Stem Cells for Uremic Calciphylaxis Patients

Study Overview
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Brief Summary
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Treatment for Uremic Calciphylaxis Patients with Human Amniotic Mesenchymal Stem Cells

Detailed Description
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Calciphylaxis is a rare, devastating disorder causing excruciatingly painful ischemic skin lesions. Sepsis due to the infection of ulcerated wounds is a common cause of death. To date, practical therapies typically include wound care, pain management, anti-infection and aggressive treatment of predisposing conditions. Drugs such as sodium thiosulfate, bisphosphonate, and cinacalcet are also suggested. However, it's still a lethal disease with 1-year mortality up to 80% for dialysis patients. Here the investigators plan to treat uremic calciphylaxis patients with human amniotic mesenchymal stem cells (hAMSCs).

Official Title
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Effects of Human Amniotic Mesenchymal Stem Cells (hAMSCs) on Wound Healing in Uremic Calciphylaxis Patients: an Open Label Single Arm Study

Conditions
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Chronic Kidney Diseases, Calciphylaxis, Calcific Uremic Arteriolopathy, Treatment

Intervention / Treatment
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* Other: Human amniotic mesenchymal stem cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 18-60 years old. Patients with chronic kidney disease who did not or had regular dialysis (hemodialysis or peritoneal dialysis). Clinical diagnosis of calciphylaxis. All subjects signed informed consent. Exclusion criteria: Patients who refuse to sign informed consent. Patients with malignant tumor or serious mental disease, cardiovascular disease, shock, abnormal liver function and secondary kidney disease. Pregnant or lactating women of childbearing age. Participation in another clinical trial with an experimental drug within 90 days prior the inclusion.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Uremic Calciphylaxis Patients<br>Human amniotic mesenchymal stem cells (hAMSCs) | Other: Human amniotic mesenchymal stem cells<br>* Intravenous infusion of hAMSCs: once every 3 weeks, a total of 6 times (if cured before the end of the planned course of treatment, it will be terminated in advance). Injection of hAMSCs to local skin lesions: once every 3 weeks, a total of 6 times (if cured before the end of the planned course of treatment, it will be terminated in advance).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Wound Healing | The Bates-Jensen Wound Assessment (BWAT) is a standardized tool for quantitative assessment of wound healing that includes the 13 items listed below: (1)Size; (2)Depth; (3)Edges; (4)Undermining or pockets; (5)Necrotic tissue type; (6)Necrotic tissue amount; (7)Exudate type; (8)Exudate amount; (9)Surrounding skin color; (10)Peripheral tissue edema; (11)Peripheral tissue induration; (12)Granulation tissue; (13)Epithelialization. Each item was rated on a scale of 1 (best) to 5 (worst). The BWAT total score is the sum of the individual items with a possible range of 13 (best) to 65 (worst). | Up to 1 year. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Wound Pain | Assessed by pain visual analog scale (VAS). 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. 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. VAS Score range 0 (best) to 100 (Worst). | Up to 1 year. | 
| Measured Quality of Life | The wound-quality of life(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. | Up to 1 year. | 
| Systemic Infection | Assessed by serum hypersensitive C-reactive protein level(hCRP, mg/L). Increased serum hCRP levels above normal ranges indicate infection. | Up to 1 year. | 
| Survival State | Survival time from the hAMSCs treatment started(momths) | From date of treatment until the date of die, assessed up to 1 year. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Chronic Kidney Diseases, Calciphylaxis, Calcific Uremic Arteriolopathy, Human Amniotic Mesenchymal Stem Cells, Rare disease (ORPHA280062)