Calciphylaxis MedDRA version: 20.0 Level: PT Classification code 10051714 Term: Calciphylaxis System Organ Class: 10027433 - Metabolism and nutrition disorders
Conditions
Interventions
Trade Name: Sodium Thiosulfate 250 mg/mL Solution for Injection
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Sodium Thiosulfate
CAS Number: 7772-98-7
Other descriptive name: SODIUM
Sponsors
Hope Pharmaceuticals Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Informed of the investigational nature of the study and sign written informed consent -Willing and able to adhere to all study-related procedures, including adherence to study medication regimen -Male or female 18 years old and over -End-stage renal disease on chronic haemodialysis -Calciphylaxis with active skin lesions(s) of any morphological appearance (including but not limited to livedo, induration, ulceration etc.) and tissue histology review consistent with calciphylaxis diagnosis. Histological features consistent with calciphylaxis will include soft tissue calcification, microthrombosis and/or fibrointimal hyperplasia of dermal arterioles. -Acute pain associated with calciphylaxis lesions with the worst non-evoked pain intensity score of greater than or equal to 5 at initial screening on the modified BPI/SF scale and for both of the 2 days preceding randomisation with the mean of the two scores greater than or equal to 6 -Females of childbearing potential must have a pregnancy test (urine or serum [if anuric]) at screening and not be pregnant and willing to use an acceptable method of contraception from screening through the last study visit (4 weeks): hormonal (oral pills, implant or injection) begun at time of screening, barrier (condom, diaphragm or cervical cap with spermicide), intrauterine device (IUD), abstinence (not having sex), being with the same sex partner or a partner who has had a vasectomy at least six months before the screening visit Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: -Peritoneal dialysis patients -Current congestive heart failure exacerbation -Baseline abnormalities related to QT prolongation (corrected QT interval >490 ms) or history of Torsade de Pointes, hypocalcaemia (serum albumin-corrected calcium 50% improvement in the worst non-evoked pain intensity score in the preceding 24 hour period of run-in phase or > 50% improvement in skin lesions during the run-in phase as determined by clinical investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: 1. The last two days (averaged over Day 20 and Day 21) of the 3-week double-blind treatment phase ;Main Objective: To compare the effects of treatment with intravenous Sodium Thiosulfate Injection vs. placebo for the proportion of patients (responders) who achieve a greater than or equal to 30% mean reduction from the last 24 hours of the 72 hour run-in phase prior to initiation of study drug in pain intensity score for the worst non-evoked pain intensity score calculated from the daily pain diaries (modified BPI/SF) over the last two days (averaged over Day 20 and Day 21) of the 3-week double-blind treatment phase in adult patients with end-stage renal disease on chronic haemodialysis. ;Secondary Objective: To compare the effects of treatment with intravenous Sodium Thiosulfate Injection vs. placebo on: - Proportion of patients who achieve improvement or stabilisation (i.e. not worsening) of index lesion at the end of the 3-week double-blind treatment phase on Day 21 based upon clinical assessment compared to the clinical assessment during run-in phase. -Occurrence of surgical debridement of skin lesions and/or amputation over the third week of the 3-week double-blind treatment phase. -Occurrence of surgical debridement of skin lesions and/or amputation over the 3-week double-blind treatment phase. -Time in days to when patient achieves a greater than or equal to 30% mean reduction from the last 24 hours of the 72 hour run-in phase prior to initiation of study drug in pain intensity score for the worst non-evoked pain intensity score calculated from the daily pain diaries (modified BPI/SF);Primary end point(s): 1. The proportion of patients (responders) who achieve a greater than or equal 30% mean reduction from the last 24 hours of the 72 hour run-in period prior to initiation of study drug in pain intensity score for the worst non-evoked pain intensity score calculated from the daily pain diaries (modified BPI/SF) ov | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Proportion of patients who achieve improvement or stabilisation (i.e. not worsening) of index lesion at the end of the 3-week double-blind treatment phase on Day 21 based upon clinical assessment compared to the clinical assessment during run-in phase 2. Occurrence of surgical debridement of skin lesions and/or amputation during the third week of the 3-week double-blind treatment phase 3. Occurrence of surgical debridement of skin lesions and/or amputation over the 3-week double-blind treatment phase 4. Time in days to when a patient achieves a greater than or equal to 30% mean reduction from the last 24 hours of the 72 hour run-in phase prior to initiation of study drug in pain intensity score for the worst non-evoked pain intensity score calculated from the daily pain diaries (modified BPI/SF) in patients who are classified as responders at endpoint;Timepoint(s) of evaluation of this end point: 1. Day 21 of the 3-week double-blind treatment phase 2. Over the third week of the 3-week double-blind treatment phase 3. Over the 3-week double-blind treatment phase 4. At the end of the 3-week double-blind treatment phase | — |
Countries
Australia, Canada, United Kingdom, United States
Contacts
Public ContactMedical Director
Hope Pharmaceuticals Ltd
Outcome results
None listed