters from the lest hand end of the line (no pain) to the point that the subject marked. The VAS score ranges from 0 (best) to 100 (worse).
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
Absolute change from baseline (week 1) to week 12 in the Pain Visual Analogue Scale (VAS) Score
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Wound Quality of Life Global Score | ||||||||
End point description |
The Wound Quality of Life questionnaire measures the disease specific, health related quality of life 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 (worst) scale with possible responses from 'not at all' to 'very much". The total score is the average of the 17 responses.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
Absolute change from baseline (week 1) to week 12 in the Wound Quality of Life Global Score.
|
||||||||
|
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Adverse events information
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Timeframe for reporting adverse events |
Adverse events reported are from the time subject received the first dose of SNF472 to the last follow up visit at week 13 or early termination visit.
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Dictionary used for adverse event reporting
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
20
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Reporting groups
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
SNF472
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||
Substantial protocol amendments (globally) |
|||
| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
||
10 Mar 2016 |
Amendment 1 |
||
08 Oct 2016 |
Amendment 2 |
||
11 Apr 2017 |
Amendment 3 |
||
Interruptions (globally) |
|||
| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
|||
| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| Study limitations included the small number of patients, lack of a control group, and open-label treatment. | |||
